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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 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 174 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00592553
The ClinicalTrials.gov registry entry for NCT00592553 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 174 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 2 interventions - of which Placebo is the first listed.
NCT00592553 reports 0 study locations.
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.
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