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 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.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 37236246) ↗
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 2 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 are able to read and sign a consent form (or a parent/guardian can sign on your behalf if you are under 18)
- You are male
- You are 5 years of age or older
- You have shown signs of Duchenne or Becker muscular dystrophy (such as muscle weakness, an unusual walking style, or difficulty getting up from the floor) before age 9, along with high levels of a muscle enzyme in your blood and ongoing difficulty walking
- A genetic test has confirmed that your dystrophy is caused by a specific type of gene change called a "nonsense mutation" in the dystrophin gene, tested by an approved laboratory
- A blood sample has been taken to confirm the presence of this specific gene change
- You are able to walk at least 75 meters (about 246 feet) on your own without any personal help or walking aids during a 6-minute walk test
- Your blood test results for liver, kidney, adrenal gland, and electrolyte levels are within normal ranges
- If you are sexually active, you are willing to abstain from sex or use contraception during the treatment period and for 6 weeks after
- You are willing and able to attend all scheduled visits and follow all study procedures and rules
Who may not be able to join:
- You have been treated with a type of antibiotic called aminoglycosides (given throughout the body, not just topically) in the past 3 months
- You started taking steroid medication (such as prednisone) in the past 6 months, or your steroid dose or schedule changed in the past 3 months
- Any medication you take for heart failure has been started, changed, or stopped in the past 3 months
- You have taken the blood-thinning medication warfarin in the past month
- You have previously received the study drug ataluren
- You have a known allergy to any ingredient in the study drug (confirm with trial site)
- You have taken part in another clinical trial involving an experimental drug in the past 2 months
- You have had a major surgery in the past 30 days
- You are currently taking immune-suppressing medication (other than steroids)
- You are currently taking part in another treatment clinical trial
- You are expected to need major surgery (for example, surgery for a curved spine) during the 12-month study period
- You need a ventilator (breathing machine) to help you breathe during the day
- You have significant heart failure symptoms or your heart scan shows serious heart muscle problems
- You have another medical, mental health, or personal condition that, in the doctor's opinion, could affect your safety, your ability to complete the study, or the accuracy of the results
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: Leone Atkinson, MD, PhD, PTC Therapeutics
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
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Change From Baseline in 6MWD at Week 48
Can't join this trial?
Data last synced from ClinicalTrials.gov: 26 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.