Reported trial results for Cystic Fibrosis

155 trials have reported results.

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

• NCT05453578 · results posted 16 July 2026

  According to the results reported on ClinicalTrials.gov, this trial tested an intravenous bacteriophage treatment (a therapy using viruses that target bacteria) against a placebo in people with *Pseudomonas aeruginosa* lung infections, likely in the context of cystic fibrosis or a similar condition. The trial ran in two stages: a small early safety-focused stage (Stage 1) with 6 participants across three dose levels, and a larger Stage 2 involving 67 participants spread across three dose groups and a placebo group. In total, 73 people started the study and 71 completed it. The reported data shows several things were measured. For unwanted events rated "Grade 2 or higher" (meaning moderate to severe), the numbers reported were: 2 out of 8 participants in the lowest dose group, 7 out of 26 in the middle dose group, 4 out of 9 in the highest dose group, and 8 out of 24 in the placebo group. The trial also tracked changes in the amount of *Pseudomonas aeruginosa* bacteria in sputum (mucus coughed up) over 30 days. The reported data shows that in the middle-dose bacteriophage group, average bacterial counts shifted by small amounts ranging from 0.0 to −0.4 (log units) at various time points up to Day 30, while in the placebo group the reported shifts ranged from +0.1 to +0.5 over the same period. The trial also used a ranking system called DOOR (Desirability of Outcome Ranking) to compare how participants fared overall by Day 8; the reported data shows that in the bacteriophage group, 25 of 26 participants fell into the lowest-priority rank (less than a 1 log reduction in bacteria, with no serious related event), compared with 18 of 24 in the placebo group, with small numbers in higher-benefit ranks in both groups. A subgroup analysis of participants whose bacteria were identified as susceptible to the four-phage cocktail reported 12 bacteriophage participants and 13 placebo participants in that subgroup, though further breakdown of their outcomes was not reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05616221 · results posted 5 January 2026

  According to the results reported on ClinicalTrials.gov, this trial (NCT05616221) tested an inhaled investigational treatment called AP-PA02 — a type of bacteriophage (a virus designed to target bacteria) — in people with a bacterial lung infection caused by *Pseudomonas aeruginosa*. A total of 48 people started the trial, divided into two cohorts (groups) and given either one of two doses of AP-PA02 or a placebo (an inactive treatment). The trial was measuring changes in the amount of *Pseudomonas aeruginosa* bacteria found in sputum (mucus coughed up from the lungs) one week after the treatment period ended. The reported data shows the primary outcome was the change in bacterial levels from the start of the trial to one week after treatment finished, measured on a logarithmic scale (a way of expressing large ranges of numbers in a more manageable form — a change of 1 on this scale represents a tenfold change in bacterial count). In Cohort A, the lower-dose AP-PA02 group showed a reported change of −0.5 (a small reduction in bacterial levels), the higher-dose group showed −0.1, and results for the Cohort A placebo group were not reported in the submitted data. In Cohort B, the higher-dose AP-PA02 group showed a change of −0.4, while the Cohort B placebo group showed a change of +1.0 (an increase in bacterial levels). Results for the Cohort B lower-dose group were not reported in the submitted data. In a separate pooled (combined) analysis across both cohorts, the AP-PA02 groups together showed a reported change of −0.6, compared to +0.1 in the combined placebo groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04375514 · results posted 17 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04375514) tested an inhaled investigational drug called ARO-ENaC across two groups of participants: healthy volunteers and people with cystic fibrosis. A total of 43 people took part — 36 healthy volunteers and 7 people with cystic fibrosis. Participants received different doses of ARO-ENaC (ranging from 20 mg to 180 mg, given over several days) or a placebo (an inactive treatment used for comparison). The trial was primarily measuring how many participants experienced adverse events — that is, any unwanted medical occurrences during the study period. The reported data shows that, among the healthy volunteers, the number of participants who experienced adverse events ranged from 2 out of 4 (in the 40 mg group) to 6 out of 8 (in the 180 mg group who also underwent a bronchoscopy, a procedure to look inside the airways). In the pooled placebo group for healthy volunteers, 10 out of 12 participants reported adverse events. Among people with cystic fibrosis, 3 out of 4 in the ARO-ENaC group and all 3 in the placebo group reported adverse events. The reported data also shows small changes in blood electrolyte levels (minerals such as potassium and sodium) across all groups, and small changes in a breathing measurement called FEV1 (the amount of air a person can forcefully breathe out in one second) in healthy volunteers — these changes appeared in both the ARO-ENaC and placebo groups. The trial also tracked how much of the drug reached the bloodstream; for healthy volunteers, the peak blood concentration of ARO-ENaC ranged from 3.33 to 56.7 ng/mL depending on dose, with the time to reach that peak ranging from 2 to 50 hours. For participants with cystic fibrosis, the peak concentration data was not reported in full detail for all time points in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04594369 · results posted 16 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04594369) looked at a medicine called brensocatib in people with a lung condition called bronchiectasis. A total of 1,721 people took part — 583 received a 10 mg daily dose of brensocatib, 575 received a 25 mg daily dose, and 563 received a placebo (a dummy treatment with no active ingredient). The main thing the trial was measuring was how often participants had "pulmonary exacerbations" — that is, flare-ups of lung symptoms serious enough for a doctor to prescribe antibiotics — over the course of a year. The reported data shows that, on average, participants in the placebo group had approximately 1.29 flare-ups per person per year. In the 10 mg brensocatib group, the reported rate was approximately 1.02 flare-ups per person per year, and in the 25 mg group it was approximately 1.04 per person per year. For the secondary outcomes, the reported data shows that the average time before a first flare-up occurred was about 37 weeks in the placebo group, compared with about 49 weeks in the 10 mg group and about 51 weeks in the 25 mg group. Around 40% of people in the placebo group had no flare-ups at all during the 52-week treatment period, compared with about 48–49% in both brensocatib groups. For severe flare-ups (those requiring hospitalisation or a drip of antibiotics), the reported rate was 0.185 per person per year in the placebo group versus 0.137 in both brensocatib groups. Lung function (measured by how much air someone can forcefully breathe out in one second) showed small changes across all three groups, with no group showing a clear improvement. On a quality-of-life questionnaire focused on breathing symptoms (scored 0–100, where higher is better), the reported average improvement from the start of the study to week 52 was about 4.8 points for placebo, 6.8 points for the 10 mg group, and 8.6 points for the 25 mg group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05865886 · results posted 18 November 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 22 people in total — 7 received a placebo (a dummy treatment with no active ingredient) and 15 received a 5 mg dose of the investigational medicine BI 1291583. All 7 placebo participants and 14 of the 15 in the active medicine group completed the study (one person in the active group did not finish). The trial was primarily measuring how often participants experienced adverse events — that is, any unwanted or unexpected health changes that occurred during the study period — and also looked at how the medicine moved through the body and whether it affected a substance in lung mucus called neutrophil elastase (an enzyme linked to lung inflammation). The reported data shows that 85.7% of participants in the placebo group and 93.3% in the BI 1291583 group experienced at least one treatment-emergent adverse event (a health change that appeared or worsened after starting the study treatment). Regarding the secondary outcome measuring neutrophil elastase activity in sputum (mucus coughed up from the lungs) at 8 weeks, the reported data shows the placebo group had an average increase of about 39.7% from their starting level, while the BI 1291583 group had an average decrease of about 66.0% from their starting level. For the blood concentration measurements in the BI 1291583 group only, the reported data shows the medicine reached a peak level of 3.33 nmol/L (nanomoles per litre, a measure of concentration) after the first dose, rising to 11.6 nmol/L at steady state (when levels in the body had stabilised with regular dosing). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03506061 · results posted 11 September 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03506061) enrolled 42 people with cystic fibrosis across two groups: 22 people who showed signs of partial lung function (based on sweat chloride levels or pancreatic status), and 20 people who carry a specific genetic variation called N1303K. Of the 42 who started, 40 completed the study. The trial was measuring several things, including how much air participants could forcefully breathe out in one second (called FEV1, expressed as a percentage of what would be expected for a healthy person of the same age and size), the level of chloride (salt) in their sweat, quality of life related to breathing, and whether laboratory-grown cells from participants could predict who might respond to the treatment (Trikafta). The reported data shows the following numbers across the two groups. For the partial-function group, the FEV1 readings were reported as 74.8% and 76% (likely before and after the study period, though the data does not label these time points explicitly). Their sweat chloride levels were reported as 66.9 and 57.8 mmol/L across the two measurement points. For the N1303K group, FEV1 was reported as 75.8% and 85.3%, and sweat chloride was reported as 109 and 107.9 mmol/L. On the breathing-related quality-of-life questionnaire (scored from 1 to 100, where higher means better), the partial-function group scored 66.4 and then 74.1, while the N1303K group scored 60.6 and then 81.4. For the laboratory cell testing outcome, the reported data shows that zero participants in the partial-function group had cells that predicted a response to treatment in the lab setting. The reported data also notes that no participants' laboratory-grown cells successfully predicted a personalised response, which was one of the primary goals of the study. It is worth noting that the data as submitted does not clearly label which measurements were taken at the start versus the end of the study period, so the figures above are presented in the order they appear in the reported results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05006573 · results posted 20 July 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05006573) enrolled 99 people with a lung condition called non-cystic fibrosis bronchiectasis — 54 received the study drug benralizumab (30 mg) and 45 received a placebo (a dummy treatment). The trial was split into two phases: a double-blind period (where neither participants nor doctors knew who received which treatment) and an open-label extension period (where everyone knew). The main thing the trial was measuring was how often participants had flare-ups (called exacerbations) of their bronchiectasis over the course of the double-blind period. It also measured secondary things such as time until a first flare-up, lung function, and quality-of-life scores using a few different questionnaires. The reported data shows that during the double-blind period, people in the benralizumab group had a reported rate of 1.44 flare-ups per year, compared with 1.27 flare-ups per year in the placebo group. For time to first flare-up, the benralizumab group reached their first flare-up at a median of 233 days, compared with 316 days in the placebo group. On a respiratory symptoms quality-of-life questionnaire (scored 0–100, where higher means better), the benralizumab group showed score changes from baseline generally ranging from around 5 to 7 points across time points, while the placebo group showed changes ranging from approximately −1.5 to 3.1 points. Lung function (measured as the volume of air a person can forcefully breathe out in one second) showed small changes in both groups across multiple time points, with the reported figures provided at several intervals throughout the trial period. Cough-related quality-of-life scores and physical functioning scores were also reported at multiple time points for both groups, with the numbers available in the full data record. The reported data shows changes across several questionnaires and time points, and a complete breakdown of each time point is available in the full ClinicalTrials.gov record. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04183790 · results posted 18 May 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04183790) involved 64 participants in the main group receiving a triple combination medicine called ELX/TEZ/IVA, plus 2 additional participants included for a specific analysis. The trial was designed for people with cystic fibrosis, and it measured safety-related events as its main focus, alongside several secondary measurements including lung function, a sweat test, quality of life, and body weight indicators. The reported data shows that, for the primary focus on safety-related events, 64 participants were recorded as having experienced treatment-emergent adverse events (that is, health issues that appeared or worsened after starting the treatment), while 7 participants experienced serious adverse events (more significant health issues requiring attention). For the secondary measurements, the reported data shows an average increase of 9.6 percentage points in a lung function test (measuring how much air someone can forcefully breathe out in one second), a decrease of 57.9 units in a sweat chloride test (a marker commonly used in cystic fibrosis monitoring — lower levels are generally considered closer to normal), an increase of 10.0 points on a respiratory quality-of-life questionnaire (scored 0–100, where higher scores indicate fewer breathing symptoms), an increase of 3.60 in BMI (a measure relating weight to height), and an increase of 0.39 in BMI-for-age z-score (a measure of how a participant's BMI compared to what is typical for their age group). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02496780 · results posted 17 March 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT02496780) enrolled 65 people across four groups: 12 received a placebo, 13 received a long-acting insulin called Levemir, 15 received a fast-acting insulin called Novolog, and 25 were healthy volunteers included for comparison. The trial was measuring how the body breaks down its own proteins — a process sometimes called "protein turnover" — in people with cystic fibrosis (CF), and whether four weeks of insulin treatment changed that rate compared to a placebo. Not everyone who started the trial finished it: by the end, 11, 9, 10, and 20 people respectively completed their group. The reported data shows that the primary outcome — the change in the body's protein breakdown rate after four weeks — was very small across all three CF treatment groups. In the placebo group, the rate shifted by −0.01 µmol per kilogram of lean body mass per minute (meaning a tiny decrease), while the long-acting insulin group showed a change of +0.022 and the fast-acting insulin group showed a change of +0.0004 in the same units. These numbers represent very small shifts from each group's starting point. For context, the unit "µmol/kg(LBM)/min" is simply a way of measuring how quickly the body is breaking down protein relative to a person's body size. The reported data also shows a secondary comparison between people with CF and healthy volunteers at the start of the study (before any treatment). CF participants had a baseline protein breakdown rate of 0.963 µmol/kg(LBM)/min, while healthy controls measured 0.921 in the same units — a modest numerical difference. No further secondary outcome figures beyond these were reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04938726 · results posted 13 February 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04938726) involved 15 people in total — 10 in the Ketone Monoester group and 5 in the Placebo group. Of those, 9 from the Ketone Monoester group and all 5 from the Placebo group completed the trial. The trial was measuring lung function, a marker of inflammation in the body, and how participants felt about their own breathing and respiratory health. The reported data shows the following numbers for the two groups. For lung function (measured as the amount of air a person can forcefully breathe out in one second, called FEV1.0), the Ketone Monoester group recorded 1.9 litres per second, compared to 2.0 litres per second in the Placebo group. For the inflammation marker Interleukin-1 Beta (a protein the body produces during inflammation), the Ketone Monoester group recorded 0.3 pg/mL (a very small unit of measurement) compared to 0.1 pg/mL in the Placebo group. For the secondary outcome — a questionnaire called the Cystic Fibrosis Questionnaire-Revised, where scores range from 0 to 100 and higher scores represent better self-reported respiratory quality of life — the Ketone Monoester group scored 70.1 and the Placebo group scored 60.3. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02038803 · results posted 6 December 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 5 adults with cystic fibrosis. The study was looking at whether participants would stick to (adhere to) their antibiotic treatment — specifically comparing an inhaled solution called Tobramycin with a dry-powder inhaler version called TOBI Podhaler. Each person acted as their own comparison point, meaning their experience with one treatment was compared to their experience with the other. It is worth noting that none of the 5 participants who started the study were recorded as having completed it. The reported data shows that 4 out of the 5 participants were recorded against the primary measure of treatment adherence. No further breakdown of what this means in practice was provided in the submitted data. For the two secondary outcomes — whether participants expressed a preference for the TOBI Podhaler, and breathing test (spirometry) results measured at the start and end of the study — no numerical results were reported to ClinicalTrials.gov. Because that data was not submitted, it is not possible to describe what those measures found. It is also worth noting that this was a very small study (5 people), and the fact that no participants were recorded as completing the trial limits what can be understood from these numbers. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT06350474 · results posted 26 November 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 477 people with cystic fibrosis — 240 in a group that stopped taking a medication called DNase (also known as dornase alfa), and 237 in a group that continued taking it. The trial was designed to find out whether stopping DNase while on a newer cystic fibrosis treatment was "non-inferior" — meaning not meaningfully worse — compared to continuing it. The main thing being measured was a breathing test result called FEV1 % predicted, which is a standard way of measuring how much air a person can forcefully breathe out, expressed as a percentage of what would be expected for someone of their age, height, and sex. Nearly all participants — 238 in the discontinue group and 236 in the continue group — completed the trial. The reported data shows that over the six-week study period, the FEV1 % predicted score changed by +0.2 percentage points in the group that stopped DNase, and by −0.2 percentage points in the group that continued it. For the secondary outcomes, the reported data shows a lung clearance index score (a measure of how efficiently air moves through the lungs — higher numbers meaning poorer airflow) changed by −0.1 in the discontinue group and 0.0 in the continue group. On a respiratory symptom diary scored from 0 to 100 (where lower scores indicate fewer symptoms), the discontinue group changed by −0.1 points and the continue group by −1.1 points. On a separate respiratory quality-of-life questionnaire also scored 0 to 100 (where higher scores indicate fewer symptoms), the discontinue group changed by −0.9 points and the continue group by 0.0 points. Two additional breathing test measurements at earlier time points in the study showed similarly small numerical differences between the groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03277196 · results posted 23 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03277196) enrolled 86 people who were given ivacaftor, a medicine used in the treatment of cystic fibrosis. Of those 86 participants, 38 had previously been on ivacaftor (described as "rollover participants") and 48 had never taken it before ("ivacaftor-naïve"). The trial was primarily looking at the safety and tolerability of the medicine — in other words, tracking any unwanted or unexpected health events that occurred while participants were taking it. A secondary measurement looked at changes in sweat chloride levels, a marker commonly used to assess how a certain protein is functioning in the body. By the end of the study, 58 participants had completed it, while 28 did not finish. The reported data shows that out of the 85 participants included in the safety analysis (one fewer than started, which may reflect a reporting adjustment not further explained in the data), 85 experienced what are called "treatment-emergent adverse events" — meaning any health event that appeared or worsened after starting the medicine. Of those, 21 participants experienced serious treatment-emergent adverse events. For the secondary measure, the reported data shows an average change in sweat chloride levels of minus 55.3 millimoles per litre (mmol/L), meaning that, on average, sweat chloride levels were lower during treatment compared to the starting point. It is important to note that these numbers describe what was observed and recorded in this particular group of trial participants — they do not on their own tell us whether these changes are good, bad, or meaningful for any individual person. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03265288 · results posted 9 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03265288) enrolled 83 people in the LAU-7b group and 83 people in the placebo group — 166 participants in total. The trial was studying a treatment called LAU-7b in people with cystic fibrosis. The main things being measured were changes in lung function (using a breathing test called FEV1, which measures how much air a person can forcefully breathe out in one second, expressed as a percentage of what would be expected for someone of their age and size) and the occurrence of side effects during the study. The trial ran for up to 28 weeks. The reported data shows that, at 24 weeks, both groups had a small average decline in their lung function score compared to when they started. In the LAU-7b group, the average change at the 24-week mark was approximately −1.18 percentage points, while in the placebo group it was approximately −1.95 percentage points. When averaged across all measurement points up to week 24, the LAU-7b group showed figures ranging from around −0.33 to −1.41 percentage points depending on the time point, while the placebo group ranged from around −1.55 to −4.07 percentage points across the same time points. Regarding side effects, the reported data shows that 74 out of 83 participants in the LAU-7b group and 64 out of 83 in the placebo group experienced at least one treatment-related adverse event (an unwanted health event recorded during the study). For the secondary outcome measuring time to first serious lung flare-up, the data was not reported as a usable number in the submitted results. The reported data also shows results for blood markers related to certain fatty acids (arachidonic acid and docosahexaenoic acid), with 15–25 participants in each group showing what the researchers called "normalisation" of these markers at various points during the trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT06350461 · results posted 1 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT06350461) enrolled 370 people with cystic fibrosis — 184 in a group that stopped using hypertonic saline (a saltwater inhaled therapy, labelled "HS-Discontinue") and 186 in a group that kept using it ("HS-Continue"). The vast majority finished the study: 181 and 183 participants respectively. The trial was primarily measuring whether stopping hypertonic saline made a difference to lung function — specifically a breathing test called FEV1 (how much air a person can forcefully breathe out in one second, expressed as a percentage of what is expected for someone of that age and size) — over six weeks. The reported data shows that for the main outcome (change in FEV1 % predicted from the start to week six), the group that stopped hypertonic saline had an average change of −0.2 percentage points, while the group that continued had an average change of +0.1 percentage points. For the secondary outcomes, a lung ventilation measure called LCI 2.5 (a score showing how efficiently air moves through the lungs — lower is generally considered better) changed by +0.1 in both groups. A respiratory symptom diary score (where lower scores indicate fewer symptoms, on a scale of 0–100) changed by −1.4 in the discontinue group and +0.2 in the continue group. Another symptom questionnaire score (where higher is better, also 0–100) changed by +0.7 and +0.5 respectively. Additional FEV1 measurements at earlier time points showed similarly small changes across both groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04853368 · results posted 16 July 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04853368) enrolled 48 people with cystic fibrosis across three groups (called cohorts). The trial tested different three-drug combinations — involving investigational medicines called galicaftor, navocaftor, ABBV-119, and ABBV-576 — in people with specific genetic types of cystic fibrosis. The study ran in two phases: a lead-in period of about a month where participants took a two-drug combination, followed by a second month where they were assigned to a triple-drug combination or, in one group, a placebo (a dummy treatment with no active ingredient). The trial measured changes in lung function and a marker called sweat chloride — a substance found at higher levels in people with cystic fibrosis, which can reflect how well a key protein involved in the condition is working. The reported data shows that for lung function (measured as the percentage of expected air blown out in one second, or "ppFEV1"), participants in Cohort 1 taking the triple combination with ABBV-119 had an average change of +2.2 percentage points from their starting level, while Cohort 2 participants on the same combination had an average change of +2.6 percentage points. The Cohort 2 placebo group had an average change of −2.0 percentage points. For sweat chloride in Cohort 3 (using ABBV-576), the reported average change from the starting level was a reduction of 24 mmol/L in one genetic subgroup and 40 mmol/L in another — noting that lower sweat chloride levels are associated with greater protein activity. Several other breathing measurements (FVC and FEF25-75) were also reported across groups, with mixed results including some negative values, but full context for interpreting those figures was not provided in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02694393 · results posted 24 June 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT02694393) involved just two participants who inhaled a treatment called nebulized nitrite — meaning a nitrite solution delivered as a fine mist to breathe in. One participant completed the trial and one did not. The trial was measuring lung function and certain markers in breath and mucus (sputum) after the inhalation. The reported data shows that the main (primary) measure was lung function, specifically a breathing test called FEV1 — short for "forced expiratory volume in one second," which is a measure of how much air a person can forcefully breathe out in one second, expressed as a percentage of what would be expected for someone of that age and size. Two FEV1 values were reported for the nebulized nitrite group: 59% and 78% of predicted. For the secondary measures, the concentration of nitric oxide in exhaled breath — a gas that can be detected in the air a person breathes out — was reported as 10 and 12 parts per billion (a measure of concentration). For the other secondary measure, the concentration of nitrite in mucus samples (sputum), no data was reported for either measurement time point. It is worth noting that with only two participants enrolled and one not completing the trial, the reported numbers are extremely limited, and no broader conclusions can be drawn from them. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05076149 · results posted 13 June 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 289 people in the ELX/TEZ/IVA group and 284 people in the VX-121/TEZ/D-IVA group at the start. The trial was comparing two combination medicines being studied for cystic fibrosis. The main thing the trial measured was a lung function test called FEV1 — specifically the amount of air a person can forcefully breathe out in one second — expressed as a percentage of what would be expected for someone of their age, height, and sex. It also measured sweat chloride levels, which is the amount of salt in sweat (a marker often tracked in cystic fibrosis). The reported data shows that, for the primary measure of lung function, both groups had very similar results: people in the ELX/TEZ/IVA group showed an average change of 0.0 percentage points, while those in the VX-121/TEZ/D-IVA group showed an average change of 0.2 percentage points. For sweat chloride levels, the ELX/TEZ/IVA group had an average decrease of 2.3 units (millimoles per litre), compared to a decrease of 5.1 units in the VX-121/TEZ/D-IVA group. When looking at the proportion of participants whose sweat chloride fell below 60 mmol/L (a commonly used threshold), the reported data shows 76.6% in the ELX/TEZ/IVA group and 85.8% in the VX-121/TEZ/D-IVA group. For the stricter threshold of below 30 mmol/L, the figures were 22.5% and 30.5% respectively. These sweat chloride results were pooled with data from a related study. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05033080 · results posted 10 June 2024

