A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
Research type
Research Study
Full title
A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
IRAS ID
269907
Contact name
Peter Barry
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-004652-38
Duration of Study in the UK
2 years, 5 months, 12 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 to evaluate the long term safety and tolerability of VX-445/tezacaftor (TEZ)/ivacaftor (IVA)in participants with CF who are homozygous or heterozygous for the F508del mutation.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.
The study plans to include 400 participants worldwide.Male and female participants with cystic fibrosis who are 12 years of age or older. The study duration is up to approximately 100 weeks ,including a Treatment Period of up to 96 weeks and a 4-week Safety Follow-up Period.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
19/NW/0540
Date of REC Opinion
6 Nov 2019
REC opinion
Further Information Favourable Opinion