NCT02107859

Study of Ataluren (PTC124) in Cystic Fibrosis

An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 61 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram \[ECG\] measurements, vital signs).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Ataluren	Ataluren will be administered per dose and schedule specified in the arm.

Timeline

• Start date: 2014-05-23
• Primary completion: 2017-06-05
• Completion: 2017-06-05
• First posted: 2014-04-08
• Last updated: 2020-03-25
• Results posted: 2020-03-25

Locations

17 sites across 7 countries: United States, Belgium, France, Israel, Italy, Spain, Sweden