VX18-445-104 VX-445/TEZ/IVA Safety & Efficacy in CF (F/G and F/RF)
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
IRAS ID
268820
Contact name
Peter Barry
Contact email
Sponsor organisation
Vertex Pharmaceuticals (Europe) Ltd
Eudract number
2018-002835-76
Duration of Study in the UK
0 years, 11 months, 16 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-445, 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 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 the other copy of a CF gene with a mutation termed a gating or residual function.
The study plans to include 250 participants worldwide. The study duration is approximately 20-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 4-weeks taking either ivacaftor, or tezacaftor and ivacaftor in combination. Then 8-weeks of treatment on either the active treatment (triple combination of VX-445, tezacaftor and ivacaftor) or control arm. You will not know which treatment you are on. You will have 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
London - Westminster Research Ethics Committee
REC reference
19/LO/1318
Date of REC Opinion
29 Oct 2019
REC opinion
Further Information Favourable Opinion