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NCT00803205 · ClinicalTrials.gov registry record · Phase 3
Study of Ataluren (PTC124™) in Cystic Fibrosis
A Phase 3 study of Cystic Fibrosis, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 238
- Enrollment target
- 20
- Study locations
NCT00803205 is a Phase 3 study of Cystic Fibrosis that has completed, run by PTC Therapeutics. The registered enrollment target is 238 participants, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (34% higher). The trial reports 20 study locations across 15 states.
The verdict
NCT00803205, a Phase 3 study of Cystic Fibrosis, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 238 participants
- Enrollment target
- 20
- Study locations
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 trial that will evaluate the clinical benefit of ataluren in adult and pediatric participants with CF due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve pulmonary function and whether the drug can safely be given for a long period of time. The study will also assess the effects of ataluren on CF pulmonary exacerbation frequency, cough frequency, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Ataluren
Study Locations (20)
California
- Miller Children's Hospital Long Beach - Long Beach
- Lucile Packard Children's Hospital - Palo Alto
- Stanford - Palo Alto
- Rady Children's Hospital - San Diego - San Diego
Florida
- University of Miami - Miami
- Miami Children's Hospital - Miami
New York
- Beth Israel Medical Center - New York
- New York Medical College - Valhalla
Alabama
- University of Alabama-Birmingham - Birmingham
Colorado
- The Children's Hospital - Aurora
Georgia
- Emory University Cystic Fibrosis Center - Atlanta
Illinois
- Children's Memorial Hospital - Chicago
Iowa
- University of Iowa - Iowa City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 238 participants |
| Start Date | 2009-09-08 |
| Est. Completion | 2011-11-12 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00803205
The ClinicalTrials.gov registry entry for NCT00803205 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 238 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (34% higher). The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Cystic Fibrosis appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT00803205 reports 20 study locations spanning 15 distinct geographic areas - top geographies include California, Florida, New York.
Frequently Asked Questions
What is clinical trial NCT00803205 about?
NCT00803205 is a clinical study titled "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 NCT00803205?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 238 participants. The study started on 2009-09-08. Estimated completion is 2011-11-12.
What conditions does trial NCT00803205 study?
This clinical trial studies the following conditions: Cystic Fibrosis.
What interventions are being tested in trial NCT00803205?
The interventions under investigation include: Placebo (DRUG), Ataluren (DRUG).
Who is sponsoring clinical trial NCT00803205?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00803205 being conducted?
This trial has 20 study locations across Alabama, California, Colorado, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.