Study of Ataluren (PTC124™) in Cystic Fibrosis
Public ClinicalTrials.gov record NCT00803205. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00803205
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 238 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 7, 2009
- Primary completion
- Nov 11, 2011
- Completion
- Nov 11, 2011
- Last update posted
- May 13, 2020
2009 – 2011
United States locations
- U.S. sites
- 21
- U.S. states
- 16
- U.S. cities
- 19
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama-Birmingham | Birmingham | Alabama | 35233 | — |
| Miller Children's Hospital Long Beach | Long Beach | California | 90806 | — |
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | — |
| Stanford | Palo Alto | California | 94304 | — |
| Rady Children's Hospital - San Diego | San Diego | California | 92123 | — |
| The Children's Hospital | Aurora | Colorado | 80045 | — |
| University of Miami | Miami | Florida | 33136 | — |
| Miami Children's Hospital | Miami | Florida | 33155 | — |
| Emory University Cystic Fibrosis Center | Atlanta | Georgia | 30322 | — |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| Johns Hopkins Children's Center | Baltimore | Maryland | 21287 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| Washington University | St Louis | Missouri | 63110 | — |
| Beth Israel Medical Center | New York | New York | 10011 | — |
| New York Medical College | Valhalla | New York | 10595 | — |
| University of North Carolina | Chapel Hill | North Carolina | 27599 | — |
| Rainbow Babies & Children's Hospital | Cleveland | Ohio | 44106 | — |
| Childrens Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | — |
| University of Texas | Tyler | Texas | 75708 | — |
| University of Washington | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 17 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00803205, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 13, 2020 · Synced Sep 3, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00803205 live on ClinicalTrials.gov.