  According to the results reported on ClinicalTrials.gov, this trial compared two combination drug regimens in people with cystic fibrosis: one called ELX/TEZ/IVA and another called VX-121/TEZ/D-IVA. Around 202 people were assigned to the first group and 196 to the second group, with the vast majority completing the trial (191 and 184 respectively). The trial's main focus was on measuring lung function — specifically a breathing test called FEV1, which measures how much air a person can forcefully breathe out in one second — and also on sweat chloride levels, a marker commonly used in cystic fibrosis monitoring. The reported data shows that for the main outcome — change in lung function (ppFEV1) — the ELX/TEZ/IVA group had an average change of +0.3 percentage points, while the VX-121/TEZ/D-IVA group had an average change of +0.5 percentage points. For sweat chloride levels, the reported data shows the ELX/TEZ/IVA group had an average change of +0.9 mmol/L (a very small increase), while the VX-121/TEZ/D-IVA group had an average change of −7.5 mmol/L (a decrease). When looking at the proportion of participants who reached a sweat chloride level below 60 mmol/L, the reported figures were 76.6% for the ELX/TEZ/IVA group and 85.8% for the VX-121/TEZ/D-IVA group. For the lower threshold of below 30 mmol/L, the reported figures were 22.5% and 30.5% respectively. These percentage figures were drawn from a pooled analysis that also included data from a separate related study. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04545515 · results posted 8 May 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04545515) enrolled 120 participants, all of whom received a combination treatment referred to as ELX/TEZ/IVA. The trial was set up in two back-to-back phases: one group of 61 participants had previously received a placebo before switching to ELX/TEZ/IVA, while another group of 59 had already been on ELX/TEZ/IVA and continued with it. In total, 110 participants completed the study and 10 did not. The trial was measuring safety and tolerability (whether unwanted health events occurred), as well as changes in two body measurements: sweat chloride levels (a marker measured through a sweat test) and a breathing test score called the Lung Clearance Index (a measure of how evenly air moves in and out of the lungs — lower scores indicate more even airflow). The reported data shows that, for the primary measure of safety and tolerability, 118 out of 120 participants experienced at least one adverse event (an unwanted health occurrence of any kind), and 13 participants experienced a serious adverse event (a more significant unwanted health occurrence). For the secondary measures, the reported data shows changes in sweat chloride from the starting point of the earlier ("parent") study: the group that had previously been on placebo showed an average change of −57.3 mmol/L, and the group that had already been on ELX/TEZ/IVA showed an average change of −57.5 mmol/L. For the Lung Clearance Index, the reported changes from the parent study starting point were −1.74 (placebo-to-treatment group) and −2.35 (continuous treatment group). These numbers represent how much each measure shifted from where participants started in the earlier study; the data does not separately report what portion of any change occurred during this specific trial period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT03525574 · results posted 8 May 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03525574) enrolled 507 participants in the main treatment group (ELX/TEZ/IVA — a triple combination of three medicines: elexacaftor, tezacaftor, and ivacaftor) plus 3 additional participants included for a specific analysis, giving a total of 510 people at the start. The trial was an open-label extension study, meaning all participants received the triple combination medicine. It was measuring safety-related events, lung function, a sweat test marker, and the number of lung flare-ups (called pulmonary exacerbations) over the study period. Of the 507 main participants, 354 completed the study and 153 did not. The reported data shows that, for the primary focus of the trial — tracking unwanted health events — 504 out of 506 participants in the safety group experienced at least one treatment-emergent adverse event (an unexpected health event that occurred during the treatment period), and 175 experienced a serious adverse event (a more significant health event). For lung function, which was measured as the percentage of expected air a person can forcibly breathe out in one second (ppFEV1), the reported changes from the start of the earlier "parent" studies were an increase of around 15.3 and 13.8 percentage points for one group of participants, and around 10.9 and 10.7 percentage points for another group. The sweat chloride test — a marker related to the underlying condition — showed reported reductions of around 47.0 and 45.3 mmol/L (a unit of concentration) in one group, and 48.2 mmol/L in both measurements for another group. The reported data also shows that 174 lung flare-up events were recorded across the relevant participant group during the study period. It is worth noting that some of the outcome data appears to include two separate measurements per group (likely reflecting different starting points or time points within the study), though the trial record does not provide full labels for each individual figure. Where details were not fully specified in the submitted data, this summary reflects only what was reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT03551691 · results posted 30 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 19 participants in total — 9 in the group that received the study drug (omeprazole) first and then a placebo, and 10 in the group that received the placebo first and then the study drug. Thirteen participants completed the trial (6 and 7 from each group respectively), while 6 did not complete it. The trial was measuring how well the body absorbs fat, and whether omeprazole — a medicine that reduces stomach acid — had any effect on that process in participants who have difficulty absorbing fat. The reported data shows the following figures for each outcome measured. For the main (primary) outcome — the proportion of fat the body absorbed, measured using a standard laboratory method — the omeprazole group recorded 80% fat absorption, compared with 77% in the placebo group. For the first secondary outcome, the level of acidity (pH) measured in the upper part of the small intestine was reported as 6.03 for the omeprazole group and 5.38 for the placebo group (a higher pH number means less acidic). For the second secondary outcome — a blood test used as another way to estimate fat absorption — the omeprazole group recorded 7.3 mg\*h/dl and the placebo group recorded 9.6 mg\*h/dl. No further context or statistical analysis details were included in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT01180634 · results posted 30 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 330 people with cystic fibrosis — 220 received an inhaled antibiotic called Aeroquin (levofloxacin) at a dose of 240 mg, and 110 received a placebo (a dummy treatment with no active ingredient). Of those who started, 210 in the Aeroquin group and 109 in the placebo group completed the study. The trial was mainly measuring how long it took for participants to experience a lung flare-up (called an exacerbation — a worsening of respiratory symptoms meeting a set of defined criteria). It also measured changes in lung function, levels of a specific bacteria (*Pseudomonas aeruginosa*) in sputum, and participants' quality of life related to their breathing. The reported data shows that, for the main measure, the median time to a lung flare-up was 58 days in the Aeroquin group and 51.5 days in the placebo group (median meaning the middle point — half of participants reached that point before this time, half after). For the secondary measures, the reported average change in a standard lung function test (FEV1 — how much air someone can breathe out in one second) showed a small increase of 0.08 percentage points in the Aeroquin group compared with 1.49 percentage points in the placebo group. The reported change in bacteria levels in sputum was a small increase of 0.04 units in the Aeroquin group versus a decrease of 0.59 units in the placebo group. On a breathing symptom quality-of-life questionnaire (scored 0–100, where higher means fewer symptoms), the reported average improvement was 4.66 points in the Aeroquin group and 4.94 points in the placebo group. The reported median time until participants needed other antibiotic treatment for their lungs was 59 days in the Aeroquin group and 55 days in the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04596319 · results posted 31 January 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 29 participants across seven groups. The groups were split into "SAD" (single ascending dose — meaning participants received one dose, with different groups receiving increasing amounts) and "MAD" (multiple ascending dose — meaning participants received several doses over time). Some participants in each type received a placebo (an inactive treatment used for comparison). The trial was measuring how often and how severely participants experienced side effects, known as "treatment-emergent adverse events" — that is, any health changes that appeared after receiving the study treatment or placebo. The reported data shows that across the five active-treatment groups, the number of participants who experienced a treatment-emergent adverse event were: 1 out of 3 in Cohort 1, 1 out of 3 in Cohort 2, 1 out of 2 in the Amendment 5 group, 0 out of 3 in Cohort 3, and 5 out of 10 in Cohort 4. Among the placebo groups, 2 out of 3 participants in the SAD placebo group and 4 out of 5 in the MAD placebo group reported such events. The reported data also shows that 1 participant across the active-treatment groups experienced a more severe event, though the breakdown by group for that figure was not fully reported in the submitted data. All 29 participants who started the trial completed it. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04058366 · results posted 16 January 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04058366) enrolled 251 participants who were taking a combination medicine called ELX/TEZ/IVA (elexacaftor/tezacaftor/ivacaftor), a treatment for cystic fibrosis. Of those, 215 completed the study and 36 did not. The trial was measuring safety-related events as its main goal, along with several secondary measures including lung function, sweat chloride levels (a marker used in cystic fibrosis monitoring), and body weight and BMI (a measure of body size based on height and weight). The reported data shows that, for the primary (main) goal — tracking unwanted health events — 241 out of the participants in Part A experienced what are called treatment-emergent adverse events (that is, any health issue that appeared or worsened during the treatment period), and 38 experienced serious adverse events. For the secondary measures, the reported data shows an average increase in lung function (the amount of air blown out forcefully in one second) of around 4.1 percentage points from the starting point of an earlier linked study, at one time point, and 3.7 percentage points at another. Sweat chloride levels changed by approximately −23.0 and −22.6 units (mmol/L) at the two time points measured, meaning the levels went down from the earlier baseline. Body weight increased by an average of 3.6 kg and 2.9 kg respectively, and BMI went up by around 1.15 and 0.83 kg/m² across the two time points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT03460704 · results posted 29 December 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03460704) enrolled 152 people in the Colistimethate Sodium (CMS) group and 135 people in the placebo group, for a total of 287 participants. The trial was measuring how often people with a lung condition called non-cystic fibrosis bronchiectasis experienced "pulmonary exacerbations" — that is, episodes where their symptoms (such as increased cough, more mucus, breathlessness, or fever) worsened significantly and a doctor prescribed antibiotic tablets or injections to treat them. The study tracked how many of these episodes each person had over the course of a year. The reported data shows that the average number of these worsening episodes per year was 0.889 in the CMS group and 0.885 in the placebo group. In plain terms, both groups experienced less than one such episode per person, on average, over the year, and the two numbers were very close to each other. It is worth noting that a portion of participants did not complete the study — 58 people in the CMS group and 46 in the placebo group — though the reasons for not completing were not detailed in the data provided here. No secondary outcome measure data was included in the information provided, so those results cannot be described here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03093974 · results posted 15 November 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03093974) looked at a inhaled medication called Colistimethate Sodium (CMS) compared to an inactive placebo, in people with a lung condition called non-cystic fibrosis bronchiectasis (a condition where the airways are permanently widened and prone to infection). A total of 177 people started in the CMS group and 200 in the placebo group, with 176 and 197 respectively included in the main analysis. The primary thing the trial measured was how often participants had a "pulmonary exacerbation" — that is, a flare-up of lung symptoms (such as increased cough, more or thicker mucus, breathlessness, or fever) serious enough that a doctor prescribed a course of antibiotic tablets or injections. The reported data shows that, on average over a year, people in the CMS group had approximately 0.58 of these flare-ups per year, while people in the placebo group had approximately 0.95 flare-ups per year. No secondary outcome measure data was included in the submitted results, so those figures are not available to report here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03104855 · results posted 12 October 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 10 people in total — 5 with cystic fibrosis (CF) and 5 healthy volunteers without CF. All 10 participants completed the study, with no dropouts. The trial was measuring how the body processes a specially labelled form of vitamin D (called D6-25(OH)D3, a tracking version of vitamin D used to follow its journey through the body). Researchers looked at how quickly the body cleared it from the blood, how long it stayed in the body, and how it was broken down — comparing the CF group to the healthy group. The reported data shows that the main measurement — how fast the body cleared the labelled vitamin D from the blood — was 397 mL/day in the CF group and 342 mL/day in the healthy group. For the secondary measurements: the total amount of vitamin D detected in the blood over time (a measure called "area under the curve") was 58.3 units in the CF group and 67.2 units in the healthy group; the time it took for the level in the blood to reduce by half was 16.2 days (CF group) and 15.8 days (healthy group); and the estimated volume of the body's fluid in which the vitamin D spread was 8.4 litres (CF group) and 7.2 litres (healthy group). Regarding calcium levels in the blood, the reported change from the start of the study to 7 days later was 0.00 mg/dL in the CF group and 0.14 mg/dL in the healthy group. The reported data also shows that the ratio of breakdown products relative to the original vitamin D compound was 0.10 in the CF group and 0.08 in the healthy group. It is worth noting that this was a very small study with only five people in each group, so these numbers reflect a limited snapshot. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗

• NCT04969224 · results posted 3 October 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 82 adults who received a combination treatment called ELX/TEZ/IVA (elexacaftor/tezacaftor/ivacaftor). Of those 82 participants, 80 completed the study and 2 did not. The trial was measuring two main things: how much participants' coughing frequency changed over the course of the study, and whether their daily step count (as a measure of physical activity) changed. The reported data shows that, on average, participants' cough frequency — measured as the number of cough events recorded each day — was reported to have reduced by 91.7% from their starting level when comparing their baseline to the average recorded between weeks 8 and 12 of the study. For the second measure, the reported data shows that participants' average daily step count increased by approximately 638 steps per day over that same period, compared to where they started. It is important to note these are the numbers as submitted to ClinicalTrials.gov, and no comparison group (such as a placebo group) was included in this trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03924947 · results posted 28 September 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03924947) ran in two separate parts and involved a total of 50 participants across both parts (26 in Part 1 and 24 in Part 2). The trial was measuring how well the digestive system absorbed fat and protein — specifically in people taking a pancreatic enzyme replacement called Creon, compared to modified versions of it (referred to as "Creon MP" in Part 1 and "Creon AAPIS" in Part 2). The main thing being measured was the "Coefficient of Fat Absorption" (CFA) — simply put, the percentage of fat from food that the body actually absorbed rather than passing out in the stool. The reported data shows the following primary outcome results for fat absorption: in Part 1, participants taking standard Creon absorbed about 85.5% of dietary fat, while those taking Creon MP absorbed about 84.6%. In Part 2, those taking standard Creon absorbed about 87.7% of dietary fat, and those taking Creon AAPIS absorbed about 88.9%. For the secondary outcomes, protein absorption (measured as the percentage of nitrogen absorbed) was reported at roughly 85% across all four groups. The reported data also shows stool fat figures — the amount of fat passed out in stools — ranging from about 34 to 48 grams across the groups, and total stool weight ranging from roughly 754 to 861 grams across the groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02725567 · results posted 7 September 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT02725567) looked at a medicine called ivacaftor (IVA) in very young children with cystic fibrosis, aged from 1 month up to 24 months old. The trial ran in two parts: Part A was a short-term phase (up to 5 days) involving 19 children aged 3 months to under 24 months, and Part B/A/B was a longer phase (up to 24 weeks) involving 43 children aged 1 month to under 24 months, of whom 40 completed that phase. The trial was primarily measuring how much of the medicine appeared in the children's blood (to understand how their bodies processed it) and tracking any unwanted health events that occurred during treatment. The reported data shows that in Part A, 10 out of 19 participants experienced treatment-emergent adverse events (that is, any health issue that appeared or worsened after starting the medicine), and 1 participant experienced a serious such event. In the longer Part B/A/B phase, 38 out of 43 participants had treatment-emergent adverse events, and 6 had serious ones. The blood concentration measurements of ivacaftor and its breakdown products (called metabolites, M1-IVA and M6-IVA) were recorded at various time points across different age subgroups, with figures ranging widely — for example, from around 129 to 2,880 nanograms per millilitre depending on the subgroup and time point. These numbers reflect how the medicine moved through the body at different ages and doses. The reported data also shows one secondary outcome: in the longer phase, the average change in sweat chloride levels (a marker commonly tracked in cystic fibrosis studies) from the start of the study was reported as minus 62.0 millimoles per litre, meaning sweat chloride levels were, on average, 62.0 units lower at the end compared to the beginning. No further breakdown of this figure by age group was reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT04599465 · results posted 3 August 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04599465) enrolled 69 people with cystic fibrosis, all of whom received a combination treatment called ELX/TEZ/IVA. Of those, 66 completed the study and 3 did not. The trial was measuring changes in blood sugar levels — specifically using a test called an oral glucose tolerance test (OGTT), where a person drinks a sugary drink and their blood sugar is measured two hours later. The study tracked these blood sugar readings over roughly 48 weeks. The reported data shows that, on average, participants' two-hour blood sugar readings after the OGTT dropped by 35 milligrams per deciliter (mg/dL) compared to where they started, measured as an average across week 36 and week 48. For context, a lower number on this test generally means lower blood sugar at that point in time. The reported data also shows that 37.7% of participants showed an improvement in how their blood sugar was categorised by week 48 — for example, moving from a diabetes classification to a lower-concern category, or from an in-between category to a normal range. On a separate safety-related measure, the reported data shows that 67 out of the 69 participants experienced at least one adverse event (an unwanted health experience during the study period), and 6 participants experienced a serious adverse event. The trial results do not allow us to draw conclusions about whether these events were caused by the treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04362761 · results posted 28 July 2023

  According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called ELX/TEZ/IVA (a combination of three medicines). The trial ran in two parts: Part A lasted 48 weeks and Part B lasted 86 weeks. In Part A, 172 people started and 159 completed it. In Part B, 50 people started, though none are recorded as having completed it — this may reflect the trial still being in progress for that group at the time results were submitted, rather than participants dropping out, but the data does not explain this further. The reported data shows that the trial's main focus in both parts was on tracking side effects and any serious medical events that came up during treatment (called "treatment-emergent adverse events" and "serious adverse events" — meaning any health issues that appeared or worsened after starting the medicine). In Part A, out of 172 people who started, 160 experienced at least one adverse event of any kind, and 26 experienced a serious adverse event. In Part B, all 50 participants experienced at least one adverse event of any kind, and 8 experienced a serious adverse event. The reported data does not include details about what those events were or how they were assessed beyond these counts. It is worth noting that this trial was designed primarily to monitor these health events rather than to measure whether the treatment improved a particular symptom, so the numbers above reflect how many people had events recorded — not an assessment of benefit. No secondary outcome measure data was included in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT05111145 · results posted 11 July 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 86 participants, all of whom received a triple-combination treatment referred to as ELX/TEZ/IVA (elexacaftor/tezacaftor/ivacaftor), a medicine used in the context of cystic fibrosis. The trial was measuring safety and tolerability — in other words, it was tracking whether participants experienced any unwanted health events (called adverse events) while taking the treatment, including serious ones. The reported data shows that out of the 86 people who started the trial, 61 participants experienced at least one "treatment-emergent adverse event" — meaning an unwanted health event that appeared or worsened after starting the treatment. Additionally, 4 participants experienced a "serious adverse event," which refers to a more significant health event such as one requiring hospitalisation. It is worth noting that the trial records show zero participants listed as having "completed" the study, though the data does not clearly explain why this is the case. No other outcome measures were reported in the submitted results. It is also important to note that adverse events recorded during a trial are not automatically caused by the treatment — they are simply health events that occurred during the study period and are reported for monitoring purposes. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04043806 · results posted 6 July 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04043806) enrolled 458 participants who were given a combination treatment referred to as ELX/TEZ/IVA. The trial was measuring the safety and tolerability of this treatment — in other words, it was looking at whether participants experienced any unwanted health events while taking it. Of the 458 who started, 412 completed the trial, while 46 did not finish. The reported data shows that the main (primary) outcome being tracked was the number of participants who experienced what are called "treatment-emergent adverse events" (TEAEs) — meaning any unwanted health events that appeared or worsened after starting the treatment — as well as "serious adverse events" (SAEs), which are more severe health events requiring significant medical attention. According to the results reported on ClinicalTrials.gov, 435 participants were reported as experiencing at least one treatment-emergent adverse event, and 75 participants were reported as experiencing at least one serious adverse event. No further breakdown of these numbers — such as what types of events occurred or how often — was available in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04537793 · results posted 28 June 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04537793) tested a combination medicine referred to as ELX/TEZ/IVA in children with cystic fibrosis. The trial ran in two parts: Part A involved 18 participants and lasted 15 days, focusing on how the medicine moved through the body and on tracking any unwanted effects. Part B involved 75 participants (74 of whom completed it) and ran for 24 weeks, continuing to track those same things as well as some measures of how the body was responding. The reported data shows that in Part A, out of 18 participants, 15 experienced what are called treatment-emergent adverse events (that is, any unwanted health event that appeared after starting the medicine), while none experienced a serious adverse event. In Part B, the reported data shows that 74 out of 75 participants experienced treatment-emergent adverse events, and 2 experienced serious adverse events. The trial also measured drug levels in the blood at set time points — these varied depending on which part of the trial and which weight group the child was in, ranging roughly from about 1.85 to 7.68 micrograms per millilitre across the different measurements reported. For Part B, the reported data shows an average change in sweat chloride (a marker commonly tracked in cystic fibrosis) of −57.9 mmol/L from the start of the study to the end, and an average change in a lung function measure called the Lung Clearance Index of −0.83 units (where a lower number means the lungs needed fewer "breath turnovers" to clear a test gas, which is the direction considered more favourable by the index's design). It is important to note that this summary only describes the numbers as submitted — it does not indicate whether these changes are clinically meaningful, and no comparison group (such as a placebo group) was included in the data reported here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03218917 · results posted 8 February 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 256 adults across three groups: 82 received a 10 mg dose of a drug called brensocatib, 87 received a 25 mg dose of brensocatib, and 87 received a placebo (a dummy treatment with no active ingredient). The trial ran for 24 weeks and was mainly measuring how long it took for participants to have their first "pulmonary exacerbation" — that is, a worsening of lung symptoms serious enough for a doctor to prescribe antibiotics. The reported data shows that for the primary measure — time to first lung flare-up — a median number of days could only be calculated for the placebo group (189 days), meaning at least half of the placebo group had experienced a flare-up by that point. The data was reported as "NA" (not available/not reached) for both brensocatib groups, which means that by the end of the 24-week study, fewer than half of participants in those groups had experienced a first flare-up. In terms of raw counts, 42 participants in the placebo group experienced a pulmonary exacerbation, compared with 26 in the 10 mg group and 29 in the 25 mg group. For the secondary measures, scores on a breathing symptom quality-of-life questionnaire (where higher is better) changed by +3.8 points for the 10 mg group, +5.9 for the 25 mg group, and +5.7 for the placebo group. A measure of lung function (how much air someone can breathe out in one second, as a percentage of what is considered normal) changed by −0.3% in both brensocatib groups and −1.8% in the placebo group. A marker of airway inflammation measured in sputum (mucus coughed up from the lungs) decreased by 2.928 units in the 10 mg group, 4.117 units in the 25 mg group, and 1.409 units in the placebo group — with a larger decrease considered a positive sign in this context. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03601637 · results posted 6 January 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03601637) involved 60 participants in total, split into two groups. Fourteen people took part in Part A, which looked at how much of the study medicine (lumacaftor/ivacaftor, often called LUM/IVA) got into the bloodstream in children, comparing two different doses. Forty-six people took part in Part B, which looked at how many participants experienced side effects and also measured a marker in sweat called sweat chloride, which is used in cystic fibrosis research. The overall numbers who completed the trial were 13 out of 14 in Part A, and 43 out of 46 in Part B. The reported data shows that in Part A, blood concentration levels of LUM and IVA were measured at two time points — a few hours after a dose, and just before the next dose. For the lower dose group, LUM levels a few hours after dosing were reported at around 14,600 ng/mL (nanograms per millilitre, a measure of how much of the medicine was in the blood), and for the higher dose group around 12,600 ng/mL. Pre-dose LUM levels were around 12,000 and 12,800 ng/mL respectively. IVA levels followed a similar pattern across the two doses. For Part B's primary measure, the reported data shows that 44 out of 46 participants experienced at least one treatment-emergent adverse event (an unwanted health event that occurred during the study period), and 5 experienced a serious adverse event. In Part A, 12 out of 14 participants had at least one such event, and none had a serious adverse event. The reported data also shows a secondary outcome from Part B: the average change in sweat chloride from the start of the study was reported as −29.1 mmol/L (meaning sweat chloride levels were, on average, 29.1 units lower by the end of the study period). Sweat chloride is a marker measured in cystic fibrosis research, and this figure simply describes the numerical change recorded — it does not on its own indicate anything about whether a treatment should be used. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04353817 · results posted 26 July 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT04353817) enrolled 121 people with cystic fibrosis — 61 received a placebo (an inactive treatment) and 60 received a combination medicine called ELX/TEZ/IVA. The trial was measuring changes in two things: a lung function test called the Lung Clearance Index (LCI2.5), which measures how evenly air moves in and out of the lungs (a lower score generally means more even airflow, with 7.5 or below considered normal), and sweat chloride levels, which reflect how a protein linked to cystic fibrosis is working in the body. The reported data shows that, on average, the LCI2.5 score changed by −0.02 in the placebo group and −2.29 in the ELX/TEZ/IVA group over the course of the study — meaning the ELX/TEZ/IVA group's scores moved further in the downward direction. For sweat chloride levels, the placebo group saw an average change of −0.9 mmol/L (millimoles per litre), while the ELX/TEZ/IVA group saw an average change of −52.1 mmol/L. These are the numbers as submitted; the trial did not report whether these differences met any pre-set statistical threshold in the data provided here. The reported data also includes information on unwanted health events that occurred during the trial. In the placebo group, 57 out of 61 participants experienced at least one treatment-emergent adverse event (an unwanted health event that occurred after starting the study treatment), compared with 48 out of 60 in the ELX/TEZ/IVA group. Serious adverse events — more significant health events — were reported in 9 participants in the placebo group and 4 in the ELX/TEZ/IVA group. These figures describe what was recorded and counted; they do not on their own indicate whether any treatment is safer or riskier than another. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01427712 · results posted 10 June 2022

