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NCT00264888 · ClinicalTrials.gov registry record · Phase 2
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
A Phase 2 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 38
- Enrollment target
NCT00264888: Completed Phase 2 study, sponsored by PTC Therapeutics.
NCT00264888 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 38 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (71% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00264888, a Phase 2 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 38 participants
- Enrollment target
Study Summary
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein. PTC124 has been shown to partially restore dystrophin production in animals with DMD due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional dystrophin protein in the muscles of patients with DMD due to a nonsense mutation.
Interventions
- DRUG PTC124
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 38 participants |
| Start Date | 2005-12 |
| Est. Completion | 2007-05 |
| Phase | Phase 2 |
What the finished NCT00264888 record still lists
NCT00264888 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 38 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (71% lower).
The record links to 0 conditions, and to 1 intervention - of which PTC124 is the first listed.
NCT00264888 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00264888 about?
NCT00264888 is a clinical study titled "Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy". In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein. PTC124 has been shown to partially restore dystrophin production in animals with DMD due to a nonsense mutation. The main purpos...
What is the current status of trial NCT00264888?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 38 participants. The study started on 2005-12. Estimated completion is 2007-05.
What interventions are being tested in trial NCT00264888?
The interventions under investigation include: PTC124 (DRUG).
Who is sponsoring clinical trial NCT00264888?
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 NCT00264888's enrollment target sits among peer trials
38 1467th of 2000 higher than 517 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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