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 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.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 34120912) ↗
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, Completed
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 (or your child's) legal guardian must be willing and able to sign a consent form agreeing to take part in the trial
- You have been diagnosed with Duchenne muscular dystrophy (DMD), confirmed by high levels of a specific muscle enzyme in the blood and a genetic test showing a change in the dystrophin gene linked to DMD
- You are able to get up from lying on the floor to standing on your own in 10 seconds or less
- You are able to walk 10 meters and climb 4 stairs as part of physical tests
- You are regularly seen by a doctor who manages your DMD care, and you are willing to let your other medical professionals know you are taking part in this study
Who may not be able to join:
- You have taken steroid tablets or injections (such as prednisone) in the last 24 weeks — note that steroid inhalers, nasal sprays, or skin creams are allowed
- You have taken another experimental drug, idebenone, or a treatment specifically targeting dystrophin in the last 4 weeks — except if you have been on a stable dose of eteplirsen for at least 24 weeks and plan to continue it
- You have taken certain medications in the last 4 weeks, including immune-suppressing drugs, warfarin, or several other specific medicines (confirm with trial site for the full list)
- You have used human growth hormone in the last 3 months
- You have another serious or ongoing medical condition not related to DMD (confirm with trial site)
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
Principal Investigator: Joanne M Donovan, Chief Medical Officer, MD, PhD, Catabasis Pharmaceuticals
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
3 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)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.