Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 3 study of Cystic Fibrosis, sponsored by PTC Therapeutics.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Spirometry was used to assess pulmonary function by measuring the percentage of predicted function, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was assessed by using current guidelines of the American Thoracic Society (ATS) and European Respiratory Society (ERS). Baseline was the average of percent-predicted FEV1 at screening and randomization.
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 |
What the finished NCT00803205 record still lists
NCT00803205 is an interventional study that assigns participants to a tested intervention. The registered 238 participants enrollment target is mid-sized for trials with a published cap, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (34% higher).
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 names 20 study sites across 15 states, led by 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.
Learn More About Clinical Trials
Similar trials for Cystic Fibrosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT00803205's enrollment target sits among peer trials
238 30th of 169 higher than 140 of 169 other Cystic Fibrosis trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Cystic Fibrosis 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
-
Assessing Tenapanor as a Treatment of CF-related Constipation.
RECRUITING · Phase 3
-
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
RECRUITING · Phase 3
-
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
ACTIVE NOT RECRUITING · Phase 3
-
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
COMPLETED · Phase 3
-
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
COMPLETED · Phase 3
-
OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis
COMPLETED · Phase 3
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT05012072 · 238 participants · NA
Reducing Pregnancy Risks: The Mastery Lifestyle Intervention
- NCT05400252 · 238 participants · NA
Whole Health in VA Mental Health: Omnis Salutis
- NCT06380517 · 238 participants · Phase 3
Dichoptic Treatment for Amblyopia in Children 4 to 7 Years of Age
- NCT06452797 · 238 participants · NA
Prognostic Estimates Among ICU Clinicians
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI