VX17-445-105 Open-label Safety and Efficacy of VX-445/TEZ/IVA in CF
Research type
Research Study
Full title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
IRAS ID
244427
Contact name
Inderpal Kalayan
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-000185-11
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 8 months, 21 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 study will test 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. The triple combination will be tested in people with either, 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, or have two copies of the CF gene mutation F508del. The study plans to include participants from VX17-445-102 and VX17-445-103 studies and all participants will receive active treatment (triple combination of VX-445, tezacaftor and ivacaftor). The study duration is 100 weeks, which includes up to 96 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
West Midlands - Edgbaston Research Ethics Committee
REC reference
18/WM/0204
Date of REC Opinion
19 Oct 2018
REC opinion
Further Information Favourable Opinion