  According to the results reported on ClinicalTrials.gov, this trial followed 24 participants who were all given a medication called LipaCreon (a pancreatic enzyme replacement treatment). Of those 24 people, 21 had their data collected and included in the safety analysis, and 17 completed the full study period. The trial was observing what unwanted reactions or symptoms participants experienced while taking LipaCreon, and also tracking a range of nutritional measurements — including body mass index (BMI, a measure of body weight relative to height), blood protein levels, albumin (a protein made by the liver), cholesterol, and triglycerides (a type of fat found in the blood) — over a period of up to several years. The reported data shows that, for the primary outcome, 1 out of 21 participants experienced what was recorded as an adverse drug reaction (an unwanted symptom or sign that could not be ruled out as being linked to the medication). For the nutritional measurements taken across multiple time points — before starting treatment, then at 4, 8, and 24 weeks, at 52 weeks, and at yearly intervals through to March 2018 — the reported BMI figures across those time points ranged from approximately 11.8 to 15.8 kg/m². Blood total protein readings ranged from about 6.1 to 8.1 g/dL, albumin readings ranged from roughly 3.6 to 4.4 g/dL, total cholesterol ranged from about 114 to 167 mg/dL, and triglycerides ranged from approximately 73 to 160 mg/dL across the various time points recorded. It is worth noting that the data as submitted does not specify which individual measurement corresponds to which exact time point, so a direct before-and-after comparison cannot be drawn from the figures available. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03746483 · results posted 6 June 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 41 people across two groups (21 in one group and 20 in the other). It was a crossover study, meaning participants tried both treatments at different times — one being MS1819, a yeast-based digestive enzyme being investigated as an alternative to the standard treatment for people who cannot properly absorb fat (a condition often linked to pancreatic disease), and the other being PERT, the standard animal-derived enzyme replacement therapy that participants were already taking before the trial. The trial was measuring how well each treatment helped the body absorb fat from food, as well as tracking any unwanted side effects. The reported data shows that the main measure used was something called the Coefficient of Fat Absorption (CFA%) — essentially, the percentage of dietary fat the body absorbed rather than passing it out in stools. A score above 80% is considered a meaningful threshold by the US Food and Drug Administration. The reported figures show that, on average, participants on MS1819 had a CFA of 55.6%, while those on PERT had a CFA of 86.2%. For stool weight (a secondary measure, where lower weight generally reflects better absorption), MS1819 was associated with an average of 1,394 grams compared to 727.3 grams for PERT. For nitrogen (protein) absorption, MS1819 showed 93.0% compared to 97.2% for PERT. Regarding unwanted events, the reported data shows 13 participants reported one or more adverse events (unexpected health issues) while on MS1819, compared to 6 while on PERT, with a total of 19 such events recorded for MS1819 and 8 for PERT. No serious treatment-related adverse events were reported for either treatment in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03219164 · results posted 16 May 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03219164) enrolled 149 people with cystic fibrosis who had a early infection with a bacteria called *Pseudomonas aeruginosa* (PA). Participants were split into two groups: 74 people received 14 days of the inhaled antibiotic aztreonam lysine for inhalation (AZLI) followed by 14 days of a placebo (an inactive treatment), and 75 people received 28 days of AZLI. The main thing the trial was measuring was how many participants in each group had no detectable PA in their cultures (mucus or airway samples) during the 28 days after treatment finished. The reported data shows that in the 14-day AZLI group, 55.9% of participants had PA-negative cultures (no bacteria detected) through 28 days after treatment. In the 28-day AZLI group, 63.4% of participants had PA-negative cultures over the same period. The trial also compared the 14-day group's result against historical data from a separate study using a different antibiotic (tobramycin), where the reported figure for the 14-day AZLI group was again 55.9%. Among those who initially cleared PA and were then followed for up to about two years to see if the bacteria came back, the reported average time to recurrence in the 14-day group was 19.3 months; for the 28-day group, this figure was either not reached or not reported as a single number in the main analysis, though a sub-group comparison reported 15.2 months for the 28-day group. It is also worth noting that not everyone completed the trial — 25 people in the 14-day group and 18 in the 28-day group did not finish. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02163681 · results posted 19 April 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 45 participants, with 44 completing the study and 1 not completing it. The trial was investigating a specialised type of MRI scan that uses a gas called hyperpolarized helium-3 to take pictures of the lungs. The goal was to see how well air was moving through different parts of the lungs, by looking at how bright or dark areas appeared on the scan. The reported data shows that trained readers examined the MRI images and sorted each participant's lungs into one of three categories: no ventilation defects (areas where air was not flowing well), small defects, or large defects. According to the results reported on ClinicalTrials.gov, 9 participants were assessed as having no defects, 13 were assessed as having small defects, and 16 were assessed as having large defects. It is worth noting that these three groups add up to 38 participants, and the results as submitted do not explain the breakdown for the remaining participants, so that data was not fully reported in the structured results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01694069 · results posted 1 February 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 6 people in total, and all 6 completed the study with no one dropping out. The trial was comparing two different ways of giving the antibiotic piperacillin-tazobactam to people with cystic fibrosis who were experiencing a lung flare-up (called a pulmonary exacerbation): one group received it as an intermittent infusion (given over a short period at regular intervals), while the other received it as a continuous infusion (given slowly and steadily over a longer period). The study was measuring things like lung function, antibiotic levels in the blood, quality of life scores, bacterial levels in sputum (mucus coughed up from the lungs), body weight, and how long it took before the next lung flare-up occurred. The reported data shows that, unfortunately, no numerical results were submitted for any of the outcome measures — not for the primary measure (change in lung function, measured as FEV1, which is the amount of air a person can forcefully breathe out in one second) nor for any of the secondary measures (antibiotic blood levels, time to next flare-up, quality of life scores, bacterial levels in sputum, or weight change). The measurements section for every outcome is empty in the data provided to ClinicalTrials.gov. Because no result figures were reported for either group across any of the planned measures, it is not possible to describe what the numbers showed. The data was simply not reported on ClinicalTrials.gov for this trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03447262 · results posted 25 January 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03447262) enrolled 481 people with cystic fibrosis who all received a triple combination of investigational medicines known as VX-659, tezacaftor, and ivacaftor. The trial was an open-label extension study, meaning participants came from two earlier trials and continued on the triple combination. The primary thing the trial was set up to measure was safety and tolerability — in other words, tracking any unwanted health events (called adverse events) that occurred while participants were taking the medicines. The reported data shows that out of 481 people who started the study, 470 experienced at least one adverse event (an unwanted health occurrence during the study period), and 99 experienced a serious adverse event (a more significant unwanted health occurrence). For the secondary measures — which looked at lung function, a salt-level test in sweat, and episodes of worsening lung symptoms — the reported data shows changes compared to each participant's starting point in their earlier trial. Lung function (measured as the volume of air a person can forcibly breathe out in one second, expressed as a percentage of what would be expected for someone of their age and size) showed reported changes ranging from approximately +10.3 to +15.1 percentage points across the different participant groups. Sweat chloride levels (a marker used in cystic fibrosis monitoring) showed reported changes ranging from approximately −45.7 to −53.5 millimoles per litre. The number of episodes of worsening lung symptoms recorded was 60 in one participant group and 84 in another. It is worth noting that only 2 of the 481 participants were recorded as having formally completed the study, with 479 noted as not completing it; the reasons for this are not detailed in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03911713 · results posted 25 January 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03911713) involved 77 people across five groups. Participants received either ivacaftor (an already-approved medicine used as a comparison) or one of four different doses of an investigational medicine called VX-561 (25 mg, 50 mg, 150 mg, or 250 mg). The trial was measuring changes in lung function, sweat chloride levels (a common marker used in cystic fibrosis monitoring), drug levels in the blood, and the number of unwanted health events that occurred during treatment. The reported data shows that for the main outcome — a breathing test called FEV1, which measures how much air a person can forcibly breathe out in one second — the ivacaftor group showed an average change of −0.8 percentage points, while the VX-561 150 mg group showed a change of +3.1 percentage points and the 250 mg group showed +2.7 percentage points. For sweat chloride levels, the reported changes were +0.9 mmol/L for ivacaftor, +3.3 mmol/L for the 150 mg VX-561 group, and −6.5 mmol/L for the 250 mg group. Regarding unwanted health events, the reported data shows that across all groups, between 4 and 23 participants experienced at least one treatment-emergent adverse event (an unwanted health event that occurred during the treatment period), and between 1 and 2 participants in each group experienced a serious adverse event. Drug concentration levels in the blood were also measured and reported across the dose groups, with higher doses generally associated with higher measured blood levels, though the 25 mg group had no detectable levels for one of the metabolites (breakdown products of the drug). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03227471 · results posted 18 January 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03227471) enrolled a total of 225 participants across six sections, labelled Parts A through F. The trial was testing a drug called VX-445, given either on its own or in combination with other medicines (tezacaftor/ivacaftor, or tezacaftor/VX-561), compared to a placebo (a dummy treatment with no active ingredient). The trial was measuring how many people experienced unwanted health events (called adverse events), as well as changes in a lung function test that measures how much air a person can breathe out forcefully in one second — known as FEV1. The reported data shows that in the earlier parts of the trial (Parts A, B and C), between 0 and 5 participants in each group experienced an adverse event, and no serious adverse events were recorded in most groups. In the later parts (Parts D, E and F), adverse events were reported in 12 out of 12 placebo participants and 49 out of 53 active-treatment participants in Part D; serious adverse events were reported in small numbers across several groups (for example, 2 in the Part D placebo group and 3 in the Part D active-treatment group). Regarding lung function, the reported data shows that participants receiving the active triple combination in Part D had increases in their FEV1 score ranging from 7.9 to 13.8 percentage points depending on the dose, compared to 0.0 percentage points in the placebo group. In Part E, the reported change was 11.0 percentage points for the triple combination versus 0.4 for the two-drug comparison treatment, and in Part F, 11.7 percentage points for the active combination versus 1.2 for placebo. The reported data also shows how much of the drug VX-445 was detected in the bloodstream at its peak across different dose levels in Part A, ranging from 0.398 to 7.07 micrograms per millilitre. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03537651 · results posted 26 November 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03537651) tested a combination treatment called TEZ/IVA (tezacaftor/ivacaftor) in people with cystic fibrosis. A total of 130 participants entered Part A of the trial (lasting up to 96 weeks), and 62 of those went on to Part B (lasting up to 192 weeks). The trial was divided into subgroups based on the specific genetic variants participants had, which is why different numbers appear across different measurements. The main thing being tracked in Part A was how many participants experienced adverse events (unexpected health events that occurred during treatment) and serious adverse events. The reported data shows that in Part A, 129 out of 130 participants experienced at least one treatment-emergent adverse event, and 31 experienced a serious adverse event. For the secondary measurements, the trial tracked changes in a lung function test called the Lung Clearance Index (LCI), where lower numbers are better — a score of 7.5 or below is considered normal. The reported data shows an average decrease (improvement in the index) of 0.95 points in one subgroup and 2.04 points in another subgroup. Sweat chloride levels, which are used as a marker in cystic fibrosis, were reported to decrease by an average of 13.8 mmol/L in one subgroup and 16.2 mmol/L in another. Participants also filled out a quality-of-life questionnaire focused on breathing symptoms (scored 0–100, with higher being better), and the reported average change was an increase of 6.4 points. No outcome data was reported for Part B in this submission. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03691779 · results posted 22 October 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03691779) looked at a combination medicine made up of three components — elexacaftor, tezacaftor, and ivacaftor (referred to together as ELX/TEZ/IVA) — and was run in two separate parts. Part A enrolled 16 participants and focused on measuring how the medicine and its breakdown products moved through the body over a 15-day period, including how much of the medicine reached the bloodstream and how it was processed. Part B enrolled 66 participants (64 of whom completed it) and ran for 24 weeks, with its main focus on tracking unwanted or unexpected health events that occurred while taking the medicine. The reported data shows that in Part A, the highest recorded levels of the three active components in the blood (a measure called peak concentration) were 6.13, 6.93, and 1.01 micrograms per millilitre respectively. The levels present in the blood just before each dose (a measure of how much remained overnight) were 2.86, 1.06, and 0.297 micrograms per millilitre. A measure of total drug exposure over a 24-hour period came in at 107, 58.4, and 8.12 hour-micrograms per millilitre for each component. Similar blood-level figures were also reported for the breakdown products the body creates from each component. For Part B, the reported data shows that 65 out of 66 participants experienced at least one treatment-emergent adverse event (an unwanted health event that occurred after starting the medicine), and 1 participant experienced a serious adverse event. It is worth noting that the data as submitted does not include details about what those adverse events were, how severe they were, or what happened as a result, so those specifics were not reported in the structured results available here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03445793 · results posted 1 October 2021

  According to the results reported on ClinicalTrials.gov, this was a small observational study (meaning researchers watched and recorded what happened rather than randomly assigning treatments) involving 5 participants, all of whom completed the study. The trial was looking at what happened when people with cystic fibrosis switched from one medication combination (lumacaftor/ivacaftor) to another (tezacaftor/ivacaftor). It tracked several measurements over 6 months, including a chemical in sweat called sweat chloride (which reflects how well a particular protein involved in cystic fibrosis is working), lung function, quality of life related to breathing, and the number of chest infections requiring antibiotics. The reported data shows that, on average, sweat chloride levels changed by 10.2 mmol/L (millimoles per litre) from the starting point to 6 months. Lung function — measured as the percentage of expected airflow a person can breathe out in one second — changed by an average of minus 2.0 percentage points over the same period. A quality-of-life score focused on breathing symptoms (where 0 is the worst and 100 is the best) changed by an average of minus 11.3 points. The reported data also shows an average of 3.2 chest infections requiring antibiotics per participant during the study period. Regarding the reasons doctors and patients gave for switching medications, questionnaire results were reported as participant counts across different response categories, though the specific category labels were not included in the submitted data, so the full context of those responses cannot be described here. It is worth noting that with only 5 participants, this was an extremely small study, and the reported figures reflect averages across a very limited group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04105972 · results posted 18 August 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT04105972) involved 175 people with cystic fibrosis. Participants were split into two groups: 88 people received a combination called TEZ/IVA (tezacaftor/ivacaftor), and 87 received a combination called ELX/TEZ/IVA (elexacaftor/tezacaftor/ivacaftor). The trial was primarily measuring changes in a questionnaire called the CFQ-R, which asks people with cystic fibrosis to rate their respiratory (breathing-related) symptoms on a scale of 0 to 100, where higher scores mean fewer symptoms. The trial also looked at changes in lung function and sweat chloride levels (a marker commonly measured in cystic fibrosis). The reported data shows that, on the CFQ-R respiratory symptom score, the TEZ/IVA group had an average change of +1.2 points, while the ELX/TEZ/IVA group had an average change of +17.1 points. For lung function — specifically how much air a person can forcefully breathe out in one second (FEV1) — the reported change was +1.0 percentage points for the TEZ/IVA group and +11.2 percentage points for the ELX/TEZ/IVA group. For sweat chloride levels, the TEZ/IVA group had an average change of −3.4 millimoles per litre, and the ELX/TEZ/IVA group had an average change of −46.2 millimoles per litre. Regarding side effects, the reported data shows that 81 participants in the TEZ/IVA group and 77 in the ELX/TEZ/IVA group experienced treatment-emergent adverse events (unexpected health events that occurred during the trial). Serious adverse events were reported in 14 participants in the TEZ/IVA group and 5 in the ELX/TEZ/IVA group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02709109 · results posted 29 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT02709109) enrolled 142 people across four groups. It was a crossover study, meaning participants tried more than one treatment in sequence, with a "washout" rest period in between. The four sequences tested were: VX-371 (an investigational inhaled drug) combined with hypertonic saline (a saltwater solution); hypertonic saline alone; VX-371 combined with an inactive placebo inhaler; and placebo alone. Each treatment period lasted 28 days. The trial's two main goals were to record how many participants experienced unexpected medical events (called adverse events) during the study, and to measure any change in lung function using a standard breathing test called FEV1 — which measures how much air a person can forcefully breathe out in one second. The reported data shows that, for adverse events, the numbers of participants who experienced at least one treatment-emergent adverse event were: 65 out of those who received VX-371 plus hypertonic saline; 66 out of those who received hypertonic saline alone; 32 out of those who received VX-371 plus placebo; and 28 out of those who received placebo alone. For serious adverse events, the reported figures were 8, 4, 6, and 3 participants respectively across those same groups. For the lung function measure (reported as the change in "percent predicted FEV1" from the start of the study to Day 28), the reported changes were very small across all groups: +0.1 percentage points for VX-371 plus hypertonic saline, −0.1 for hypertonic saline alone, −0.8 for VX-371 plus placebo, and +0.8 for placebo alone. The reported data also shows that VX-371 was detectable at low levels in participants' blood and urine during the treatment periods, with concentration figures varying across different time points and between the two VX-371 groups. These concentration measurements were reported in very small units (picograms per millilitre). The trial did not appear to report whether these blood and urine concentration differences between groups were considered meaningful, so no further interpretation of those figures is available from the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04058353 · results posted 2 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT04058353) enrolled 258 people with cystic fibrosis — 126 in a control group who continued taking an existing cystic fibrosis medicine (either ivacaftor alone or tezacaftor/ivacaftor), and 132 in a group who switched to a triple-drug combination called elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA). The trial was measuring changes in three things: lung function (specifically how much air someone can forcefully breathe out in one second, expressed as a percentage of what is expected for a healthy person of similar age and size — called ppFEV1), a sweat chloride level (a marker commonly measured in cystic fibrosis, where salt concentration in sweat is tested), and a self-reported quality-of-life score focused on breathing symptoms (scored from 0 to 100, where higher means fewer symptoms). The reported data shows that in the triple combination group, ppFEV1 changed by an average of +3.7 percentage points, compared to +0.2 percentage points in the control group. For sweat chloride, the triple combination group saw an average change of −22.3 mmol/L (a fall in the level), while the control group saw a change of +0.7 mmol/L. On the breathing symptom quality-of-life questionnaire, the triple combination group reported an average change of +10.3 points on the 0–100 scale, compared to +1.6 points in the control group. These are the numbers as submitted by the trial sponsor; no other outcome data was reported in the structured results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03566550 · results posted 5 April 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03566550) looked at how food moves through the digestive system in people with cystic fibrosis compared to people without the condition. It used MRI (medical imaging) scans taken at several points after eating to track things like how quickly the stomach empties, how much water is present in the small bowel, and how long it takes for food to reach the large bowel. A total of 12 people with cystic fibrosis and 13 people without (the control group) started the study; all 12 in the cystic fibrosis group finished, and 12 of the 13 in the control group finished. The reported data shows the following numbers across the two groups. For the main measurement — the time for food to travel from the mouth to the start of the large bowel — the cystic fibrosis group recorded an average of 330 minutes, compared to 210 minutes in the control group. For stomach emptying (the time for half the stomach contents to move on), the reported figures were 97 minutes for the cystic fibrosis group and 80 minutes for the control group. The reported data also shows that the amount of water measured in the small bowel (adjusted for body size, expressed as a combined value across all time points) was 62 units in the cystic fibrosis group versus 34 units in the control group, and the measured colon volume (similarly adjusted) was 186 units versus 123 units. A gut symptom questionnaire scored the cystic fibrosis group at an average of 16 out of 100 and the control group at 7 out of 100, where a higher number indicates more frequent or severe symptoms. One additional measurement (water content in a specific part of the large bowel) was listed but no figures were reported for it. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02677701 · results posted 1 March 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT02677701) involved people with cystic fibrosis who were also taking an inhaled antibiotic called tobramycin. A total of 62 people were assigned to receive azithromycin (an additional antibiotic taken by mouth) and 57 were assigned to receive a placebo (a dummy pill with no active ingredient). Of those, 56 in the azithromycin group and 52 in the placebo group completed the trial. The main thing the trial was measuring was whether lung function — specifically a breathing test result called FEV1, which measures how much air someone can forcefully breathe out in one second — changed over the course of treatment. The reported data shows that, for the primary (main) outcome, the azithromycin group had an average relative change in lung function of +1.69%, while the placebo group had an average relative change of −1.95% over the study period. For a secondary (additional) outcome measuring lung function change only during the tobramycin treatment weeks, the azithromycin group showed a change of +0.44% compared to −0.91% in the placebo group. The reported data also shows results from two symptom questionnaires. On one scale (where lower scores mean fewer symptoms), the azithromycin group's average score changed by −2.3 points and the placebo group's by +0.6 points. On another scale (where higher scores mean fewer symptoms), the azithromycin group changed by +1.0 points and the placebo group by −0.5 points. A separate pre-specified measurement tracked bacteria levels in sputum (mucus coughed up); the azithromycin group showed a change of +0.3 units on a logarithmic scale, while the placebo group showed −0.5 units — though the data does not include further context to explain this difference. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02730793 · results posted 12 January 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT02730793) enrolled a total of 5 participants, split into two groups: 2 people received "Standard Therapy" and 3 people received "Study Therapy." The trial was designed to look at lung flare-ups (called pulmonary exacerbations) in people with cystic fibrosis — specifically, episodes serious enough to need intravenous (IV, meaning delivered directly into a vein) antibiotics. It also set out to measure things like time until a first serious flare-up, sinus and nasal quality of life, overall cystic fibrosis quality of life, lung function, and changes in bacteria found in sputum (mucus coughed up) and nasal swabs, all tracked over roughly 140–168 days. The reported data shows that of the 5 participants who started the trial, 2 in the Standard Therapy group and 1 in the Study Therapy group completed it; 2 participants in the Study Therapy group did not complete the trial. The reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of the outcome measures — this includes the primary outcome (number of serious lung flare-ups) and all six secondary outcomes (time to first flare-up, sinus and nasal quality of life scores, cystic fibrosis quality of life scores, lung function measurements, and bacteria culture results). In other words, the actual measurement data was not reported. Given that only 5 people were enrolled and results were not submitted for any outcome, it is not possible to draw any conclusions from this trial's data as recorded on ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01140451 · results posted 19 October 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT01140451) enrolled 191 participants — 95 in the group that received ataluren throughout the study ("Ataluren/Ataluren"), and 96 who started on placebo before switching to ataluren ("Placebo/Ataluren"). The trial was an extension study that followed participants for up to 96 weeks (roughly two years). By the end, 57 participants in the first group and 54 in the second group had completed the full study period. The trial was primarily measuring how many participants experienced medical events or abnormal laboratory test results while taking the study drug, and it also tracked a lung function test called FEV1 — a measure of how much air a person can breathe out in one second, expressed as a percentage of what would normally be expected for someone of that height and age. The reported data shows that essentially all participants in both groups experienced at least one treatment-emergent adverse event (any medical occurrence recorded during the study period) — 100% in the Ataluren/Ataluren group and 97.9% in the Placebo/Ataluren group. Serious adverse events were reported in 14.7% and 18.8% of participants respectively. Regarding laboratory abnormalities (unusual blood or urine test results), the reported data shows these occurred in 13 participants in the first group and 18 in the second group for one category of tests, with higher numbers across other test categories. For lung function, the Ataluren/Ataluren group started with a baseline FEV1 of about 60.6% of predicted, and by week 96 this had changed by approximately −3.1%. The Placebo/Ataluren group started at about 56.5% and showed a change of approximately −2.1% by week 96. In a separate analysis looking only at those who completed the full 96 weeks, the reported change from baseline was −1.63% for the Ataluren/Ataluren group and −5.13% for the Placebo/Ataluren group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00630812 · results posted 9 October 2020

  According to the results reported on ClinicalTrials.gov, this trial involved people with cystic fibrosis and compared two groups: one taking mannitol 400mg and one taking a control (an inactive comparison treatment). A total of 318 people entered the randomisation stage — 192 in the mannitol group and 126 in the control group — and 305 moved into the double-blind treatment period (where neither participants nor researchers knew who was receiving which treatment). Of those, 153 in the mannitol group and 107 in the control group completed the full 26-week treatment period. The trial's main focus was on measuring lung function, specifically a breathing test called FEV1 (the amount of air a person can forcefully breathe out in one second), as well as lung flare-ups (called pulmonary exacerbations), hospital stays, and antibiotic use. The reported data shows that for the main measure — the average change in FEV1 (in millilitres) from the start of the trial over 26 weeks — the mannitol group showed a reported average increase of approximately 107 mL, while the control group showed a reported average increase of approximately 52 mL. Among the subset of participants who were also using a separate inhaled medication called dornase, the reported figures were approximately 79 mL for the mannitol group and 35 mL for the control group. For the secondary measure of FEV1 as a percentage of what would be predicted for a healthy person of similar age and size, the mannitol group showed a reported change of 3.14 percentage points compared with 0.72 percentage points in the control group, at 26 weeks. The reported data also shows counts of lung flare-ups, hospital stays, and antibiotic courses over the 26-week period, broken down by how many participants experienced zero, one, two, or three such events in each group. For example, for protocol-defined lung flare-ups, 156 participants in the mannitol group and 98 in the control group reported having none, while 21 in the mannitol group and 18 in the control group reported having one. Similar breakdowns were reported for hospitalisations and antibiotic use, though the data as submitted does not include a single summary rate figure that would allow straightforward comparison between groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02134353 · results posted 3 September 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 423 adults with cystic fibrosis — 209 in the experimental arm (inhaled mannitol) and 214 in the control arm. The main thing the trial was measuring was the change in a lung function test called FEV1 (the amount of air a person can forcefully breathe out in one second, measured in millilitres) over 26 weeks. A number of secondary measurements were also taken, including another lung function measure called FVC (the total amount of air breathed out forcefully), as well as information about lung flare-ups (called pulmonary exacerbations), antibiotic use, and hospital days. The reported data shows that, on average, the experimental arm's FEV1 changed by plus 63 mL from their starting point, while the control arm's FEV1 changed by plus 8 mL from their starting point — these figures represent averages across measurements taken at 6, 14, and 26 weeks. For the secondary lung function measure (FVC), the reported data shows the experimental arm averaged a change of plus 28 mL, while the control arm averaged a change of minus 12 mL. For antibiotic days due to flare-ups, the reported averages were 8.1 days in the experimental arm and 5.5 days in the control arm. For the measure of time to first flare-up, no numerical results were reported in the data. The hospital days figures were reported as counts of participants across several categories, but the data as submitted does not include enough labelling to describe those numbers plainly without risk of misrepresentation, so that detail has not been summarised here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02951182 · results posted 28 August 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02951182) looked at a triple combination of investigational medicines being tested in people with cystic fibrosis. A total of 73 participants took part across two parts of the study and several treatment groups, including groups receiving different doses of a triple combination therapy, a two-drug combination (tezacaftor/ivacaftor, or TEZ/IVA), and a placebo (a dummy treatment with no active ingredient). Every participant who started the trial also completed it. The trial was primarily measuring how many participants experienced unwanted health events (called adverse events) after taking the medicines, and also tracking changes in a lung function test called FEV1 — which measures how much air a person can forcefully breathe out in one second. The reported data shows that for the primary safety measure, adverse events were reported in 9 out of 11 placebo participants, all 9 participants in the Cohort 1A triple combination group, all 9 in the low-dose triple combination group, 15 out of 18 in the high-dose triple combination group, all 6 in the TEZ/IVA group, and 15 out of 20 in the Part 2 triple combination group. Serious adverse events (more significant health concerns) were reported in 0 placebo participants, 2 in the high-dose triple combination group, 2 in the TEZ/IVA group, and 1 in the Part 2 triple combination group. For the lung function measure, the placebo group showed an average change of +1.4 percentage points, the pooled low-dose triple combination groups showed +10.0, the high-dose triple combination showed +12.0, the TEZ/IVA group showed −2.5, and the Part 2 triple combination group showed +9.5 percentage points. The reported data also shows changes in sweat chloride levels (a marker used in cystic fibrosis monitoring) and scores on a quality-of-life questionnaire focused on breathing symptoms (scored 0–100, where higher means fewer symptoms). The placebo group's sweat chloride changed by +1.6 mmol/L on average, while the triple combination groups showed reductions of −20.7 and −33.1 mmol/L, and the Part 2 triple combination showed −31.3 mmol/L; the TEZ/IVA group showed a change of +2.1 mmol/L. For the quality-of-life score, the placebo group changed by +2.2 points, the pooled low-dose triple combination groups by +18.3, the high-dose triple combination by +20.7, TEZ/IVA by −7.8, and the Part 2 triple combination by +12.3 points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02712983 · results posted 14 August 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02712983) enrolled 107 participants across three groups (called Cohorts A, B and C), each testing different doses or dosing schedules of an inhaled antibiotic called TIP (tobramycin inhalation powder). Within each cohort, participants were randomly assigned to receive TIP alone, a mix of TIP and placebo capsules (dummy medicine), or placebo only. The trial was primarily measuring whether TIP changed the amount of a lung bacteria called *Pseudomonas aeruginosa* in participants' sputum (phlegm) over 29 days, reported as a laboratory unit called log10 CFU — essentially a score representing how much bacteria was present. Secondary measures included how often participants had lung flare-ups (called pulmonary exacerbations), how long those flare-ups lasted, and the rate at which they occurred over the study period. The reported data shows that, at the start of the study, bacteria levels across all groups ranged from roughly 5.73 to 7.67 log10 CFU. By Day 29, the reported change from baseline in bacteria levels for the pooled TIP group was −2.04 log10 CFU (meaning the score went down by that amount), compared with data not fully reported for all comparison groups in the submitted results. For lung flare-ups, the reported data shows that in the pooled groups, 29 out of 44 TIP participants, 27 out of 42 TIP/placebo participants, and 11 out of 21 placebo participants experienced at least one flare-up during the study. The reported rate of flare-ups per year of study time was 1.21 for the pooled TIP group, 1.35 for the pooled TIP/placebo group, and 1.41 for the pooled placebo group. The reported average duration of flare-ups was 19.0 days (pooled TIP), 15.2 days (pooled TIP/placebo), and 14.5 days (pooled placebo). For time to first flare-up, the data was not reported for most groups except pooled placebo, where the figure was 173 days. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03125395 · results posted 7 August 2020

