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NCT01826487 · ClinicalTrials.gov registry record · Phase 3
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
A Phase 3 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 230
- Enrollment target
NCT01826487: Completed Phase 3 study, sponsored by PTC Therapeutics.
NCT01826487 is a Phase 3 study that has completed, run by PTC Therapeutics. The registered enrollment target is 230 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (70% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01826487, a Phase 3 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 230 participants
- Enrollment target
Study Summary
Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.
Primary Outcome
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirm
Interventions
- DRUG Placebo
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 230 participants |
| Start Date | 2013-03-26 |
| Est. Completion | 2015-08-20 |
| Phase | Phase 3 |
What the finished NCT01826487 record still lists
NCT01826487 is an interventional study that assigns participants to a tested intervention. The registered 230 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (70% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01826487 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01826487 about?
NCT01826487 is a clinical study titled "Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)". Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to...
What is the current status of trial NCT01826487?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 230 participants. The study started on 2013-03-26. Estimated completion is 2015-08-20.
What interventions are being tested in trial NCT01826487?
The interventions under investigation include: Placebo (DRUG), Ataluren (DRUG).
Who is sponsoring clinical trial NCT01826487?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01826487's enrollment target sits among peer trials
230 1304th of 2000 higher than 692 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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