NCT01140451

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

A Phase 3 Extension Study of Ataluren (PTC124®) in Subjects With Nonsense-Mutation-Mediated Cystic Fibrosis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 191 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This Phase 3, open-label, safety and efficacy study will be performed at sites in North America, Europe, and Israel. The study will enroll up to approximately 208 participants with nmCF who participated in a previous Phase 3 study of ataluren (PTC124-GD-009-CF \[Study 009\], NCT00803205). Participants will receive study drug 3 times per day (TID) (at breakfast, lunch, and dinner) for approximately 48 weeks (approximately 1 year). Study assessments will be performed at clinic visits every 8 weeks.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Ataluren	Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Timeline

• Start date: 2010-08-12
• Primary completion: 2013-12-02
• Completion: 2013-12-02
• First posted: 2010-06-09
• Last updated: 2020-10-19
• Results posted: 2020-10-19

Locations

31 sites across 11 countries: United States, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, United Kingdom