Reported trial results for Muscular Dystrophy
Every Muscular Dystrophy 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.
58 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
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NCT03179631 · results posted 10 March 2026
According to the results reported on ClinicalTrials.gov, this trial enrolled 360 participants in total — 184 in the ataluren group and 176 in the placebo group. The trial ran in two back-to-back phases, each lasting 72 weeks, for a combined total of about three years. The main thing being measured was how far participants could walk in six minutes (known as the "6-minute walk distance" or 6MWD), which is a standard way of tracking physical function over time in conditions that affect movement. A secondary measure looked at how long it took participants to walk or run 10 metres. The reported data shows that, over the first 72-week (double-blind) phase, walking distance declined in both groups. In the ataluren group, the average distance walked in six minutes fell by approximately 82 metres (in the more specific "mITT" sub-group) or about 53 metres (in the broader "ITT" group) from where it started. In the placebo group, the reported declines were approximately 90 metres and 67 metres respectively. For the rate of change week by week, the ataluren group declined at roughly 1.14 metres per week (mITT) or 0.74 metres per week (ITT), compared with 1.25 and 0.94 metres per week in the placebo group. On the 10-metre walk/run test, the time to complete it increased (meaning it took longer) by about 3.1 seconds in the ataluren group and about 3.8–3.9 seconds in the placebo group across both analysis 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT05185622 · results posted 24 October 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT05185622) enrolled 54 children and young people across six treatment groups, ranging in size from 6 to 16 participants per group. All 54 participants who started the trial completed it — none dropped out. The trial was measuring a range of unwanted medical events (called adverse events) that occurred during the study, as well as any changes in participants' height over 12 weeks. The reported data shows that, when it came to any adverse event occurring during the study, the numbers across the six groups were 7, 9, 6, 4, 3, and 12 participants respectively. For adverse events that investigators considered possibly or probably related to the study drug, the reported numbers were 1, 7, 3, 4, 1, and 8 participants across the six groups. Severe adverse events — meaning those serious enough to limit a person's daily activities or require hospitalisation — were reported in 0, 0, 0, 1, 0, and 1 participant(s) across the groups. Similarly, serious adverse events (a stricter category including things like hospitalisation or life-threatening events) were also reported in 0, 0, 0, 1, 0, and 1 participant(s). Importantly, the reported data shows that no participant in any group stopped the treatment because of an adverse event. Regarding height, the reported average change over 12 weeks across the six groups was 1.98 cm, 2.35 cm, 0.55 cm, 2.29 cm, 0.60 cm, and 1.27 cm 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03692312 · results posted 11 September 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT03692312) looked at a drug called tideglusib in people with congenital myotonic dystrophy type 1 (a genetic muscle condition present from birth). A total of 53 people took part — 27 received tideglusib and 26 received a placebo (a dummy treatment with no active ingredient). Of those, 25 in each group completed the study. The trial measured changes in several rating scales filled out by clinicians and caregivers, as well as a short walking test, to see how symptoms and overall condition changed over time. The reported data shows the following for the main (primary) outcome — a clinician-completed symptom severity scale scored from 0 to 44, where a lower (more negative) number means greater improvement: the tideglusib group had an average change of −1.65, while the placebo group had an average change of −3.40. For the secondary outcomes, on a 7-point clinician "improvement" scale (where 1 = very much improved and 7 = very much worse), the tideglusib group scored 3.11 and the placebo group scored 2.77. On a caregiver concern scale (0–30, lower is better), tideglusib showed a change of −1.74 versus −6.47 for placebo. On a caregiver-completed symptom severity scale (0–44), the changes were −0.18 for tideglusib and −3.36 for placebo. On a clinician severity scale (1–7), changes were −0.20 and −0.12 respectively. Finally, on the 10-metre walk/run test (measured in seconds, where a negative number means faster), the tideglusib group improved by −0.54 seconds and the placebo group by −0.19 seconds. 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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NCT04281485 · results posted 8 June 2025
According to the results reported on ClinicalTrials.gov, this trial enrolled 114 boys across two groups: 79 in Cohort 1 and 35 in Cohort 2. The trial was measuring changes over 52 weeks (about one year) in physical function, muscle protein levels, and a blood marker related to muscle. The main thing being tracked was performance on the North Star Ambulatory Assessment (NSAA) — a 17-task test of everyday physical skills scored from 0 (unable to do the task independently) to 34 (full independent function), with higher scores meaning better physical ability. By the end of the study, 69 participants in Cohort 1 and 28 in Cohort 2 had completed the trial. The reported data shows that, on the primary measure, the average NSAA score changed by +1.46 points in Cohort 1 and +1.37 points in Cohort 2 from their starting scores over 52 weeks. For the secondary measures, muscle biopsy results showed that the level of a specific muscle protein (mini-dystrophin, measured by a laboratory method called LC-MS) changed by +85.88% of the normal level in Cohort 1 and +0.13% in Cohort 2. When looking at the proportion of muscle fibres showing this protein under a microscope, Cohort 1 showed a change of +50.15 percentage points, while Cohort 2 showed a change of −1.31 percentage points. A blood marker called creatine kinase (a substance that can reflect muscle activity) changed by +0.68 units per litre in Cohort 1 and +1.06 units per litre in Cohort 2. On the NSAA skill-based measures, the reported data shows that roughly 55% of skills that could potentially be gained were gained in Cohort 1, compared to 42% in Cohort 2. When looking at skills either improved or maintained, both groups reported a similar proportion — around 88–89% of the 17 assessed skills. 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 ↗
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NCT04572893 · results posted 21 May 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT04572893) tested an investigational drug called MYK-491 in people with a type of heart condition called dilated cardiomyopathy (DCM) — where the heart's main pumping chamber becomes enlarged and weakened. Participants were grouped based on whether their condition was linked to specific gene changes (in genes called MYH7 or TTN, or other genetic causes) or had no identified genetic cause. The trial ran in two stages, called Part A and Part B. In Part A, 41 people started (12 in the MYH7 group, 14 in the TTN group, 5 in the DCM-genetic group, and 10 in the non-genetic group), and 40 completed it. Of those, 19 went on to Part B, which was still ongoing at the time the results were submitted — so no Part B completions were recorded. The reported data shows that in Part A, the primary measurements tracked things like unwanted medical events (called adverse events), serious medical events, changes in physical examinations, changes in vital signs (such as blood pressure and heart rate), and certain laboratory test results. According to the results reported on ClinicalTrials.gov, adverse events of any kind were reported in 9 out of 12 participants in the MYH7 group, 9 out of 14 in the TTN group, 4 out of 5 in the DCM-genetic group, and 4 out of 10 in the non-genetic group. The reported data shows that no participants in any group experienced a serious adverse event in Part A. Clinically significant changes in vital signs were reported for zero participants across all groups, and clinically significant changes in physical examinations were reported for one participant in the TTN group only. A secondary measurement looked at how long the heart spent actively pumping with each beat (called Left Ventricular Ejection Time, or LVET, measured in milliseconds). The reported data shows changes from each participant's starting point varied across groups and time points — for example, in Part B the MYH7 group showed an average change of around 38 milliseconds at one time point and 25 milliseconds at another, while the TTN group showed around 44 milliseconds, and the non-genetic group around 20 milliseconds. Some Part B group figures were not 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04428476 · results posted 24 February 2025
According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called deramiocel and enrolled 13 participants. The trial was measuring two main things over 12 months: how often participants experienced unwanted health events (called adverse events) while on the treatment, and whether there were any changes in arm and hand function over time. The arm function was measured using a scoring tool called the Performance of the Upper Limb Test, Version 2 (PUL 2.0), which gives a score from 0 to 42, where a higher score means better arm and hand function. Notably, the reported data shows that none of the 13 participants completed the trial as planned. The reported data shows that 11 out of 13 participants experienced at least one adverse event (an unwanted health event that occurred during treatment) over the 12-month period. Breaking these down by how serious they were: 6 participants had mild events, 3 had moderate events, and 2 had severe events requiring medical attention. No life-threatening events or deaths were reported. Regarding arm function, the reported data shows an average change of −1.8 points on the 0–42 PUL 2.0 scale from the start of the trial to 12 months, meaning scores on average went down slightly over that period. For the longer-term outcomes — such as adverse events and arm function scores at 24, 36, 48, and 60 months — the data was not reported on ClinicalTrials.gov, so no figures are available for those 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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NCT02485938 · results posted 9 January 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT02485938) enrolled 25 people in total — 12 in the usual care group and 13 in the CAP-1002 (an experimental cell therapy) group. The trial was looking at a range of safety-related measurements, including whether any serious heart-related events occurred shortly after treatment, how often unwanted medical events were reported, and how certain blood test values changed over 6 and 12 months. The reported data shows that when it came to the main heart-related safety events tracked — such as sudden unexpected death or a major heart event within 72 hours of the infusion procedure — zero participants in either group experienced any of these. Regarding general unwanted medical events, 10 out of 12 usual care participants and 12 out of 13 CAP-1002 participants had at least one reported medical event during the study period. Serious medical events (those involving hospitalisation, disability, or being life-threatening) were reported in 1 usual care participant and 3 CAP-1002 participants. For the blood test measurements — including salts (sodium, potassium, chloride), albumin (a protein), glucose (blood sugar), and various blood cell counts — the reported data shows small changes from starting values at the 6-month and 12-month check-ins in both groups, though the specific meaning of those changes is not stated 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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NCT04060199 · results posted 11 December 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04060199) enrolled 77 participants in total — 38 received viltolarsen and 39 received a placebo (an inactive treatment used for comparison). Most participants completed the trial: 36 in the viltolarsen group and 38 in the placebo group. The trial was measuring a physical ability test called the "Time to Stand" (TTSTAND) — specifically, how quickly a participant could get up from lying flat on the floor to a standing position. This was converted into a speed score (rises per second), and the trial tracked how that score changed from the start of the study. The reported data shows a series of measurements of change in that standing-up speed score across different time points for both groups. For the viltolarsen group, the reported changes from baseline were 0.026, 0.027, 0.027, and 0.009 rises per second across the measured time points. For the placebo group, the corresponding reported changes were 0.013, 0.014, 0.027, and 0.013 rises per second. In plain terms, a positive number means participants were getting up from the floor slightly faster compared to when they started the trial. The data as submitted does not include labels identifying which specific time points each measurement corresponds to, so a full time-by-time comparison 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT03760029 · results posted 19 September 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT03760029) enrolled 312 participants in total across three groups: 99 children who could walk and were under 6 years old, 177 participants who could walk and were aged 6 or older, and 36 participants who could not walk. The study followed participants for up to 30 months and tracked key physical milestones — specifically, the age at which participants lost the ability to walk, stand, or feed themselves. It also used a standardised 17-item physical function test called the Northstar Ambulatory Assessment (NSAA), which scores movement abilities on a scale of 0 to 34, where higher scores reflect greater physical function. The reported data shows that, based on a statistical estimation method (Kaplan-Meier, which uses the available data to estimate the point in time when half the group reached a milestone), the age at which participants who could walk and were 6 or older lost the ability to walk was estimated at 14.4 years; the same figure was reported for loss of the ability to stand in that group. For non-ambulatory participants, those ages were estimated at 11.1 years (walking) and 11.0 years (standing). Loss of ability to self-feed was only estimable for non-ambulatory participants, reported at 20.0 years; for all ambulatory groups, this figure was listed as not available in the reported data. Regarding the NSAA physical function scores, the reported changes from the starting point varied by group and time: younger ambulatory participants (under 6) showed score increases of +1.3, +2.7, and +4.1 points at 6, 12, and 18 months respectively, while older ambulatory participants (6 and over) showed score decreases of −1.0, −2.1, and −3.6 points at the same 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 ↗
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NCT04632940 · results posted 26 August 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04632940) enrolled 73 participants in total — 37 in the pamrevlumab group and 36 in the placebo group — across a 52-week double-blind period followed by a 52-week open-label extension period. The trial was measuring physical functioning in people with Duchenne muscular dystrophy, a condition that progressively affects muscle strength and movement. The main thing being tracked was a score from a 17-item movement assessment called the North Star Ambulatory Assessment (NSAA), which rates everyday physical activities on a scale from 0 (worst) to 34 (best). Several movement-based tests were also tracked as secondary measures. The reported data shows that, by week 52 of the double-blind period, both groups showed a decline in their NSAA total score from where they started. The pamrevlumab group had an average decline of approximately 3.0 points, while the placebo group had an average decline of approximately 2.5 points. For the stair-climbing speed test, the pamrevlumab group declined by about 1.9 cm/second on average, compared with about 3.8 cm/second in the placebo group. For the 10-metre walk/run test, the pamrevlumab group declined by about 0.18 metres/second and the placebo group by about 0.20 metres/second. The time taken to stand up from lying down increased (meaning it took longer) by about 2.2 seconds in the pamrevlumab group and about 1.9 seconds in the placebo group. For the measure tracking time until participants could no longer walk, the reported data shows the median value was not able to be calculated for either group 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT04956289 · results posted 20 August 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04956289) enrolled 20 boys or young men receiving the drug viltolarsen at a dose of 80 mg/kg. Half of the participants (10) were able to walk independently (described as "ambulant") and half (10) were not (described as "non-ambulant"). All 20 participants completed the trial. The trial's primary focus was on tracking unwanted medical events — known as "adverse events" — that occurred during treatment, rather than measuring physical function or other health outcomes. The reported data shows that 19 out of 20 participants experienced at least one treatment-emergent adverse event (meaning a medical event that appeared or worsened after starting the drug). When these events were grouped by how serious they were, the reported numbers show 7 participants had events classed as mild and 12 had events classed as moderate, with none recorded at the highest severity level. Of the 19 participants with adverse events, 17 had events whose worst recorded outcome fell into the lower-severity categories, and 2 had events with a more notable outcome recorded, though no further detail on outcomes was provided in the structured data. Separately, 4 participants had adverse events that were considered possibly related to the study drug itself — 3 of these were mild and 1 was moderate. The reported data also shows that, when looking at what action was taken in response to adverse events, 16 participants required no change to their treatment, 2 had their dose reduced or treatment temporarily stopped, and 1 had their treatment permanently stopped; no action was reported for the remaining participant in that breakdown. No statistical analysis was performed on any of these figures, and fuller details were noted as being available in the trial's separate adverse events section. 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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NCT04003974 · results posted 10 July 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04003974) enrolled 80 people in total — 40 who took losmapimod (15 mg twice daily) and 40 who received a placebo (a dummy treatment with no active ingredient). The trial was looking at a condition called facioscapulohumeral muscular dystrophy (FSHD), and its main goal was to measure whether losmapimod changed the activity of a protein called DUX4 in muscle tissue. DUX4 is thought to play a role in FSHD, and its activity was estimated by looking at how certain genes behaved in muscle samples taken before and after the treatment period. The reported data shows that, for the primary outcome — the change in DUX4 activity measured in muscle biopsies — the losmapimod group showed a change of 0.83 units (in a laboratory measurement called "delta Ct") from their starting point, while the placebo group showed a change of 0.40 units. A higher number in this measurement indicates a reduction in DUX4 activity, though the clinical meaning of this difference was not described in the submitted results data. Regarding unintended medical events (called adverse events) that occurred during the trial, the reported data shows that 29 out of 40 participants in the losmapimod group and 23 out of 40 in the placebo group experienced at least one such event. Two participants in the losmapimod group experienced a serious adverse event, compared to none in the placebo group. No participant in either group stopped taking the study drug early because of an adverse event, and no cases of a specific liver concern being monitored were reported in 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04371666 · results posted 12 March 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04371666) looked at a medicine called pamrevlumab compared to a placebo (an inactive dummy treatment) in people with a muscle-wasting condition. A total of 49 people were assigned to the pamrevlumab group and 49 to the placebo group for the main 52-week treatment period. The trial measured changes in upper limb function, breathing capacity, grip strength, and heart pumping function over that year. The reported data shows that for the primary measure — upper limb function, scored on a 42-point scale where higher scores mean better function — both groups showed a small decline from their starting scores over 52 weeks. The pamrevlumab group's score changed by approximately −2.0 points and the placebo group's by approximately −2.1 points. For breathing, the reported data shows the pamrevlumab group's lung capacity (as a percentage of what would be predicted for a healthy person of the same age, sex and height) changed by about −8.3 percentage points, compared with about −6.0 percentage points in the placebo group. Grip strength in both hands declined by roughly 7.6 newtons (a unit of force) in the pamrevlumab group, while the placebo group showed almost no change (around −0.07 and −0.01 newtons). For heart pumping function, the pamrevlumab group changed by about −0.5 percentage points and the placebo group by about −1.1 percentage points. The peak breathing flow measure changed by approximately −4.9 percentage points in the pamrevlumab group and −4.5 percentage points 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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NCT02836418 · results posted 22 December 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT02836418) enrolled 8 participants, all of whom received at least one dose of the study drug, ATYR1940, and all 8 completed the trial. The study was primarily measuring safety-related outcomes — that is, it was tracking whether participants experienced any unwanted medical events, abnormal test results, or other changes while taking the study drug, rather than measuring whether the drug treated a condition. The reported data shows that out of the 8 participants, 7 experienced what are called "treatment-emergent adverse events" (TEAEs) — meaning unwanted medical occurrences that appeared after starting the study drug — while none experienced a "serious adverse event" (a more severe category, such as hospitalisation or a life-threatening event). Three participants were reported as developing antibodies against the study drug itself (known as anti-drug antibodies). Two participants had a particular blood test result — a "Jo-1 antibody" level — that reached or exceeded a threshold of 1.5 units per millilitre, which under the trial's rules required them to stop taking the study drug. One participant had a laboratory test abnormality that was recorded as an adverse event. Two participants had an abnormal heart tracing (ECG) result that was recorded as an adverse event. No participants were reported as having a lung function event recorded as an adverse event. The reported data shows only these counts from a very small group of 8 people, and no further numerical detail beyond these figures was included in the submitted results. This type of early-phase trial is typically focused on monitoring and recording these kinds of measurements, rather than drawing 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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NCT03777319 · results posted 23 October 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT03777319) enrolled just two participants — one child with Duchenne muscular dystrophy (DMD) who received spironolactone, and one who received prednisolone (a steroid). Both participants completed the trial. The trial was designed to compare these two medicines in boys with DMD who had not previously taken steroids, by measuring muscle-related outcomes over time. The reported data shows that for the main physical test — how long it took each participant to walk 100 metres — the child on spironolactone improved by 0.6 seconds (a shorter time), while the child on prednisolone improved by 5.3 seconds. For the blood salt (electrolyte) levels monitored monthly as a safety measure, the readings across both participants stayed within a similar range throughout the study (for example, sodium levels were generally between 139 and 142 mmol/L for both). For the secondary muscle strength measurements (using a device that measures how hard a person can push with their arms and legs), the reported data shows the child on prednisolone recorded scores ranging from roughly 3.3 to 5.3 kg across different time points, while the child on spironolactone recorded scores of 0 kg at several time points and up to 4.1 and 2.8 kg at later ones. It is important to note that with only one participant in each group, the reported data shows individual observations only — no broader conclusions about groups of people can be drawn from numbers this small. 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 ↗
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NCT03167255 · results posted 18 November 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03167255) enrolled 16 boys with Duchenne muscular dystrophy — 8 in a lower-dose group (40 mg/kg) and 8 in a higher-dose group (80 mg/kg) of the investigational drug NS-065/NCNP-01. All 16 participants completed the trial. The trial was measuring changes in physical movement tasks — specifically how long it took participants to stand up from the floor, run or walk 10 metres, and climb 4 stairs — and comparing those changes to a matched group of historical control participants from an earlier related trial. The trial also recorded how many participants experienced side effects that were considered related to the treatment. The reported data shows that, for the "time to stand" test, the combined group of participants showed small changes compared to the historical controls across multiple time points, ranging from around −0.11 seconds to +0.54 seconds (where a negative number means it took less time, and a positive number means it took more time). For the "run/walk 10 metres" test, the reported changes compared to historical controls ranged from approximately −0.88 seconds to −0.46 seconds across time points for the combined group, meaning participants in the trial took less time than the historical controls at those points. For the "climb 4 stairs" test, the combined group changes ranged from approximately +0.01 seconds to +1.05 seconds compared to historical controls. Regarding side effects, the reported data shows that all 16 participants experienced at least one treatment-related adverse event (an unwanted health event that occurred during the study). Of these, 1 participant (in the 80 mg/kg group) experienced a serious adverse event, and 2 participants (both in the 80 mg/kg group) experienced a severe adverse event. No participants in the 40 mg/kg group were reported to have experienced a serious or severe adverse event. The data does not provide further detail about the nature of these events within this results report. 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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NCT02439216 · results posted 23 September 2022
According to the results reported on ClinicalTrials.gov, this trial tested a medicine called CAT-1004 across three separate stages (called Part A, Part B, and Part C). Part A was a short 7-day open-label phase (meaning everyone knew what they were receiving) involving 17 participants across three dose levels. Part B was a 12-week double-blind phase (where neither participants nor researchers knew who received the medicine or a dummy treatment called a placebo) involving 31 participants. Part C was a longer open-label phase running up to 138 weeks, involving 31 participants — though only 13 of those completed it. The trial measured things like muscle changes visible on MRI scans, physical movement tasks, and tracked unwanted health events that occurred during the study. The reported data shows that the main thing being measured in Part B was a specific MRI reading of lower leg muscles — higher numbers suggest more muscle damage or fat build-up. At the start of the study, the groups receiving the two CAT-1004 doses showed readings of 2.04 and 1.26 milliseconds above baseline respectively, while the placebo group showed a change of 0.34 milliseconds. By week 12, the reported changes from the start of the study were −0.25 milliseconds for the lower dose group, +0.01 milliseconds for the higher dose group, and 0.00 milliseconds for the placebo group. For the physical movement tests at week 12 — including a 10-metre walk/run, climbing 4 stairs, and standing up from lying down — the reported data shows small changes in speed across all groups, with the placebo group generally showing slightly larger reductions in speed compared to the CAT-1004 groups in Part C. It is worth noting that all the changes recorded across these physical tests were very small in size. The reported data also shows that during Part A, some participants experienced treatment-related unwanted health events (2 out of 5 in the lowest dose group, 5 out of 6 in each of the two higher dose groups), while in Parts B and C, the number of such events reported was zero across all groups. Serious unwanted health events data was only partially 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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NCT01603407 · results posted 12 August 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT01603407) looked at three different steroid treatment approaches for boys with Duchenne Muscular Dystrophy (DMD): daily prednisone, daily deflazacort, and intermittent (not every day) prednisone. A total of 196 participants were enrolled across the three groups — 65, 65, and 66 respectively — and by the end of the study, 54, 54, and 56 had completed it. The trial measured lung function, movement ability, how satisfied participants were with their treatment, and several other physical measures over up to 36 months. The reported data shows the following results averaged across follow-up visits. For lung function (how much air participants could breathe out forcefully), all three groups recorded similar figures: 1.4 litres for daily prednisone, 1.4 litres for daily deflazacort, and 1.5 litres for intermittent prednisone. For the speed at which participants could rise from the floor, the reported figures were 0.24 rises per second for both daily groups and 0.18 for the intermittent group. On a treatment satisfaction questionnaire scored from 0 to 100 (where higher means more satisfied), scores were 71.2, 67.8, and 65.1 for the three groups respectively. For the secondary measures, a 17-item movement ability scale (maximum score 34) returned averages of 23.7, 24.0, and 20.7; the distance walked in six minutes averaged 384.95 metres, 384.17 metres, and 346.81 metres; and ankle joint flexibility averaged 4.39, 3.29, and 2.67 degrees across the three groups. These numbers are what the trial sponsor submitted to ClinicalTrials.gov, and no information about the reasons behind any differences between groups 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03439670 · results posted 13 July 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03439670) enrolled a total of 121 participants across six treatment groups, with group sizes ranging from 15 to 30 people. The trial was measuring how a drug called vamorolone compared to a placebo (an inactive treatment), focusing on a physical ability test called the "Time to Stand" test. This test measures how quickly a person can rise from the floor, recorded in "rises per second" — essentially, a higher number means rising more quickly. The reported data shows that the primary outcome compared changes in the Time to Stand test score from the start of the trial to 24 weeks later, looking specifically at Treatment Group 1 (vamorolone at 6.0 mg/kg/day) versus Treatment Group 2 (placebo). According to the results reported on ClinicalTrials.gov, the placebo group's score changed by -0.007 rises per second (a very slight decline), while the vamorolone group's score changed by +0.054 rises per second (a slight increase). No further outcome measure results were included in the submitted data for the remaining four treatment groups, so those figures were not reported in the structured results available. It is also worth noting that not all participants who started the trial finished it — for example, 2 people did not complete it in Treatment Group 1 and 4 did not complete it in Treatment Group 2, with similar small numbers across other 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03783923 · results posted 27 June 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03783923) enrolled 11 participants in total — 5 in the deflazacort group and 6 in the placebo group — for the first 26-week placebo-controlled phase. The trial was measuring how a medicine called deflazacort compared to a dummy treatment (placebo) across a range of physical movement tasks, including climbing and descending stairs, walking, and getting up and moving around. The number of people who completed the trial was very small: only 1 in the deflazacort group and 2 in the placebo group finished the first phase, and no participants completed the subsequent 26-week open-label extension phase (where everyone received deflazacort). The reported data shows the following figures for the deflazacort group only (no comparative placebo numbers were reported for the outcome measures). For the primary measure — the time taken to climb 4 stairs — the starting (baseline) average was 5.476 seconds, and the reported change after 26 weeks was −0.200 seconds (a very small decrease). For the secondary movement measures: the 2-minute walk test started at an average of 135.4 metres, with a reported change of +2.0 metres; the "timed up and go" test (standing up and walking a short distance) started at 11.93 seconds and changed by −9.70 seconds (a decrease); descending 4 stairs started at 3.66 seconds and changed by +0.10 seconds; and the 10-metre run/walk started at 8.53 seconds and changed by −0.40 seconds. The reported data shows no figures for the lung capacity (Forced Vital Capacity) measure — that result was not reported. It is worth noting that because so few participants completed the trial, the reported numbers come from a very small group of people, which the trial's own records reflect. 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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NCT03703882 · results posted 21 June 2022
According to the results reported on ClinicalTrials.gov, this trial enrolled 131 boys with Duchenne muscular dystrophy (DMD) — 88 in the treatment group (Dose 1) and 43 receiving a placebo (an inactive dummy treatment). By the end of the study, 85 in the treatment group and 37 in the placebo group completed it. The trial ran for 52 weeks and was measuring physical function — specifically, whether the boys' ability to move and perform everyday physical tasks changed over time. The main tool used was something called the North Star Ambulatory Assessment (NSAA), a 17-item checklist where a trained clinician scores a child on activities like walking, jumping, climbing steps, and getting up from the floor, with a total possible score ranging from 0 (most limited) to 34 (best function). The reported data shows that on the NSAA total score, both groups showed a small decline from their starting scores over the 52 weeks. The treatment group's score changed by an average of −1.5 points, while the placebo group's score changed by an average of −1.8 points. For the secondary movement tests — a 10-metre walk/run, getting up from lying down, and climbing four stairs — both groups also showed small declines from baseline, with the reported numbers being very similar between the treatment and placebo groups across all three tests. For the safety-related outcome, the reported data shows that 85 out of 88 participants in the treatment group and 41 out of 43 in the placebo group experienced at least one adverse event (an unwanted health occurrence recorded during the study). Serious adverse events were recorded for 61 participants in the treatment group and 14 in the placebo group, though the data as submitted does not include further breakdown of 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03796637 · results posted 5 April 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03796637) enrolled 6 participants, all of whom completed the study. All 6 were in a single group receiving a medicine called ataluren. The trial was measuring levels of a protein called dystrophin in muscle tissue, using two different laboratory methods. Dystrophin is a protein found in muscles, and the trial tracked how much of it could be detected after treatment. The reported data shows that dystrophin levels were measured in two specific muscles — the gastrocnemius (calf muscle) and the tibialis anterior (a muscle in the lower leg) — as well as across muscle locations combined. For the primary measurement method (a laboratory technique called electrochemiluminescence), the reported average dystrophin levels were 0.0844, 0.1002, and 0.1054 nanograms per milligram of muscle tissue, for the gastrocnemius, tibialis anterior, and combined locations respectively. The reported data also notes that any readings too low for the test to measure precisely were recorded as half of the lowest detectable amount (0.25 nanograms per milligram). For the secondary measurement method (a different laboratory technique called immunohistochemistry, which uses staining to detect the protein), the reported average dystrophin levels were 0.28817, 0.26578, and 0.30483 nanograms per milligram for the same muscle groups in the same order. No other outcome figures 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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NCT01976091 · results posted 1 April 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT01976091) involved six participants in total, divided into three small groups testing a treatment called SRP-9004. One person was in Cohort 1A, three were in Cohort 1B, and two were in Cohort 2. All six participants received at least one dose of the study drug, and all six completed the trial. The trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) after receiving the treatment. A secondary measure looked at whether participants' ability to walk changed, using a standard test where they walk as far as they can in six minutes. The reported data shows that when it came to adverse events — that is, any unwanted medical occurrences during the study — all six participants across the three groups experienced at least one. When looking only at serious adverse events (those involving hospitalisation, life-threatening situations, or significant disruption to daily life), the reported data shows none occurred in Cohort 1A, two out of three occurred in Cohort 1B, and one out of two occurred in Cohort 2. For the six-minute walk test, the reported data shows that Cohort 1A's result was not reported, Cohort 1B participants walked on average 66 metres less than they did at the start of the trial, and Cohort 2 participants walked on average 29 metres more than they did at the start. It is worth noting that this was a very small trial with only six participants across all groups, so the numbers reflect a very 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 ↗ · Linked publication on PubMed ↗
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NCT03531788 · results posted 18 February 2022
According to the results reported on ClinicalTrials.gov, this trial looked at two types of arm support devices — the Armon Ayura (made by Kinova) and the JAECO WREX — designed to help people move their arms more easily. A total of 18 people were enrolled: 12 in the Armon Ayura group and 6 in the JAECO WREX group. Of those, 17 completed the trial (11 in the Armon Ayura group and all 6 in the JAECO WREX group). The trial measured arm movement using a wrist-worn activity tracker, and also asked participants to set personal goals and rate how well those goals were met with and without the device. The reported data shows that when it came to everyday arm movement effort (measured as "activity counts" — essentially how much the wrist was moving), the Armon Ayura group showed an average change score of 270.61 counts, compared to 26.75 for the JAECO WREX group. For arm position movement across three directions (side-to-side, up-and-down, and in-and-out from the body), the reported numbers varied between the two groups and across the three directions, with figures ranging from around 1,280 to over 69,000 activity counts. For the personal goal ratings — scored on a scale where 0 means performance was the same with and without the device and 4 means much better than expected with the device — the reported data shows scores broadly between 2.72 and 3.5 across three goals for 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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NCT00577577 · results posted 6 January 2022
According to the results reported on ClinicalTrials.gov, this trial enrolled 69 people in total — 34 in the IPLEX™ group and 35 in the placebo group. The trial was measuring physical activity and movement in people over a 24-week period. The main things being measured included how far participants could walk in six minutes, how many steps they took each day, how quickly they walked during their busiest periods of the day, how much time they spent inactive, and how fast they could climb up and down four stairs. Of the 69 who started, 59 completed the trial (29 in the IPLEX™ group and 30 in the placebo group), with five people in each group not completing it. The reported data shows the following changes from the start of the trial to week 24. For the six-minute walk test, the IPLEX™ group walked an average of 12.40 metres more than at the start, while the placebo group walked an average of 20.11 metres more. For daily step count, both groups recorded fewer steps per day than at the start — the IPLEX™ group averaged 140 fewer steps per day, and the placebo group averaged 58 fewer steps per day. For the "peak activity" measure (steps per minute during the fastest 30-minute walking period), the IPLEX™ group showed a change of +0.5 steps per minute and the placebo group +1.1 steps per minute. For the "sustained activity" measure (steps per minute over the busiest continuous 20 minutes), the IPLEX™ group showed a change of −0.9 steps per minute and the placebo group +0.8 steps per minute. For time spent inactive, the IPLEX™ group spent 0.4% less time inactive, while the placebo group showed virtually no change (0.0%). For the stair-climbing test, the reported data shows changes of 0.0 seconds (IPLEX™) and −0.5 seconds (placebo) for going up, and −0.1 seconds (IPLEX™) and −0.5 seconds (placebo) for going down — where a negative number means it took less time than at the start. 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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NCT02606136 · results posted 30 December 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT02606136) enrolled 21 people who all received at least one dose of the study drug, pamrevlumab. The trial ran for up to 104 weeks (about two years), with an optional extension period of up to 208 weeks. Of the 21 who started the main study, 15 completed it and then entered the extension phase; however, none completed the extension phase. The trial was measuring changes over time in lung function, heart function, and upper body strength and movement in participants. The reported data shows that, on average, participants' lung function — measured in several different ways — declined over the 104-week period. The main measure, called percent predicted forced vital capacity (a way of comparing how much air someone can breathe out compared to what would be expected for a healthy person of the same age, sex and height), showed an average annual change of −4.17 percentage points. Two other breathing measurements also showed declines: the amount of air forcefully breathed out in one second changed by −8.32 percentage points, and peak exhalation flow changed by −7.13 percentage points. The reported data also shows changes in other measures: heart pumping function (left ventricular ejection fraction) changed by −2.73 percentage points on average; an upper limb movement score (out of a maximum of 42, where higher means better function) changed by −4.14 points; and grip strength in the dominant and non-dominant hands changed by −2.52 and −1.31 newtons respectively. It is worth noting that this was a single-group study with no comparison group, so all figures reflect changes within the one group of 21 participants receiving pamrevlumab. Because there was no placebo or untreated comparison group reported here, the data alone does not allow a conclusion about what caused these changes. 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 ↗
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NCT01856868 · results posted 22 December 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT01856868) involved 7 participants, all of whom received a treatment called epicatechin — a natural compound found in foods like dark chocolate and green tea. All 7 participants completed the trial, and none dropped out. The trial was measuring changes in certain proteins found in muscle tissue over 8 weeks. These proteins are involved in processes like how muscles produce energy and how muscle growth is regulated. The reported data shows the levels of six different muscle proteins, measured before and after the 8-week treatment period using a laboratory technique that measures protein amounts in relative units (a scale used to compare before and after, rather than an absolute measurement). For a protein called PGC1alpha (linked to how cells produce energy), the reported level went from 0.55 before treatment to 0.86 afterwards. For AMPK (another energy-related protein), the figures were 0.76 before and 1.06 after. For LKB1 (also related to energy processes in cells), the reported figures were 0.67 before and 0.88 after. For Mitofillin (a protein associated with cell energy structures), the reported figures were 1.05 before and 1.33 after. For follistatin (a protein involved in muscle growth), the figures were 0.83 before and 1.11 after. For myostatin (a protein that can limit muscle growth), the reported figures went from 0.79 before treatment to 0.51 afterwards. It is worth noting that this was a very small study with only 7 participants and no comparison group (such as a placebo group), which the reported data does not address further. 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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NCT02239224 · results posted 11 August 2021
According to the results reported on ClinicalTrials.gov, this trial enrolled 20 people in total across four groups. Three participants received a low dose of the study drug ATYR1940 (0.3 mg/kg), six received a medium dose (1.0 mg/kg), six received a higher dose (3.0 mg/kg), and five received a placebo (an inactive treatment given for comparison). The trial was primarily measuring a range of safety-related observations — including any unwanted medical events that occurred after dosing, blood and urine test results, physical examination findings, vital signs such as heart rate and blood pressure, and whether participants developed certain antibodies in response to the study drug. The reported data shows that every participant who received at least one dose experienced at least one treatment-emergent adverse event (that is, any unwanted medical occurrence that happened after the first dose) — this was recorded for all 3 participants in the low-dose group, all 6 in the medium-dose group, all 6 in the higher-dose group, and all 5 in the placebo group. Regarding antibodies that the body might produce against the study drug, the reported data shows 2 out of 3 participants in the low-dose group, 1 out of 6 in the medium-dose group, and 3 out of 6 in the higher-dose group tested positive, while none in the placebo group did. No participants in any group tested positive or borderline for a specific antibody (Jo-1) that would have required them to stop receiving the drug. Clinically significant laboratory abnormalities were recorded for 2 participants in the medium-dose group and none in the other groups. Physical examination abnormalities and vital-sign-related events were each recorded for only 1 participant 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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NCT02740972 · results posted 12 July 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT02740972) looked at a drug called NS-065/NCNP-01, which was being tested in people with Duchenne muscular dystrophy (DMD) — a genetic condition that affects muscle. The trial ran in two parts: a four-week blinded phase (where some participants received a placebo and others received one of two doses of the drug — 40 mg/kg or 80 mg/kg), followed by a 20-week open-label phase (where all participants received one of the two doses and everyone knew what was being given). A total of 16 participants took part across both phases. The trial was primarily measuring how often unwanted side effects occurred, and whether muscle biopsies showed any change in the amount of a protein called dystrophin — the protein that is missing or faulty in DMD. The reported data shows that, when it came to side effects (called adverse events), 3 out of 5 placebo participants, 4 out of 6 in the lower-dose group, and 4 out of 5 in the higher-dose group experienced at least one adverse event during the first phase. In the second open-label phase, 5 out of 8 participants in the lower-dose group and 7 out of 8 in the higher-dose group reported at least one adverse event. No serious adverse events of the highest severity category were recorded. For dystrophin protein levels measured in muscle biopsies (using a lab technique called Western blot), the reported data shows that after 24 weeks of treatment, participants in the lower-dose group had dystrophin levels at about 5.4% of what is seen in people without DMD, and the higher-dose group had about 3.7% — compared to baseline (starting) readings of around 5.7% and 5.9% respectively. Using a different measurement method (mass spectrometry), post-treatment dystrophin levels were reported at 2.1% of normal for the lower-dose group and 4.2% for the higher-dose group. A further method measuring the percentage of muscle fibres showing dystrophin (immunofluorescence) reported 14.2% for the lower dose and 34.8% for the higher dose after treatment. The reported data also shows that a measure of the genetic "skipping" process the drug is designed to trigger (measured in muscle tissue RNA) was 17.4% for the lower-dose group and 43.9% for the higher-dose group after 24 weeks. Muscle strength, measured in pounds using a standardised testing method, showed small changes from baseline across both dose groups, with the numbers reported as slightly negative (indicating a small decrease) in most muscle groups tested, though the size of these changes varied across the different muscles assessed. 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 ↗
