Is a
Clinical Study attributes
NCT Number
Health Conditions in Trial
Trial Recruitment Size
610
Trial Sponsor
Clinical Trial Start Date
May 23, 2014
0Primary Completion Date
June 5, 2017
0Study Completion Date
June 5, 2017
0Clinical Trial Study Type
Interventional0
Interventional Trial Purpose
Treatment0
Intervention Type
Drug0
Intervention Name
Ataluren0
Interventional Trial Phase
Phase 30
Participating Facility
Official Name
An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)0
Last Updated
March 25, 2020
0Allocation Type
NA0
Intervention Model
Single Group Assignment0
Masking Type
None (Open Label)0
Study 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).
Timeline
No Timeline data yet.
Further Resources
No Further Resources data yet.
