VX-659 Bioavailability study in Cystic Fibrosis subjects - Part D
Research type
Research Study
Full title
A Phase 1, Randomized, Double blind, Placebo controlled, Dose Escalation, and Bioavailability Study Evaluating the Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Subjects With Cystic Fibrosis
IRAS ID
215537
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2016-003048-35
Duration of Study in the UK
0 years, 4 months, 1 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 trial is split into 4 parts, A, B, C and D. Parts A-C involve dosing in healthy volunteers and Part D dosing in CF patients. Parts A- C will be the first time VX-659 will be administered to man, both alone and in combination with IVA and IVA/TEZ, Part D will be the first time VX-659 will be administered to CF patients. This application is for Part D only.
This objective of Part D is to evaluate the safety and tolerability of VX-659. CF patients will receive multiple oral doses of VX-659 suspension or placebo for 14 days, a combination of TEZ/IVA on the morning and a dose of IVA in the evening. Procedures will include physical examinations, vital sign measurements, pulse oximetry, eye exam, ECG (electrocardiogram), sweat chloride test, spirometry, urine test, pregnancy test, DNA test, and blood tests. Participation in Part D could last for approximately 10 weeks (including screening and follow-up).
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
16/SC/0515
Date of REC Opinion
21 Nov 2016
REC opinion
Favourable Opinion