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NCT00847379 · ClinicalTrials.gov registry record · Phase 2
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 173
- Enrollment target
NCT00847379 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 173 participants.
The verdict
NCT00847379, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 173 participants
- Enrollment target
Study Summary
Duchenne/Becker muscular dystrophy (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 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 173 participants |
| Start Date | 2009-01-31 |
| Est. Completion | 2010-05-24 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00847379
The ClinicalTrials.gov registry entry for NCT00847379 describes a study currently listed as terminated, categorized as Phase 2. The registered enrollment target is 173 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.
NCT00847379 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00847379 about?
NCT00847379 is a clinical study titled "Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)". Duchenne/Becker muscular dystrophy (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 los...
What is the current status of trial NCT00847379?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 173 participants. The study started on 2009-01-31. Estimated completion is 2010-05-24.
What interventions are being tested in trial NCT00847379?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT00847379?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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