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NCT01009294 · ClinicalTrials.gov registry record · Phase 2
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 6
- Enrollment target
NCT01009294 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 6 participants.
The verdict
NCT01009294, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 6 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 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.
Interventions
- DRUG Ataluren
- DRUG Chronic Corticosteroid Therapy
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2010-01-13 |
| Est. Completion | 2010-03-23 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01009294
The ClinicalTrials.gov registry entry for NCT01009294 describes a study currently listed as terminated, categorized as Phase 2. The registered enrollment target is 6 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 Ataluren is the first listed.
NCT01009294 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01009294 about?
NCT01009294 is a clinical study titled "Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/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 NCT01009294?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 6 participants. The study started on 2010-01-13. Estimated completion is 2010-03-23.
What interventions are being tested in trial NCT01009294?
The interventions under investigation include: Ataluren (DRUG), Chronic Corticosteroid Therapy (DRUG).
Who is sponsoring clinical trial NCT01009294?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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