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NCT04054375 · results posted 8 July 2021
According to the results reported on ClinicalTrials.gov, this trial enrolled 20 participants, all of whom completed the study with no drop-outs. The trial involved a single group who received a weekly steroid treatment. It was measuring several health markers in people with dysferlinopathy (a rare inherited muscle condition), including blood sugar levels, a longer-term blood sugar measure (HbA1c), cholesterol levels, a muscle enzyme called creatine kinase (a substance released into the blood when muscle is damaged), lung function, and physical ability scores. Two sets of measurements were recorded for each marker — most likely taken at two different time points — though the data as submitted does not label these as specific time points. The reported data shows the following before-and-after figures for the group. Fasting blood sugar was 93 mg/dL at the first measurement and 102 mg/dL at the second. The HbA1c (a percentage reflecting average blood sugar over time) was 5.2% then 5.3%. Cholesterol was 182 mg/dL then 185 mg/dL. The creatine kinase (muscle enzyme) level was 1,574 units/L at the first measurement and 1,047 units/L at the second. Lung function, measured as a percentage of the expected breathing capacity, was 80% then 79%. For the secondary outcome — a physical ability score called the Northstar Assessment for Dysferlinopathy, rated out of 58 where higher means better function — the reported scores were 18.4 and 18.6 at the two time points. It is worth noting that because there was only one group in this trial (no comparison group), the reported figures reflect measurements within that single group only. Whether any differences between the two time points are meaningful is not described in the submitted data, and no comparative or statistical information was provided for 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02246478 · results posted 4 June 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT02246478) tested a drug called TAS-205 across three different dose levels — low, middle, and high — compared to a placebo (a dummy treatment with no active ingredient). A total of 21 people took part, split into four groups: 6 received the placebo, and 5 each received the low, middle, or high dose. The trial ran in two phases — a single-dose phase and a multiple-dose phase — and all 21 participants completed the first phase. In the second phase, one person in the placebo group and one in the high-dose group did not finish. The trial was primarily measuring how often unwanted events (called adverse events) occurred, and also tracked how the drug moved through the body and its effect on a specific chemical marker in urine. The reported data shows that in the primary outcome — counting how many participants experienced adverse events — no adverse events were recorded in the placebo group. In the low-dose group, 3 participants had adverse events in one category and 1 in another; in the middle-dose group, 1 and 3 participants respectively; and in the high-dose group, 1 and 1 participants. For the secondary outcomes tracking how the drug was absorbed into the bloodstream, the reported peak blood concentration of TAS-205 increased with higher doses across multiple measurement time points, ranging from 839 to 4,660 ng/mL depending on the dose and timing. Similarly, the overall amount of drug the body was exposed to over time (measured as area under the curve) also increased with dose, ranging from approximately 2,525 to 9,452 ng·hr/mL. The reported data also shows measurements of a urine marker (a ratio involving a substance called prostaglandin E2 metabolite) across all groups and time points, with values ranging from 0.76 to 1.27 across the different groups. The trial notes that some data were not analysed due to missing inspection records. 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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NCT03907072 · results posted 20 May 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT03907072) tested an investigational treatment called WVE-210201 in people with Duchenne muscular dystrophy. A total of six participants were enrolled — two received a lower dose (3 mg/kg), two received a higher dose (4.5 mg/kg), and two received a placebo (an inactive substance used for comparison). The trial was designed to measure things like changes in levels of a protein called dystrophin (which is reduced or absent in Duchenne muscular dystrophy), as well as physical ability tests such as a walking assessment and stair-climbing speed. Notably, none of the six participants completed the study — all six are recorded as not having finished. The reported data shows that, despite the study having defined primary and secondary outcome measures, no numerical results were submitted for any of them. This includes the dystrophin level measurements, the walking ability scores, the upper limb strength assessments, and the stair-climb test. The data was not reported for any of these outcomes across any of the three groups. Because no outcome numbers were submitted to ClinicalTrials.gov for this trial, it is not possible to describe what the measurements showed. The early stopping of all participants and the absence of reported results mean there is simply no data available from this source to summarise further. 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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NCT03038399 · results posted 20 May 2021
According to the results reported on ClinicalTrials.gov, this trial looked at a drug called vamorolone, given daily by mouth to boys aged 4–7 years who had Duchenne muscular dystrophy (DMD). The boys were divided into four dose-level groups, with 11, 12, 12, and 11 participants starting in each group respectively — 46 boys in total. Most boys finished the study (10, 11, 11, and 9 in each group), with a small number not completing it (1, 1, 1, and 2 respectively). The trial ran over a 24-month period and was primarily focused on tracking any unwanted health events (called adverse events) that occurred during treatment, using a standard medical checklist known as CTCAE version 4.03. The reported data shows that across the dose groups, the number of participants who experienced treatment-related adverse events varied. The results list five groups for this outcome measure (including an overall combined group): 4, 14, 29, and 1 participants in the individual dose groups, and 39 participants across all groups combined, were reported as having experienced a treatment-related adverse event. The reported data also shows the total count of individual adverse events recorded: 15, 34, 203, and 5 events in each dose group, with 302 events recorded across all groups combined. A second, very similar set of figures was also submitted (14, 34, 202, 5, and 300 events), though no explanation for the slight difference between the two sets of numbers was provided in the reported data. It is worth noting that the reporting of adverse events tells us what was recorded and counted during the trial — it does not on its own tell us whether those events were serious, minor, or how they compared to what might be expected without treatment. No other outcome measures beyond these adverse event counts were included in the structured data 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 ↗
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NCT03400852 · results posted 21 February 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT03400852) looked at a medicine called MNK-1411 in people with Duchenne muscular dystrophy (DMD), a condition that weakens the muscles over time. The trial had two stages: a blinded period (where neither doctors nor participants knew who was getting the real medicine or a dummy pill) and an open-label period (where everyone knew the medicine being given). In the blinded period, 29 participants started on MNK-1411 and 15 started on placebo (the dummy pill). Of those, 20 and 9 respectively finished that stage. In the open-label period, 24 participants started on MNK-1411, but only 2 completed it. The trial measured movement and strength abilities using several physical tests. The reported data shows the following numbers for the main test — how quickly participants could walk or run 10 metres. At the start, the MNK-1411 group averaged 5.9 seconds and the placebo group averaged 7.8 seconds; at a later time point, those figures were 5.4 seconds and 8.7 seconds respectively. For a movement ability score (called the North Star Ambulatory Assessment, rated out of 34 where higher means better ability), the MNK-1411 group started at 17.9 and later reached 20.5, while the placebo group started at 17.1 and later measured 16.6. The reported data also shows times for climbing four stairs — starting at 8.52 seconds (MNK-1411) versus 8.47 seconds (placebo), and later at 4.71 seconds versus 15.09 seconds. For rising from lying flat on the back, the starting times were 11.14 seconds (MNK-1411) and 15.03 seconds (placebo), later recorded as 7.65 seconds and 24.89 seconds. Muscle strength (measured in units called Newtons using a device that tests pushing and pulling force) started at 26.61 (MNK-1411) versus 29.87 (placebo), and at 24 weeks was recorded as 33.64 versus 25.27. 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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NCT01207908 · results posted 20 January 2021
According to the results reported on ClinicalTrials.gov, this trial looked at boys with Duchenne muscular dystrophy and compared two groups: one receiving IGF-1 (a growth-related protein) added to standard steroid treatment, and one receiving standard steroid treatment alone. A total of 44 boys took part — 22 in each group. The trial ran for 6 months and measured how well participants could walk, how fast they were growing in height, and how well they could perform everyday physical tasks. The reported data shows that for the main measurement — how far participants could walk in 6 minutes compared to the start of the trial — the IGF-1 group's walking distance increased by an average of 3.4 metres, while the standard treatment group's distance decreased by an average of 5.1 metres. For height growth rate, the IGF-1 group increased by 2.60 cm per year on average, while the standard treatment group's growth rate changed by -0.06 cm per year (essentially no change). For the physical function score — called the North Star Ambulatory Assessment, which rates a person's ability to do 17 movement tasks on a scale of 0 to 34, where higher means better — both groups showed a small decline from their starting scores: the IGF-1 group by 0.94 points and the standard treatment group by 0.4 points. It is worth noting that 5 participants in the IGF-1 group and 1 in the standard treatment group did not complete the trial, and the reasons for this were not detailed in the data 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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NCT03039686 · results posted 21 December 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT03039686) looked at a medicine called RO7239361 in boys with Duchenne muscular dystrophy (DMD), a condition that causes progressive muscle weakness. A total of 166 boys took part — 56 received a placebo (a dummy treatment with no active medicine), 55 received a lower dose of RO7239361, and 55 received a higher dose. The trial had two phases: a 48-week blinded period (where no one knew who was getting which treatment) and an open-label period (where participants knew they were receiving the active medicine). The main thing the trial was measuring was change in a movement and function score called the North Star Ambulatory Assessment (NSAA), which rates 17 physical tasks on a scale of 0 to 34, with higher scores meaning better physical ability. The reported data shows that at the start of the trial, average NSAA scores were similar across all three groups — around 23.1 for the placebo group, 24.5 for the low-dose group, and 22.7 for the high-dose group. After 48 weeks, all three groups showed a decline in their scores: the placebo group dropped by approximately 3.0 points, the low-dose group by approximately 3.4 points, and the high-dose group by approximately 2.4 points. For the stair-climbing speed test, all groups also showed a small decline over 48 weeks — the placebo and low-dose groups both declined by 0.15 stairs per second, while the high-dose group declined by 0.07 stairs per second. For the time taken to stand up from lying down, small declines in speed were also reported across all three groups, ranging from 0.02 to 0.05 units per second. 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 ↗
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NCT01557400 · results posted 25 November 2020
