Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Public ClinicalTrials.gov record NCT01140451. 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 Extension Study of Ataluren (PTC124®) in Subjects With 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 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.
Study identification
- NCT ID
- NCT01140451
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 191 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
- Aug 12, 2010 (Actual)
- Primary completion
- Dec 2, 2013 (Actual)
- Completion
- Dec 2, 2013 (Actual)
- Last update posted
- Oct 19, 2020
2010 – 2013
United States locations
- U.S. sites
- 15
- U.S. states
- 13
- U.S. cities
- 15
| 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 | — |
| The Children's Hospital | Aurora | Colorado | 80045 | — |
| University of Miami | Miami | Florida | 33136 | — |
| Emory University Cystic Fibrosis Center | Atlanta | Georgia | 30322 | — |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | — |
| Johns Hopkins Children's Center | Baltimore | Maryland | 21287 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| Washington University | St Louis | Missouri | 63110 | — |
| St. Vincent's Hospital | 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 | — |
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 16 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01140451, 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 Oct 19, 2020 · Synced Oct 19, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01140451 live on ClinicalTrials.gov.