Reported trial results for Rare Disease

Every Rare Disease trial Voxsanity tracks that has reported results to a public registry, newest first. These trials have finished, so they are not recruiting. Not medical advice.

37 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

NCT04046224 · results posted 14 April 2026

According to the results reported on ClinicalTrials.gov, this trial (NCT04046224) tested a gene therapy called ST-920 in people with Fabry disease, a rare inherited condition. The trial enrolled 33 participants in total, spread across nine groups that received either different doses of ST-920 or the same high dose but were selected based on specific characteristics (such as having heart or kidney involvement, being female, or having particular antibody levels). The trial was measuring how often unwanted health events occurred after receiving ST-920, and how levels of a specific enzyme — called alpha-galactosidase A (alpha Gal-A), which is deficient in Fabry disease — changed in the blood over 52 weeks. The reported data shows that across the four initial dose-testing groups (2 people each in the lowest two doses, 3 in the next, and 2 in the highest), every participant who started the trial completed it, and no unwanted health events related to ST-920 were recorded in those groups. In the later expansion groups, the reported data shows that all five participants in the cardiac group, four of five in the female group, two of two in the renal group, all seven in the antibody-negative group, and all five in the antibody-positive group experienced at least one health event considered related to ST-920. Serious health events were recorded in one participant each in the renal, antibody-negative, and antibody-positive groups. The reported data shows no participant in any group left the study early because of a health event. Regarding the enzyme level outcome, the reported data shows increases in alpha Gal-A enzyme activity in the blood from baseline at the 24-week and 52-week time points across the groups where figures were provided — for example, increases ranging from around 16 to 76 units (nmol/h/mL) at 24 weeks, and figures of approximately 50, 31, and 163 units reported at 52 weeks for the first three dose groups (data for the remaining groups at 52 weeks was not reported in the submitted results). These numbers represent changes from each participant's starting level, not absolute values. 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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NCT03635073 · results posted 19 March 2026

According to the results reported on ClinicalTrials.gov, this trial (NCT03635073) enrolled 156 participants who were given a study medicine called soticlestat. The trial was measuring a range of things, including how often participants experienced unexpected medical events (called adverse events) while taking the medicine, changes in behaviour and everyday living skills (using two questionnaires — the Vineland Adaptive Behavior Scale and the Aberrant Behavior Checklist), thoughts of self-harm (using a standard rating scale), and changes in certain blood test results. The reported data shows that none of the 156 participants were recorded as having "completed" the study, with all 156 listed under "not completed" — no further explanation for this was provided in the submitted data. The reported data shows that 91% of participants experienced at least one adverse event (an unexpected medical occurrence) during the study. On the behaviour questionnaire for participants aged 6 and over (ABC-C), the average total score at the start of the study was 36.8 out of a possible 174, and the average change from that starting point was −16.3 points, where a negative number indicates a reduction in the scores being measured. Changes were also reported across individual sections of both behaviour questionnaires and across everyday living skill categories; these ranged from small negative numbers (indicating reductions in the scores being tracked) to small positive numbers, depending on the specific area measured. For the blood test results and the self-harm rating scale, numbers were reported for individual categories, though the submitted data did not include a summary explanation linking those numbers to an overall conclusion. 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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NCT05998395 · results posted 3 February 2026

According to the results reported on ClinicalTrials.gov, this trial enrolled just one participant. The study was looking at a drug called ruxolitinib as a potential treatment for a very rare condition called Kohlmeier-Degos disease affecting the central nervous system (CNS) — meaning the brain and spinal cord. The main thing being measured was whether MRI scans (brain and spine imaging) showed that existing lesions (areas of abnormality) stayed stable or shrank, or whether no new lesions appeared, after the participant took ruxolitinib for between 13 and 73 weeks. The reported data shows that the one participant who started the trial did not complete it. Regarding the primary outcome — stable or no new lesions on MRI — the data as submitted appears to reflect measurements at different time points. The reported numbers show values of 1, 0, and 0 for one set of measurements, and 0, 0, and 1 for another set. However, the way these figures are presented in the submitted data makes it difficult to clearly interpret what they represent at each specific time point, and no further breakdown or explanation was provided alongside them. Because the single participant did not complete the trial, and given the very small size of the study (one person), the reported data is extremely 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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NCT04221451 · results posted 28 January 2026

According to the results reported on ClinicalTrials.gov, this trial (NCT04221451) looked at a medicine called venglustat in people with certain rare inherited conditions affecting the nervous system. The main group studied had a condition called Niemann-Pick disease type C (referred to as the "primary population"), while a smaller "secondary population" included people with related conditions such as GM1 or GM2 gangliosidosis, sialidosis type 1, and galactosialidosis. In the first phase of the trial (up to about two years), 19 participants received a placebo (a dummy treatment with no active ingredient), 40 received venglustat, and 16 were in the secondary population group receiving venglustat. Most participants who started this phase completed it. A second, longer phase then followed for those who continued, though the reported data shows that none of those participants had fully completed that extended phase at the time results were submitted. The reported data shows that one of the key things measured in the primary population was the change in a biological marker called GM2 — a substance found in the fluid surrounding the brain and spine — which was used as a signal of disease activity. After about two years, the placebo group showed an average reduction of around 11% in this marker, while the venglustat group showed an average reduction of around 48%. The trial also measured how quickly arm and hand function changed over time using a peg-placing test; the reported data shows the placebo group's score worsened at a rate of about 0.95% per year, compared to about 2.49% per year in the venglustat group — noting that a higher number on this test indicates greater difficulty. For participants in the secondary population with GM2 gangliosidosis, the reported data shows reductions in relevant biological markers ranging from roughly 43% to 82%. For those with GM1 gangliosidosis, reductions in some markers of around 32% to 80% were reported, though one marker showed an increase of about 17%. For the sialidosis type 1 and galactosialidosis groups, the data was 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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NCT05612139 · results posted 26 January 2026