  According to the results reported on ClinicalTrials.gov, this trial involved a single group of participants who received a combination treatment called lumacaftor/ivacaftor (LUM/IVA). Around 57–60 participants were enrolled across two related study sets, 47 completed the trial, and 10 did not finish. The trial was measuring safety (by counting how many participants experienced unwanted health events) as well as a range of physical measurements including sweat chloride levels, body weight, and body mass index (BMI — a measure that relates a person's weight to their height). The reported data shows that, for the primary (main) outcome — safety — 56 out of participants experienced what are called treatment-emergent adverse events (that is, any unwanted health event that occurred during the treatment period), and 15 experienced serious adverse events (more significant unwanted health events). For the secondary (additional) outcomes, the reported data shows an average change in sweat chloride of –29.6 millimoles per litre (a reduction), an average increase in weight of 6.0 kilograms, and an average increase in BMI of 0.30 kg/m². The trial also reported changes in age-adjusted scores for both weight and BMI (these scores compare a child's measurements to what is typical for their age) — weight-for-age increased by an average of 0.23 and BMI-for-age increased by an average of 0.27 on these scales. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02565914 · results posted 16 June 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02565914) looked at a combination treatment referred to as TEZ/IVA, which is used in the context of cystic fibrosis. The trial ran in three separate parts (Part A, Part B, and Part C). Part A enrolled 1,044 participants, Part B enrolled 464, and Part C enrolled 204. The main thing the trial was set up to measure was how many participants experienced unwanted health events — called adverse events (unexpected or unwanted medical occurrences) and serious adverse events (more significant medical occurrences) — while taking the treatment. The reported data shows that in Part A, out of 1,042 participants included in the safety analysis, 995 experienced at least one adverse event and 351 experienced at least one serious adverse event. In Part B, out of 463 participants in the safety analysis, 427 had at least one adverse event and 136 had at least one serious adverse event. In Part C, out of 204 participants, 168 had at least one adverse event and 44 had at least one serious adverse event. These numbers describe how often events were recorded — they do not on their own tell us whether the treatment caused them. The trial also measured changes in lung function, specifically in a breathing test called FEV1 (how much air a person can forcefully breathe out in one second). The reported data shows that, depending on which previous study participants had come from and what treatment they had received before, the change in this lung-function measure during Part A ranged from around 2.0 percentage points up to 7.5 percentage points. The data was reported across several sub-groups, and the figures varied between those groups; no lung-function data was reported for Parts B or C in the results provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03525444 · results posted 19 May 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03525444) enrolled 403 people in total — 203 in the placebo group and 200 in the group receiving the triple combination treatment called VX-445/TEZ/IVA (also known as elexacaftor/tezacaftor/ivacaftor). The trial was measuring several things in people with cystic fibrosis, including how well their lungs were working, how often they had breathing flare-ups (called pulmonary exacerbations), a chemical in their sweat called sweat chloride (which is a marker related to cystic fibrosis), their quality of life related to breathing symptoms, and their body mass index (BMI, a measure of weight relative to height). The reported data shows the following changes from the start of the trial to its end. For the main measure — lung function, specifically how much air a person can forcefully breathe out in one second (expressed as a percentage of what would be expected for someone of that age and size) — the placebo group showed a change of −0.2 percentage points, while the treatment group showed a change of +13.6 percentage points. A similar pattern was seen in a related secondary lung function measure (−0.4 versus +13.9 percentage points). The reported data also shows that the placebo group had 113 breathing flare-up events during the trial, compared with 41 in the treatment group. For sweat chloride levels, the placebo group changed by −0.4 mmol/L and the treatment group changed by −42.2 mmol/L. On a breathing-related quality-of-life questionnaire (scored 0–100, where higher means fewer symptoms), the placebo group's score changed by −2.7 points and the treatment group's score changed by +17.5 points. Finally, BMI changed by +0.09 kg/m² in the placebo group and +1.13 kg/m² in the treatment group. These figures are the numbers submitted to ClinicalTrials.gov and describe what was measured and recorded during this specific trial, in this specific group of participants, under the conditions of that study. No conclusions should be drawn about what these numbers might mean for any individual person reading this summary. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02139306 · results posted 14 May 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 279 people with cystic fibrosis — 140 in the ataluren group and 139 in the placebo (dummy treatment) group. The main thing the trial was measuring was a lung function test called FEV1, which captures how much air a person can forcibly breathe out in one second. This was expressed as a percentage of what would normally be expected for someone of that age, height, and sex. The trial also tracked how often participants had chest flare-ups (called pulmonary exacerbations), scores on a cystic fibrosis-specific quality-of-life questionnaire focused on breathing symptoms, changes in body mass index (a measure of body weight relative to height), and the number of participants who experienced unwanted health events during the study. The reported data shows that for the main lung function measure, the ataluren group had an average change of −1.396 percentage points from their starting level over 48 weeks, while the placebo group had an average change of −1.992 percentage points — meaning both groups showed a small decline. For chest flare-ups, the reported rate was 0.95 events per 48 weeks in the ataluren group compared with 1.127 in the placebo group. The breathing symptoms questionnaire score (where higher means better) changed by −0.760 points in the ataluren group and −1.032 in the placebo group. Body mass index changed by +0.296 kg/m² in the ataluren group and +0.361 kg/m² in the placebo group. Regarding unwanted health events, 133 of 140 participants in the ataluren group and 135 of 139 in the placebo group experienced at least one such event during the study; serious unwanted health events were reported in 40 participants taking ataluren and 46 taking placebo. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02456103 · results posted 15 April 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02456103) enrolled 246 participants, all of whom received the study drug ataluren. The trial was designed for people with cystic fibrosis caused by a specific type of genetic change called a nonsense mutation. The study ran for 48 weeks and was measuring things like unwanted medical events that occurred during treatment, changes in blood and urine test results, and how well participants' lungs were working over time. The reported data shows that out of 245 participants in the "as-treated" group, 222 experienced at least one unwanted medical event (called a treatment-emergent adverse event) during the study period. Of those, 147 had events that were considered serious — meaning they led to hospitalisation, were life-threatening, caused significant disability, or resulted in death. Separately, no participants were reported to have had a clinically meaningful abnormal result in their blood or urine laboratory tests. For lung function, the reported data shows that participants' average lung function (measured as a percentage of what would be expected for someone of their age, height, and sex) was around 60% at the start of the study, with a reported change of approximately 0.015 percentage points at week 24. Similar small changes were reported for two other lung function measures. The rate of lung flare-ups (pulmonary exacerbations) over the 48-week period was reported as approximately 1.05 per person. It is worth noting that the data shows zero participants were recorded as having "completed" the trial in the usual sense, and the full context behind some of the individual numbers was not broken down further in the submitted data. Where additional detail was not provided, it has not been assumed here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03447249 · results posted 13 March 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03447249) enrolled 382 people in total — 190 in the placebo group and 192 in the group receiving the triple combination treatment called VX-659/TEZ/IVA (also referred to as the "TC" group). The trial was measuring several things in people with cystic fibrosis, including how well their lungs could push out air in one second (a standard lung function test called FEV1), how often they had serious flare-ups of lung symptoms, the level of salt (chloride) in their sweat, their quality of life related to breathing, and their body weight relative to height (BMI). The reported data shows that, for the main measurement — lung function expressed as a percentage of what would be expected for a healthy person of the same age and size — the placebo group's score changed by −1.0 percentage points, while the VX-659/TEZ/IVA group's score changed by +13.0 percentage points. For the secondary lung function measurement (assessed at a different time point), the reported figures were −0.8 and +13.4 percentage points respectively. The reported data also shows that the number of serious lung flare-up events recorded during the trial was 116 in the placebo group compared with 17 in the treatment group. For sweat chloride — a marker often used in cystic fibrosis — the placebo group's level changed by −0.1 mmol/L, while the treatment group's level changed by −44.6 mmol/L. On the breathing-related quality-of-life questionnaire (scored from 0 to 100, with higher scores meaning fewer symptoms), the placebo group's score changed by −1.5 points and the treatment group's by +18.6 points. Finally, for BMI, the placebo group changed by −0.05 kg/m² and the treatment group by +1.06 kg/m². These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03559062 · results posted 11 February 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03559062) enrolled 67 people in total across three groups: 10 received a placebo (a dummy treatment), 54 received a combination called TEZ/IVA (tezacaftor/ivacaftor), and 3 received ivacaftor alone. The trial was looking at a lung function measure called the Lung Clearance Index (LCI2.5) — essentially a test of how efficiently air moves through the lungs, where a lower number generally reflects more even airflow. It also looked at sweat chloride levels (a common marker measured in cystic fibrosis), a quality-of-life questionnaire focused on breathing symptoms, and the number of people who experienced side effects. All measurements were taken over 8 weeks. The reported data shows that, in the TEZ/IVA group, the average LCI2.5 score changed by −0.51 units from the start to week 8. Sweat chloride levels changed by an average of −12.3 mmol/L (millimoles per litre) over the same period. Scores on the respiratory symptoms questionnaire — which runs from 0 to 100, with higher scores meaning fewer symptoms — changed by an average of +2.3 points in the TEZ/IVA group. These figures represent average changes from each participant's starting point; the data for the placebo and ivacaftor-only groups was not reported for these outcome measures. The reported data shows that, when it came to side effects (called adverse events), 8 out of 10 people in the placebo group, 41 out of 54 in the TEZ/IVA group, and 2 out of 3 in the ivacaftor group experienced at least one treatment-emergent adverse event. No serious adverse events were reported in any of the three groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03633526 · results posted 5 February 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03633526) enrolled 16 participants, all of whom completed the study with no drop-outs. All 16 participants received a combination of three investigational medicines referred to as VX-659, tezacaftor (TEZ), and ivacaftor (IVA). The trial was primarily focused on measuring how these medicines were absorbed into the bloodstream — specifically, how much of each drug was present in the blood at its peak level, just before the next dose was due, and over a six-hour window after dosing. The reported data shows the following peak blood concentration levels (Cmax) for the three main drugs: VX-659 reached values of 1.81 and 2.55 micrograms per millilitre (mcg/mL) across measurement points, TEZ reached 4.53 and 5.22 mcg/mL, and IVA reached 0.536 and 0.733 mcg/mL. For the pre-dose (trough) levels — that is, how much remained in the blood just before the next dose — VX-659 was 0.358 and 0.367 mcg/mL, TEZ was 0.897 and 0.740 mcg/mL, and IVA was 0.289 and 0.283 mcg/mL. The six-hour exposure figures (a measure of total drug in the blood over that period) were 5.41 and 8.55 for VX-659, 15.5 and 19.3 for TEZ, and 1.64 and 2.95 for IVA. The trial also measured breakdown products (metabolites) of TEZ and IVA in the blood, with similar concentration and exposure figures reported for those as well. It is worth noting that the data as submitted does not clearly label which specific measurement point or time period each pair of numbers corresponds to, so the reported figures above reflect the values as listed without further distinction. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00789867 · results posted 18 January 2020

