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NCT00592553 · ClinicalTrials.gov registry record · Phase 2
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 174
- Enrollment target
NCT00592553: Completed Phase 2 study, sponsored by PTC Therapeutics.
NCT00592553 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 174 participants, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (31% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00592553, a Phase 2 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 174 participants
- Enrollment target
Study Summary
DMD/BMD is a genetic disorder that 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 during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 13 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. This study is a Phase 2b trial that will evaluate the clinical benefit of ataluren in boys with DMD/BMD due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.
Primary Outcome
The 6MWD test was performed in a 30 meters long flat corridor, where the participant was 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.
Interventions
- DRUG Placebo
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 174 participants |
| Start Date | 2008-02-29 |
| Est. Completion | 2009-12-31 |
| Phase | Phase 2 |
What the finished NCT00592553 record still lists
NCT00592553 is an interventional study that assigns participants to a tested intervention. The registered 174 participants enrollment target is mid-sized for trials with a published cap, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (31% higher).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT00592553 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00592553 about?
NCT00592553 is a clinical study titled "Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)". DMD/BMD is a genetic disorder that 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 during childhood...
What is the current status of trial NCT00592553?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 174 participants. The study started on 2008-02-29. Estimated completion is 2009-12-31.
What interventions are being tested in trial NCT00592553?
The interventions under investigation include: Placebo (DRUG), Ataluren (DRUG).
Who is sponsoring clinical trial NCT00592553?
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 NCT00592553's enrollment target sits among peer trials
174 396th of 2000 higher than 1,604 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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