Evaluating VX-659 Combination Therapy safety & efficacy in CF patients
Research type
Research Study
Full title
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis
IRAS ID
226533
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2016-003585-11
Duration of Study in the UK
0 years, 7 months, 7 days
Research summary
Cystic Fibrosis (CF) is a genetic disease caused by mutations in the gene encoding a cell transport protein (ion channel). This ion channel transports chloride ions in and out of cells in multiple organ systems. When ions are not transported correctly the salt and water balance in cells and tissues is not controlled, leading to the production of sticky mucus in the lungs, airways and intestines.
Vertex is developing treatments to help increasing production of the ion channel and increase transport of chloride ions. IVA and Orkambi are two drugs that have been approved for treatment. Vertex is also developing TEZ and now VX-659. VX-659 increases the production of the ion channel, but in a different way to the current treatments. It is hoped that by using VX-659 as well as current treatments the production of the ion channel will be increased even further.
This study follows a Phase 1 study testing the safety and tolerability of VX-659 in healthy volunteers and some CF patients. This study aims to test the safety and efficacy of VX-659 combination therapy in CF patients. The study is in 3 parts with 2 treatment periods.
Part 1 F508del/minimal function genotype participants will receive VX-659 (high, mid or low dose) with tezacaftor/ivacaftor, VX-659 with ivacaftor or placebo.only in period 1 for 4 weeks. Then, in period 2 (4 days) either tezacaftor/ivacaftor, ivacaftor, or placebo only.
Part 2 F508del/F508del genotype participants will receive VX-659 (high dose level) with tezacaftor/ivacaftor,VX-659 with ivacaftor or tezacaftor/ivacaftor in period 1 for 4 weeks. Then, in period 2 (4 weeks) they will receive tezacaftor/ivacaftor.
Part 3 F508del/gating genotype participants will have a run in period of 4 weeks receiving tezacaftor/ivacaftor, receive VX-659 (high dose level ) with tezacaftor/ivacaftor or tezacaftor/ivacaftor in period 1 for 4 weeks . Then, in period 2 (4 weeks) they will receive tezacaftor/ivacaftor.
REC name
London - Westminster Research Ethics Committee
REC reference
17/LO/0683
Date of REC Opinion
1 Jun 2017
REC opinion
Further Information Favourable Opinion