  According to the results reported on ClinicalTrials.gov, this trial involved 35 people in total, split across three groups based on how much of the study treatment (pGM169/GL67A) they received — 17 people received the 20 ml dose, 10 received the 10 ml dose, and 8 received the 5 ml dose. All 35 participants completed the trial. The trial was measuring several body responses after receiving the treatment, including body temperature, levels of certain white blood cells in the blood (leukocytes and neutrophils), and breathing-related measurements — specifically how much air a person could breathe out and how evenly air moved through the lungs. The reported data shows the following results across the three dose groups. For maximum body temperature, the figures were 38.6°C in the 20 ml group, 38.0°C in the 10 ml group, and 37.4°C in the 5 ml group. White blood cell counts (leukocytes) were reported as 15.8, 14.1, and 12.8 (in billions of cells per litre), and neutrophil counts — another type of white blood cell — were 13.9, 11.3, and 9.8 respectively. For breathing measurements, the reported drops in how much air participants could forcefully breathe out (FEV1) were 24.6%, 17.5%, and 16.8% across the three groups, while drops in total breath capacity (FVC) were 20.7%, 13.7%, and 14.7%. A measure of how evenly air moves through the lungs (Lung Clearance Index) showed changes of 0.75, 0.32, and 0.32 across the groups from highest to lowest dose. It is worth noting that the reported data does not include information about what was considered a normal or expected range for these measurements, nor does it include detail about how these figures compared to participants' starting points before receiving the treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03068312 · results posted 6 January 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03068312) involved 38 people in total, split into two groups of 19. It was a crossover study, meaning everyone took both ivacaftor (a cystic fibrosis medication) and a placebo (a dummy treatment with no active ingredient) at different times during the trial — one group took ivacaftor first, the other took placebo first. Importantly, all 38 participants completed the study with no drop-outs reported. The main thing the trial was measuring was a lung function test called the Lung Clearance Index 2.5 (LCI2.5). In plain terms, this is a measure of how evenly air moves in and out of the lungs — generally, a lower number suggests more even airflow distribution. The reported data shows that, on average, participants' LCI2.5 scores changed by +0.20 (a small increase) while they were taking the placebo, and by −0.46 (a small decrease) while they were taking ivacaftor. These are the changes from each starting point, so one number went slightly up and the other went slightly down over the course of each treatment period. No secondary outcome measures were included in the data provided, so results for any other measurements from this trial were not reported in the information available here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03229252 · results posted 30 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03229252) enrolled 91 people across three groups: 31 received a placebo (an inactive treatment), 15 received a lower dose of SPX-101 (60 mg twice daily), and 45 received a higher dose of SPX-101 (120 mg twice daily). The trial was measuring changes in lung function — specifically a test called "percent predicted FEV1," which is a standard way of measuring how much air a person can forcefully breathe out in one second compared to what would be expected for someone of their age and size. Most participants completed the study, with 2 people leaving the placebo group early, none leaving the low-dose group, and 6 leaving the high-dose group. The reported data shows that for the main measure — the change in lung function from the start of the trial — the placebo group recorded an average change of 1.633 units, the low-dose SPX-101 group recorded 0.800 units, and the high-dose SPX-101 group recorded 0.890 units. All three groups showed a small upward change from their starting point, with the placebo group's reported change being slightly higher than either SPX-101 group. For one of the secondary measures — the number of participants who experienced any adverse events (unwanted or unexpected health occurrences during the trial) — the reported data shows 20 out of 31 placebo participants, 11 out of 15 low-dose participants, and 30 out of 45 high-dose participants had at least one such event. For laboratory tests (blood chemistry, blood counts, and urine analysis), the reported data shows no participants in any group had notable changes flagged, with the exception of 1 participant in the low-dose group for urinalysis. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02544451 · results posted 29 October 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02544451) looked at a combination medicine called lumacaftor/ivacaftor (LUM/IVA) in children with cystic fibrosis. The trial had two main groups in Treatment Period 1 (which ran for 96 weeks): 143 participants who had already been taking LUM/IVA before the trial began, and 96 participants who had previously been on a placebo (a dummy treatment with no active ingredient) before switching to LUM/IVA. A small separate observational group of 6 participants was also included. The primary focus of the trial was to track any side effects or unwanted health events that occurred while participants were taking the medicine. The reported data shows that in Treatment Period 1, 142 out of 143 participants in the LUM/IVA-to-LUM/IVA group, and 94 out of 96 in the placebo-to-LUM/IVA group, experienced at least one treatment-emergent adverse event (that is, any unwanted health event that occurred after starting or continuing the medicine). Serious adverse events — meaning more significant health events — were reported in 43 participants in the first group and 29 in the second group. In the small observational group, 1 participant experienced a serious adverse event. The trial also measured several other things. Both groups showed a reduction in sweat chloride levels (a marker used in cystic fibrosis) of around 22.8–22.9 units. A lung function measure called Lung Clearance Index 2.5 decreased by about 0.85–0.86 in both groups (a lower number on this measure is generally considered to reflect better lung function). Body mass index (a measure of weight relative to height) increased by approximately 1.78 kg/m² in the first group and 2.04 kg/m² in the second. Scores on a quality-of-life questionnaire focused on breathing symptoms increased by about 7.4 and 6.6 points respectively out of a possible 100 (where higher scores indicate fewer symptoms). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03460990 · results posted 17 October 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03460990) involved 111 people with cystic fibrosis — 57 in one group who received a two-drug combination called TEZ/IVA, and 54 in another group who received a three-drug combination called VX-659/TEZ/IVA (also referred to as the "triple combination"). All participants who started the trial completed it. The trial was primarily measuring changes in lung function, specifically a test called FEV1 — which measures how much air a person can forcefully breathe out in one second — expressed as a percentage of what would normally be expected for someone of that age and size. The reported data shows that, for the primary lung function measure, the TEZ/IVA group had an average change of +0.3 percentage points, while the VX-659/TEZ/IVA triple combination group had an average change of +10.2 percentage points. For a secondary measure — the level of salt (chloride) in sweat, which is a marker commonly tracked in cystic fibrosis — the TEZ/IVA group showed an average change of +1.5 millimoles per litre, while the triple combination group showed an average change of −47.2 millimoles per litre. A questionnaire measuring how participants felt about their respiratory symptoms (scored from 0 to 100, where higher means fewer symptoms) showed an average change of +3.0 points in the TEZ/IVA group and +16.5 points in the triple combination group. Regarding side effects, the reported data shows that 31 out of 57 participants in the TEZ/IVA group and 33 out of 54 in the triple combination group experienced treatment-related side effects of any kind; no serious side effects were recorded in the TEZ/IVA group, while 2 participants in the triple combination group experienced serious side effects. The reported data also includes blood concentration levels of the individual medicines measured during the trial, though the figures varied across the different substances and time points. These drug level measurements were a secondary outcome, and the numbers were reported only for informational purposes as part of the trial's data collection. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02730208 · results posted 21 August 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02730208) enrolled 41 people with cystic fibrosis — 20 received a combination medicine called TEZ/IVA (tezacaftor/ivacaftor) and 21 received a placebo (a dummy treatment with no active ingredient). All 20 people in the TEZ/IVA group completed the study, while 20 out of 21 in the placebo group completed it. The main thing the trial was measuring was change in a lung CT scan score called the Brody/CF-CT score, which rates the visible structural damage to the lungs on a scale from 0 to 219 — a higher number means more damage is visible on the scan. The reported data shows that, on average, the Brody/CF-CT score changed by 0.90 points in the TEZ/IVA group and by 2.38 points in the placebo group over the course of the study. Both figures represent small increases from baseline (meaning slightly higher scores, suggesting slightly more visible lung changes on the scan), though the numbers reported are the average changes and no further breakdown was provided in the submitted data. As a secondary measure, the trial also recorded how many participants experienced adverse events (unexpected health problems during the study) and serious adverse events. The reported data shows that all 20 participants in the TEZ/IVA group and all 21 in the placebo group had at least one adverse event reported, while serious adverse events were reported in 8 participants in the TEZ/IVA group and 13 in the placebo group. It is important to note that these numbers alone do not tell us whether any differences between the groups are meaningful, and no conclusions about whether the treatment works or is safe can be drawn from these figures alone. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01316276 · results posted 26 July 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 206 participants, all of whom received a treatment called LAI 590 mg once daily. Of those, 139 people completed the study and 67 did not finish. The trial was measuring tolerability and safety-related observations over a period of up to 672 days (roughly two years), looking at things like unwanted events experienced by participants, changes in blood test results, lung function, and basic body measurements such as heart rate, breathing rate, and blood pressure. The reported data shows that 183 out of 206 participants experienced at least one treatment-emergent adverse event (that is, an unwanted event that appeared or worsened after starting the study treatment). Of these, 92 participants experienced a serious adverse event, and 21 participants stopped taking the study drug permanently because of an adverse event. Regarding blood test results, the reported data shows small numbers of participants had notable abnormalities — for example, 13 participants had significant abnormalities in overall lab values, with further breakdowns reported across blood cell counts and chemistry tests, with individual figures ranging from 1 to 23 participants depending on the specific measurement. For lung function, the reported data shows that across different time points, between 1 and 6 participants at any given assessment had a drop of more than 15% in a measure of how much air they could breathe out in one second. For body measurements, the reported data shows an average decrease in breathing rate of 0.8 breaths per minute, an average decrease in heart rate of 0.9 beats per minute, and an average increase in systolic blood pressure (the top number in a blood pressure reading) of 2.3 mmHg from the start of the study to day 672. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02443688 · results posted 25 July 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02443688) enrolled 200 adults across three groups: 66 people received the higher dose of CTX-4430 (100 mg), 67 received the lower dose (50 mg), and 67 received a placebo (a dummy treatment with no active ingredient). The trial ran for 48 weeks and was primarily measuring changes in lung function, specifically a breathing test called FEV1 — which measures how much air a person can forcefully breathe out in one second, expressed as a percentage of what would be expected for someone of their age and size. A number of other outcomes were also tracked, including how often participants had lung flare-ups (called pulmonary exacerbations). The reported data shows that, for the main outcome, lung function (ppFEV1) declined across all groups over the 48 weeks. The 100 mg CTX-4430 group showed an average decline of 1.30 percentage points, the 50 mg group declined by 3.76 percentage points, and the placebo group declined by 2.69 percentage points. For lung flare-ups, the reported annualised rate (the estimated number of flare-ups per year) was 1.57 in the 100 mg group, 1.46 in the 50 mg group, and 1.56 in the placebo group. The reported data also shows that 25 people in the 100 mg group and 26 in the 50 mg group had no flare-up during the study, compared with 20 in the placebo group. Other breathing measures and flare-up timing data were also reported, with figures varying across the groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02566044 · results posted 17 July 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02566044) enrolled 16 people in total — six in Cohort 1 (who received one dose level of the inhaled investigational medicine QBW276), six in Cohort 2 (who received a higher dose level), and four who received a placebo (an inactive treatment). All 16 participants completed the study. The trial was measuring how the drug moved through the body after inhalation, and also looking at any unwanted side effects. It is important to note that the study was stopped early before it was fully completed. The reported data shows that for the safety outcome — which counted the number of participants who experienced any unwanted effects — zero participants in Cohort 1 and zero in the placebo group had such events recorded in two of the reported categories, while six participants in Cohort 2 and two in Cohort 1 had events recorded in one category. The data on how the drug moved through the body (measured by peak levels in the blood, how quickly those peak levels were reached, and how much of the drug was present over time) showed that the drug and its breakdown products were detectable in the bloodstream after inhalation. For example, peak blood levels of one breakdown product (QBP545) ranged from roughly 3.88 to 7.80 nanograms per millilitre across the two cohorts and time points measured. For the secondary outcome — a breathing test called FEV1, which measures how much air a person can forcefully breathe out in one second — the reported change from the starting measurement was minus 0.1 percentage points for both Cohort 1 and Cohort 2, and minus 2.7 percentage points for the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02875366 · results posted 17 June 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02875366) enrolled 70 people with cystic fibrosis — 36 in the placebo group and 34 in the group receiving a combination treatment called lumacaftor/ivacaftor (LUM/IVA). All 36 people in the placebo group completed the study, while 31 of the 34 in the LUM/IVA group completed it (3 did not finish). The trial was measuring whether LUM/IVA had any effect on exercise capacity over 24 weeks, primarily by tracking changes in "VO2max" — a measure of the maximum amount of oxygen the body can use during intense exercise, tested on a specialised exercise machine. The reported data shows that for the primary outcome — the percentage change in VO2max from the start of the study to week 24 — the placebo group saw a change of −3.5% and the LUM/IVA group saw a change of −6.6%, meaning both groups showed a decline on average, with the LUM/IVA group's decline being somewhat larger. For the secondary outcomes, exercise duration changed by +0.4% (placebo) versus −2.8% (LUM/IVA) in relative terms, and by −2.1 seconds (placebo) versus −17.4 seconds (LUM/IVA) in absolute terms. The absolute change in VO2max was −1.3 ml/kg/min for placebo and −2.7 ml/kg/min for LUM/IVA. For oxygen consumption at the "anaerobic threshold" — the point during exercise where the body starts to struggle to keep up with oxygen demand — the placebo group showed a change of +94.6 ml/min, while the LUM/IVA group showed a change of −55.1 ml/min. In percentage terms, those figures were +9.4% (placebo) and +1.8% (LUM/IVA). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02444234 · results posted 4 June 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 11 adults in total — six in one group and five in the other. It used a "crossover" design, meaning each participant received both forms of the antibiotic tedizolid at different times, with a washout break of at least two days in between. One group took tedizolid by mouth first, then by drip (intravenous infusion); the other group did it in reverse order. The trial was measuring how the drug moved through the body — specifically, how much of it reached the bloodstream and the lungs' mucus (sputum), and how quickly. The reported data shows the following numbers for the blood (plasma) measurements: the highest concentration of tedizolid recorded in the blood (called "peak plasma concentration") was 2.22 mg/litre for the tablet form and 2.92 mg/litre for the drip form. The total drug exposure in the blood over time (a measure called "area under the curve," or AUC — essentially a way of adding up the drug level across all the time points) was 22.1 mg·h/mL for the tablet and 20.7 mg·h/mL for the drip. The time it took to reach that peak blood level was 2.5 hours for the tablet and 1.36 hours for the drip. The reported data also shows measurements from sputum (the mucus coughed up from the lungs). The peak concentration of tedizolid found in sputum was 1.08 mg/litre for the tablet form and 1.196 mg/litre for the drip form. The total drug exposure in sputum over time was 15.04 mg·h/mL for the tablet and 13.53 mg·h/mL for the drip. The time to reach peak sputum levels was 4 hours for the tablet and 3 hours for the drip. No other outcome data was reported beyond these figures. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00558844 · results posted 4 June 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 41 people across five groups. Participants received one of three doses of an inhaled antibiotic medicine called Arikayce™ (also known as liposomal amikacin for inhalation) — at 70 mg, 140 mg, or 560 mg — or a matching dummy treatment (placebo) for 28 days. The main thing the trial was set up to measure was how many participants in each group experienced unwanted medical events (called adverse events) during treatment. Secondary measurements looked at how the drug moved through the body — how much appeared in the blood, in mucus coughed up from the lungs (sputum), and in urine — as well as a standard lung-function test called FEV1, which measures how much air a person can forcefully breathe out. The reported data shows that in the primary outcome — the count of participants who had a treatment-emergent adverse event — 14 out of 15 participants in the 560 mg Arikayce™ group, all 7 in the 560 mg placebo group, all 7 in the 70 mg Arikayce™ group, 4 out of 5 in the 140 mg Arikayce™ group, and 6 out of 7 in the combined placebo group experienced at least one such event. For the secondary measures, the reported data shows that higher doses of Arikayce™ were associated with higher measured levels of the drug in blood, sputum, and urine. For example, peak blood concentration (the highest level detected in the blood) was reported as 1.59 mg/L for the 560 mg group compared with 0.216 mg/L and 0.375 mg/L for the 70 mg and 140 mg groups respectively on the first measurement time point. Sputum concentrations were also notably higher in the 560 mg group. For lung function (FEV1), baseline values and small changes from baseline across time points were reported for each group; no figures for some later time points were included in the submitted data. It is worth noting that this was a small, early-phase trial with between 5 and 15 people per group, so the numbers are very limited. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02354859 · results posted 21 March 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 119 people with cystic fibrosis — 60 in the gallium group and 59 in the placebo group. All 60 participants in the gallium group completed the study, while 57 of the 59 in the placebo group did. The trial was primarily measuring how many participants showed a meaningful improvement in lung function (specifically, a 5% or greater relative increase in a breathing test called FEV1, which measures how much air a person can forcefully breathe out in one second) from the start of the study to day 28. It also tracked a range of other measurements out to day 56, including breathing test results, levels of a particular bacteria in sputum (mucus coughed up from the lungs), respiratory symptoms, and the occurrence of unwanted health events. The reported data shows that by day 28, 22 out of 60 participants in the gallium group and 17 out of 59 in the placebo group had that 5% or greater improvement in their breathing test result. For the secondary measurements at day 56, the gallium group showed an average relative change in the breathing test of +1.89% compared to −0.05% in the placebo group. For the bacteria levels in sputum, the gallium group showed an average reduction of 1.06 units (on a log scale) compared to a reduction of 0.33 units in the placebo group. On the respiratory symptom score (where lower scores indicate fewer symptoms, on a scale of 0–100), the gallium group showed an average increase of 3.03 points, while the placebo group showed an average decrease of 0.98 points — note that on this scale, a lower score reflects improvement. The reported data also shows that unwanted health events (called adverse events) occurred in 57 participants in each group over the 56-day follow-up. Serious unwanted health events (serious adverse events) were reported in 11 participants in the gallium group and 9 in the placebo group. The rate of all unwanted health events was reported as 0.84 per participant per week in the gallium group and 1.03 in the placebo group, while serious events occurred at a rate of 0.02 and 0.03 per participant per week respectively. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02088216 · results posted 18 February 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 161 people with bronchiectasis (a lung condition where the airways become permanently widened and prone to infection). Participants were split into two groups: 81 people received a medicine called N-acetylcysteine, and 80 people received a control (comparison) treatment. The trial ran for 12 months and was primarily measuring how often participants experienced flare-ups — periods when their symptoms got noticeably worse, sometimes requiring antibiotics. The reported data shows that, over the 12 months, the N-acetylcysteine group had a median (middle value) of 1 flare-up, compared to a median of 2 flare-ups in the control group. For the secondary measurements, the N-acetylcysteine group showed an average reduction in daily mucus (sputum) volume of about 6.5 mL, while the control group showed a larger average reduction of about 18.3 mL. A symptom-impact questionnaire (scored 0–40, where higher means worse impact) changed by an average of −3.79 points in the N-acetylcysteine group and −1.44 points in the control group. The number of participants whose sputum tested positive for a particular bacterium (*Pseudomonas aeruginosa*) changed by 8 people in the N-acetylcysteine group and 5 in the control group. Two measures of lung airflow (how much air a person can breathe out forcefully in one second) showed small changes in both groups, and the reported data shows these changes were modest across both groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02412111 · results posted 8 January 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 156 people with cystic fibrosis. All participants first took ivacaftor alone for four weeks (a "run-in" period), after which 153 continued into the main eight-week comparison phase. In that phase, participants were split into two groups: 76 took a combination of two drugs (VX-661 plus ivacaftor) and 75 took ivacaftor on its own. The trial was primarily measuring changes in lung function, specifically a test called FEV1 — the amount of air a person can forcefully breathe out in one second — expressed as a percentage of what would be expected for someone of their age and size. The reported data shows that, on average, the combination group's FEV1 score changed by +0.5 percentage points from their baseline (starting point), while the ivacaftor-alone group changed by +0.2 percentage points. For the secondary outcomes, a quality-of-life questionnaire focused on breathing symptoms (scored 0–100, where higher is better) showed a reported change of +0.7 points in the combination group and −2.1 points in the ivacaftor-alone group. A sweat chloride test — a common marker measured in cystic fibrosis studies — showed a reported change of −7.9 millimoles per litre in the combination group and −2.1 millimoles per litre in the ivacaftor-alone group. Regarding reported side events, 50 participants in the combination group and 54 in the ivacaftor-alone group experienced adverse events (unexpected health events recorded during the trial), while 4 and 7 participants in those groups respectively experienced serious adverse events. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02504827 · results posted 27 November 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 12 people who all received an intravenous (given through a vein) combination of two antibiotics called ceftazidime and avibactam. Eleven of the 12 participants completed the study, and one did not finish. The trial was measuring how much of the medication ended up in the blood and in the sputum (the mucus coughed up from the lungs), to understand how the drug moves through the body. The reported data shows that the peak concentration of the medication in the blood — that is, the highest level recorded in the bloodstream after a dose — was 85.87 mg/L (milligrams per litre, a measure of how much drug was present in a set volume of fluid). The reported peak concentration reached in the sputum was 2.5 mg/L. No other outcome figures were included in the data submitted to ClinicalTrials.gov, so additional details beyond these two measurements were not reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02742519 · results posted 19 November 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02742519) looked at a medicine called ivacaftor in people with cystic fibrosis. The trial had two parts. In Part 1, 14 participants took part in a crossover design — meaning each person took ivacaftor for 8 weeks and a placebo (a dummy treatment with no active ingredient) for 8 weeks, though in different orders. In Part 2, a separate group of 10 participants took ivacaftor in an open-label period of 32 weeks (meaning everyone knew what they were taking). The trial was primarily measuring changes in something called the Lung Clearance Index (LCI2.5) — a measure of how evenly air moves in and out of the lungs, where a lower number generally indicates more even airflow. The reported data shows that for the main measurement (LCI2.5), participants in the ivacaftor group showed an average change of −0.53 units from their starting point, compared to −0.07 units in the placebo group, over 8 weeks. For the secondary measurements: a blood marker related to the pancreas (immunoreactive trypsinogen) changed by −15.5 ng/mL in the ivacaftor group versus −9.3 ng/mL in the placebo group. A marker related to pancreatic digestive function in stool (fecal elastase-1) changed by +23.1 mcg/g in the ivacaftor group versus −17.0 mcg/g in the placebo group. Body weight changed by +1.1 kg (ivacaftor) versus +0.9 kg (placebo), and BMI changed by +0.32 kg/m² (ivacaftor) versus +0.13 kg/m² (placebo). The reported data also shows that 11 participants in each of the placebo and ivacaftor crossover groups, and 6 participants in the open-label group, experienced at least one adverse event (an unwanted health event recorded during the trial); no serious adverse events were reported in any group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02679729 · results posted 5 November 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02679729) tested an inhaled investigational medicine called AZD5634. A total of 63 people took part across two parts of the study. In Part A, participants received one of seven increasing inhaled doses of AZD5634 (ranging from 10 micrograms up to 1,692 micrograms), or a placebo (a dummy treatment with no active ingredient). In Part B, a smaller group of 6 participants received both an inhaled dose and a dose given directly into a vein (intravenously). The trial was primarily measuring how many participants experienced adverse events (unwanted effects) at each dose level, and also tracking how the medicine moved through the body — for example, how much of it entered the bloodstream and how quickly it was cleared. The reported data shows that, for the primary outcome, the number of participants with reported adverse events was: 1 person at the 10 µg dose, 0 at 27 µg, 0 at 81 µg, 1 at 216 µg, 1 at 648 µg, 0 at 1,296 µg, 2 at 1,692 µg, and 3 among those who received the placebo. In Part B, 2 participants receiving the intravenous dose and 1 receiving the inhaled dose had reported adverse events. For the secondary outcomes — which tracked how much of the medicine reached the bloodstream — the reported peak blood concentration (the highest level detected) rose as the inhaled dose increased, from 0.016 nmol/L at 81 µg up to 0.532 nmol/L at 1,692 µg, while the intravenous dose in Part B produced a notably higher peak of 6.07 nmol/L. The reported data also showed that the total amount of medicine absorbed over time (measured as "area under the curve") followed a similar pattern of increase with dose. For the lowest dose group (81 µg), the overall area-under-the-curve figure was not reported. In Part B, the proportion of the inhaled dose that was estimated to reach the bloodstream (called "bioavailability") was reported as approximately 2.9%. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02823470 · results posted 27 September 2018

  According to the results reported on ClinicalTrials.gov, this trial looked at whether a "smart pill bottle" — a special medication container that can send reminders or track when it is opened — made a difference to how consistently people took a cystic fibrosis medicine called lumacaftor/ivacaftor (LUM/IVA). A total of 24 people took part: 9 used a deactivated (switched-off) smart bottle, which acted as the control group, and 15 used an activated (switched-on) smart bottle, which was the test group. The reported data shows that none of the participants were recorded as having formally "completed" the study under the trial's own classification, though all 24 were recorded as having started. The main thing the trial measured was how often participants took their medication as prescribed, expressed as a percentage. The reported data shows that the control group had a median adherence of around 85%, while the test group had a median adherence of around 92%. When looking at the first 12 weeks specifically, the reported figures were approximately 89% for the control group and 97% for the test group. These figures were described by the researchers as medians (the middle value in a set of numbers) rather than averages, because the groups were small. The reported data also shows how many people in each group took their medication at least 80% of the time over 12 weeks: 4 out of 9 in the control group and 13 out of 15 in the test group. For the higher threshold of at least 90% of the time, the numbers reported were 2 out of 9 in the control group and 12 out of 15 in the test group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03534986 · results posted 6 September 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT03534986) enrolled 32 participants across three groups, each using a different airway clearance vest device: the AffloVest compared against "The Vest," the inCourage, and the SmartVest. Thirty-two people started the trial and 30 completed it — two participants in the AffloVest vs. SmartVest group did not finish. The trial was measuring how different vest devices affected several aspects of breathing, including the normal volume of air moved during breathing, how fast air can be breathed out, and how much air the lungs can hold and push out forcefully. The reported data shows five breathing measurements were taken at baseline (before any device was used) and then compared across two broader groupings — an AffloVest arm and a combined compressor-based vest arm. For the normal volume of air moved with each breath (Tidal Volume), the reported figures were 0.93 litres at baseline, 1.00 litres for the AffloVest arm, and 1.07 litres for the compressor arm. For maximum speed of breathing out (Peak Expiratory Flow), the figures were 8.19, 8.28, and 8.13 litres per second respectively. For the total amount of air that can be forcefully breathed out (Forced Vital Capacity), the numbers were 4.29, 4.25, and 4.12 litres. For the air pushed out in the first second of a forceful breath (FEV1), the figures were 3.51, 3.46, and 3.30 litres. For airflow measured in the middle portion of a forceful breath (FEF25–75%), the reported values were 3.71, 3.54, and 3.19 litres per second. The reported data shows only the numerical measurements as submitted to ClinicalTrials.gov, without any statistical analysis or comparison conclusions being available in the structured data provided. No additional secondary outcome data was included in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00908830 · results posted 6 August 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 10 people in total — 5 who had received a lung transplant and had cystic fibrosis (CF), and 5 who had received a lung transplant for a reason other than cystic fibrosis (non-CF). All 10 participants completed the study with none dropping out. The trial was measuring how a medication called mycophenolic acid (MPA) — a drug commonly used after transplants to help prevent the body from rejecting the new organ — moved through the body over time. Specifically, it tracked how much of the drug and its breakdown product (called mycophenolic acid glucuronide, or MPAG) were present in the blood over a 12-hour period after a dose. The reported data shows that the total amount of MPA detected in the blood over 12 hours (a measure called "area under the curve", or AUC — essentially a way of capturing overall drug exposure) was 47.7 mg·h/L in the CF group and 83.1 mg·h/L in the non-CF group. For the breakdown product MPAG, the reported figures were 569 mg·h/L in the CF group and 911 mg·h/L in the non-CF group. These numbers suggest the CF group had lower measured drug exposure compared to the non-CF group, though the trial does not draw conclusions about what this means for treatment outcomes. The reported data also shows how much the drug levels varied — both between different patients and within the same patient across multiple measurement visits. In the CF group, the variation between patients was reported at around 31.2%, and within individual patients across visits at around 16.6–37.3%. In the non-CF group, the between-patient variation was around 36.3% and within-patient variation around 13.8–27.8%. These figures are a way of describing how consistent or inconsistent drug levels were across and within individuals; higher percentages mean more variability. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02508207 · results posted 27 July 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02508207) enrolled 34 people in total — 7 received a placebo (an inactive treatment used for comparison) and 27 received a combination treatment called TEZ/IVA (tezacaftor/ivacaftor). All 34 participants completed the study. The trial was measuring several things over approximately 28–29 days, including how well the lungs cleared mucus, how well the lungs moved air, acidity levels in the small bowel, and the level of chloride (a salt marker) in sweat. The reported data shows the following changes from the start of the study to day 28 or 29. For mucus clearance in the lungs (the primary — that is, main — thing being measured), the placebo group showed a change of −0.5 percentage points and the TEZ/IVA group showed a change of −0.9 percentage points. For lung airflow (how much air participants could force out in one second, compared to what is typical for someone of their age and size), the placebo group showed a change of −0.4 percentage points while the TEZ/IVA group showed a change of +2.4 percentage points. For small bowel acidity, the placebo group changed by +0.3 pH minutes and the TEZ/IVA group by −0.2 pH minutes. For sweat chloride levels, the placebo group changed by −0.2 millimoles per litre and the TEZ/IVA group by −8.4 millimoles per litre. Regarding unwanted health events (called adverse events), the reported data shows that 0 participants in the placebo group and 0 in the TEZ/IVA group experienced serious adverse events, while 2 participants in the placebo group and 24 in the TEZ/IVA group experienced any adverse events of any kind. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00846053 · results posted 24 July 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT00846053) involved 21 people with cystic fibrosis, divided into two groups: 13 people whose lung function was considered stable, and 8 people whose lung function was declining rapidly. The trial was measuring how a specialised imaging scan could track the movement of a substance into the lungs, as well as looking at levels of a particular protein (neutrophil elastase, an enzyme found in airway mucus) in sputum (phlegm) samples. Ten of the 13 people in the stable group completed the study, while all 8 in the rapidly declining group completed it. The reported data shows that the main measurement — called the "kinetic influx constant" or Ki, which is a number describing how quickly a tracer substance moved into lung tissue during scanning — was 0.009 mL/min/mL in the stable lung function group and 0.013 mL/min/mL in the rapidly declining lung function group. For the secondary measurement, the reported data shows that levels of neutrophil elastase in sputum were 2,179 micrograms per microgram of protein in the stable group and 1,656 micrograms per microgram of protein in the rapidly declining group. These are the figures as submitted; no further breakdown or comparison statistics were reported in the structured data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02750501 · results posted 18 July 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 44 participants in a single group — meaning everyone received the same treatment with no comparison group. Of those, 39 were treated and 36 completed the study. The trial was measuring changes in the levels of omega-3 fatty acids (specifically DHA and EPA — types of healthy fats) in participants' red blood cells and blood plasma after using a device called RELiZORB, which is a cartridge used during tube feeding. Measurements were taken at the start of the study and again after 90 days. The reported data shows that the main thing being measured — the omega-3 index in red blood cells (a way of checking how much DHA and EPA are stored in red blood cells) — increased by 5.01 percentage points over 90 days. Looking at the individual components, DHA in red blood cells increased by 3.28 percentage points and EPA increased by 1.73 percentage points. The reported data also shows that DHA and EPA levels in the blood plasma increased by 93.73 percentage points over the same period. Additionally, the ratio of omega-6 to omega-3 fatty acids in red blood cells (a measure where a lower number is generally considered more balanced) decreased by 2.51 percentage points. Regarding unexpected serious device-related events (called Unanticipated Adverse Device Effects — meaning serious harms unexpectedly linked to the device), the data reports figures of 10, 2, and 8 participants across what appear to be different categories, though the breakdown of those categories was not fully detailed in the reported data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02759562 · results posted 5 July 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02759562) looked at a medicine called andecaliximab compared to a placebo (an inactive dummy treatment) in people with a lung condition. The trial had two phases: an 8-week double-blind period (where neither participants nor researchers knew who received which treatment) and a follow-on open-label period (where everyone knew what was being given). A very small number of people took part — just 3 in the andecaliximab group and 3 in the placebo group during the double-blind phase. The main thing being measured was a standard breathing test called FEV1 (how much air a person can forcefully breathe out in one second), expressed as a percentage of what would be expected for a healthy person of the same age and size. The reported data shows that for the primary outcome — change in the pre-bronchodilator FEV1 percentage (measured before any inhaled airway-opening medicine) from the start to Week 8 — the andecaliximab group showed a change of −2.10 percentage points, while the placebo group showed a change of +2.90 percentage points. For the secondary outcomes, the post-bronchodilator FEV1 percentage (measured after airway-opening medicine) changed by +1.01 percentage points in the andecaliximab group and −1.45 percentage points in the placebo group. When these changes were looked at in relative terms (as a percentage of the starting value), the pre-bronchodilator result was −3.85% for andecaliximab and +4.41% for placebo, and the post-bronchodilator result was +2.28% for andecaliximab and −1.98% for placebo. It is important to note that only 6 people in total completed the double-blind phase, and none completed the open-label phase. The reported data shows such a very small number of participants that no broad conclusions can be drawn from these figures alone. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02516410 · results posted 12 June 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02516410) enrolled 168 people with cystic fibrosis — 85 in the placebo group and 83 in the group receiving the combination treatment VX-661/IVA (also known as tezacaftor/ivacaftor). All 85 placebo participants completed the study, while 81 of the 83 in the treatment group did so. The trial's main focus was measuring any change in lung function over 12 weeks, specifically a breathing test called FEV1 — the amount of air a person can forcefully breathe out in one second — expressed as a percentage of what would be expected for someone of that age, sex, and height. The reported data shows that, on average, the placebo group's FEV1 percentage changed by −0.1 percentage points from their starting point, while the VX-661/IVA group's changed by +1.0 percentage point. In relative terms (that is, as a proportion of each person's own starting lung function score), the placebo group showed a 0.1% relative change and the treatment group showed a 2.1% relative change. For the secondary measures, a quality-of-life questionnaire focused on breathing symptoms (scored 0–100, where higher means fewer symptoms) showed an average change of +3.8 points in the placebo group and +5.9 points in the VX-661/IVA group. The number of lung flare-up (pulmonary exacerbation) events recorded was 23 in the placebo group and 22 in the treatment group, with both groups recording approximately 0.97–0.98 such events per year. Average body mass index (BMI, a measure of weight relative to height) changed by +0.22 kg/m² in the placebo group and +0.14 kg/m² in the treatment group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT02392234 · results posted 12 June 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 248 people with cystic fibrosis across six groups, each receiving different sequences of three treatments — a combination of VX-661 and ivacaftor (VX-661/IVA), ivacaftor (IVA) alone, or a placebo (a dummy treatment with no active medicine). The trial ran in two back-to-back 8-week periods, and it was measuring changes in lung function, quality of life, sweat chloride levels, and the number of people who experienced unwanted health events during the study. The reported data shows that the main thing being measured was the change in a lung function test called ppFEV1 — essentially, how much air a person can forcibly breathe out in one second, expressed as a percentage of what would be expected for someone of their age and size. Over the study period, the placebo group showed an average change of −0.3 percentage points, the ivacaftor-alone group showed a change of +4.4 percentage points, and the VX-661/IVA combination group showed a change of +6.5 percentage points. For a secondary measure of respiratory symptoms and quality of life (scored from 0 to 100, where higher means fewer symptoms), the reported changes were −1.0 points for placebo, +8.7 for ivacaftor alone, and +10.1 for the combination. Sweat chloride levels (a marker used in cystic fibrosis monitoring) changed by −0.4 mmol/L for placebo, −4.9 mmol/L for ivacaftor alone, and −9.9 mmol/L for the combination. The reported data also shows that unwanted health events during treatment were recorded in 126 participants on placebo, 114 on ivacaftor alone, and 117 on the combination, with serious events reported in 14, 10, and 8 participants respectively across those groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02823964 · results posted 17 May 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02823964) involved 25 participants who all received a treatment called Liprotamase. There was only one group in the study — meaning there was no comparison or placebo group. Of the 25 who started the trial, 20 completed it, and 5 did not finish. The trial was measuring safety by looking at how many participants experienced adverse events (that is, unwanted health events or abnormal test results) during the study. The reported data shows that the primary outcome — tracking the number of participants who experienced adverse events, including any unusual results from clinical checks or laboratory tests — recorded figures of 25 and 9 participants. However, the data as submitted does not clearly label which number refers to which specific category of adverse event, so it is not possible to say with certainty what each figure represents beyond what is provided. No secondary outcome measure data was reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02734810 · results posted 11 May 2018

  According to the results reported on ClinicalTrials.gov, this trial was made up of two parts — Part A and Part B. A total of 15 people were enrolled in Part A, of whom 13 completed the study and 2 did not finish. No participants were enrolled in Part B. The trial was measuring safety, specifically by counting how many participants experienced one or more adverse events (that is, any unwanted or unexpected health events that were recorded during the study). The reported data shows that, out of the 15 people in Part A, 3 participants experienced at least one adverse event during the study. No data was reported for Part B, as no one was enrolled in that group. No other outcome measures — such as whether the treatment had any effect on a condition — appear to have been reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02070744 · results posted 13 April 2018

