VX16-661-115 Efficacy and Safety of TEZ/IVA in 6 to 11 Years With CF
Research type
Research Study
Full title
A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
IRAS ID
241642
Contact name
Donald Urquhart
Contact email
Sponsor organisation
Vertex Pharmaceuticals (Europe) Limited
Eudract number
2016-004479-35
Duration of Study in the UK
0 years, 7 months, 8 days
Research summary
Cystic Fibrosis (CF) is a genetic disease caused by mutations in the gene encoding a cell transport protein (ion channel). This ion channel transports chloride ions in and out of cells in multiple organ systems. When ions are not transported correctly the salt and water balance in cells and tissues is not controlled, leading to the production of sticky mucus in the lungs, airways and intestines.
This clinical study is designed to evaluate the safety and efficacy of tezacaftor in combination with /ivacaftor in subjects with CF aged 6 through 11 years, with either two copies of a F508del mutation (homozygous) or one copy of a F508del mutation (heterozygous) and with a residual function mutation.
Approximately 65 male and female subjects will be enrolled, of which up to 15 participants will have one copy of the F508del mutation and with a residual function mutation. The remaining participants will have two copies the F508del mutation. Each subject will participate in the study for 16 weeks: 4-week Screening Period, 8-week Treatment Period, and 4-week Safety Follow-up Period. Subjects (who are homozygous for F508del will receive either active tezacaftor/ivacaftor or matching placebo respectively. Subjects who are heterozygous for F508del with a residual function mutation) will receive either active tezacaftor/ivacaftor or active ivacaftor/placebo for tezacaftor. Both groups will be randomised 4:1 to the tezacaftor/ivacaftor arm or respective blinding arm.
REC name
West of Scotland REC 1
REC reference
18/WS/0041
Date of REC Opinion
24 Apr 2018
REC opinion
Further Information Favourable Opinion