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 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.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 30135256) ↗
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 must have already completed the treatment period in the previous related Phase 3 clinical trial (called PTC124-GD-020-DMD).
- You (and/or your parent or legal guardian, if you are a minor) must have signed and dated a consent form showing you have been fully informed about the trial.
- If you are sexually active, you must be willing to either avoid sexual intercourse or use a barrier or medical form of contraception during the study treatment period and for 6 weeks after it ends.
- You must be willing and able to attend all scheduled visits, take the study drug as directed, follow study procedures, and complete required tests and restrictions.
Who may not be able to join:
- You cannot join if you have a known allergy or sensitivity to any of the ingredients in the study drug, including substances such as refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol, crospovidone, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate.
- You cannot join if you are currently taking part in any other clinical trial that involves a treatment or therapy.
- You may not be able to join if you have a current or past medical condition, mental health condition, behavioral issue, substance misuse problem, abnormal test results, or any other health concern that the trial doctor believes could put you at risk, prevent you from completing the trial, or affect the reliability of the results (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: Francesco Bibbiani, M.D., 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
Number of Participants With Treatment-Emergent Adverse Events (TEAEs); Number of Participants With Abnormalities in Clinical Laboratory Parameters
Can't join this trial?
Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.