  According to the results reported on ClinicalTrials.gov, this trial tested two investigational medicines — VX-661 (also called tezacaftor) and ivacaftor (IVA) — used together or with placebo (a dummy treatment with no active ingredient) in people with cystic fibrosis. The trial had two phases: a 12-week controlled phase (PC Phase), where participants were randomly assigned to receive either active medicines or placebos, and a 48-week open-label extension phase (OLE Phase), where participants could continue on the active combination. In total, 40 people entered the controlled phase across four groups (6, 5, 15, and 14 participants respectively), and 27 people entered the extension phase. The reported data shows that, in the controlled phase, the primary thing being tracked was the number of participants who experienced adverse events (any unwanted medical occurrence during the study) and serious adverse events (those involving hospitalisation, life-threatening situations, or similar significant medical events). According to the results reported on ClinicalTrials.gov, all participants in the active and placebo groups experienced at least one adverse event during the 12-week phase. Serious adverse events were reported in 1 out of 6 participants in one active group, 2 out of 5 in the placebo group, 4 out of 15 in another active group, and 5 out of 13 in the other placebo group. In the extension phase, 25 out of 27 participants reported at least one adverse event, and 6 reported a serious adverse event. The reported data shows that a secondary measure — how much lung function changed, specifically the amount of air a person can forcibly breathe out in one second (called FEV1, expressed as a percentage of what would be expected for someone of that age and size) — was also tracked. In the 12-week controlled phase, the group taking VX-661 100 mg once daily plus ivacaftor showed an average increase of 3.0 percentage points in this lung function measure, compared to 1.9 percentage points in their matched placebo group. The group taking the lower dose of VX-661 with ivacaftor showed an average increase of 0.9 percentage points, compared to a small decrease of 0.1 percentage points in their matched placebo group. In the 48-week extension phase, participants on the active combination showed an average increase of 2.7 percentage points from their starting point at that phase. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗

• NCT01531673 · results posted 13 April 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 190 participants across 13 different treatment groups. It was testing different doses of an investigational drug called VX-661 (also known as tezacaftor), both on its own and combined with another drug called ivacaftor, compared to a placebo (an inactive dummy treatment). The trial was looking at people with cystic fibrosis and was measuring two main things: any unwanted medical events (called adverse events) that occurred during the study, and changes in sweat chloride levels — a standard measurement used in cystic fibrosis research where a lower level of chloride in sweat is considered a marker of how the affected protein may be functioning. The reported data shows that across the groups receiving active treatment, most participants completed the study, with only a small number — between zero and one person per group — not finishing. For adverse events (unwanted medical occurrences during the study, whether or not related to the drug), the numbers of participants who experienced them ranged from 2 out of 16 in one group up to 30 out of 33 in the combined placebo group. For sweat chloride changes, the reported data shows that the placebo groups had little change or a slight increase in sweat chloride levels (ranging from around −0.86 to +10.18 mmol/L). The reported data shows that most active treatment groups had reductions in sweat chloride levels, with the combination of VX-661 100 mg once daily and ivacaftor 150 mg twice daily (Group 3b) showing the largest reported average reduction of approximately −20.43 mmol/L. Other active treatment groups showed smaller reported reductions, generally in the range of −4.76 to −10.46 mmol/L. For Group 7 — a separate group of participants — the placebo group's sweat chloride increased by an average of 10.18 mmol/L, while the VX-661 100 mg group showed a reported average decrease of −7.02 mmol/L. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗

• NCT01222273 · results posted 29 March 2018

  According to the results reported on ClinicalTrials.gov, this trial involved 7 participants, all of whom completed the study. All participants had cystic fibrosis (CF) along with a related allergic lung condition called ABPA (allergic bronchopulmonary aspergillosis), which involves an immune reaction to a common mould called *Aspergillus*. The trial was investigating whether taking a vitamin D supplement (cholecalciferol) over 24 weeks would change certain immune and allergy-related measurements in these patients. The reported data shows that all 7 participants had a detectable immune response to *Aspergillus* in a specific type of immune cell (CD4+ T-cells), which was the main thing the trial set out to measure — though the data does not report whether this response went up, down, or stayed the same compared to before treatment. For the secondary measurements, the reported data shows an average total IgE level (a marker associated with allergic reactions, measured in standard units called IU/mL) of 312.6, and an average *Aspergillus*-specific IgE level (a more targeted allergy marker) of 11.73 kUA/l by the end of the study period. It is worth noting that the reported figures represent the values recorded, but no before-and-after comparison data was included in the results as submitted. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT00322868 · results posted 23 February 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 21 adults who each took a 30 mg daily dose of a medication called pioglitazone. Twenty of the 21 participants completed the study, and one did not. The trial was measuring changes in the composition of sputum (mucus coughed up from the lungs) — specifically looking at the number and type of immune cells present, as well as levels of certain proteins linked to inflammation, before and after treatment. The reported data shows the following before ("Baseline") and after ("Post-Treatment") taking pioglitazone. The total white cell count in sputum went from 6.93 to 6.81 (measured on a logarithmic scale, meaning very small numerical differences can represent larger real-world changes). The neutrophil count — a type of immune cell — went from 6.88 to 6.75 on the same scale, and neutrophils as a share of all white cells went from about 82% to about 74%. A protein called active elastase (which can break down lung tissue) went from 2.03 to 1.96, while two inflammation-related proteins — TNFα and IL-1β — went from 1.74 to 1.69 and from 4.05 to 3.99 respectively (all on logarithmic scales). No information about whether these changes were considered statistically meaningful was included in the reported data. It is worth noting that this was a single-group study with no comparison group receiving a placebo or different treatment, so the reported figures reflect measurements taken from the same participants at two different time points. No data from a control group was reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗

• NCT02465450 · results posted 9 February 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02465450) tested a drug called JBT-101 (also known as lenabasum) across two treatment periods. In the first period, 85 people took part, split across three groups: 26 received a low dose (1 mg once daily), 24 received a medium dose (5 mg once daily), and 35 received a placebo (a dummy treatment with no active ingredient). In the second period, 81 people took part across three different groups: 30 received 20 mg once daily, 28 received 20 mg twice daily, and 23 received a placebo twice daily. The main thing the trial was set up to measure was how many participants in each group experienced adverse events — that is, unwanted or unexpected health changes that arose during the treatment period. The reported data shows that, for the primary outcome, the number of participants who experienced adverse events during treatment was: 14 out of 26 in the 1 mg once-daily group, 13 out of 24 in the 5 mg once-daily group, and 15 out of 35 in the matching placebo group. In the second period, 21 out of 30 in the 20 mg once-daily group, 19 out of 28 in the 20 mg twice-daily group, and 14 out of 23 in the placebo twice-daily group reported adverse events. The trial also measured the level of lenabasum in participants' blood (in units called ng/mL) at day 84 as a secondary outcome. The reported data shows average blood levels of 249.77 ng/mL for the 20 mg once-daily group, 360.80 ng/mL for the 20 mg twice-daily group, and 0.00 ng/mL for the placebo group. Blood level data for the 1 mg and 5 mg groups was not reported for this outcome. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT02514473 · results posted 23 October 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02514473) enrolled 204 people with cystic fibrosis — 101 in the placebo group and 103 in the group receiving a combination treatment called lumacaftor/ivacaftor (LUM/IVA). Of those who started, 98 in each group completed the 24-week study. The trial was primarily measuring a lung test called the Lung Clearance Index (LCI2.5), which reflects how evenly air moves in and out of the lungs — a lower score on this test indicates more even airflow. The trial also tracked a number of secondary measures, including sweat chloride levels (a marker used in cystic fibrosis monitoring), body mass index (BMI), a quality-of-life questionnaire score focused on breathing symptoms, and a related lung airflow measure called LCI5.0. The reported data shows the following changes from the starting point over 24 weeks: for the primary lung measure (LCI2.5), the placebo group's score went up by 0.08, while the LUM/IVA group's score went down by 1.01. For sweat chloride (averaged across Day 15 and Week 4), the placebo group's level rose by 0.8 mmol/L, while the LUM/IVA group's level fell by 20.0 mmol/L; at Week 24 specifically, the placebo group rose by 3.2 mmol/L and the LUM/IVA group fell by 21.6 mmol/L. For BMI, the placebo group increased by 0.27 kg/m² and the LUM/IVA group by 0.38 kg/m². The reported data shows the quality-of-life respiratory score (where higher means fewer symptoms) rose by 3.0 points in the placebo group and 5.5 points in the LUM/IVA group. For the secondary lung measure (LCI5.0), the placebo group's score rose by 0.08 while the LUM/IVA group's fell by 0.36. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT01486199 · results posted 25 September 2017

  According to the results reported on ClinicalTrials.gov, this trial involved two groups: 10 children with cystic fibrosis (CF) and 10 adult controls (people without CF). The trial was measuring two things in the lungs — how quickly a substance is absorbed through the lung lining (called "absorptive clearance") and how quickly tiny particles are swept out of the airways by the lungs' natural cleaning system (called "mucociliary clearance"). Both were tracked by having participants inhale a harmless radioactive tracer and then measuring how much cleared from the lungs over 80 minutes. The children with CF were also followed up approximately two years later, while the adult control group was only involved at the initial study day. The reported data shows that at the baseline study day, the absorptive clearance rate for the CF children was 34.0% cleared per 80 minutes, compared with 17.7% for the adult controls. For mucociliary clearance, the CF children showed 22.8% cleared per 80 minutes, while the adult controls showed 31.4% cleared per 80 minutes. At the two-year follow-up — which only involved the CF children — the reported absorptive clearance rate was 28.6% per 80 minutes, and the mucociliary clearance rate was 22.0% per 80 minutes. No two-year follow-up data was reported for the adult control group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02106832 · results posted 1 September 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 521 adults across four groups: 171 received ciprofloxacin (an antibiotic) inhaled as a dry powder on a 28-days-on/28-days-off schedule ("Cipro 28"), 176 received the same antibiotic on a 14-days-on/14-days-off schedule ("Cipro 14"), 86 received a placebo (inactive treatment) on the 28-day schedule, and 88 received a placebo on the 14-day schedule. The trial was measuring things such as how long it took before participants had their first serious chest flare-up (called an exacerbation — a significant worsening of lung symptoms requiring antibiotic tablets or injections), how often flare-ups happened over 48 weeks, and several other measures including quality of life scores and whether bacteria in the lungs were cleared. The reported data shows that for the main (primary) outcome — the time to first serious flare-up comparing the Cipro 28 group to the placebo groups combined — no numerical result was reported in the submitted data, so that figure is not available here. For the secondary outcomes, the reported data shows that when looking at the number of participants who had at least one serious flare-up over 48 weeks: 46 out of 115 participants in the Cipro 28 group, 40 out of 108 in the Cipro 14 group, and 41 out of 101 in the combined placebo group experienced such an event. Regarding bacteria being cleared from the lungs by the end of treatment, around 35% of participants in both ciprofloxacin groups had their bacteria cleared, compared to about 40% in the placebo group. On a quality-of-life questionnaire about respiratory symptoms (scored 1–100, where lower scores mean better health), the Cipro 28 group's score changed by −8.92 points, the Cipro 14 group by −9.02 points, the Placebo 28 group by −2.91 points, and the Placebo 14 group by −11.50 points from the start of the trial. The reported data also shows that new bacteria (not present at the start) were detected at the end of treatment in roughly 62–68% of participants across all groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00531882 · results posted 28 July 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 25 participants across three groups. Ten people received pioglitazone (a diabetes-related medicine), ten received simvastatin (a cholesterol-related medicine), and five received ibuprofen (a common anti-inflammatory painkiller). All 25 participants completed the study with no drop-outs. The trial was measuring how each of these medicines affected the number of a type of white blood cell — called neutrophils (or PMNs) — that travel to the lining of the mouth. Neutrophil counts were measured using a non-invasive mouthwash technique at three points: before treatment started (baseline), during treatment, and during a recovery period after treatment ended. The reported data shows the results as a percentage change in the average neutrophil count in the mouth between the baseline period and the treatment period. For the pioglitazone group, the average count increased by 6.4%. For the simvastatin group, the average count decreased by 19.6%. For the ibuprofen group, the average count decreased by 28.4%. The reported data also notes that a statistical test (a paired t-test, which compares two sets of measurements from the same group of people) was used to assess whether those changes were meaningful, though the specific test results and any recovery-period data were not included in the figures submitted to ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01859390 · results posted 8 June 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01859390) enrolled 73 children and young people with cystic fibrosis — 36 in the AquADEKs-2 group (a specialised multivitamin) and 37 in the Control Multivitamin group. The trial ran for 18 weeks and was primarily measuring changes in a substance in sputum (mucus coughed up from the lungs) called myeloperoxidase, or MPO, which is linked to inflammation. A number of secondary measurements were also recorded, including lung function, body mass index (BMI), the time until a lung flare-up occurred, and how many unwanted health events were reported. The reported data shows that for the primary measure — the change in sputum MPO levels over 16 weeks — the AquADEKs-2 group had an average change of −0.10 (on a logarithmic scale, meaning a small decrease), while the Control Multivitamin group had an average change of +0.03 (a small increase). For the secondary measures: lung function (measured as FEV1 % predicted, a standard way of assessing how much air someone can forcefully breathe out) changed by −0.76 in the AquADEKs-2 group and −2.20 in the Control Multivitamin group. BMI change was +0.16 kg/m² in the AquADEKs-2 group and +0.13 kg/m² in the Control Multivitamin group. The median time to a first lung flare-up was 102 days in the AquADEKs-2 group and 96 days in the Control Multivitamin group. Regarding unwanted health events, 33 participants in the AquADEKs-2 group and 34 in the Control Multivitamin group reported at least one adverse event; 8 and 13 participants respectively reported at least one serious adverse event. The rate of adverse events was 0.34 per participant-week (AquADEKs-2) versus 0.37 (Control Multivitamin), and serious adverse event rates were 0.04 versus 0.05 per participant-week. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01764841 · results posted 24 May 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 416 people in total across four groups. Participants received either an inhaled antibiotic called ciprofloxacin (delivered as a dry powder inhaler, or DPI) or a placebo (an inactive treatment), and were placed on one of two dosing schedules — either 28 days on/off or 14 days on/off. The trial ran for 48 weeks and was primarily measuring how long it took before participants had their first serious lung flare-up (called an exacerbation), defined as a worsening of at least three symptoms alongside fever or fatigue and the need for antibiotic tablets or injections. Around 334 people completed the study, with the remainder not finishing for various reasons. The reported data shows that for the primary outcome — time to first flare-up — the ciprofloxacin 28-days on/off group had a reported median of 336 days before their first flare-up, compared with 186 days in the pooled placebo group. A median figure means the point at which half the group had experienced an event. The result for the ciprofloxacin 14-days on/off group was listed as "not available" in the reported data. For the secondary outcomes, the reported data shows the number of participants who experienced flare-ups varied across groups over 48 weeks. Regarding pathogen eradication (clearing bacteria detected at the start of the trial), the reported figures ranged from 24.1% to 39.0% across the ciprofloxacin groups and 16.7% to 33.3% in the placebo group, depending on the bacteria type. On a respiratory quality-of-life questionnaire (scored 0–100, where lower is better), the reported average change from the starting score ranged from −8.17 to −7.20 points in the ciprofloxacin groups and from −4.23 to +2.78 points in the placebo groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01931839 · results posted 12 May 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01931839) enrolled 1,163 participants in total across seven groups. It was split into two parts — Part A and Part B — and tested a combination of two investigational medicines, lumacaftor (LUM) and ivacaftor (IVA), given at different doses, against a placebo (a dummy treatment with no active ingredient). A small observational group of 19 people was also included. The trial was measuring, as its primary focus, how many participants experienced adverse events (unwanted medical occurrences during the study) and serious adverse events (more severe events such as hospitalisation or life-threatening situations) while taking the treatments. A key secondary focus was tracking changes in lung function, measured by a breathing test called FEV1 — essentially how much air a person can forcefully breathe out in one second. The reported data shows that in Part A, adverse events of any kind were recorded in 331 out of 335 participants in the LUM 600 mg once-daily/IVA group, 177 out of 178 in its placebo group, 333 out of 340 in the LUM 400 mg twice-daily/IVA group, and 176 out of 176 in its placebo group. Serious adverse events were recorded in 156, 77, 143, and 89 participants in those same groups respectively. In Part B, adverse events were recorded in 52 out of 55 participants in the active treatment group and 57 out of 60 in the placebo group, with serious adverse events in 18 and 21 participants respectively. For the lung function measure in Part A, the reported data shows small increases from baseline across all groups at the time points provided — for example, absolute changes at one measured point ranged from roughly +2.5 to +3.4 percentage points across the active and placebo groups. In Part B, the reported data shows small negative changes from baseline (slight decreases) at all measured time points across both the active and placebo groups, ranging from around −2 to −5.4 percentage points in the active group and −1.8 to −3.4 percentage points in the placebo group. The data for several of the later time points (weeks 36 through 72) was not reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02015663 · results posted 29 March 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02015663) involved 32 people in total — 16 in a group taking Tobramycin Inhalation Powder once daily, and 16 taking it twice daily. The trial was measuring things related to lung function and respiratory health in participants, including how much air they could forcefully breathe out (a common way of checking how well the lungs are working), as well as levels of a specific bacteria called *Pseudomonas aeruginosa* in their sputum (mucus coughed up from the lungs), and whether participants needed to be hospitalised for breathing-related reasons. It is worth noting that very few participants completed the study — only 1 in the once-daily group and 4 in the twice-daily group finished, while 15 and 12 respectively did not complete it. The reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of the outcome measures — neither the primary measure (change in lung function, specifically FEV1 percent predicted) nor any of the secondary measures (other lung function tests, bacteria levels in sputum, or time to first hospitalisation). This means the actual measurements for these outcomes were not reported in the structured results data available on the registry. Because no outcome numbers were provided in the submitted data, it is not possible to describe what the trial found in terms of the measurements it set out to collect. The reasons for the large number of participants not completing the study and the absence of reported results data are not explained in the information available on ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00528190 · results posted 23 March 2017

  According to the results reported on ClinicalTrials.gov, this trial involved 35 people in total — 18 received a medicine called itraconazole and 17 received a placebo (a dummy treatment with no active ingredient). The trial ran over 24 weeks and was measuring how many participants in each group had a serious lung flare-up (called a respiratory exacerbation) that was bad enough to need antibiotics given through a drip (intravenously). The reported data shows that out of the 18 people in the itraconazole group, 4 experienced a lung flare-up requiring intravenous antibiotics during the 24-week period. In the placebo group, 5 out of 17 people experienced the same. Secondary outcome measure results were not reported in the data available, so no further figures can be described here. Almost all participants finished the trial — all 18 in the itraconazole group completed it, and 16 out of 17 in the placebo group completed it, with one person not finishing for reasons not detailed in the reported data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02598128 · results posted 20 January 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02598128) enrolled 34 participants and looked at a device called RELiZORB, which is an enzyme cartridge used with tube feeding. The trial was structured in three periods: a baseline period where participants continued their usual tube feeding (Period A), a crossover period where participants were split into two groups — one receiving a placebo first then RELiZORB, the other receiving RELiZORB first then placebo (Period B) — and a final period where everyone used RELiZORB alongside their usual feeding (Period C). The trial measured two main things: the number of participants who experienced adverse events (unwanted health occurrences) or unexpected device-related effects, and the level of certain fatty acids (DHA and EPA, types of fats important for the body) in participants' blood over 24 hours. The reported data shows that during the various periods, the number of participants who experienced at least one adverse event was: 4 during the usual feeding baseline period, 6 during the placebo phase, 1 during the RELiZORB phase, and 2 during the final period using RELiZORB with usual feeding. No unexpected device-related effects were reported in the final period. For the fatty acid blood levels, the reported data shows an average measured value of approximately 537 ug\*h/mL (micrograms multiplied by hours per millilitre — a way of capturing the total amount of fatty acid in the blood over time) for the RELiZORB group, compared with approximately 192 ug\*h/mL for the control group. The reported data also shows responses about breakfast habits after overnight tube feeding during the final period: 11 participants reported eating no breakfast, 14 reported a small breakfast, 6 reported a normal breakfast, 1 reported "other," and none reported eating a big breakfast. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01897233 · results posted 5 December 2016

  According to the results reported on ClinicalTrials.gov, this trial looked at a combination medicine called lumacaftor/ivacaftor (also known as LUM/IVA) in children with cystic fibrosis. The trial had two parts. Part A ran for 14 days and involved 10 children aged 6 to 11 years (5 aged 6–8 and 5 aged 9–11); all 10 completed this part. Part B ran for 24 weeks and involved 58 children, of whom 54 completed it and 4 did not. Part A was mainly focused on measuring how much of the medicine got into the bloodstream, while Part B tracked medical events that occurred during treatment. The reported data shows that in Part A, the amount of lumacaftor measured in the blood 4 hours after a dose was 15,200 nanograms per millilitre (ng/mL) on Day 1 and 24,500 ng/mL on Day 14. For ivacaftor, the corresponding figures were 1,920 ng/mL on Day 1 and 622 ng/mL on Day 14. A separate measure of overall drug exposure over a dosing period (essentially the total amount of drug in the blood over time) was reported as 387,600 ng·hr/mL for lumacaftor and 6,838 ng·hr/mL for ivacaftor. In Part A, 4 out of 10 participants experienced at least one adverse event (an unexpected medical occurrence during the study) and no serious adverse events were recorded; 3 out of 10 experienced a particular category of adverse event, again with no serious adverse events in that category. In Part B, the reported data shows that 55 out of 58 participants experienced at least one adverse event, and 4 out of 58 experienced a serious adverse event. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02178540 · results posted 23 May 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 45 participants, all of whom completed the study with no drop-outs. The trial involved people with cystic fibrosis (CF) — and their carers where relevant — who had never used a specific inhaler device called the TOBI Podhaler before and had received no training on it. The aim was to test whether the written instructions for use (IFU) that come with the device clearly communicated how to use it correctly. Participants were asked to inhale one dose (the contents of four capsules containing a placebo, meaning no active medicine) using the device, while researchers watched for mistakes or near-mistakes. The reported data shows that researchers tracked six specific types of critical errors — agreed upon with the US Food and Drug Administration — to see whether any mistakes could be traced back to the instructions being unclear or confusing. According to the results reported on ClinicalTrials.gov, across the six critical error categories, the numbers of participants whose errors were attributed to a failure to understand the instructions were: 0, 0, 0, 3, 0, and 6. This means that for four of the six error types, no participants were recorded as having made that mistake due to unclear instructions, while for the remaining two error types, 3 and 6 participants respectively were recorded as having errors attributed to a lack of clarity in the instructions. No further breakdown of what each specific error category involved was provided in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01082367 · results posted 27 April 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 51 people in total — 26 in one group and 25 in the other. The trial was looking at an inhaled antibiotic called TOBI (tobramycin inhaled solution) in people who had a bacterial infection called *Pseudomonas aeruginosa* (a common lung bacteria in people with cystic fibrosis). It used a "crossover" design, meaning one group received TOBI first and then a placebo (a dummy treatment with no active medicine), while the other group received the placebo first and then TOBI. The main thing the trial was measuring was how many participants no longer had the bacteria detectable in their sputum (mucus) or throat swabs after the first round of treatment. The reported data shows that after the first treatment cycle, 84.6% of participants in the TOBI-first group had no detectable bacteria, compared with 24.0% in the placebo-first group. For a secondary measure — looking at results 28 days after the second treatment cycle was finished — the reported figures were 92.3% bacteria-free in the TOBI-first group and 83.3% in the placebo-first group. A further secondary measure looked at how many participants were bacteria-free by the end of the entire double-blind (neither participants nor researchers knew who received which treatment) phase of the trial; the reported data shows 76.0% in the TOBI-first group and 47.8% in the placebo-first group were free of the bacteria at that point. It is worth noting that this was a relatively small trial, and the numbers of participants who completed each stage varied. These percentages describe what was observed and recorded in this specific group of trial participants only. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02415959 · results posted 17 February 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 70 people in total, split evenly across five groups of 14. Four groups received different doses of an immediate-release form of a pancreatic enzyme capsule called Creon IR (labelled low, medium, high, and maximum dose), while the fifth group received the standard delayed-release/gastric-resistant form of Creon already on the market. The trial's main goal was to measure how well each treatment allowed the body to absorb fat from food — a figure called the Coefficient of Fat Absorption (CFA), which is simply the percentage of eaten fat that the body takes in rather than passing out in stools. Secondary measurements included how well nitrogen (a marker of protein) was absorbed, how much fat appeared in stools, and total stool weight. The reported data shows that for the main measurement — fat absorption — the five groups recorded the following percentages: Creon IR Low Dose 71.0%, Creon IR Medium Dose 70.9%, Creon IR High Dose 71.8%, Creon IR Maximum Dose 75.9%, and the standard Creon 92.3%. For nitrogen absorption, the reported figures were 71.0%, 73.2%, 76.2%, 79.9%, and 84.8% respectively across the same groups. The reported data shows that stool fat over 72 hours was 87.5 g, 87.1 g, 84.1 g, 73.0 g, and 23.5 g for each group in the same order, and total stool weight over 72 hours was 889.0 g, 905.3 g, 793.8 g, 755.7 g, and 545.7 g respectively. Regarding side effects, the trial also tracked how many participants in each group experienced adverse events (unwanted health changes noticed during treatment). The reported numbers were: 10 participants in the Creon IR Low Dose group, 9 in the Medium Dose group, 7 in the High Dose group, 9 in the Maximum Dose group, and 7 in the standard Creon group. No further breakdown of what those events were is included in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01684410 · results posted 8 February 2016

