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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: Clinical Trial Phase 2 study, sponsored by PTC Therapeutics.
NCT00947193 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 13 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
A plasma FVIII/FIX activity response was defined as an end-of-treatment (Day 14) activity of ≥1%.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2009-10-14 |
| Est. Completion | 2011-08-30 |
| Phase | Phase 2 |
Why NCT00947193 stopped before completion
NCT00947193 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT00947193 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00947193's enrollment target sits among peer trials
13 1947th of 2000 higher than 53 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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