VX17-445-103 Efficacy and Safety of VX-445/TEZ/IVA in CF Homozygous
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 Homozygous for the F508del Mutation (F/F)
IRAS ID
244419
Contact name
Edward Nash
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-000184-89
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 5 months, 31 days
Research summary
Cystic fibrosis (CF) is a chronic lifelong disease resulting from mutations in one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Mutations in this CF-causing gene can affect the amount or the function of the protein made from this gene. VX-445, tezacaftor and ivacaftor are a triple combination of therapies targeted at improving the function of this protein. This triple combination has shown positive effects in some patients with CF. This clinical trial will evaluate if the triple combination will treat CF in men and women with CF who are 12 years of age or older, and who have two copies of the CF gene mutation F508del. The study plans to include approximately 100 participants globally, who will be assigned to either active treatment group (triple combination of VX-445, tezacaftor and ivacaftor) or a group receiving tezacaftor and ivacaftor only. The total study duration is approximately 16 weeks: a 4 week screening period, followed by a 4 week “run-in” period in which all participants will be given tezacaftor and ivacaftor, followed by the 4 week treatment period where half of the participants are assigned to the active treatment group (triple combination of VX-445, tezacaftor and ivacaftor) and the other half to tezacaftor and ivacaftor alone, finishing with an additional 4 weeks of safety follow-up. The study will check if the triple combination can improve lung function and other measures of health in people with CF.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
18/NE/0201
Date of REC Opinion
12 Sep 2018
REC opinion
Further Information Favourable Opinion