  According to the results reported on ClinicalTrials.gov, this trial involved 30 people in total, split evenly into three groups of 10: one group received a lower dose of Alpha-1 HC (100 mg), one received a higher dose (200 mg), and one received a placebo (a dummy treatment with no active ingredient). All 30 participants completed the trial. The study was measuring how often unwanted health events — called adverse events — occurred, as well as changes in two breathing measurements taken before and after inhaling the study product. The reported data shows that the primary thing being tracked was how many people in each group experienced any adverse event. In the 100 mg group, 100% of participants had at least one adverse event reported; in the 200 mg group, 80% did; and in the placebo group, 60% did. For the breathing measurements, the trial also tracked changes in two lung function tests. The first, FEV1 (roughly, how much air a person can forcefully breathe out in one second), changed by +1.5% from the starting point in the 100 mg group, −2.1% in the 200 mg group, and +0.5% in the placebo group, at week 3. The second measure, FVC (the total amount of air a person can breathe out in one go), changed by +1.2% in the 100 mg group, −2.3% in the 200 mg group, and −0.9% in the placebo group at week 3. It is worth noting that this was a very small trial with only 10 people per group, and the figures above simply describe what was recorded — they do not on their own tell us whether any of these differences are meaningful. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01775137 · results posted 6 November 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 45 participants, all of whom received tobramycin inhalation powder (an antibiotic inhaled as a dry powder). The study ran over 12 treatment cycles and was primarily measuring how many participants experienced unwanted medical events (called adverse events) during that time. It also tracked changes in lung function, levels of a specific bacteria in sputum (mucus coughed up from the lungs), and the use of other antibiotics during the study period. Of the 45 who started, 34 completed the trial and 11 did not finish. The reported data shows that out of 45 participants, 39 experienced at least one adverse event during the study. Of those, 18 were described as mild, 19 as moderate, and 11 as severe. Ten participants experienced a serious adverse event (a more significant medical event such as one requiring hospitalisation), and 1 participant died. Regarding lung function — measured as how much air a person can breathe out in one second — the reported data shows small fluctuations across the 12 cycles, with changes ranging from a relative increase of around 5% early in the study to a relative decrease of around 7.5% at later points. Levels of a bacteria called *Pseudomonas aeruginosa* in sputum samples showed reductions from baseline across most of the cycles measured. The reported data also shows that approximately 78% of participants used additional antibiotic medicines at some point during the study. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01225211 · results posted 5 October 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01225211) enrolled 311 participants across four groups (called cohorts), testing different doses of two investigational drugs — lumacaftor (LUM) and ivacaftor (IVA) — alone and in combination. The trial was designed to look at how well the drugs were tolerated (by tracking unwanted medical events), and also to measure changes in two physical markers: sweat chloride levels (a marker used in cystic fibrosis monitoring) and a lung function test called percent predicted FEV1, which measures how much air a person can forcefully breathe out in one second. The reported data shows the following numbers. For adverse events (any unwanted medical occurrence during the study), the figures varied across groups: in Cohort 1, 12 placebo participants and 29 LUM-only participants reported adverse events during the first period, with 14–15 reporting events during the combination period. In Cohorts 2 and 3, between 7 and 37 participants per group reported adverse events depending on the dose and period. In Cohort 4, 53 of 63 placebo participants and 52 of 62 active-drug participants reported adverse events, with serious adverse events reported in 5 placebo and 9 active-drug participants. For sweat chloride, changes from the comparison point ranged from approximately +0.3 to −9.1 mmol/L across the different dose combinations, compared with small increases (around +0.5 to +1.6 mmol/L) in placebo groups. For the lung function measure in Cohort 4, the placebo group showed a change of −1.23 percent predicted FEV1, while the active drug group showed a change of −0.62 percent predicted FEV1 — both figures representing small decreases from baseline. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01807949 · results posted 1 September 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01807949) involved 563 people with cystic fibrosis, split across three groups: 187 received a placebo (a dummy treatment with no active ingredient), 187 received a combination called lumacaftor 600 mg once daily plus ivacaftor 250 mg twice daily, and 189 received lumacaftor 400 mg twice daily plus ivacaftor 250 mg twice daily. The trial ran for 24 weeks and was primarily measuring changes in lung function, specifically a breathing test called FEV1 — which measures how much air a person can forcefully breathe out in one second, expressed as a percentage of what would be expected for someone of their age, sex, height and race. The reported data shows that, for the main outcome (change in that lung function score from the start to week 24), the placebo group's score changed by −0.15 percentage points on average, while the two active treatment groups changed by +2.46 and +2.85 percentage points respectively. For a secondary measure looking at the same lung function result expressed differently (as a relative percentage change), the placebo group showed 0.00%, while the two active groups showed approximately 4.4% and 5.3%. The reported data also shows changes in body mass index (BMI): the placebo group's BMI changed by +0.07 kg/m², compared with +0.48 and +0.43 in the active groups. On a respiratory symptom quality-of-life questionnaire (scored 0–100, where higher means fewer symptoms), all three groups showed some improvement from their starting scores, with the placebo group rising by 2.81 points and the two active groups rising by 5.02 and 5.66 points. Regarding lung flare-ups (called pulmonary exacerbations), the reported rate per year was 1.18 events in the placebo group, compared with 0.82 and 0.67 in the active treatment groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01807923 · results posted 31 August 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01807923) enrolled 559 people across three groups: 187 received a placebo (a dummy treatment with no active ingredient), 185 received a combination called LUM 600 mg once daily with IVA 250 mg twice daily, and 187 received LUM 400 mg twice daily with IVA 250 mg twice daily. The trial was measuring changes in lung function, body weight, breathing-related quality of life, and the rate of lung flare-ups (called pulmonary exacerbations) over 24 weeks in people with cystic fibrosis. The main thing being measured was a breathing test called FEV1 — essentially how much air a person can forcefully breathe out in one second — expressed as a percentage of what would normally be expected for someone of the same age, sex, height, and race. The reported data shows that for the primary measure — the change in that lung function score from the start of the trial to week 24 — the placebo group's score shifted by –0.44 percentage points, the LUM 600/IVA group's score shifted by +3.59 percentage points, and the LUM 400/IVA group's score shifted by +2.16 percentage points. For the secondary measures, the reported data shows small increases in BMI (a measure of body weight relative to height) across all three groups (placebo +0.19, LUM 600/IVA +0.35, LUM 400/IVA +0.32 kg/m²). On a breathing symptoms quality-of-life questionnaire scored from 0 to 100 (where higher means fewer symptoms), the placebo group's score rose by 1.10 points, the LUM 600/IVA group's by 4.98 points, and the LUM 400/IVA group's by 2.60 points. The proportion of participants whose lung function score improved by 5% or more was reported as 22.3% in the placebo group, 46.4% in the LUM 600/IVA group, and 36.8% in the LUM 400/IVA group. The reported rate of lung flare-ups per year was 1.07 for the placebo group, 0.77 for the LUM 600/IVA group, and 0.71 for the LUM 400/IVA group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00431964 · results posted 19 August 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 263 participants in total — 131 in the active treatment group and 132 in the placebo (dummy treatment) group. The trial was measuring changes in lung function over approximately six months (168 days). Lung function was assessed using a measure called FEV1, which stands for the amount of air a person can forcefully breathe out in one second — essentially a snapshot of how well the airways are working. The goal was to see whether there was a difference in how much this figure changed between the two groups over the course of the study. The reported data shows that, by the end of the treatment period, the active group's FEV1 had increased by an average of 0.08 litres from where it started, while the placebo group's FEV1 had increased by an average of 0.06 litres. The reported data does not include any further statistical detail — such as whether this difference between the two groups was considered meaningful from a statistical standpoint — so no further breakdown can be described here. It is also worth noting that the trial report shows that most participants completed the study: 125 out of 131 in the active group and 124 out of 132 in the placebo group reached the end. No additional outcome measures beyond the primary lung function result were included in the structured data submitted to ClinicalTrials.gov, so no further results can be described. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01117012 · results posted 7 July 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01117012) enrolled 192 people in total — 89 in the "Placebo/VX-770" group (meaning they received a dummy treatment first, then switched to the active drug VX-770, also known as ivacaftor) and 103 in the "VX-770/VX-770" group (meaning they received VX-770 throughout). The trial was measuring safety events and several aspects of lung function and quality of life over a period of up to 96 weeks in people with cystic fibrosis. Of the 192 who started, 179 completed the study. The reported data shows that, across all 190 participants who received VX-770 at any point, 70 experienced what are classified as serious adverse events — that is, medical events serious enough to involve hospitalisation, be life-threatening, or cause significant disability, among other criteria. The remaining participants experienced non-serious adverse events, which covers any other unintended medical occurrence noticed during the study. For lung function — measured as the amount of air a person can forcefully breathe out in one second (called FEV1) — the Placebo/VX-770 group showed an annualised rate of decline of −0.30 percentage points per year, while the VX-770/VX-770 group showed −1.23 percentage points per year. When looking at the overall change in lung function from the starting point, the Placebo/VX-770 group showed increases of around +9.3 percentage points at week 48 and +9.8 at week 96, while the VX-770/VX-770 group showed smaller changes of −0.44 and +0.16 at those same time points. On a respiratory symptom questionnaire (scored 0–100, where higher means fewer symptoms), the Placebo/VX-770 group reported changes of +7.43 at week 48 and +10.05 at week 96, compared with −1.94 and +0.77 for the VX-770/VX-770 group. The reported rate of lung flare-ups (called pulmonary exacerbations) was 0.463 events per year in the Placebo/VX-770 group and 0.658 in the VX-770/VX-770 group, with those flare-ups lasting an average of 10.89 days per year and 17.27 days per year respectively. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01429259 · results posted 27 March 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 30 participants, all of whom completed the study with no drop-outs. The trial was looking at how the antibiotic meropenem moves through the body when it is given as a slow, three-hour drip (called a prolonged infusion) in people with cystic fibrosis. Specifically, researchers measured two things about how the drug behaves in the body: how quickly the body clears the drug away, and how widely the drug spreads through the bloodstream. The reported data shows that, on average across the group, the body cleared meropenem at a rate of 0.36 litres per hour per kilogram of body weight — in plain terms, this is a measure of how fast the drug is removed from the body. The reported data also shows that the drug spread through the central part of the bloodstream at a volume of 0.21 litres per kilogram of body weight, which reflects how much of the body that portion of the drug was distributed into. These two figures come from a type of mathematical modelling that estimates drug behaviour across the whole group. The trial also listed three secondary measures — side effects and safety monitoring, how practical and burdensome patients or parents found the three-hour drip (using a cystic fibrosis-specific questionnaire), and how drug levels related to lung function — however, no numerical results for any of these secondary outcomes were reported in the data submitted to ClinicalTrials.gov, so those findings are not available here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01519661 · results posted 10 February 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 157 participants, all of whom received a treatment called Tobramycin Inhalation Powder (TIP), an inhaled antibiotic powder. Of those who started, 96 completed the study and 61 did not finish. The trial was measuring how often participants experienced unwanted medical events (called adverse events), as well as tracking changes in lung function and levels of a bacteria called *Pseudomonas aeruginosa* — a common bacteria found in the lungs of people with cystic fibrosis — over the course of treatment. The reported data shows that 85.4% of participants experienced at least one treatment-emergent adverse event (meaning an unwanted medical event that occurred after starting the study drug), 31.2% experienced a serious adverse event, and no deaths (0.0%) were reported. For lung function, the trial tracked several breathing measurements over time. One key measure — how much air a person can forcefully breathe out in one second (called FEV1) — showed small changes across different time points, ranging from a +0.8% change at one point to a −3.5% change at another, compared to where participants started. The reported data also shows that levels of *Pseudomonas aeruginosa* bacteria in sputum (mucus coughed up from the lungs) decreased from baseline at most time points measured, with the largest reported reduction being −1.6 units on a logarithmic scale (a way of expressing very large number ranges in a compressed form). The trial also measured how much of the antibiotic was needed to inhibit the bacteria in the lab, tracking this across several time points. The reported data shows that this figure remained relatively stable at most points but showed higher values at later time points for one measure, which may indicate changes in bacterial sensitivity — however, the data as reported does not include further interpretation of this finding. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00416182 · results posted 14 November 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 16 people in total — 8 received a drug called Pulmozyme (also known as dornase alfa) and 8 received a placebo (a dummy treatment with no active ingredient). The trial was looking at whether Pulmozyme, delivered directly into the sinuses after surgery, could reduce sinus disease in people with cystic fibrosis over a 12-month period. Not everyone finished the study: 5 people in the Pulmozyme group and 6 in the placebo group completed it, with 3 and 2 people respectively not completing the trial (the reasons were not detailed in the reported data). The reported data shows the following numbers across the main and secondary measurements. For sinus appearance on CT scans (scored on a 0–24 scale, where a higher number means more disease), the Pulmozyme group's score changed by 5.875 units and the placebo group's score changed by 3 units — both representing a reduction from baseline, meaning less disease was seen at one year in both groups. For the appearance of the nasal passages as assessed by a surgeon using a camera (scored 0–2, where lower is better), the Pulmozyme group's score changed by +0.2 units and the placebo group's score changed by −1 unit from baseline. On a sinusitis symptom survey (scored 0–24, where lower is better), the Pulmozyme group's score reduced by 8.5 units and the placebo group's by 3 units. For lung function — measured as how much air a person can forcefully breathe out in one second — the reported change over the year was +0.8 percentage points for the Pulmozyme group and +5.4 percentage points for the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01614470 · results posted 29 October 2014

  According to the results reported on ClinicalTrials.gov, this trial looked at a medicine called ivacaftor in people with cystic fibrosis. The trial had two parts. In Part 1, 39 participants were split into two groups in a crossover design — meaning everyone received both ivacaftor and a dummy pill (placebo) at different times, each for 8 weeks, with a break in between. In Part 2, 36 participants took ivacaftor openly for a further 16 weeks. The trial was measuring lung function (using a breathing test called FEV1, which measures how much air a person can blow out in one second), as well as body weight relative to height (BMI), and the level of chloride in sweat — a marker commonly tracked in cystic fibrosis. The reported data shows the following numbers for the main lung function measure in Part 1: at the start, participants' average FEV1 was around 76–79% of what would be expected for a person of their age, sex, and height. During the 8-week period on ivacaftor, the average change from that starting point was +8.1 percentage points, while during the placebo period it was -5.9 percentage points. For Part 2, looking at participants who took ivacaftor for a total of 24 weeks, the reported average change in FEV1 from their baseline was +13.5 percentage points. For sweat chloride in Part 1, the reported average change was -55.8 mmol/L on ivacaftor compared with -5.6 mmol/L on placebo; over 24 weeks in Part 2 it was reported as -59.2 mmol/L. For BMI, the reported average change at 8 weeks in Part 1 was +0.75 kg/m² on ivacaftor versus +0.04 kg/m² on placebo, and over 24 weeks in Part 2 the reported average change was +1.26 kg/m². These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01417481 · results posted 23 October 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 15 people in total who completed the study. It used a "crossover" design, meaning each participant received both glycine (an amino acid supplement) and a placebo (an inactive substitute) at different times, with a two-week break in between. One group started with glycine for eight weeks and then switched to placebo, while the other group did the opposite. The trial was primarily measuring changes in the levels of certain inflammation-related proteins in the blood and in sputum (phlegm) after eight weeks on each treatment. It also looked at changes in respiratory symptoms such as cough severity, appetite, breathlessness, and energy levels. The reported data shows that for the inflammation-related proteins measured in the blood, the numbers (expressed as a mathematical transformation of percentage change from the starting point) ranged from around −0.44 to +0.30 in the glycine group and from around −0.29 to +0.23 in the placebo group across the different proteins tested. For one specific blood protein (TNF-alpha), the reported figure was −0.39 in the glycine group and +0.20 in the placebo group. For inflammation-related proteins measured in sputum, the reported figures were similarly small and varied in direction across the different proteins in both groups. For the secondary symptom scores, results were expressed as a percentage of each participant's starting score — a figure below 100% means the score went down from the start, while above 100% means it went up. The reported data shows figures such as 81.1% (glycine) versus 89.1% (placebo) for one symptom category, and 89.1% (glycine) versus 132.1% (placebo) for another, with other symptom scores sitting closer together between the two groups. It is worth noting that this was a very small trial, and the reported data does not include enough detail to draw broad conclusions. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01755455 · results posted 6 October 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 22 people in total, split into two groups of 11. It was a "crossover" trial, meaning everyone took both treatments at different times — one group started with a placebo (a dummy pill with no active ingredient) for six weeks, then switched to ferrous sulfate (an iron supplement) for six weeks, while the other group did the reverse. There was a four-week break between the two phases to allow the body to return to its starting point. The trial was measuring changes in iron-related markers in the blood and in sputum (mucus coughed up from the lungs). The reported data shows the following changes from the starting point. For the main measurement — haemoglobin (a protein in red blood cells that carries oxygen, measured in grams per decilitre) — the placebo group showed a change of +0.12 gm/dl, while the ferrous sulfate group showed a change of +0.09 gm/dl. For iron levels in the blood, the placebo group showed a change of +13.7 mcg/dl, compared to −4.2 mcg/dl in the ferrous sulfate group. For transferrin saturation (a measure of how much iron is being carried in the blood), the placebo group showed a change of +4.7%, compared to −1.8% in the ferrous sulfate group. For iron measured in sputum, the placebo group showed a change of +0.13 ng/mg, while the ferrous sulfate group showed a change of +0.42 ng/mg. It is worth noting that these are the numbers as submitted, and no further context — such as whether the differences between groups were considered meaningful — was included in the structured data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00486837 · results posted 21 August 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 72 people in total — 37 in a group receiving an inhaled treatment aimed at reaching the outer (peripheral) parts of the lungs, and 35 in a group where the treatment was aimed at the larger, more central (bronchial) airways. By the end of the study, 28 people in each group had completed it. The trial was measuring changes over four weeks in several markers found in mucus samples coughed up by participants (called "induced sputum"), including levels of an enzyme called free elastase, a protective protein called alpha-1-antitrypsin, immune proteins, bacteria counts, and certain immune cells called neutrophils. The reported data shows the following changes from the start of the trial to week four. For the main (primary) measure — free elastase levels in the mucus — the peripheral deposition group showed a change of −7.42 µg/mL (a decrease), while the bronchial deposition group showed a change of +6.11 µg/mL (an increase). For the secondary measures: the protective protein alpha-1-antitrypsin changed by +16,741 µg/mL in the peripheral group and +6,469 µg/mL in the bronchial group; immune protein fragments (IgG) changed by +0.005 µg/mL and −0.48 µg/mL respectively; total bacteria in the mucus changed by approximately +22,673,276 and +5,021,353 colony-forming units per gram respectively; a specific bacteria called Pseudomonas changed by −40.1 and −22.3 colony-forming units per gram; and the percentage of neutrophil immune cells changed by −20.8% and −8.2% respectively. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01747330 · results posted 15 July 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 40 infants or young children who were given a pancreatic enzyme supplement called Creon Micro (in the form of tiny spheres) over a period of 84 days (about 12 weeks). All 40 participants who started the trial also completed it. The trial was measuring several things: changes in body weight and height, how often participants had bowel movements, what their stools looked like, and how well caregivers felt their child accepted the treatment. The reported data shows that, on average, participants gained 0.75 kg in body weight and grew 0.026 metres (about 2.6 cm) in height over the 84-day period. During the treatment period, the average number of bowel movements per day was reported as 2.1. Stools were described as normal in consistency on about 55.4% of days, as recorded by caregivers. When caregivers were asked how well their child accepted the treatment, 37.5% rated acceptance as "very good," 52.5% rated it as "good," 10% rated it as "moderate," and none rated it as "unsatisfactory." Regarding unwanted effects, the reported data shows that 16 out of 40 participants experienced at least one adverse event (an unwanted or unexpected health event noted during the trial), though the data does not provide further detail on what those events were. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00591864 · results posted 9 July 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 89 participants, with 84 completing the study and 5 not completing it. All participants were imaged using two different breast scanning methods — MBI (Molecular Breast Imaging) and MRI (Magnetic Resonance Imaging). The trial was measuring how well each method could detect breast cancer, both at the level of individual patients and at the level of individual tumours, as well as how often each method correctly identified people who did *not* have cancer. The reported data shows that for the primary outcome — detecting cancer at the patient level — 25 out of 27 women who were confirmed to have breast cancer (through surgery or biopsy) were identified. For the first secondary outcome, looking at individual tumours rather than whole patients, 32 out of 36 confirmed tumours were detected. For the second secondary outcome, known as specificity (meaning how often the test correctly gave a "no cancer" result for people who truly did not have cancer), 38 out of 37 participants were recorded — noting that this figure as reported appears inconsistent, since the number detected cannot exceed the total, and this data was reported as submitted to ClinicalTrials.gov without further clarification. It is unclear from the submitted data which numbers correspond to which imaging method, as the measurements appear to reflect a combined group rather than being broken down separately for MBI and MRI. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00885365 · results posted 20 June 2014