According to the results reported on ClinicalTrials.gov, this trial enrolled 94 participants, all of whom received at least one dose of the study drug, ataluren. Of those 94, 37 completed the study and 57 did not complete it. The trial was measuring unwanted medical events (called adverse events) that occurred during treatment, as well as a range of physical function tests — including how far participants could walk in six minutes, how well they could stand up from lying down, how quickly they could walk or run 10 metres, and lung function measurements. The reported data shows that 91 out of 94 participants experienced at least one treatment-emergent adverse event (an unwanted medical occurrence that began or got worse after taking the study drug). Of those, 31 participants experienced a serious adverse event — meaning one that led to hospitalisation, was life-threatening, or caused significant disability — and 26 participants experienced a non-serious adverse event. For the physical function tests, the reported data shows changes from the starting point over time that were generally in the negative direction — for example, the distance walked in six minutes decreased by figures ranging from approximately 42 metres to 134 metres at various time points. Similarly, scores on a physical function rating scale decreased over time, and the time taken to stand from lying down and to walk 10 metres generally increased (meaning tasks took longer). For lung function, small decreases in measured values were reported across most time points. The reported data shows results across a single group of participants taking ataluren, with no comparison group included in this 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 ↗
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NCT03726996 · results posted 14 October 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT03726996) enrolled 4 children diagnosed with Infantile Neuroaxonal Dystrophy (INAD), a rare and serious neurological condition. The trial was measuring several things: changes in the children's ability to move and control their bodies (using two different movement tests), changes in everyday thinking and behaviour skills (using a standardised assessment), and two specific body measurements related to safety — changes in a heart electrical reading called the Q-T interval (a measure of how the heart's electrical activity behaves over time) and liver enzyme levels in the blood. None of the 4 children completed the trial. The reported data shows that for the three outcome measures looking at movement and cognitive (thinking and behaviour) function, no numerical results were submitted to ClinicalTrials.gov — meaning those figures were not reported. For the heart-related measurement, the reported data shows that 1 out of 4 participants had a prolonged Q-T interval (an change in the heart's electrical pattern) recorded at some point during the trial. For the liver enzyme measurement, the reported data shows that 0 out of 4 participants had abnormal values recorded at any point 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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NCT02819557 · results posted 28 August 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT02819557) enrolled 14 participants, all of whom received the study drug ataluren. All 14 participants completed the trial without anyone withdrawing early. The trial was measuring two main things: how the body processes ataluren (its behaviour in the bloodstream over time), and a range of safety-related observations including any unwanted medical events, blood and urine test results, and heart tracing (ECG) results. The reported data shows that, of the 14 participants, all 14 experienced at least one treatment-emergent adverse event — that is, an unwanted medical occurrence that began or got worse after taking the study drug. Eight participants experienced what were classified as adverse events of moderate severity, five experienced mild ones, and five experienced events that were considered "related" to the study drug according to the investigator. One participant experienced a serious adverse event (a more significant medical event as defined by the study). No participants stopped the study because of an adverse event. The reported data also shows that no participants had clinically meaningful abnormal results on blood tests, urine tests, or heart tracings, and no participants experienced a dose-limiting toxicity (a level of liver or kidney-related concern that would require stopping or reducing the dose). For the drug's behaviour in the bloodstream, the reported peak concentration of ataluren in the blood after the morning dose was around 15.95 micrograms per millilitre (a measure of how much of the drug was present at its highest point), reached at approximately 3.84 hours after 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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NCT02090959 · results posted 11 August 2020
According to the results reported on ClinicalTrials.gov, this trial enrolled 219 participants, all of whom received the study drug ataluren (218 were included in the main safety analysis). The trial was a single-group study — meaning there was no comparison group — designed primarily to track the number of participants who experienced adverse events (unexpected or unwanted medical occurrences) while taking ataluren. It also measured changes over roughly 2.75 years (144 weeks) in physical abilities commonly assessed in people with Duchenne muscular dystrophy, such as how far someone could walk in 6 minutes and how quickly they could stand up, walk 10 metres, or climb four stairs. Notably, only 68 of the 219 participants who started the study completed it, with 151 not completing it; the data does not explain the reasons in this summary. The reported data shows that out of 218 participants in the safety analysis, 202 experienced at least one treatment-emergent adverse event (a medical occurrence that happened or got worse from the first dose up to six weeks after the last dose). Of those, 87 were described as drug-related adverse events, 71 were classed as serious adverse events, and 44 were graded as severe. Two participants experienced an adverse event graded as life-threatening, and one death was reported. Additionally, 29 participants had abnormal results in certain blood or urine laboratory tests that were being specifically monitored. For the physical ability tests at week 144, the reported data shows that on average, participants walked about 98 metres less in the 6-minute walk test compared to when they started, took approximately 5 seconds longer to stand up from lying down, about 2 seconds longer to walk or run 10 metres, and around 4 seconds longer to climb four stairs. It is important to note that because this trial had no comparison group, the reported numbers alone cannot tell us what would have happened without the study drug. The reported data shows changes over time in a single group of 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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NCT01826487 · results posted 4 August 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT01826487) enrolled 230 boys or young men with Duchenne muscular dystrophy — 115 in a placebo group and 115 in an ataluren group. Of those, 228 were included in the main analysis group (114 per group), and most participants completed the 48-week study (111 on placebo, 110 on ataluren). The trial's main measurement was how far participants could walk in six minutes (known as the "six-minute walk distance" or 6MWD), comparing where each group started to where they were at 48 weeks. Several secondary measurements were also taken, including how quickly participants could walk or run 10 metres, climb or descend four stairs, and a broader physical function score. The reported data shows that both groups walked a shorter distance by week 48 than they did at the start. The placebo group's average walking distance fell by about 61 metres, while the ataluren group's average fell by about 48 metres — a difference of roughly 13 metres between the two groups. For the secondary measure of how long it took for walking distance to drop by 10% and stay there, the reported median time was 56 days in the placebo group and 164 days in the ataluren group for participants who started with a walking distance under 300 metres; for the other distance subgroups, the data was not reported as a single figure. On the 10-metre walk/run test, the placebo group slowed by an average of about 3.5 seconds and the ataluren group by about 2.3 seconds. For climbing four stairs, the placebo group slowed by about 4.5 seconds versus about 2.7 seconds in the ataluren group, and for descending four stairs, the figures were about 4.0 seconds versus about 2.2 seconds. A broader physical function score (rated 0–100, where higher is better) fell by an average of 8.4 points in the placebo group and 6.3 points in the 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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NCT01009294 · results posted 29 July 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT01009294) enrolled 6 participants, all of whom received at least one dose of the study drug, ataluren. None of the 6 participants completed the trial — all 6 did not finish, though the reasons are not detailed in the submitted data. The trial was measuring a range of things related to upper limb (arm and hand) function, daily living activities, joint movement, and muscle strength, as well as tracking any unwanted medical events (called adverse events) that occurred while participants were taking the study drug. The reported data shows that 1 out of 6 participants experienced a treatment-emergent adverse event (that is, an unwanted medical occurrence that began or got worse after starting the study drug), while no participants experienced a serious adverse event (defined as events such as hospitalisation, life-threatening situations, or significant lasting disability). For the arm and hand function tests, the reported data shows individual participant scores across tasks such as moving objects, picking up small items, and simulated feeding — with times ranging from around 7 seconds to 120 seconds depending on the task and hand used. On the Brooke Upper Extremity scale (where 1 means full arm movement and 6 means very little useful hand function), scores of 3 were reported. Daily living scores on the EK scale (where 0 is the most independent and 30 the least) ranged from 0 to 3 across individual categories for participants. Joint movement measurements and muscle strength figures were also reported across different joints and muscle groups, though given the very small number of participants, the data was not reported as a group average — only as individual 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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NCT00847379 · results posted 15 July 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT00847379) enrolled 173 participants across three groups who received the study drug ataluren at different doses, or a placebo followed by ataluren. Specifically, 59 participants were in the high-dose ataluren group throughout, 57 were in the low-dose ataluren group (then switched to high-dose), and 57 started on placebo before switching to high-dose ataluren. The trial was primarily measuring whether participants experienced any unwanted medical events (called adverse events) or abnormal results on blood and urine tests during the study period. The reported data shows that across all 173 participants, 149 experienced at least one treatment-emergent adverse event (meaning an unwanted medical occurrence during or shortly after the study period). When broken down by group, this was 52 out of 59 in the high-dose group, 49 out of 57 in the low-dose group, and 48 out of 57 in the placebo-then-high-dose group. Serious adverse events were reported in 10, 10, and 22 participants in those same groups respectively, with 3 participants across all groups experiencing events that were considered possibly related to the study drug. No participants in any group were reported to have clinically significant abnormal laboratory results, according to the investigators' judgement. The reported data also shows results from a walking test (where participants walked as far as they could in 6 minutes) and ankle-worn step counters measured over 60 weeks. All three groups showed a decrease from their starting distance in the 6-minute walking test by week 60 — the high-dose group's average distance fell by about 17 metres, the low-dose group by about 11 metres, and the placebo-then-high-dose group by about 7 metres. Similarly, the step-counting data generally showed small decreases in daily step counts across all groups by week 60, though the specific numbers varied 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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NCT03627494 · results posted 10 July 2020
