Overview

Study of Ataluren (PTC124) in Cystic Fibrosis

Status:
Terminated
Trial end date:
2017-06-05
Target enrollment:
Participant gender:
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).
Phase:
Phase 3
Details
Lead Sponsor:
PTC Therapeutics