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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 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 38 participants.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00264888
The ClinicalTrials.gov registry entry for NCT00264888 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 38 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which PTC124 is the first listed.
NCT00264888 reports 0 study locations.
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.
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