According to the results reported on ClinicalTrials.gov, this trial involved 14 participants, all of whom completed the study with no dropouts. The trial was examining a device called JTIN — an intramedullary nail (a rod inserted into the bone) — and was looking at how often unwanted medical events occurred during procedures involving it, as well as several measures related to how the implant and the treated bones performed over the follow-up period. The reported data shows that in 4% of procedures, at least one adverse event (an unwanted medical occurrence) that was considered certainly or possibly related to JTIN was recorded. On the secondary measures, the reported data shows that 100% of the implanted nails remained in place without needing to be surgically replaced, and bone union (where the treated bone knitted back together) was reported to have been achieved in 100% of procedures. For the measure described as "post-treatment fracture-free survival" — meaning the proportion of patients who did not experience a new fracture in the treated bone during the follow-up period — the reported figure was 35.7%. The trial's reporting does not clarify over what specific timeframe this last figure was measured. It is worth noting that this was a small study of only 14 people, and the results reported here are those submitted by the trial sponsor 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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NCT05358717 · results posted 13 January 2026

According to the results reported on ClinicalTrials.gov, this trial (NCT05358717) enrolled 159 people across three groups — 53 received a placebo (a dummy treatment with no active ingredient), 52 received a 5 mg dose of PTC518, and 54 received a 10 mg dose of PTC518. The trial was studying PTC518, an investigational oral tablet being tested in people with Huntington's disease. The main things being measured were how many participants experienced unexpected medical events (called adverse events) after taking the study drug, and how much a specific protein linked to Huntington's disease — called total huntingtin protein, or tHTT — changed in the blood after three months of treatment. The reported data shows that for the primary outcomes, 46 out of 53 placebo participants, 43 out of 52 in the 5 mg group, and 49 out of 54 in the 10 mg group experienced at least one adverse event during the study. Regarding the blood tHTT protein levels at three months, the placebo group showed a change of approximately −1.4%, while the 5 mg group showed approximately −17.4% and the 10 mg group approximately −28.7% — meaning the protein level appeared lower in the PTC518 groups compared to placebo. By 12 months, the reported protein changes were approximately +8.2% for placebo, −14.2% for the 5 mg group, and −28.4% for the 10 mg group. A similar protein measured in spinal fluid (mutant huntingtin) also showed reported changes at 12 months: approximately −15.6% for placebo, −23.7% for the 5 mg group, and −23.3% for the 10 mg group. For a brain structure called the caudate (which can shrink in Huntington's disease), the reported volume changes at 12 months were approximately +4.4% for placebo, +5.0% for the 5 mg group, and +5.7% for the 10 mg group. A combined score measuring movement, thinking, and daily functioning (called the cUHDRS, where a higher score means better functioning) changed by approximately −0.64 points in the placebo group, −0.61 in the 5 mg group, and −0.92 in the 10 mg group at 12 months. 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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NCT03936777 · results posted 17 November 2025

According to the results reported on ClinicalTrials.gov, this trial (NCT03936777) enrolled 412 participants who received an open-label (meaning everyone knew what treatment they were getting) dose of a medicine called ZX008. Of those, 360 completed the study and 52 did not. The trial was measuring how often unwanted medical events (called adverse events) occurred during treatment, and also tracking how participants' overall wellbeing, thinking, behaviour, and movement were rated over time by both parents/carers and the medical team running the trial. The reported data shows that the primary thing being measured — the percentage of participants who experienced a treatment-emergent adverse event (that is, any unwanted medical occurrence that appeared or worsened after starting treatment) — was reported as 75.5% of participants. This means roughly three in four people in the trial had at least one such event recorded. The data does not break down what types of events occurred or how serious they were in this summary. For the secondary measures, participants were rated on a 7-point scale covering overall function, thinking, behaviour, and movement — where 1 means "very much improved" and 7 means "very much worse." The reported data shows the number of participants falling into each category across different rating points during the study, but a full visit-by-visit breakdown was not straightforward to summarise from the submitted figures alone. The reported numbers varied across the scale categories at different time points for both parent/carer and investigator ratings. 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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NCT04200664 · results posted 10 June 2025

According to the results reported on ClinicalTrials.gov, this trial (NCT04200664) looked at hearing and balance function in people with a condition called idiopathic superficial siderosis (iSS) — a rare condition where iron deposits build up around the brain and spinal cord. The study planned to include three groups: people with iSS, people with age-related hearing loss, and a healthy control group. However, the reported data shows that only the iSS group enrolled any participants — 11 people started the study and 10 completed it. No participants were enrolled in the other two groups, so no comparison data between groups was reported. The reported data shows the results only for the 11 iSS participants. For the pure-tone hearing test — a standard test where a person indicates the quietest sounds they can hear — the average threshold across both ears was reported as 57.3 decibels hearing level (a higher number means quieter sounds are harder to hear). For the tympanogram, a test of how the eardrum moves, all 11 participants returned a normal result. For the auditory brainstem response test, which measures how the hearing nerve and brain respond to sound, 5 participants had an abnormal result, 2 had a normal result, and 4 were inconclusive. For otoacoustic emissions, a test of the tiny hair cells inside the ear, 3 were abnormal, 3 were normal, and 5 were inconclusive. For a speech-in-noise listening test, 4 were abnormal, 1 was normal, and 6 were inconclusive. For balance and vestibular (inner ear) testing, several sub-measures were reported as counts of participants with normal, abnormal, or inconclusive results, though the specific labels for each sub-measure were not 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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NCT04612790 · results posted 29 November 2024

According to the results reported on ClinicalTrials.gov, this trial (NCT04612790) looked at a medicine called benralizumab compared to a placebo (a dummy treatment with no active ingredient) in people with a skin condition called bullous pemphigoid. In the first part of the trial (the "double-blind period," lasting up to 36 weeks), 34 people received benralizumab and 33 received placebo — neither the participants nor the researchers knew who was getting which treatment. A smaller follow-on "open-label extension" period then took place, in which 16 and 18 participants respectively continued or crossed over to benralizumab. The trial's main question was how many participants reached complete remission (no active disease) while off steroid tablets for at least two months by week 36. The reported data shows that for the main outcome, approximately 11.1% of participants in the benralizumab group met this remission definition at week 36, compared with about 5.3% in the placebo group. For the secondary outcomes, around 23.8% of the benralizumab group remained free of a disease relapse (a meaningful worsening of symptoms) up to week 36, versus about 19.8% in the placebo group. Both groups showed a reduction in a skin disease severity score (called the BPDAI activity score) from their starting point — the benralizumab group's score fell by about 53.3 points and the placebo group's by about 52.8 points, on a scale of 0 to 360. The reported data also shows that scores for itch (rated 0–30, with higher meaning worse) fell by about 5.6 points in the benralizumab group and about 16.6 points in the placebo group. Steroid tablet use over the 36 weeks was reported as approximately 71.4 mg/kg in the benralizumab group and 62.7 mg/kg in the placebo group. It is worth noting that a large proportion of participants did not complete the double-blind period (18 in the benralizumab group and 14 in the placebo group), and no participants were recorded as completing the open-label extension period, so the data should be interpreted with that in mind. 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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NCT03474640 · results posted 22 November 2024

