Study Evaluating the Efficacy & Safety of VX-445/Tezacaftor/Ivacaftor
Research type
Research Study
Full title
A Phase 3b, Randomized, Double blind, Controlled Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
IRAS ID
269475
Contact name
Damian Downey
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2019-001735-31
Duration of Study in the UK
1 years, 2 months, 1 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.
This study will help researchers learn if the study drugs VX-445/TEZ/IVA is safe and effective in the tested doses. 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 two copies of the CF gene mutation F508del (F/F Type).
The total study duration is approximately 36 weeks (4 weeks for the Screening Period, 4 weeks for the TEZ/IVA Run-in Period, 24 weeks for the Treatment Period, and 4 weeks for the Safety Follow-up Period. There will be approximately 158 people participating in this study.REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
19/NW/0482
Date of REC Opinion
13 Sep 2019
REC opinion
Further Information Favourable Opinion