VX18-445-110 VX-445/TEZ/IVA Open-label in CF (F/G and F/RF)
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 Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Duration of Study in the UK
2 years, 8 months, 29 days
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. This study will test the 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 will include participants from study VX18-445-104 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.
London - Westminster Research Ethics Committee
Date of REC Opinion
29 Oct 2019
Further Information Favourable Opinion