Trial 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
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.
View full results on ClinicalTrials.gov ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Muscular Dystrophy Trial, Terminated
Who may be able to join
AI generated eligibility 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
Who might be able to join this trial:
- You are a boy between 6 and just under 12 years old at the time of screening
- You (and/or your parent or legal guardian) are willing and able to give written consent to participate
- You have been diagnosed with Duchenne Muscular Dystrophy (DMD) and a genetic test has confirmed the specific gene change causing it, including information about a section of DNA called "exon 44"
- Your lung breathing capacity (measured by a standard breathing test) is above 45% of what would be expected for someone your age and size
- You have been taking steroid medications (like prednisone, prednisolone, or deflazacort) at a stable dose for at least 6 months, with no major dose changes in the last 3 months (other than adjustments for weight changes)
- You are able to walk between 270 and 450 metres in a 6-minute walking test, with consistent results across two tests done within 3 months before joining the trial
- You are able to stand up from the floor in less than 10 seconds without any aids or braces
- You are able to have an MRI scan of the muscles in your thighs
- You agree to have a yearly flu vaccination during the study
- Your kidney function is within an acceptable range (confirmed by a blood test)
- Your blood counts (platelets, haemoglobin, and white blood cells) and certain minerals in your blood are within acceptable levels
- You have no history of liver disease, and your liver function tests are within acceptable limits
Who may not be able to join:
- You have another illness (besides DMD) that also causes muscle weakness or problems with movement
- You have a severe intellectual or behavioural condition (such as severe autism or severe cognitive impairment) that would prevent you from completing the study assessments, in the doctor's opinion
- You have previously received the study drug, pamrevlumab
- Your body mass index (BMI) is very high (38 or above) or you weigh more than 117 kg
- You have had a serious allergic or anaphylactic reaction to certain types of antibody-based medicines in the past, or you are known to be allergic to the study drug or any of its ingredients
- You have taken another experimental drug within 30 days before screening, or certain approved DMD treatments within a specific timeframe before screening (confirm with trial site)
- You need continuous breathing support (ventilation) for 16 or more hours a day
- You have poorly controlled asthma or another ongoing lung condition (such as bronchitis or recurring pneumonia) that may affect your breathing, in the doctor's opinion
- You were hospitalised due to breathing failure within the 8 weeks before screening
- You have severe, uncontrolled heart failure, needed IV heart medications or were hospitalised for a heart problem in the 8 weeks before screening, or have severely reduced kidney function
- You have a heart rhythm problem that requires medication to control it
- You have any other significant heart condition identified by tests or examination
- The study doctor believes, for any reason, that you would not be able to fully complete the study
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.