VX11-770-110 Phase 3 study of ivacaftor for CF R117H-CFTR Mutation
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis who Have the R117H-CFTR Mutation
IRAS ID
106669
Contact name
Steve Cunningham
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2012-000387-19
Clinicaltrials.gov Identifier
Research summary
Cystic fibrosis (CF) is a disease that weakens its sufferers over a long period of time and leads to premature mortality. It affects approximately 30,000 individuals in the United States and 36,000 in the European Union. There is currently no cure. CF is caused by mutations (changes) in the CF transmembrane conductance regulator gene (CFTR). This study will provide information on the efficacy, safety, and pharmacokinetics (PK) of a drug called ivacaftor in subjects with CF who have one of over 1800 mutations in the CFTR gene that have been identified. A minimum of 40 (maximum of approximately 80) subjects with CF, aged 6 years or older, will be randomised to receive ivacaftor 150 mg or placebo every 12 hours. Ivacaftor or placebo will be administered in tablet form. Study treatment will last up to 24 weeks. Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in patients with CF.
REC name
North of Scotland Research Ethics Committee 1
REC reference
12/NS/0052
Date of REC Opinion
1 Jun 2012
REC opinion
Favourable Opinion