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NCT01140451 · ClinicalTrials.gov registry record · Phase 3
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
A Phase 3 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 191
- Enrollment target
NCT01140451: Completed Phase 3 study, sponsored by PTC Therapeutics.
NCT01140451 is a Phase 3 study that has completed, run by PTC Therapeutics. The registered enrollment target is 191 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01140451, a Phase 3 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 191 participants
- Enrollment target
Study Summary
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients 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 3 extension trial that will evaluate the long-term safety of ataluren in adult and pediatric participants with nonsense mutation CF (nmCF), as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology. Funding source for this study is the FDA OOPD.
Primary Outcome
A TEAE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship that occurred or worsened in the period extending from the first dose of study drug to 6 weeks after the last dose of study drug. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immedi
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 191 participants |
| Start Date | 2010-08-12 |
| Est. Completion | 2013-12-02 |
| Phase | Phase 3 |
What the finished NCT01140451 record still lists
NCT01140451 is an interventional study that assigns participants to a tested intervention. The registered 191 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT01140451 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01140451 about?
NCT01140451 is a clinical study titled "Extension Study of Ataluren (PTC124) in Cystic Fibrosis". Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients with the di...
What is the current status of trial NCT01140451?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 191 participants. The study started on 2010-08-12. Estimated completion is 2013-12-02.
What interventions are being tested in trial NCT01140451?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT01140451?
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 NCT01140451's enrollment target sits among peer trials
191 1402nd of 2000 higher than 599 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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