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 (NCT00847379) enrolled 173 participants across three groups who received the study drug ataluren at different doses, or a placebo followed by ataluren. Specifically, 59 participants were in the high-dose ataluren group throughout, 57 were in the low-dose ataluren group (then switched to high-dose), and 57 started on placebo before switching to high-dose ataluren. The trial was primarily measuring whether participants experienced any unwanted medical events (called adverse events) or abnormal results on blood and urine tests during the study period. The reported data shows that across all 173 participants, 149 experienced at least one treatment-emergent adverse event (meaning an unwanted medical occurrence during or shortly after the study period). When broken down by group, this was 52 out of 59 in the high-dose group, 49 out of 57 in the low-dose group, and 48 out of 57 in the placebo-then-high-dose group. Serious adverse events were reported in 10, 10, and 22 participants in those same groups respectively, with 3 participants across all groups experiencing events that were considered possibly related to the study drug. No participants in any group were reported to have clinically significant abnormal laboratory results, according to the investigators' judgement. The reported data also shows results from a walking test (where participants walked as far as they could in 6 minutes) and ankle-worn step counters measured over 60 weeks. All three groups showed a decrease from their starting distance in the 6-minute walking test by week 60 — the high-dose group's average distance fell by about 17 metres, the low-dose group by about 11 metres, and the placebo-then-high-dose group by about 7 metres. Similarly, the step-counting data generally showed small decreases in daily step counts across all groups by week 60, though the specific numbers varied between 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 17389552) ↗
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, 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 phase of the previous related study (called PTC124-GD-007-DMD).
- You must be able to sign a consent form agreeing to take part (if you are under 18, a parent or guardian must also sign, and you may be asked to sign an assent form).
- 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 treatment period and for 6 weeks afterward.
- You must be willing and able to attend scheduled visits, take the study drug as planned, follow study procedures, have laboratory tests done, and stick to any study rules.
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 (confirm with trial site for the full list of ingredients).
- 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, history of alcohol or drug misuse, abnormal test results, or abnormal heart test (ECG) findings that the study doctor believes could affect your safety, your ability to complete the study, or the accuracy of the study 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: Leone Atkinson, M.D., Ph.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 (AEs); Number of Participants With Clinically Significant Abnormal Laboratory Parameters
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.