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NCT00234663 · ClinicalTrials.gov registry record · Phase 2
PTC124 for Cystic Fibrosis
A Phase 2 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 24
- Enrollment target
NCT00234663: Completed Phase 2 study, sponsored by PTC Therapeutics.
NCT00234663 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 24 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (82% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00234663, a Phase 2 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 24 participants
- Enrollment target
Study Summary
In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional CFTR protein in the cells of patients with CF due to a nonsense mutation.
Interventions
- DRUG PTC124
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2005-09 |
| Est. Completion | 2006-08 |
| Phase | Phase 2 |
What the finished NCT00234663 record still lists
NCT00234663 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (82% lower).
The record links to 0 conditions, and to 1 intervention - of which PTC124 is the first listed.
NCT00234663 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00234663 about?
NCT00234663 is a clinical study titled "PTC124 for Cystic Fibrosis". In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation....
What is the current status of trial NCT00234663?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 24 participants. The study started on 2005-09. Estimated completion is 2006-08.
What interventions are being tested in trial NCT00234663?
The interventions under investigation include: PTC124 (DRUG).
Who is sponsoring clinical trial NCT00234663?
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 NCT00234663's enrollment target sits among peer trials
24 1756th of 2000 higher than 217 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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