According to the results reported on ClinicalTrials.gov, this trial tested a drug called toripalimab in people with advanced cancers. The trial had two parts: Part A looked at three different dose levels of the drug (80 mg, 240 mg, and 480 mg, given every 14 days) to examine how the body responded to each dose, while Part B enrolled participants grouped by their cancer type — including sarcoma (59 people), biliary tract cancer (42 people), gastric cancer (29 people), neuroendocrine cancer (22 people), oesophageal cancer (11 people), and other tumours (3 people). In total, 177 people took part across all groups. The reported data shows that the primary thing being tracked was the number of participants who experienced treatment-related side effects, as recorded using a standard medical checklist. Across the dose groups in Part A, side effects were reported in 2 out of 3 people (80 mg), 5 out of 8 (240 mg), and 5 out of 7 (480 mg) participants. In Part B, side effects were reported in 33 of 59 sarcoma participants, 27 of 42 biliary tract participants, 15 of 29 gastric cancer participants, 14 of 22 neuroendocrine participants, 7 of 11 oesophageal participants, and 2 of 3 participants in the "other tumours" group. For tumour response in Part B — meaning whether scans showed a tumour shrinking — the reported data shows that in the sarcoma group, 5 participants had a measurable reduction in tumour size, 3 in the oesophageal group, 3 in the neuroendocrine group, 2 in the biliary tract group, 1 in the gastric cancer group, and none in the "other tumours" group. The reported median time until the disease progressed (got worse) or death ranged from 1.4 months (other tumours) to 6.4 months (neuroendocrine), while the reported median overall survival ranged from 1.4 months (other tumours) to 5.7 months (oesophageal cancer). No Part A participants were reported as having a measurable tumour reduction. 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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NCT04316143 · results posted 15 October 2024

According to the results reported on ClinicalTrials.gov, this trial (NCT04316143) tested a medicine called zamicastat across four different daily doses: 50 mg, 100 mg, 150 mg, and 200 mg. The trial followed a stepped approach, where participants moved through each dose level in sequence — 29 people started at the 50 mg stage, 27 continued to the 100 mg stage, 21 to the 150 mg stage, and 19 reached the 200 mg stage. The trial was primarily measuring how the drug moved through the body — specifically how much of it got into the bloodstream, how high the levels peaked, and how quickly that peak was reached. These are called pharmacokinetic (or "drug movement") measurements. The reported data shows that as the dose increased, the amount of zamicastat detected in the blood over a 24-hour period also increased. For example, at the 50 mg dose, the total drug exposure over 24 hours was reported as 155 ng·h/mL (a unit that reflects how much drug was in the blood and for how long), while at the 200 mg dose this figure was reported as 1,570 ng·h/mL. The reported peak blood level of zamicastat was 13.3 ng/mL at 50 mg and 134 ng/mL at 200 mg. The time it took to reach that peak was reported as approximately 2 to 4 hours depending on the dose. Similar measurements were also taken for the drug's breakdown products (metabolites), and those figures were also reported across the dose groups. Data for the 150 mg group was not reported for several of these measurements. 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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NCT04966741 · results posted 10 July 2024

According to the results reported on ClinicalTrials.gov, this trial (NCT04966741) enrolled 12 children in total — 7 with a condition called PPL (Pomc, Pcsk1, or Lepr deficiency) and 5 with a condition called BBS (Bardet-Biedl syndrome). Both are rare genetic conditions linked to obesity. The trial was testing a medicine called setmelanotide over 52 weeks (about one year), and the main things being measured were changes in body weight relative to height — specifically something called BMI (body mass index) and a related score that compares a child's BMI to others of the same age and sex. The reported data shows that, looking at the primary (main) measures after 52 weeks: in the PPL group, 85.7% of participants (roughly 6 out of 7) had a meaningful reduction in their BMI score relative to their peers, compared with 80% (4 out of 5) in the BBS group. In terms of overall BMI change, the PPL group showed an average reported reduction of about 25.6%, while the BBS group showed an average reported reduction of about 9.7%. For the secondary (additional) measures, the reported data shows that bone age — a measure of skeletal development taken from hand and wrist X-rays — increased by an average of about one year in the PPL group and 0.7 years in the BBS group over the study period. A child development questionnaire (the ASQ-3) was also completed; results for most participants in both groups were reported as remaining "above" the cutoff (meaning on track developmentally), with no data reported showing a shift to the lowest category for either 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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NCT03679598 · results posted 9 July 2024

According to the results reported on ClinicalTrials.gov, this trial (NCT03679598) enrolled 63 people in total — 32 received a medicine called alvelestat (also known as MPH966) twice a day for 12 weeks, and 31 received a placebo (a dummy treatment with no active ingredient). The trial was set up to measure two main things: changes in a blood substance called desmosine/isodesmosine (a marker linked to tissue breakdown in the lungs) and how many participants experienced at least one unwanted health event (called a treatment-emergent adverse event) during the study. A number of additional blood markers related to inflammation and immune cell activity were also tracked as secondary measurements. The reported data shows that, for the primary blood marker (desmosine/isodesmosine), the within-individual percentage change was approximately 10.1% in the alvelestat group and 8.6% in the placebo group. Regarding unwanted health events, 25 out of 32 participants (about 78%) in the alvelestat group and 23 out of 31 participants (about 74%) in the placebo group experienced at least one such event during the study period. For the secondary blood markers, the reported data shows a range of small numerical changes across both groups — for example, one marker (NE activity) changed by approximately −16.8 units in the alvelestat group compared with +4.0 units in the placebo group, while other markers showed smaller or mixed differences. It is worth noting that the data as submitted does not clearly label each individual secondary marker result, so a full breakdown of every marker cannot be provided 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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NCT03434418 · results posted 17 October 2023