According to the results reported on ClinicalTrials.gov, this trial tested a medicine called GSK3439171A across three separate parts (Part A, Part B, and Part C). The trial was designed as an early-phase study to measure how the body responded to different doses of the medicine. Part A involved 18 participants across six small groups, each receiving different dose combinations or a placebo (a dummy treatment with no active ingredient) in a rotating sequence. Part B involved 36 participants across four groups receiving varying doses or placebo over up to 34 days. Part C involved 12 participants split into two groups comparing the medicine taken on a full versus empty stomach. The trial's primary focus was on recording any adverse events (AEs) — that is, any unexpected medical occurrences during the study — and any serious adverse events (SAEs), which are more severe occurrences such as hospitalisation or life-threatening situations. The reported data shows that in Part A, adverse events were recorded in 1 participant in the placebo group, 0 in the 5 mg group, 2 in the 10 mg group, 2 in the 30 mg group, 2 in the 60 mg group, 1 in the 120 mg group, and 1 in the 180 mg group. No serious adverse events were reported in Part A for any of the groups where data was provided. The reported data for Parts B and C adverse event numbers was truncated in the available submission and therefore cannot be fully described here — that detail was not completely 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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NCT00592553 · results posted 7 April 2020
According to the results reported on ClinicalTrials.gov, this trial enrolled 174 boys with Duchenne muscular dystrophy — a condition caused by a specific type of genetic change called a nonsense mutation. Sixty participants received a high dose of a medicine called ataluren, 57 received a low dose, and 57 received a placebo (a dummy treatment with no active ingredient). The trial ran for 48 weeks. The main thing being measured was how far participants could walk in six minutes (known as the "6-minute walk distance" or 6MWD) — a standard way of tracking movement ability in these trials. The reported data shows that over the 48 weeks, the distance walked in six minutes changed in all three groups. On average, the high-dose ataluren group's walking distance decreased by about 42 metres, the placebo group's decreased by about 43 metres, and the low-dose ataluren group's decreased by about 13 metres. For the secondary measures — which included how long it took to stand up from lying down, walk or run 10 metres, climb four stairs, and descend four stairs — the reported data shows that times increased (meaning tasks took longer) across all three groups by the end of the study. Muscle strength, measured in pounds of force across several muscle groups in the arms and legs, also showed small changes across all groups; the specific numbers varied by muscle group and are listed in the full data set on ClinicalTrials.gov. Where baseline (starting point) figures appear duplicated in the raw data, this reflects how the data was structured in the submission, and readers should refer to the full record for complete 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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NCT02285062 · results posted 30 March 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT02285062) enrolled 570 people with lymphoma, split into two groups of 285. One group received a drug called lenalidomide combined with a standard chemotherapy regimen known as R-CHOP (referred to as R²-CHOP), while the other group received a dummy pill (placebo) combined with the same R-CHOP chemotherapy. The trial was primarily measuring how long participants went without their disease getting worse or dying — a measure called "progression-free survival." It also tracked a number of secondary measures, including overall survival, how long people remained free of key negative events, how many achieved a complete response (where scans showed no detectable sign of disease), and how many had any measurable response to treatment. The reported data shows that for the primary outcome — progression-free survival — as well as for overall survival, event-free survival, and duration of complete response, the values were recorded as "NA" (not available) in the submitted results. This means those particular figures were not reported in the structured data submitted to ClinicalTrials.gov, so no numbers for those outcomes can be described here. For the secondary outcomes where numbers were provided, the reported data shows that 69.1% of participants in the lenalidomide plus R-CHOP group and 64.9% in the placebo plus R-CHOP group achieved a complete response — meaning scans showed no detectable sign of their lymphoma at the time of assessment. For any measurable response (either complete or partial shrinkage of the lymphoma), the reported rate was 90.9% in both groups. It is worth noting that more participants left the treatment period early in the lenalidomide group (72 people) compared with the placebo group (44 people), though the reasons for this are not detailed in the structured data provided. The reported data shows only what was measured and counted at the time of the data cut-off, and the missing figures for several key outcomes mean a complete picture is not available from this submission 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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NCT02907619 · results posted 25 November 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT02907619) enrolled 59 participants in total, split across three groups of roughly 20 people each (19 in Group 1, 20 in Group 2, and 20 in Group 3). The trial used a "crossover" design — meaning participants moved through different treatment sequences over time — and it was primarily measuring a range of safety-related outcomes, such as whether people had to reduce their dose or stop treatment due to unwanted medical events (called adverse events), whether they experienced severe adverse events, and whether their blood test results fell outside the normal range. The reported data shows that no participants across any of the three groups needed to have their dose reduced or temporarily stopped due to adverse events. For severe adverse events that emerged during treatment, 4 out of 59 participants in total experienced one (3 from Group 1 and 1 from Group 3); however, when only events considered by the investigator to be directly related to the treatment were counted, the number was zero across all groups. One participant (from Group 3) left the study due to an adverse event, though again, no departures were recorded when only treatment-related events were considered. For blood tests, no abnormalities were found in blood cell counts or clotting measures for most participants, though some small numbers had abnormal clotting results (up to 2 participants in total across one category). The reported data shows that liver function test results above or below the normal range were notably common — 47 out of 59 participants had at least one result outside the normal range for one liver-related measure, and 56 out of 59 for another. The data does not provide further detail about the nature or significance of those liver test findings. 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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NCT02413190 · results posted 5 November 2019
According to the results reported on ClinicalTrials.gov, this trial enrolled 94 participants, all of whom had a condition called facioscapulohumeral muscular dystrophy (FSHD), a type of muscular dystrophy that affects the muscles of the face, shoulders, and upper arms. All 94 participants completed the study with no dropouts. The trial was measuring bone mineral density — a measure of how dense and strong bones are — in people with FSHD, to see how it compared to what would typically be expected for people of the same age and sex without the condition. The reported data shows that bone mineral density was measured using something called a Z-score. A Z-score is simply a way of comparing a person's result to the average result seen in a healthy reference group of the same age and sex — a score of zero means exactly average, a negative score means below average, and a positive score means above average. The reported average Z-score for the FSHD group was −0.4, meaning the group's bone mineral density was slightly below the average seen in the healthy reference population. It is worth noting that the study only included one group (people with FSHD) and compared their results against existing reference data rather than against a separate comparison group within the trial itself. No secondary outcome measures were included in the submitted results 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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NCT01197300 · results posted 20 September 2019
According to the results reported on ClinicalTrials.gov, this trial was an extension study involving children with osteoporosis (weakened bones) who were also being treated with steroid medications (glucocorticoids). A total of 25 children took part — 10 who had received zoledronic acid (the study drug) during the earlier core study, and 15 who had received a placebo (a dummy treatment with no active ingredient). All 10 children in the zoledronic acid group completed the study, while 13 of the 15 in the placebo group completed it. The trial was primarily looking at the longer-term safety profile of zoledronic acid over an additional 12 months, and also tracked changes in bone measurements and certain chemical markers in the blood. The reported data shows that, regarding the primary focus of the study — monitoring for unwanted health events — 7 out of 10 children in the zoledronic acid group and 12 out of 13 completing children in the placebo group experienced at least one adverse event (an unwanted or unexpected health occurrence noted during the study). Serious adverse events (more significant health concerns) were reported in 3 children in the zoledronic acid group and none in the placebo group. No deaths were reported in either group. For the secondary measures, both groups showed increases in bone mineral content (a measure of the amount of mineral in the bones) in the spine and across the whole body over 18 and 24 months from the start of the original study. The reported data also shows changes in two blood markers related to bone activity — P1NP and BSAP — with the zoledronic acid group showing larger decreases in these markers compared to the placebo group at both 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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NCT02760277 · results posted 23 July 2019
According to the results reported on ClinicalTrials.gov, this trial looked at a drug called vamorolone in 48 boys aged 4 to 7 years with Duchenne muscular dystrophy (DMD), a condition that causes progressive muscle weakness. The boys were divided into four groups of 12, each receiving a different daily dose of vamorolone (0.25, 0.75, 2.0, or 6.0 milligrams per kilogram of body weight) for 24 weeks. All 48 boys completed the trial. The trial was measuring how many unwanted health events (called adverse events) occurred, how the boys performed on a muscle function test (how quickly they could stand up from the floor), and changes in body weight score and certain blood markers. The reported data shows that in terms of adverse events — any health problems or worsening of symptoms recorded during the trial — 10, 10, 11, and 11 participants (out of 12 in each group) experienced at least one such event across the four dose groups. The total number of these events across the groups was 48, 44, 54, and 73 respectively. Serious adverse events were reported in 1, 2, 4, and 5 participants across the groups, with 2 participants in the highest dose group experiencing events that led to stopping the drug. For the stand-up speed test, the reported data shows the boys' scores at the start of the trial ranged from 0.18 to 0.24 rises per second across groups, and at 24 weeks ranged from 0.22 to 0.24 rises per second. Body weight scores and a blood sugar marker (HbA1c, a measure of average blood sugar levels) were also tracked, with the reported changes appearing small across all groups; the full figures for each time point are included in the trial 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02310763 · results posted 23 July 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT02310763) enrolled 120 participants across three groups, who took part in a two-period study running for up to 96 weeks. The trial was testing domagrozumab, given at three different dose levels (5 mg/kg, 20 mg/kg, and 40 mg/kg), compared against a placebo (an inactive treatment). The primary focus of what was being measured was the number of participants who experienced unwanted medical events — called