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 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.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 41879641) ↗
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 must be male
- You must be 5 years old or older
- You must have been diagnosed with Duchenne Muscular Dystrophy based on physical signs and symptoms
- Your Duchenne Muscular Dystrophy must be caused by a specific type of gene change called a "nonsense point mutation" in the dystrophin gene
- You must have been taking steroid medication (prednisone, prednisolone, or deflazacort) by mouth or injection for at least 12 months before starting the study, with no major changes to your dose for at least 3 months before starting
- You must be able to walk at least 150 meters (about 490 feet) in a 6-minute walking test
- You must be able to complete certain timed movement tests within 30 seconds
- You must be willing and able to attend all scheduled visits and follow all study rules and procedures
Who may not be able to join:
- You have had a change in heart-protective medication within 1 month before starting the study
- You are currently receiving certain antibiotics (aminoglycosides or vancomycin) given through a drip (IV)
- You have previously used or are currently using a drug called ataluren
- You have a known allergy or sensitivity to any ingredient in the study drug
- You have taken part in another drug trial or received an experimental treatment within the last 6 months
- You have had major surgery in the last 12 weeks, or are expected to need major surgery during the 72-week study period
- You need a breathing machine (ventilator) during the day, or you use an invasive breathing machine through a tube in your throat
- You have uncontrolled signs of heart failure
- Your blood test results show that your kidneys may not be working properly (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: Vinay Penematsa, MD, PTC Therapeutics, Inc.
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
4 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population; DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population; DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population; DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population
Can't join this trial?
Data last synced from ClinicalTrials.gov: 22 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.