According to the results reported on ClinicalTrials.gov, this trial enrolled 17 people, all of whom received the drug osimertinib. The trial was measuring how well the drug could shrink tumours in participants, as well as tracking how long it took for their disease to worsen and how long they survived overall. Of the 17 who started, 2 completed the study and 15 did not complete it (the reasons for this are not detailed in the reported data). The reported data shows that 8 out of 17 participants had their tumours shrink by a meaningful amount — meaning their scans showed either a large reduction in tumour size or the complete disappearance of detectable tumours. For the secondary measures, the reported data shows that the middle value (median) for progression-free survival — that is, the time before the disease worsened or a participant died — was 10.5 months. The reported overall survival figure — the time from starting treatment until death from any cause — was 13.8 months at the median. Regarding unwanted side effects, the reported data shows that 13 of the participants who received treatment experienced adverse events (unexpected or unwanted health changes noted during the trial), though the breakdown of specific types and severities involves multiple figures across different categories. 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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NCT02034110 · results posted 21 August 2023

According to the results reported on ClinicalTrials.gov, this trial (NCT02034110) enrolled a total of 206 participants across eight different cancer types: anaplastic thyroid cancer (36 people), biliary tract cancer (43 people), gastrointestinal stromal tumour (1 person), low-grade glioma — a type of brain tumour (13 people), high-grade glioma (45 people), adenocarcinoma of the small intestine (3 people), hairy cell leukaemia (55 people), and multiple myeloma (10 people). The trial was primarily measuring the "overall response rate" — that is, the percentage of participants in each cancer group whose tumours showed a meaningful shrinkage or disappeared completely during treatment. The reported data shows the following overall response rates for the cancer groups that had a primary outcome reported: for anaplastic thyroid cancer, the reported figure was 53–56% of participants; for biliary tract cancer, it was 47–53%; for gastrointestinal stromal tumour, it was 0% (noting only one participant was enrolled in this group); for adenocarcinoma of the small intestine, it was 67% (with only three participants in this group); for low-grade glioma, the reported range was 62–69%; and for high-grade glioma, it was 31–33%. The reported data shows a range of figures for some groups, which likely reflects measurements taken at different points or using slightly different counting methods as noted in the submitted results. No primary outcome figures were reported for the hairy cell leukaemia or multiple myeloma groups in the data submitted. It is worth noting that some of these groups were very small — for example, only one person was enrolled in the gastrointestinal stromal tumour group and three in the small intestine cancer group — which means the percentages for those groups are based on very few people. The reported data does not allow any broader conclusions to be drawn about who these results might apply to. 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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NCT02651675 · results posted 13 July 2023

According to the results reported on ClinicalTrials.gov, this trial enrolled nine participants in total, split across three groups: Cohort 1 (3 people), Cohort 2 (3 people), and a Cohort 2 Expansion group (3 people). Eight of the nine participants completed the study — one person in the Cohort 2 Expansion group did not finish. The trial was measuring how the investigational product (a gene therapy delivered via a viral vector) behaved in the body, including tracking unwanted events (called adverse events) linked to the treatment, changes in blood fat levels such as LDL cholesterol, and how much of the viral carrier could be detected in participants' urine and blood. The reported data shows that, for the primary outcome — the number of participants who experienced an adverse event linked to the investigational product — 1 out of 3 participants in Cohort 1, all 3 out of 3 in Cohort 2, and 2 out of 3 in the Cohort 2 Expansion group had such an event recorded. For the secondary outcomes related to blood fat levels, the reported data shows percentage changes from each participant's starting (baseline) levels: LDL cholesterol (a type of fat in the blood often called "bad cholesterol") changed by approximately +7.2% in Cohort 1, +27.4% in Cohort 2, and +9.0% in the Cohort 2 Expansion group. Changes in other blood fat measurements (total cholesterol, HDL cholesterol, triglycerides, and others) were also reported across the three groups, with figures varying considerably between cohorts; for example, one measure showed changes ranging from +32.6% in Cohort 1 to +54.2% in Cohort 2 and −5.5% in the Expansion group. The reported data also shows that detectable amounts of the viral carrier (the vehicle used to deliver the gene therapy) were found in both urine and blood samples for participants in Cohort 1 and Cohort 2, while the reported figure for the Cohort 2 Expansion group was zero for both. Because this was a very small, early-phase trial with only three people per group, the numbers reflect a preliminary exploration rather than a broad population study, and not all detail for every sub-measure was fully 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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NCT04179890 · results posted 25 May 2023

According to the results reported on ClinicalTrials.gov, this trial enrolled 255 participants in the "Uncommon EGFR Mutation Cohort" (people whose lung cancer had a less common type of gene change called an EGFR mutation) and 207 participants in the "Sequencing Cohort" (people with a more common type of EGFR mutation who received two targeted medicines one after the other — first afatinib, then osimertinib). Of those who started, 246 and 191 participants respectively completed the study. The trial was primarily measuring how long participants stayed on their targeted cancer medicine (called an EGFR-TKI, a type of tablet that targets a specific gene change in cancer cells). The reported data shows that, for the primary measure — time on treatment — the median (the middle value when all results are lined up) was approximately 9.9 months for the Uncommon EGFR Mutation Cohort and 27.7 months for the Sequencing Cohort. For the secondary measures, the reported data shows that 96 participants in the Uncommon Mutation Cohort had a recorded tumour response (meaning scans showed their tumour shrank or disappeared) to their treatment. In the Sequencing Cohort, 131 participants had a recorded tumour response to first-line afatinib, and 75 had a recorded tumour response to second-line osimertinib. The reported median overall survival (how long, on average, participants lived from the start of treatment) was 24.4 months in the Uncommon Mutation Cohort and 36.5 months in the Sequencing Cohort. 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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NCT04240886 · results posted 19 May 2023

According to the results reported on ClinicalTrials.gov, this trial (NCT04240886) enrolled 21 adults who received the study drug fosmanogepix (also called APX001) as part of Cohort A. The trial was investigating a treatment for serious fungal infections, and it tracked what happened to participants over roughly six weeks (up to Day 42). Of the 21 people who started the treatment phase, 11 completed it and 10 did not. The trial measured things like survival, how the fungal infection responded, and what medical events occurred during the study. The reported data shows that 25% of participants (that is, roughly 1 in 4) died at some point between their first dose and Day 42. When an independent review committee assessed how each participant's fungal infection had responded by the end of their treatment, the reported data shows that 20% had a complete response (all signs of infection resolved), 20% had a partial response (improvement but not full resolution), 10% had a stable response (little change), 30% had disease progression (the infection got worse), and 20% had died. Combining these categories, 40% were counted as "treatment successes" (complete or partial response) and 60% were counted as "treatment failures" (stable, worsening, or death). The reported data also shows that all 21 participants experienced at least one adverse event (an unexpected medical occurrence during the study), and 13 of the 21 experienced a serious adverse event — meaning a medical event considered significant, such as one requiring hospitalisation. Five participants had a clinically significant abnormality in vital signs (such as blood pressure, heart rate, or temperature), and 12 participants had a clinically significant abnormality in laboratory test results. These figures describe what was observed and recorded; they do not on their own tell us what caused these 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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NCT02702115 · results posted 26 January 2023