adverse events — during the first 48 weeks, as well as results from blood tests checking things like blood cell counts, clotting, and liver function. The reported data shows that by week 49, the number of participants who experienced any adverse event was 38 out of those on placebo, 66 on the 5 mg/kg dose, 57 on the 20 mg/kg dose, and 59 on the 40 mg/kg dose. Serious adverse events were reported in 14 (placebo), 18 (5 mg/kg), 14 (20 mg/kg), and 16 (40 mg/kg) participants. Only 1 participant — in the highest dose group — stopped the study due to an adverse event. For the blood tests, no abnormalities in blood cell counts were recorded across any group. Some coagulation (blood clotting) abnormalities were reported — 1 to 2 participants per group for one measure, and between 3 and 13 for another. Liver function abnormality numbers were not fully reported for the middle two dose groups, though the data was not reported rather than confirmed as zero. No liver function abnormalities of a certain category were recorded for placebo or the highest dose group. The reported data shows that dose reductions or temporary stops due to adverse events occurred in 8 participants on placebo, 4 on the 5 mg/kg dose, 4 on the 20 mg/kg dose, and none on the 40 mg/kg dose. It is worth noting that the numbers across some outcome categories appear higher than the number of people enrolled, which may reflect the crossover design of the trial where participants moved between treatments across the two periods — however, the data as submitted was not explained further 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02710591 · results posted 18 July 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT02710591) involved 20 people in total, split evenly across four groups (called Cohorts 1 to 4), with 5 participants in each. All 20 participants completed the study without anyone dropping out. The trial was looking at a medicine called rimeporide, and the main thing it set out to measure was how many participants experienced unwanted health events (known as adverse events) while taking the study drug. It also tracked how the drug moved through the body over time — a type of measurement known as pharmacokinetics (PK), which simply means measuring how much of the drug is present in the blood and when. The reported data shows that, when it came to unwanted health events, the numbers varied across the four cohorts. In Cohort 1, 2 out of 5 participants had at least one such event; in Cohort 2, it was 1 out of 5; in Cohort 3, it was all 5 out of 5; and in Cohort 4, it was 4 out of 5. Of those, events considered possibly related to the study drug were reported for 0 participants in Cohorts 1 and 2, 0 in Cohort 3, and 2 in Cohort 4. One serious unwanted event was reported in Cohort 3, and none in the other cohorts. No events leading to death were included in the figures reported here, and several other categories — such as events leading to withdrawal — were not separately reported in the submitted data. The reported data also shows the blood-level measurements of rimeporide. The peak blood concentration (the highest amount of drug detected in the blood, called Cmax) was reported as 1,286 ng/mL for Cohort 1, 1,987 ng/mL for Cohort 2, 3,013 ng/mL for Cohort 3, and 3,819 ng/mL for Cohort 4 on Day 1. Similar figures were recorded at the Week 4 timepoint: 1,361, 2,077, 2,817, and 3,909 ng/mL respectively — suggesting the four cohorts received increasing doses, though the specific dose amounts were not included in the submitted results 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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NCT00799266 · results posted 5 July 2019
According to the results reported on ClinicalTrials.gov, this trial enrolled 34 people in total — 18 received zoledronic acid (a medication given by infusion) and 16 received a placebo (an inactive substance). The trial was designed to measure changes in bone density in the lower spine over 12 months, using a standardised score called a "Z-score" (a way of comparing a person's bone density to others of a similar age and sex). Bone mineral content (BMC, a measure of the amount of mineral in the bones) and certain blood markers related to bone activity were also tracked at 6 and 12 months. The reported data shows that, for the main outcome — change in lower spine bone density Z-score at 12 months — the zoledronic acid group had an average increase of 0.582 Z-score units, while the placebo group had an average increase of 0.168 units. At 6 months, the reported increases were 0.447 for the zoledronic acid group and 0.157 for the placebo group. For bone mineral content in the lower spine, the reported data shows average increases of 4.11 grams (zoledronic acid) versus 2.13 grams (placebo) at 6 months, and 6.45 grams versus 4.30 grams at 12 months. For total body bone mineral content, the reported figures were 129.3 grams versus 95.2 grams at 6 months, and 220.8 grams versus 140.1 grams at 12 months. Two blood markers of bone activity — P1NP and BSAP — showed average decreases in the zoledronic acid group and average increases in the placebo group at both 6 and 12 months, though what this means clinically is not described 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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NCT02760264 · results posted 2 January 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT02760264) tested four different daily doses of a drug called vamorolone in boys aged 4 to under 7 years old who had Duchenne muscular dystrophy (DMD), a condition that causes progressive muscle weakness. A total of 48 boys took part, with 12 assigned to each of the four dose groups (0.25, 0.75, 2.0, and 6.0 mg per kilogram of body weight per day). All 48 boys completed the trial. The trial was primarily looking at unwanted side effects (called adverse events) that occurred during treatment, and also measured certain markers in the blood related to how the body handles sugar and hormones. The reported data shows that across the four dose groups, the total number of treatment-related adverse events (any unwanted health change recorded after starting the drug) was 16, 18, 13, and 11 events respectively. The number of participants in each group who experienced at least one such event was 13, 13, 11, and 9 out of 12. Regarding blood sugar levels (fasting glucose), the reported data shows small changes from the start of the study to week 2, ranging from a decrease of about 5.8 mg/dL to a very slight increase of 0.2 mg/dL across the groups. For insulin (a hormone that helps control blood sugar), changes from the start to week 2 ranged from a small decrease in the lowest dose group to an increase of 2.78 units in the highest dose group. A morning cortisol reading (a hormone that reflects how the body's stress response system is working) was also reported at the end of the study period, with values of 10.4, 9.8, 7.3, and 3.0 micrograms per decilitre across the four dose groups from lowest to highest dose — noting that percentage-change data for cortisol from the start of the study 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01462292 · results posted 16 October 2017
According to the results reported on ClinicalTrials.gov, this trial (NCT01462292) enrolled 51 participants in total, split across three groups: 16 received a placebo, 17 received a lower dose of the study drug GSK2402968 (3 mg per kilogram of body weight per week), and 18 received a higher dose (6 mg per kilogram per week). All 51 participants completed the study. The trial was measuring whether the study drug had any effect on physical abilities in participants, primarily by looking at how far they could walk in six minutes, and secondarily by measuring things like how long it took them to stand up from the floor, climb four stairs, walk ten metres, and how strong certain muscles were. The reported data shows that for the main measure — the six-minute walk test — the placebo group walked an average of about 11 metres less than they did at the start of the study, the lower-dose group walked about 20 metres less, and the higher-dose group walked about 16 metres more than they did at the start. For the secondary measures, the reported data shows that all three groups took longer to rise from the floor and to climb stairs compared to the start of the study (meaning their times went up). For the ten-metre walk, the changes reported were very small across all groups. For overall muscle strength, the lower-dose group showed an average increase of about 2.67 pounds of force, the higher-dose group about 1.14 pounds, and the placebo group about 0.32 pounds — though some individual muscle group results varied across the groups. It is important to note that these numbers describe average changes within each group as reported, and the trial data does not draw conclusions about what caused any differences between the groups. Some secondary muscle strength figures were not fully reported for all individual muscle groups 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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NCT01982695 · results posted 21 March 2017
According to the results reported on ClinicalTrials.gov, this trial enrolled 23 people in total — 12 in a group taking a medicine called lisinopril and 11 in a group taking a medicine called losartan. All 12 people in the lisinopril group completed the study, while 10 out of 11 completed it in the losartan group (one person did not finish). The trial was measuring how well the heart pumps blood, using an ultrasound scan of the heart called an echocardiogram. Specifically, it looked at something called "ejection fraction" — this is simply the percentage of blood that the heart pushes out with each beat. The reported data shows that after 12 months, the average ejection fraction in the lisinopril group was 54.6%, and in the losartan group it was 55.2%. No other outcome measures were reported in the submitted results 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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NCT01916109 · results posted 12 February 2016
According to the results reported on ClinicalTrials.gov, this trial enrolled 4 participants, all of whom received a combination of three treatments: gemcitabine, carboplatin, and panitumumab (together referred to as GCaP). The trial was measuring what is called a "pathologic complete response" — this means looking at tissue removed during bladder surgery to see whether any cancer cells remained, either in the bladder itself or in nearby lymph nodes. Three of the four participants completed the study, and one did not complete it (the reason was not reported in the data). The reported data shows that, of the 4 participants who started the trial, 3 showed a pathologic complete response — meaning that when their removed tissue was examined after surgery, no remaining cancer cells were found in the bladder or lymph nodes. No other outcome measures (such as secondary outcomes) were included in the submitted results data, so no further numbers are available to describe. It is worth noting that this was a very small trial with only 4 participants, so the reported numbers reflect a very limited group of people. The data as submitted does not include information on side effects or other measures of how participants fared overall. 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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NCT00654784 · results posted 29 July 2011
According to the results reported on ClinicalTrials.gov, this trial enrolled 21 people with Duchenne muscular dystrophy (DMD) — 13 received a daily dose of idebenone (450 mg) and 8 received a placebo (a dummy treatment with no active ingredient). All 21 participants completed the study. The trial was primarily measuring changes in heart muscle function over 52 weeks, specifically looking at how well a particular part of the left side of the heart — the inferolateral wall — was squeezing during each heartbeat. This was measured using a specialised ultrasound technique called Colour Doppler Myocardial Imaging. The trial also intended to measure breathing function, muscle strength, and safety, though those results were not reported in the data submitted to ClinicalTrials.gov. The reported data shows that, when looking at the primary heart function measurement, the idebenone group had a reported change of approximately 104.4% from their starting point, while the placebo group had a reported change of approximately 28.9%. These figures represent the relative percentage change in the heart wall's squeezing motion from the beginning of the study to week 52. No further breakdown of what these numbers mean in practical terms was provided in the submitted data. As noted above, the results for breathing function, muscle strength, and safety outcomes were not reported in the data 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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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.