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NCT00759876 · ClinicalTrials.gov registry record · Phase 2
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
NCT00759876 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 36 participants.
The verdict
NCT00759876, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
Study Summary
Duchenne muscular dystrophy (DMD) 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 in approximately 10-15% 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 2a extension trial that will evaluate the long-term safety of ataluren in boys with nonsense mutation DMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, strength, and other important clinical and laboratory measures.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2008-08-13 |
| Est. Completion | 2010-05-17 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00759876
The ClinicalTrials.gov registry entry for NCT00759876 describes a study currently listed as terminated, categorized as Phase 2. The registered enrollment target is 36 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 Ataluren is the first listed.
NCT00759876 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00759876 about?
NCT00759876 is a clinical study titled "Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)". Duchenne muscular dystrophy (DMD) 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 walkin...
What is the current status of trial NCT00759876?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2008-08-13. Estimated completion is 2010-05-17.
What interventions are being tested in trial NCT00759876?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT00759876?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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