According to the results reported on ClinicalTrials.gov, this trial (NCT02702115) tested an investigational gene therapy called SB-318 in people with a rare inherited condition affecting the body's ability to break down certain complex sugars. Participants were placed into three groups, each receiving a different dose of SB-318. One person was enrolled in the lowest-dose group (Cohort 1), two people were enrolled in the middle-dose group (Cohort 2), and no participants were enrolled or completed the highest-dose group (Cohort 3). The trial was measuring things such as unwanted medical events that occurred after treatment, changes in a specific enzyme level in the blood, changes in certain sugar-like substances measured in urine, and how quickly the viral carrier used to deliver the therapy cleared from the body. The reported data shows that all participants who started the trial — one in Cohort 1 and two in Cohort 2 — experienced at least one treatment-emergent adverse event (that is, an unwanted medical event that happened after receiving the treatment). For the enzyme activity measure, the reported change from the starting level was −1.200 units for Cohort 1 and −2.515 units for Cohort 2, meaning the measured enzyme levels were lower at follow-up than at the start for both groups. For the urine sugar-like substance (GAG) levels at 24 months, the reported changes from baseline varied across the different types measured — for example, one GAG measure showed a change of +0.510 for Cohort 1 and −0.090 for Cohort 2, while another showed larger changes of +30.20 and +3.60 respectively. No data was reported for Cohort 3 for these measures. The reported data also shows that by week 24, all enrolled participants (one in Cohort 1, two in Cohort 2) had cleared the viral carrier from their bodily fluids as measured by a laboratory test. 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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NCT02358538 · results posted 7 September 2022

According to the results reported on ClinicalTrials.gov, this trial enrolled 30 people across four groups defined by their type of epilepsy syndrome: 7 with CDKL5, 2 with CSWS, 10 with Lennox-Gastaut syndrome, and 11 with PCDH19. The trial was measuring how often seizures occurred over a 28-day period, comparing that rate to each participant's starting (baseline) rate, and tracking this at around 3 months, 26 weeks, and through a 52-week extension period. Not everyone completed the full study — by the end of the 52-week extension, 4 CDKL5, 0 CSWS, 2 Lennox-Gastaut, and 2 PCDH19 participants had finished. Because the CSWS group had no participants complete the extension, many of that group's results were not reported. The reported data shows that changes in seizure frequency varied considerably between groups and across time points. For the CDKL5 group, the reported median percentage change in seizure frequency was around −47% at 3 months, −38% at 26 weeks, and −44% at the 52-week extension point (where negative numbers indicate fewer seizures compared to baseline). For the Lennox-Gastaut group, the reported median changes were smaller reductions of around −10% at 3 months and −9% at 26 weeks, with the 52-week extension figure not reported. For the PCDH19 group, reported median changes were around −26% at 3 months and −25% at 26 weeks, with the 52-week extension figure also not reported. On the secondary measure of seizure-free days per 28-day period, the reported data shows mean percentage increases in seizure-free days for CDKL5 (around +12% at 3 months and +21% at the extension), PCDH19 (around +8% at 3 months and +17% at the extension), and Lennox-Gastaut (small decreases early, then +16% at the extension). Clinician and patient impression questionnaires were also completed, but the reported data for those measures was described as qualitative rather than numerical scores, so direct comparisons are 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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NCT02985710 · results posted 7 October 2021

According to the results reported on ClinicalTrials.gov, this trial (NCT02985710) enrolled a total of 102 people across two groups: 69 in the "Sudoscan Only" group and 33 in the "Sudoscan Plus" group. The trial was measuring whether a device called Sudoscan — which checks how well the skin conducts a small electrical signal as an indirect measure of sweating ability — could detect nerve-related abnormalities in people with Fabry disease, a rare inherited condition. Specifically, the trial looked at whether the device's readings matched up with clinical signs of a type of nerve damage called small fiber polyneuropathy (damage to the smallest nerves in the body, which can affect sweating and sensation). The reported data shows that for the primary outcome — identifying participants with abnormal skin conductance readings who also had clinical symptoms of small fiber nerve damage — 42 out of 69 participants in the Sudoscan Only group and 12 out of 33 participants in the Sudoscan Plus group had results recorded in this category. For the secondary outcome — detecting a particular pattern in the skin conductance readings in the hands and/or feet that might distinguish Fabry disease from other conditions — the reported data shows 21 out of 69 participants in the Sudoscan Only group and 16 out of 33 participants in the Sudoscan Plus group had this pattern detected. It is worth noting that 4 participants in the Sudoscan Only group did not complete the trial, while all 33 participants in the Sudoscan Plus group completed it; the reasons for non-completion were not reported in the data 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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NCT02317562 · results posted 20 April 2021

According to the results reported on ClinicalTrials.gov, this trial (NCT02317562) enrolled 19 participants, all in a single group called the "I10E Arm." The trial was measuring a "responder rate" at the end of the study — that is, how many participants met certain criteria related to their disability score (a standard scale used to track how a nerve condition called CIDP affects daily functioning) and whether their treatment needed to be changed. Of the 19 people who started, only 5 completed the study, while 14 did not finish. The reported data shows that 15 out of 19 participants were counted as "responders" based on the study's definition — meaning their disability score either stayed the same, decreased, or increased by only 1 point, without needing a change in their CIDP treatment. However, the study's own documentation notes that this trial was ended earlier than planned, and a large number of participants left before it was finished. Because of this, the researchers stated that the responder rate figure is biased and, in their words, "not interpretable." This means the number should be treated with significant caution. The reported data should therefore be understood in that context — the early termination and high dropout rate mean the primary result cannot be reliably understood from this trial alone. No secondary outcome data appears to have been 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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NCT00004418 · results posted 23 October 2020

