VX17-659-102 Efficacy and Safety of VX-659/TEZ/IVA in CF Het-min
Research type
Research Study
Full title
A Phase 3, Randomised, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
IRAS ID
241180
Contact name
Peter Barry
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2017-004132-11
Duration of Study in the UK
1 years, 0 months, 22 days
Research summary
Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Changes in this gene can affect the amount of the protein made from this gene or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky and causes problems in the lungs and other organs. VX-659, tezacaftor and ivacaftor are a triple combination of therapies targeted at improving the function of this protein. Early studies have suggested that this triple combination may improve health in some patients with CF.
This clinical trial will evaluate how well the triple combination may treat CF in men and women with CF who are 12 years of age or older, and who have certain mutations in their CF genes. In this study, we are testing this triple combination in people with one copy of a CF gene with a mutation termed F508del and one copy of a CF gene with a mutation termed a minimal function mutation. The study plans to include 360 participants worldwide, who will be equally divided to receive either the active treatment (triple combination of VX-659, tezacaftor and ivacaftor) or placebo (inactive pills). The study duration is 32 weeks including a 4 week screening period where doctors will assess participant’s health to ensure they are suitable to enter the part of the trial when these new medications are used. This will be followed by 24 weeks of treatment and a follow-up visit after 4 weeks. The study will check if the triple combination can improve lung function and other measures of health in people with cystic fibrosis.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
18/NE/0104
Date of REC Opinion
26 Apr 2018
REC opinion
Further Information Favourable Opinion