  According to the results reported on ClinicalTrials.gov, this trial compared two inhaled antibiotic treatments — Bramitob and TOBI — both of which contain the same antibiotic (tobramycin) delivered by nebuliser. The trial enrolled 159 people in the Bramitob group and 165 in the TOBI group, though slightly different numbers were counted depending on how the analysis was done. The main thing the trial was measuring was a breathing test called FEV1 — short for "forced expiratory volume in one second" — which captures how much air a person can forcefully breathe out in one second, expressed as a percentage of what would be typical for someone of the same age and size. The trial ran in cycles of four weeks on treatment followed by four weeks off treatment. The reported data shows that, for the primary measurement (change in FEV1 at the end of the treatment period), the Bramitob group showed an average improvement of about 6.99 percentage points from their starting point, while the TOBI group showed an average improvement of about 7.51 percentage points. For the secondary breathing measurements taken at weeks 2, 4, and 8, the reported numbers were broadly similar between the two groups across all time points. For example, changes in the absolute volume of air breathed out (measured in litres) were reported as roughly 0.18–0.20 litres for Bramitob and 0.19–0.22 litres for TOBI during the treatment weeks, falling to around 0.16 litres each at week 8 (the off-treatment check). Other breathing measures — including how much air could be forcefully exhaled overall (FVC) and airflow in the middle part of a breath (FEF 25–75%) — showed similarly small changes in both groups across all time points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01404234 · results posted 1 May 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 61 people, all of whom received a treatment called aztreonam lysine for inhalation (AZLI). Two people did not complete the trial. The study was looking at a treatment given in repeated 28-day courses, and was measuring things like how many people stopped taking it early due to side effects or tolerability concerns, as well as changes in lung function, symptom scores, bacterial levels in mucus, and how often participants needed extra antibiotics or a hospital stay for breathing problems. The reported data shows that none of the 61 participants (0%) stopped taking the study drug early because of safety or tolerability concerns. When it came to lung function — measured as FEV1 % predicted, which compares a person's breathing capacity to what would be expected for someone of similar age, sex, and body size — the reported changes from the starting point across the three treatment courses were increases of 4.73, 1.72, and 1.65 percentage points respectively. A symptom questionnaire score (where higher means fewer symptoms, on a scale of 0 to 100) showed changes of 8.66, 9.38, and 5.90 points across the three courses. Levels of a particular bacteria (*Pseudomonas aeruginosa*) in mucus samples showed reductions of 2.6, 2.0, and 1.2 units (measured on a scientific scale) across the courses. Around 42.6% of participants used additional antibiotics during one period and 57.4% during another, while 18% of participants were reported to have been hospitalised at least once for a breathing-related event, compared to 82% who were not. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01313624 · results posted 16 April 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 266 adults split into two groups: 134 people received the active treatment (aztreonam for inhalation solution, or AZLI) throughout the study, while 132 people started on a placebo (an inactive treatment) before switching to the active treatment in a later phase. The trial was measuring changes in respiratory symptoms using a quality-of-life questionnaire (called the QOL-B), which uses a scale of 0 to 100 where a higher score means better quality of life for breathing-related symptoms. It also tracked how long it took before participants experienced a significant worsening of their respiratory condition (called a "protocol-defined exacerbation"). The reported data shows that, at Day 28 (the end of the first treatment course), the AZLI-AZLI group had an average increase of 7.4 points on the respiratory symptoms scale, while the Placebo-AZLI group had an average increase of 5.7 points. At Day 84 (the end of the second course), the reported data shows the AZLI-AZLI group again recorded an average increase of 7.4 points, compared to 4.7 points for the Placebo-AZLI group. Regarding the time to a significant worsening of respiratory symptoms, the reported data shows the Placebo-AZLI group had a reported figure of 120 days, while the figure for the AZLI-AZLI group was not reported in the submitted data. It is worth noting that not all participants who started the trial completed it — in the first phase, 38 people in the AZLI-AZLI group did not complete it, compared to 10 in the Placebo-AZLI group. These dropout numbers were much smaller in the second phase of the trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01314716 · results posted 16 April 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 274 people in total — 136 in one group and 138 in the other. Participants were adults with a lung condition called bronchiectasis and were randomly assigned to receive either the inhaled antibiotic aztreonam lysine for inhalation (AZLI) or a placebo (an inactive treatment) during a blinded phase, meaning neither participants nor their doctors knew which they received. Both groups then went on to receive the active treatment in a later open-label phase (where everyone knew what was being given). The trial's main focus was on measuring changes in respiratory symptoms using a standardised questionnaire called the QOL-B, which scores symptoms on a scale of 0 to 100, where a higher score means fewer or less troublesome symptoms. The reported data shows that, after 28 days (the end of the first treatment course), the group that received AZLI from the start saw their respiratory symptom score rise by an average of 8.2 points, while the group that received placebo first saw an average rise of 3.2 points. By day 84 (the end of the second course, by which time both groups were on the active treatment), the reported changes from the starting point were 5.6 points for the original AZLI group and 3.9 points for the group that started on placebo. These numbers simply describe how much the scores moved — they do not on their own indicate whether any change is meaningful for daily life. For the secondary outcome measuring how long it took participants to experience a significant worsening of their condition (a "protocol-defined exacerbation"), the reported data shows the values were not available (listed as "NA" in the submitted results). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01327703 · results posted 10 April 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 87 people in total (42 in one group and 45 in the other). It was a crossover study, meaning every participant tried both treatments — one called Panzytrat® and one called Kreon® — in different orders, with each treatment period lasting 15 days. Both are pancreatic enzyme replacement products. The main thing the trial was measuring was how well each treatment allowed the body to absorb fat from food, using a calculation called the "coefficient of fat absorption" (CFA) — essentially, the percentage of fat eaten that was absorbed rather than lost in stools. By the end of the second treatment period, three participants had left the study before finishing. The reported data shows that for the primary measure — percentage of fat absorbed — participants taking Panzytrat® had a reported average of 78.27%, while those taking Kreon® had a reported average of 80.35%. For the secondary measures, the average number of stools per day was reported as 4.5 for Panzytrat® and 4.2 for Kreon®. The proportion of stools described as having normal consistency was reported as approximately 64.4% for Panzytrat® and 63.5% for Kreon®. The reported average total stool weight over the collection period was 521.6 grams for Panzytrat® and 484.0 grams for Kreon®, and the average weight per individual stool sample was 131.7 grams versus 124.0 grams respectively. The reported data also shows that abdominal symptoms such as pain and flatulence were tracked across the full 15-day period. The proportion of days on which participants reported any abdominal symptoms was broadly similar between the two treatments across the different symptom categories measured, with figures generally ranging from roughly 0.2 to 0.47 (meaning symptoms were noted on between about one-fifth and just under half of days, depending on the symptom type and severity level). The trial did not appear to report which of these differences, if any, were considered meaningful — that information was not included in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01059565 · results posted 11 March 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 101 people in total — 49 in the AZLI (aztreonam lysine for inhalation) group and 52 in the placebo group — though one person in the AZLI group did not end up receiving treatment. The trial ran in two back-to-back phases: a 24-week period where participants were randomly assigned to either AZLI or a placebo (an inactive treatment), followed by a further 24-week period where all participants received AZLI. The main thing the trial was measuring was how lung function — specifically a breathing test called FEV1 (the maximum amount of air a person can breathe out in one second, compared to what is typical for someone of the same age, sex and body size) — changed over the first 24 weeks. The reported data shows that, for the primary measure of lung function change over 24 weeks, the AZLI group had an average change of +0.16% compared to a starting point, while the placebo group had an average change of −0.75%. For the secondary measures, the total number of antibiotic courses taken for breathing-related events was 54 in the AZLI group and 73 in the placebo group. A symptom questionnaire score (rated on a scale of 0–100, where higher means fewer symptoms) showed an average change of 2.97 units in the AZLI group and 2.79 units in the placebo group. Other breathing measurements — including how much air could be exhaled in total (FVC) and airflow in the middle portion of a breath (FEF25-75) — also showed numerical differences between the two groups, with the AZLI group recording +0.77% and +1.40% changes respectively, compared to +0.17% and −0.55% in the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01537666 · results posted 4 March 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 25 people in total. Eighteen were healthy volunteers, divided into three groups receiving different doses of an inhaled antibiotic called AeroVanc (16 mg, 32 mg, or 80 mg). The remaining seven participants had cystic fibrosis (CF) and received either the 32 mg or 80 mg inhaled dose. All 25 participants completed the study. The trial was primarily looking at how many people experienced adverse events (unwanted medical occurrences) after taking the study drug, and it also measured how the drug moved through the body by tracking its levels in the bloodstream over time. The reported data shows that, for the primary outcome — tracking adverse events — the numbers varied across groups. Among healthy volunteers, 2 out of 6 in the 16 mg group, 5 out of 6 in the 32 mg group, and 2 out of 6 in the 80 mg group reported at least one adverse event, compared with 2 out of an unspecified number who received an intravenous (IV, meaning into a vein) comparison dose. Among CF patients, 6 out of those in the 32 mg group and 5 in the 80 mg group reported at least one adverse event. The reported data also shows a subset of those were considered possibly related to the study drug (2, 3, 2, 1, 4, and 4 participants respectively across the six groups), though no breakdown of the nature or severity of those events is included here. For the blood-level measurements in healthy volunteers, the peak drug concentration in the bloodstream was much lower for all inhaled AeroVanc doses compared with the IV dose — for example, 108.82 ng/ml at the lowest inhaled dose rising to 617.83 ng/ml at the highest, versus 10,028.33 ng/ml for the IV dose. The time it took to reach that peak was around 1.3 to 2.1 hours for the inhaled doses and about 0.9 hours for the IV dose. The estimated time for blood levels to fall by half was broadly similar across all groups, ranging from about 7.2 to 8.6 hours. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00628134 · results posted 7 November 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled 8 adults in total, split into two groups of 4. It used a "crossover" design — meaning each person tried both treatments at different times: one group inhaled a surfactant aerosol (a substance that can coat the airways) first and then a saltwater (saline) aerosol, while the other group did it in the opposite order. All 8 participants completed the study. The trial was measuring how evenly an inhaled aerosol spreads through the lungs, using a special imaging technique (a nuclear medicine gamma camera) that tracks a radioactive tracer to see where the aerosol ends up in the lungs. The reported data shows two main measurements. The first was the change in the ratio of aerosol found in the central (inner) part of the lung versus the outer (peripheral) part, tracked over 30 minutes after inhalation. For the saline group, this ratio changed by −0.05, while for the surfactant group it changed by +0.10. The second measurement looked at the change in the percentage of the aerosol dose found in the outer part of the lung over the same 30-minute period. The reported data shows a change of −3 percentage points for the saline group and −8 percentage points for the surfactant group. No further statistical detail or explanation of these differences was reported in the structured data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01179347 · results posted 26 March 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled 463 participants in total — 155 in the placebo group and 308 in the group receiving the active treatment (referred to as "Tio R5 qd"). The trial ran in two back-to-back 12-week stages: a blinded phase (where neither participants nor researchers knew who received which treatment) and an open-label phase (where everyone knew). The trial was measuring lung function in people with cystic fibrosis, focusing on how much air participants could breathe out and how fully their lungs could fill and empty. The reported data shows that the two main lung function measures — how much air could be forcefully breathed out in one second (FEV1), assessed both over a four-hour window and just before a dose — showed small changes from the starting point in both groups. For the four-hour average measure, the placebo group showed a change of 0.87 percentage points, while the active treatment group showed a change of 2.51 percentage points. For the pre-dose measure, the placebo group showed a change of 0.72 percentage points and the active treatment group showed 2.12 percentage points. Secondary measures of how much air the lungs could hold (FVC) and airflow through the middle portion of the lungs also showed small numerical differences between the groups. Regarding lung flare-ups (pulmonary exacerbations) during the blinded period, the reported data shows that 7.8% of placebo participants and 8.9% of active treatment participants experienced at least one such episode. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00909532 · results posted 21 August 2012

  According to the results reported on ClinicalTrials.gov, this trial (NCT00909532) enrolled 161 people with cystic fibrosis — 78 in the placebo group and 83 in the group receiving 150 mg of ivacaftor twice daily. The trial was measuring several things over up to 48 weeks, including lung function (using a breathing test called FEV1, which measures how much air a person can forcefully breathe out in one second), a quality-of-life questionnaire focused on breathing (CFQ-R respiratory score), a sweat chloride test (a marker of how well a certain protein in the body is working), the time until a serious lung flare-up occurred, and body weight. The reported data shows that for the primary measure — change in lung function at 24 weeks — the placebo group showed an average change of -0.2 percentage points, while the ivacaftor group showed an average change of +10.4 percentage points. At 48 weeks, the reported figures were -0.4 for placebo and +10.1 for ivacaftor. For the breathing-related quality-of-life score (measured on a 0–100 scale), the placebo group's average change was around -2.1 to -2.7 points, while the ivacaftor group averaged +6.0 points at both time points. The sweat chloride test showed an average change of around -0.7 for the placebo group and -48.7 millimoles per litre for the ivacaftor group at both time points. For body weight, the placebo group averaged a change of roughly +0.2 to +0.4 kilograms, compared with approximately +3.0 to +3.1 kilograms in the ivacaftor group. Regarding lung flare-ups, the reported data shows the proportion of participants who had not experienced a flare-up was broadly similar between groups early on, with the ivacaftor group showing a somewhat higher proportion remaining flare-up free by the later time points measured. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00909727 · results posted 21 August 2012

  According to the results reported on ClinicalTrials.gov, this trial enrolled 52 people in total — 26 received a placebo (a dummy treatment with no active ingredient) and 26 received a medicine called ivacaftor (150 mg twice daily). All 26 participants in the ivacaftor group completed the study, while 22 of the 26 in the placebo group did so. The trial was measuring several things over up to 48 weeks, with the main focus being lung function — specifically a breathing test called FEV1, which measures how much air a person can forcefully breathe out in one second, expressed as a percentage of what would be expected for someone of their age and size. The reported data shows that, for the primary measure (change in lung function up to Week 24), the placebo group's FEV1 score changed by an average of +0.1 percentage points from their starting level, while the ivacaftor group's score changed by an average of +12.6 percentage points. At Week 48, the reported figures were +0.7 percentage points for the placebo group and +10.7 percentage points for the ivacaftor group. The trial also measured a quality-of-life questionnaire score (on a scale of 0–100 for breathing-related wellbeing), with the ivacaftor group showing a reported change of around +6.3 points at Week 24 and +6.1 points at Week 48, compared with +0.3 and +1.0 points respectively for the placebo group. Additionally, a sweat chloride test (a marker related to how the condition affects salt in the body) showed a reported change of around −55.5 mmol/L for the ivacaftor group versus −1.2 mmol/L for placebo at Week 24, and −56.0 versus −2.6 mmol/L at Week 48. Finally, average weight gain was reported as 3.7 kg for the ivacaftor group versus 1.8 kg for the placebo group at Week 24, and 5.9 kg versus 3.1 kg at Week 48. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00388505 · results posted 24 July 2012

  According to the results reported on ClinicalTrials.gov, this trial compared two forms of the antibiotic tobramycin in people with cystic fibrosis who had a lung infection caused by a bacterium called *Pseudomonas aeruginosa*. One group received tobramycin as an inhaled powder (TIP) and the other received it as an inhaled liquid solution (TOBI). A total of 308 people were assigned to the powder group and 209 to the liquid group, with 225 and 171 respectively completing the study. The trial measured a range of things including unwanted medical events that occurred during treatment, the amount of drug that entered the bloodstream, lung function, bacteria levels in sputum (mucus coughed up from the lungs), hearing, and how satisfied patients were with their treatment. The reported data shows that when it came to unwanted medical events during the study, 278 out of 308 people in the powder group and 176 out of 209 in the liquid group experienced at least one such event. Serious events (those involving hospitalisation, life-threatening situations, or similar) were recorded for 85 people in the powder group and 61 in the liquid group. For lung function — measured as the amount of air a person can forcibly breathe out in one second, expressed as a percentage of what is typical — both groups started at around 52–53% at the beginning of the study. By the end of the third treatment cycle, the reported change from the starting point was a small decrease of 0.4% in the powder group and 1.6% in the liquid group. Bacteria levels in sputum were reduced from baseline in both groups across the treatment cycles. Regarding how much of the drug entered the bloodstream, the levels measured were low in both groups, ranging from undetectable before dosing up to around 1.4 µg/mL (micrograms per millilitre) at peak in the powder group and 1.2 µg/mL in the liquid group. On the patient satisfaction survey (scored 0–100, with higher meaning more satisfied), one area — relating to convenience — showed a notably higher reported score for the powder group (75.4) compared with the liquid group (20.19). The reported data also shows that a decrease in hearing was recorded in a small proportion of participants in both groups, with the exact percentages varying depending on the frequency of sound tested. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00645788 · results posted 3 July 2012

  According to the results reported on ClinicalTrials.gov, this trial enrolled 288 people in total across four groups. Participants received either one of two doses of an inhaled antibiotic powder called Ciprofloxacin DPI (32.50 mg or 48.75 mg), or a matching inactive powder (placebo) for each dose. The trial was measuring lung function and bacterial levels in sputum (mucus coughed up from the lungs) in people with a lung condition involving a bacteria called *Pseudomonas aeruginosa*. Around 74–93 people in each active treatment group and 21–47 in each placebo group finished the study. The reported data shows that the main thing being measured was a lung function test called FEV1 — essentially how much air a person can forcefully breathe out in one second, expressed as a percentage of what would normally be expected for someone of that age and size. At the main measurement point (around days 28–30), the 32.50 mg group showed a change of −1.0%, the 48.75 mg group −0.62%, and both placebo groups showed −2.2%, compared to where each group started. The reported data also shows changes in the amount of *Pseudomonas aeruginosa* bacteria detected in sputum samples over time, with both active dose groups showing larger reductions in bacterial levels compared to the placebo groups at several time points. For the outcome measuring the time to a first lung flare-up requiring treatment, the data was not reported for any of the four groups. Other lung function measures (FVC and FEF 25–75%) were also tracked at multiple time points, with various small changes recorded across all groups; the specific numbers varied by visit and group as reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00930982 · results posted 30 January 2012

  According to the results reported on ClinicalTrials.gov, this trial enrolled 124 people in total — 60 received an inhaled form of the antibiotic ciprofloxacin (called Ciprofloxacin Inhale or BAYQ3939) and 64 received a placebo (an inactive inhaled treatment). The trial was primarily measuring how much the amount of bacteria in participants' sputum (mucus coughed up from the lungs) changed after 29 days of treatment. It also tracked a range of secondary measures, including lung function, how long it took before participants needed antibiotic treatment for a flare-up of their condition, and self-reported quality of life. The reported data shows that, for the primary outcome — change in bacterial load in sputum — the ciprofloxacin inhale group had an average reduction of 2.94 units (measured on a logarithmic scale, which is a way of expressing very large or small numbers more simply) compared to a reduction of 0.32 units in the placebo group. For the secondary lung function measures (how much air participants could breathe out), the reported changes from baseline were small in both groups across multiple time points, with figures generally close to zero in both directions. The time-to-flare-up data was reported as "not available" in the submitted results. For self-reported quality of life, the reported scores on both questionnaires were broadly similar between the two groups across the measurement time points, with no large numerical differences noted in the data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00680316 · results posted 17 June 2011

  According to the results reported on ClinicalTrials.gov, this trial (NCT00680316) compared two groups: one receiving Dornase Alfa and one receiving a placebo (an inactive treatment used for comparison). The study was very small — only 3 people took part in total, with 1 person in the Dornase Alfa group and 2 people in the placebo group. All 3 participants completed the study. The trial was designed to measure lung function in young children with cystic fibrosis using a technique called forced oscillometry, which tests how the lungs respond to gentle air pressure. It also looked at symptom scores reported by parents and preschool-aged children using a cystic fibrosis-specific questionnaire. The reported data shows that no numerical results were submitted for any of the outcome measures — neither the primary measure (a lung function reading called reactance at 8 Hz) nor any of the secondary measures (other lung function readings at different frequencies, or the questionnaire symptom scores). The data fields for all measured outcomes are empty in the results as lodged on ClinicalTrials.gov. This means it is not possible to describe what the numbers showed, as no figures were reported. Because no outcome data was reported and the number of participants was extremely small, the reported information does not allow any conclusions to be drawn about the treatment being studied. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00112359 · results posted 21 April 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 164 people in total — 84 received a placebo (an inactive treatment given three times daily) and 80 received 75 mg of aztreonam lysine for inhalation (AZLI), also three times daily. The trial was measuring several things in people with cystic fibrosis who had a lung infection caused by a bacteria called *Pseudomonas aeruginosa*. These included changes in lung function, the amount of bacteria in sputum (mucus coughed up from the lungs), and how participants rated their own respiratory symptoms. Of those who started, 57 people in the placebo group and 67 in the AZLI group completed the trial; the data was not reported explaining all individual reasons for non-completion. The reported data shows the following numbers at the end of the study period. For lung function — measured as the percentage change in how much air a person can forcefully breathe out in one second (called FEV1) — the placebo group showed a change of approximately −2.4%, while the AZLI group showed a change of approximately +7.9%. For the amount of *Pseudomonas aeruginosa* bacteria measured in sputum, the placebo group showed a small increase (a log change of +0.07) while the AZLI group showed a decrease (a log change of −1.38 — meaning roughly a large reduction in the measured bacterial count). For self-reported respiratory symptoms, scored on a scale of 0 to 100 where higher means fewer symptoms, two separate time-point measurements were reported: one showed changes of approximately +1.0 (placebo) versus +7.0 (AZLI), and another showed changes of approximately −5.7 (placebo) versus +0.6 (AZLI). The trial also tracked other bacteria in sputum samples and measured how much of the drug was needed to inhibit the target bacteria, though those results were reported as group-wide figures rather than individual participant outcomes. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00706004 · results posted 18 April 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 9 people, all of whom received a medication called lubiprostone (at a dose of 24 micrograms, twice daily). Seven of the 9 participants completed the trial, and 2 did not finish. The trial was measuring bowel movement frequency and a range of other measures related to constipation symptoms and general health, across multiple study visits. The reported data shows that the average number of spontaneous (unassisted) bowel movements per week was 9.7 at one point in the study, 10.8 at another, and 8.8 at a third. For constipation symptoms, participants filled out a standard questionnaire called the PAC-SYM, where a score of 0 means no symptoms and 4 means the most severe — the reported scores across the study visits were 1.18, then 0.54, and then 0.44. Stool consistency was measured using the Bristol Stool Scale, which goes from 1 (most constipated) to 7 (loose) — scores reported were 3.4, 3.6, and 3.3 across visits. Body mass index (a measure of body weight relative to height) stayed around 24.0–24.3 kg/m² across visits. The number of participants who reported experiencing side effects was 5 at one visit and 3 at another. Blood sodium levels were also measured, coming in at 137.4 and 139.2 nmol/L across two time points. It is worth noting that because multiple measurements were reported for some outcomes without clear labels for each time point, the exact timing of each figure was not specified in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00737100 · results posted 13 April 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 510 people across three groups: 168 received a placebo (dummy treatment), 166 received a lower dose of tiotropium Respimat (2.5 micrograms), and 176 received a higher dose (5 micrograms). The vast majority of participants finished the trial — around 161, 159, and 169 people respectively in each group. The trial was measuring changes in lung function over 12 weeks, specifically looking at how well participants could breathe out air, using several different breathing tests. The reported data shows the following numbers for the two main measures at week 12. For the first primary measure — how much air participants could forcefully breathe out over one second, averaged across a four-hour window — the placebo group's score changed by −1.74 percentage points from their starting point, while the 2.5 microgram group changed by +1.20 and the 5 microgram group by +1.65 percentage points. For the second primary measure — the same breathing-out test but taken at a single "trough" point (the lowest point before the next dose) — the placebo group changed by −1.44 percentage points, compared with +0.81 for the 2.5 microgram group and +0.78 for the 5 microgram group. The reported data also shows results from several secondary (additional) lung function measures at week 12. For the total amount of air breathed out in one breath (FVC), averaged across four hours, changes were −1.30 for placebo, +0.53 for the lower dose, and +1.81 percentage points for the higher dose. A measure of airflow through the middle part of a breath showed changes of −1.40, +2.78, and +3.94 percentage points respectively. A measure of air trapped in the lungs (RV/TLC ratio) showed very small changes of −0.01, 0.00, and +0.04 percentage points across the three groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00128492 · results posted 11 March 2011

  According to the results reported on ClinicalTrials.gov, this 18-month trial involved 274 participants in total — 85 people received a dose of the inhaled antibiotic aztreonam lysine for inhalation (AZLI) twice a day, and 189 received it three times a day. The trial was measuring a range of safety-related signals, including unwanted health events (called adverse events), changes in a breathing test (called FEV1, which measures how much air a person can forcefully breathe out in one second), changes in heart rate and blood pressure, and levels of a bacteria called *Pseudomonas aeruginosa* in participants' sputum (mucus coughed up from the lungs). The reported data shows that the vast majority of participants experienced at least one unwanted health event during the study — 83 out of 85 in the twice-a-day group, and 185 out of 189 in the three-times-a-day group. Serious events that led to hospitalisation or death were recorded in 38 people in the twice-a-day group and 100 in the three-times-a-day group. Regarding the breathing test, 9 participants in the twice-a-day group and 14 in the three-times-a-day group showed a drop of 15% or more in their FEV1 score at some point during treatment. Changes in heart rate and blood pressure across both groups were small — generally less than a few beats per minute or a few millimetres of mercury — though the reported data does not include enough detail to describe these figures more precisely at every individual time point. The reported data also shows changes in the levels of *Pseudomonas aeruginosa* bacteria in participants' sputum over the course of the study. In the twice-a-day group, bacterial levels appeared to decrease by between 0.20 and 0.47 units (on a logarithmic scale, meaning each unit represents a tenfold change) depending on the time point measured; in the three-times-a-day group, reductions ranged from 0.53 to 0.81 units. The number of participants found to have other bacteria or fungi in their sputum was also tracked across both groups at various points in the study, with the reported figures varying across time points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00712166 · results posted 20 December 2010

  According to the results reported on ClinicalTrials.gov, this trial (NCT00712166) enrolled 157 people with cystic fibrosis — 81 in a placebo group and 76 receiving a treatment called AZLI (aztreonam lysine for inhalation) at a dose of 75 mg, taken three times daily. The trial's main goal was to measure changes in respiratory symptoms — things like coughing, congestion, and wheezing — using a patient-reported questionnaire called the CFQ-R Respiratory Symptoms Scale. This scale runs from 0 to 100, where a higher score means fewer symptoms and a better quality of life. Scores were collected at the start of the study (Day 0) and again at Days 14, 28, and 42. The reported data shows that for the primary outcome — the change in respiratory symptom scores from the start to Day 28 — the placebo group's score increased by an average of 1.41 points, while the AZLI group's score increased by an average of 3.22 points. At Day 14, the reported increases were 0.28 points (placebo) and 3.65 points (AZLI). By Day 42, the reported figures were closer together: 2.91 points (placebo) and 3.02 points (AZLI). For the physical functioning scale at Day 28, the placebo group's score changed by −0.69 points (a slight decrease) while the AZLI group's score increased by 1.79 points. Regarding additional antibiotics needed during the study, 21 participants in the placebo group and 19 in the AZLI group required extra antipseudomonal antibiotic treatment. The reported data also shows that 3 participants in the placebo group and 8 in the AZLI group were hospitalised during the study period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00775528 · results posted 1 October 2010

  According to the results reported on ClinicalTrials.gov, this trial involved 19 people who were given a pancreatic enzyme supplement called pancrelipase delayed-release capsules. Eighteen of the 19 participants completed the study, and one did not finish. The trial was measuring two main things: how many participants experienced unwanted health events (called "treatment emergent adverse events" — meaning any new or worsening health problem that appeared after starting the study medication), and also tracking details about participants' diet and the fat content in their stools. The reported data shows that 9 out of the 19 participants experienced at least one such health event after starting the study medication — this was the primary (main) thing being measured. For the secondary (additional) measures, the reported data shows that, on average, the fat content in participants' stools was 28.11% of the dry solid weight per bowel movement. The average daily fat intake across participants over the three-day measurement period was reported as 46.61 grams, and the average total daily calorie intake over the same period was reported as 1,239.71 kilocalories (a standard unit of food energy). It is worth noting that this trial had only one group — everyone received the same treatment — so there was no comparison group reported in this data. This means the reported numbers describe what was observed in that single group only. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00690820 · results posted 10 March 2010

  According to the results reported on ClinicalTrials.gov, this trial involved 17 people in total (8 in one group and 9 in the other), with 16 completing the full study. It used a "crossover" design, meaning each participant took both the active treatment (pancrelipase, a digestive enzyme capsule) and a placebo (dummy capsule) during two separate periods — so everyone experienced both. The trial was primarily measuring how well the body absorbed fat from food, and also looked at protein absorption, the amount of fat passed in stools, stool weight, how often participants went to the toilet, and the number of days without flatulence (gas). The reported data shows that, for the main measure — fat absorption — participants recorded an average of 47.4% absorption during the placebo period and 82.8% during the pancrelipase period (where a higher number means more fat was absorbed). For protein absorption, the reported figures were 45.0% with placebo and 80.3% with pancrelipase. The reported data also shows that the total amount of fat passed in stools over three collection days averaged 182.9 grams with placebo compared to 58.1 grams with pancrelipase (where lower means less fat lost in stools). Total stool weight averaged 436.5 grams with placebo and 161.4 grams with pancrelipase. For the remaining secondary measures, the reported data shows that average daily toilet visits were 3.46 with placebo and 1.88 with pancrelipase, and the percentage of days with no flatulence was 36.3% with placebo compared to 45.0% with pancrelipase. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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Not medical advice. These are results as reported by the trial sponsor to a public registry, summarised in plain English. They describe what a trial recorded, not whether a treatment is suitable for any person. Verify anything important against the original registry record and discuss what it means for you with your doctor.