According to the results reported on ClinicalTrials.gov, this trial enrolled 126 participants, all of whom completed the study with none dropping out. The trial involved a single treatment group who received a combination called GTO/GTE (types of dietary oils). The study was measuring two main things: changes in the blood levels of certain fatty acids known as very long chain fatty acids (VLCFAs — a type of fat that builds up in the body in a condition called Adrenoleukodystrophy, or ALD), and whether any participants showed brain changes on MRI scans linked to childhood ALD. The reported data shows that, on average, blood levels of a specific fatty acid (called C26:0) changed by 0.41 mcg/mL (micrograms per millilitre — a measure of how much of the substance was in the blood) from the starting point before treatment. For the secondary measure, the reported data shows that zero out of 126 participants had an MRI abnormality of the type associated with childhood ALD during the study period. It is worth noting that this trial did not include a comparison group (for example, a group receiving no treatment), so the numbers above reflect only what was observed in the one group 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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NCT02293460 · results posted 9 March 2020

According to the results reported on ClinicalTrials.gov, this trial (NCT02293460) enrolled 43 participants in a single group — meaning everyone received the same treatment, with no comparison group. The trial was measuring whether participants showed a meaningful improvement on a disability scale called the adjusted INCAT score, which runs from 0 (no disability) to 9 (the highest level of disability). A "responder" was defined as someone whose score dropped by at least 1 point from their starting point by the end of the study. By the time the study finished, 37 of the 43 participants had completed it, and 6 did not complete it for reasons not detailed in the reported data. The reported data shows that, out of the group analysed, 32 participants were classified as "responders" — meaning their adjusted INCAT disability score fell by at least 1 point by the end of the study. No further breakdown of how much scores changed, or results for any secondary outcome measures, were included in the data submitted to ClinicalTrials.gov, so those figures cannot be reported here. It is worth noting that because this trial had only one group and no control or comparison group, the 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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NCT02465528 · results posted 21 October 2019

According to the results reported on ClinicalTrials.gov, this trial (NCT02465528) enrolled 22 people in total across four groups based on their cancer type: 1 person with Anaplastic Large Cell Lymphoma (ALCL, a blood cancer), 4 with Inflammatory Myofibroblastic Tumour (IMT, a rare soft-tissue tumour), 12 with Glioblastoma (GBM, an aggressive brain tumour), and 5 with other cancers that share a specific gene change called ALK-positive. The trial was measuring how the drug ceritinib performed across these different cancer types, looking at things like how many participants had their disease controlled or reduced, and how long those responses lasted. Notably, none of the 22 participants completed the study as planned. The reported data shows that the primary measure — disease control at 16 weeks (meaning tumour was shrinking, partially reduced, or at least stable) — varied considerably by cancer group. The ALCL group showed 100% disease control and 100% response rate, though this was based on only one participant. The IMT group reported 75% disease control and 75% response rate across its four participants. The GBM group reported 0% disease control across 12 participants. The "other ALK-positive tumour" group reported 40% disease control but 0% response rate across 5 participants. For how quickly a response appeared, the reported data shows the single ALCL participant responded at around 7 weeks, while the IMT group averaged around 16 weeks. Duration of response figures were not reported for any group. For the measure tracking how long participants went without their disease worsening (progression-free survival), data was not reported for the ALCL group, while the GBM and "other" groups recorded approximately 1.7 weeks each, and the IMT figure was not reported. The reported data also shows that deaths during treatment and the 30-day follow-up period were 0% in the ALCL and IMT groups, 25% in the GBM group, and 20% in the "other ALK-positive" 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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NCT01908816 · results posted 12 August 2019

According to the results reported on ClinicalTrials.gov, this trial enrolled 270 people in total across four groups defined by their eye condition: 93 people with choroidal neovascularisation (abnormal blood vessel growth under the retina, or CNV), 84 with macular oedema (fluid swelling in the central part of the retina, or ME), 58 with rubeosis iridis/neovascular glaucoma (abnormal blood vessels on the iris combined with raised eye pressure, or RI/NVG), and 35 with proliferative diabetic retinopathy/vitreous (PDR/V). The trial was measuring the number of adverse events (unwanted medical occurrences) as its main focus, and also tracked changes in vision, retinal thickness, and other eye-related measures. The reported data shows the trial was stopped early, and as a result only a basic descriptive summary of the findings was carried out rather than a full statistical analysis. The reported data shows that for the primary measure — counting participants who experienced adverse events — the numbers varied across groups. For serious adverse events specifically, 11 participants in the CNV group, 15 in the ME group, 31 in the RI/NVG group, and 14 in the PDR/V group were recorded as having experienced them. For the secondary measures, vision scores (measured in letters on a standard eye chart) showed an average change from the starting point of +7.3 letters in the CNV group and +4.5 letters in the ME group at one time point, and +5.7 and +5.2 letters respectively at another. Retinal thickness (the swelling in the back of the eye) showed average reductions from baseline of around 54–56 micrometres in the CNV group and 84–102 micrometres in the ME group across two time points, where a reduction indicates a decrease in swelling. Results for the other groups were also reported but the data as submitted does not include enough context to describe all individual measurements in full detail. It is important to note that because the trial ended early, no completed participants were recorded in the data, and the sponsor noted that only descriptive analysis was possible — meaning no formal conclusions were drawn 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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NCT02432144 · results posted 30 July 2019

According to the results reported on ClinicalTrials.gov, this trial (NCT02432144) enrolled 12 participants, all of whom received the study treatment called UX003. Eleven of the 12 participants completed the trial, and one did not finish. The trial was measuring two main things: first, how many participants experienced unwanted medical events (called treatment-emergent adverse events, or TEAEs) while on the treatment; and second, how levels of a substance called dermatan sulfate — a type of sugar-like molecule that can build up in the body — changed in participants' urine over time. The reported data shows that all 12 participants experienced at least one TEAE during the study. Of those, 9 participants had mild-to-moderate events (graded as Grade 1 or 2), and 3 participants had severe events (Grade 3). Four participants experienced what are classified as "serious" adverse events — meaning events that led to hospitalisation or were otherwise considered medically significant. One participant stopped the trial due to an adverse event. No life-threatening (Grade 4) or fatal (Grade 5) events were reported. For the urine measurement, the reported data shows a progressive percentage decrease in dermatan sulfate levels over the course of the study, starting at around −62% from the beginning and reaching approximately −89% by the final time point 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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NCT01920477 · results posted 6 June 2019

