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NCT00947193 · ClinicalTrials.gov registry record · Phase 2
Study of Ataluren (PTC124) in Hemophilia A and B
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 13
- Enrollment target
NCT00947193 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 13 participants.
The verdict
NCT00947193, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 13 participants
- Enrollment target
Study Summary
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting. The lack of FVIII/FIX can produce bleeding episodes that cause damage of the bone, muscles, joints, and tissues. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of the disease in approximately 10-30% of participants with hemophilia and results in severe manifestations. Ataluren (PTC124) is an orally delivered, investigational drug that acts to overcome the effects of the premature stop codon, potentially enabling the production of functional FVIII/FIX. This study is a Phase 2a trial evaluating the safety and efficacy of ataluren in participants with HA or HB due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase FVIII/FIX activity levels.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2009-10-14 |
| Est. Completion | 2011-08-30 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00947193
The ClinicalTrials.gov registry entry for NCT00947193 describes a study currently listed as terminated, categorized as Phase 2. The registered enrollment target is 13 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.
NCT00947193 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00947193 about?
NCT00947193 is a clinical study titled "Study of Ataluren (PTC124) in Hemophilia A and B". Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting. The lack of FVIII/FIX can produce bleeding episodes that cause damage of the bone, musc...
What is the current status of trial NCT00947193?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 13 participants. The study started on 2009-10-14. Estimated completion is 2011-08-30.
What interventions are being tested in trial NCT00947193?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT00947193?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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