According to the results reported on ClinicalTrials.gov, this trial enrolled 35 people in total — 17 received a medicine called ofatumumab and 18 received a placebo (a dummy treatment with no active ingredient). The trial was studying a skin condition and looking at whether participants could reach a point where their disease was under control ("remission") while taking only a low dose of steroid tablets (10 mg per day or less). The trial ran to 60 weeks, and only a small number of participants — 2 in the ofatumumab group and 1 in the placebo group — completed it in full. The reported data shows that for the main goal of the trial — the number of participants who achieved a sustained (lasting) remission on a low steroid dose all the way through to week 60 — the answer was zero in both groups. For a related main measure, the total number of days participants spent in remission while on a low steroid dose was reported as 168 days on average for the ofatumumab group and 122 days for the placebo group. For one of the secondary (additional) goals, the reported data shows that approximately 17.6% of participants in the ofatumumab group and 5.6% in the placebo group achieved remission on a low steroid dose at the week 60 mark. The "time to remission" figures were listed as not able to be calculated in either group. One secondary measure — whether anyone achieved remission while completely off steroids — could not be analysed at all because no participants were able to stop their steroid medication during the trial. The reported data shows this was a relatively small trial with a high number of participants who did not complete the full study period, which is noted 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.

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NCT01197378 · results posted 24 July 2018

According to the results reported on ClinicalTrials.gov, this trial enrolled 60 people, all of whom were given a medicine called cysteamine bitartrate. The trial was looking at two main things: how often participants experienced unwanted health events (called adverse events) while taking the medicine, and how the medicine behaved in the body over time — specifically, how much of the medicine could be detected in the blood, and how much of a substance called cystine (which builds up in the cells of people with cystinosis, a rare inherited condition) was present in white blood cells. Of the 60 who started, 59 received the treatment and 53 completed the study. The reported data shows that 58 out of 59 participants who received the medicine experienced at least one adverse event — that is, an unwanted health occurrence during the study period. Of these, 37 participants had adverse events that the study doctor considered possibly, probably, or definitely related to the medicine. Breaking these down by how serious they were: 24 participants had mild events, 32 had moderate events, 3 had severe events, and the data does not report any life-threatening events separately in the figures provided. Regarding the blood levels of the medicine, the reported measurements across different time points ranged from 0.17 to 0.48 mg/L. For the white blood cell cystine levels — a way of tracking how much of the stored substance remained in cells — the reported figures across different time points ranged from 0.65 to 1.68 units (measured as nmol half-cystine per mg of protein). 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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NCT01733316 · results posted 24 May 2017

According to the results reported on ClinicalTrials.gov, this trial enrolled 41 participants, all of whom had cystinosis — a rare condition where a substance called cystine builds up harmfully in the body's cells. The trial compared two forms of a medicine: an older version called Cystagon® (taken four times a day) and a newer delayed-release version called RP103 (taken twice a day). The main thing the trial was measuring was the level of cystine inside white blood cells at different times of day — specifically, whether there was a difference between morning and non-morning readings — as a way of tracking how well each medicine was keeping cystine levels under control across the day. The reported data shows that during the Cystagon® phase, the average difference between morning and non-morning white blood cell cystine readings (on a mathematical log scale used to smooth out the numbers) was -0.229, meaning morning readings tended to be higher than non-morning ones. During the RP103 phase, that average difference was 0.080, meaning the gap between morning and non-morning readings was much smaller. The trial also recorded the number of participants who experienced adverse events (unexpected medical occurrences during the study). The reported data shows that during the Cystagon® phase, 31 out of 41 participants had at least one adverse event, compared with 38 out of 41 during the RP103 phase and 32 out of 38 during a longer follow-up period. Serious adverse events were reported for 4 participants during the Cystagon® phase, 20 during the RP103 phase, and 18 during the longer follow-up phase. A smaller group of participants who reported bad breath as a side effect took part in an additional sub-study measuring how the medicine moved through the body and its relationship to breath odour; those measurements were also reported but involved only a subset of participants and the exact number in that sub-group was not reported 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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NCT00958841 · results posted 26 July 2016

According to the results reported on ClinicalTrials.gov, this trial enrolled 118 people across several types of neuroendocrine tumours (NETs) — a group of tumours that can arise in various parts of the body and are often identified by particular chemicals they produce in the blood. All participants received a long-acting injectable medicine called pasireotide LAR 60mg. The trial was measuring whether participants' blood levels of specific tumour-related chemicals either returned to normal, or fell by more than half, after six months of treatment. Ninety participants reached the six-month point, and 22 completed the full study. The primary goal — looking at a combined group of pancreatic NET patients — could not be fully assessed because fewer patients than planned (20 out of an intended 34) could be included in that analysis. The reported data shows that, among the tumour types where enough patients were enrolled to allow analysis, the percentage who met the response target at six months varied. For gastrinoma patients, 46.2% were recorded as responders. For prolactinoma patients, 21.6% were recorded as responders. For Nelson's syndrome patients (a condition involving a particular tumour of the pituitary gland), 50% were recorded as responders. In terms of actual patient numbers, the reported data shows 5 out of 8 gastrinoma patients, 9 out of 7 prolactinoma patients (note: these figures are as submitted to ClinicalTrials.gov and appear inconsistent in the source data), and 6 out of 6 Nelson's syndrome patients met the response criteria. For other tumour types — including VIPoma and glucagonoma — the reported responder rate was 0%, and some tumour types had too few participants to be formally analysed at all. 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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NCT00634049 · results posted 11 January 2016

According to the results reported on ClinicalTrials.gov, this trial enrolled 149 participants, with 146 completing the study and 3 not completing it. All participants received the antifungal medicine isavuconazole. The trial was measuring how participants with serious, invasive fungal infections responded to treatment across several different types of fungal infections, including *Aspergillus*, *Mucorales*, and various other fungal species. Participants were grouped according to their type of infection and other factors (such as whether they had kidney problems), and responses were assessed at set timepoints — day 42, day 84, and at the end of treatment. The reported data shows that the main (primary) outcome — an overall "success" rating judged by an independent review committee — varied considerably depending on the type of fungal infection. For example, among participants with *Aspergillus* infection who did not have kidney problems, 50% were rated as having an overall successful response, compared with 25% of those who did have kidney problems. For *Mucorales* infections treated as the first-line approach, 14.3% were rated as successful overall, while for those whose infection had not responded to a previous treatment, the figure was 9.1%, and for those who could not tolerate previous treatments it was 0%. For other fungal types, overall success rates ranged from roughly 9% to 47%, depending on the subgroup. The secondary outcomes — which looked separately at symptom improvement (clinical response), fungal clearance (mycological response), and imaging changes (radiological response) — were also reported across these same subgroups, with clinical response rates generally higher than the overall combined measure, and radiological response rates generally lower. 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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NCT00004980 · results posted 9 July 2012

According to the results reported on ClinicalTrials.gov, this trial looked at a brain stimulation technique called repetitive Transcranial Magnetic Stimulation (rTMS) — a procedure that uses magnetic pulses directed at the head — as a potential treatment for auditory hallucinations (hearing voices or sounds that aren't there). A total of 50 people took part: 27 were assigned to receive active rTMS and 23 received a sham (dummy/placebo) version that mimicked the procedure without the active stimulation. Of those, 25 in the active group and 21 in the placebo group completed the trial. The reported data shows that the main thing being measured was a "Hallucination Change Score" (HCS), rated on a scale where 0 means hallucinations stopped, 10 means no change, and 20 means they became twice as severe. After nine sessions, the active rTMS group reported an average score of 5.85, while the placebo group reported an average of 8.61. For a secondary measure looking at how often hallucinations occurred (on a 0–9 scale where higher means more frequent), the active group showed an average change of 1.42 points from their starting point, compared to 0.15 for the placebo group. On a separate "Clinical Global Improvement" scale (where 1 is dramatically improved and 7 is dramatically worsened), the active group averaged 2.84 and the placebo group averaged 3.79. The reported data also shows that 14 out of 27 participants in the active rTMS group were classified as "responders" (meaning their HCS score reached 5 or below), compared to 4 out of 23 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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NCT00004500 · results posted 1 May 2012

According to the results reported on ClinicalTrials.gov, this trial involved 69 newborn babies in total — 38 who received a treatment called lucinactant and 31 who received standard care. The trial was measuring how long babies needed help breathing from a machine (called mechanical ventilation), as well as tracking deaths and whether babies developed air leaks around the lungs during their care. The reported data shows that babies in the lucinactant group spent an average of 10.2 days on the breathing machine, compared to 8.1 days in the standard care group. For the secondary outcomes, no deaths were recorded in either group. The reported data also shows that 2 babies in the lucinactant group experienced air leaks around the lungs (such as air trapped in the lung tissue or around the heart), while no air leaks were recorded in the standard care group. It is worth noting that not all babies who started the trial were followed up at the one-year mark — 24 from each group were included in that follow-up. 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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NCT00390858 · results posted 15 August 2011

According to the results reported on ClinicalTrials.gov, this trial enrolled 40 children and teenagers who had too much iron building up in their bodies — 20 younger children (under 12 years old) and 20 adolescents (aged 12 and over). The trial was measuring how iron levels in the liver changed over time, how quickly the body eliminated iron overall, and what unintended medical events occurred during the study. Of the 40 who started, 24 completed the full study period (11 younger children and 13 adolescents). The reported data shows that, for the primary measure of iron stored in the liver, the average level at the start of the study was 6.25 units (mg of iron per gram of liver dry weight) in younger children and 5.73 units in adolescents. By the end of the study period examined, these figures had changed to 5.46 and 4.66 units respectively — representing an average decrease of 0.9 units in the younger group and 1.10 units in the adolescent group. For the secondary measure of how fast iron was being removed from the body overall, the reported rate was approximately 0.43 mg per kilogram of body weight per day in younger children and 0.41 in adolescents. A separate secondary measure looked at a blood marker of iron levels called serum ferritin; the reported data shows a relative change of approximately 62% in younger children and 55% in adolescents over the course of the study. Regarding unintended medical events, all 20 younger children and all 20 adolescents experienced at least one such event during the study, though the data does not detail the nature of these events in the summary 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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NCT00303329 · results posted 9 May 2011

According to the results reported on ClinicalTrials.gov, this trial enrolled 184 people in total — 85 with a condition called beta-thalassemia (a blood disorder affecting haemoglobin) and 99 with other rare anaemias (conditions where the blood cannot carry enough oxygen). The trial was measuring the safety of a medicine called deferasirox, which is used to remove excess iron from the body, as well as tracking how iron levels in the liver and blood changed over the course of the study. Not everyone finished the trial: 50 of the beta-thalassemia group and 37 of the rare anaemias group completed it. The reported data shows that, for the primary outcome — counting how many participants experienced any unwanted medical events (called adverse events) or serious adverse events, or died — all 85 beta-thalassemia participants and all 99 rare anaemias participants had at least one adverse event recorded. Serious adverse events (those involving hospitalisation, life-threatening situations, lasting disability, or death) were reported in 36 of the 85 beta-thalassemia participants and 50 of the 99 rare anaemias participants. Deaths were reported for 2 participants in the beta-thalassemia group and 12 in the rare anaemias group. For the secondary outcomes, the reported data shows that iron levels in the liver (measured by biopsy) fell by an average of 5.17 units in the beta-thalassemia group and 5.10 units in the rare anaemias group. A different liver iron measurement method showed a small average increase of 0.19 units in the beta-thalassemia group and a decrease of 2.04 units in the rare anaemias group. A blood marker of iron build-up called serum ferritin also changed over the study — starting at around 4,321 in the beta-thalassemia group and 3,269 in the rare anaemias group, and ending with average decreases of 612 and 382 units 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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NCT00010439 · results posted 17 September 2010

According to the results reported on ClinicalTrials.gov, this trial involved 10 participants, all of whom received a medication called alendronate (a type of drug commonly used to support bone strength). The trial was measuring whether participants showed an increase in bone mineral density — essentially, how dense and solid their bones were — at the lower spine and/or hip after 12 months of treatment, compared to measurements taken before treatment began. All 10 participants started the trial and all 10 completed it. The reported data shows that all 10 out of 10 participants had an increase in bone mineral density at the lower spine and/or hip by the 12-month mark, compared to their measurements before treatment. For the secondary outcomes — which looked at additional measurements — the reported data shows that 9 out of 10 participants were assessed for fractures before and after treatment (with 1 participant recorded separately in that count). The data also shows that 9 participants were analysed for changes in something called the "mineral apposition rate" — a measure from bone biopsies (small samples of bone tissue) of how quickly new bone was being laid down — with an average change reported from a higher rate (1.9 micrometres per day) to a near-normal rate (1.2 micrometres per day). Additionally, 9 participants were analysed for changes in biochemical markers — substances in blood and urine used to track bone activity — with the results described as showing average insignificant changes in those markers. 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.