A Phase 1 Study of VX-117 in Healthy Subjects
Research type
Research Study
Full title
A Phase 1, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-117 in Healthy Subjects
IRAS ID
258573
Contact name
Firas Almazedi
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-004699-36
Duration of Study in the UK
0 years, 10 months, 15 days
Research summary
VX-117 is a next generation CFTR (cystic fibrosis transmembrane conductance regulator) corrector. Cystic Fibrosis is caused by reduced quantity and/or function of CFTR protein. VX-117 improves the processing and trafficking of mutated CTFR protein, thereby increasing the quantity of functional CFTR protein. It is hoped that VX-117 used in combination with other cystic fibrosis drugs (tezacaftor and ivacaftor) will produce an effective treatment for Cystic Fibrosis. \n\nThis will be a double-blind, placebo-controlled, dose escalation study to evaluate the safety and tolerability of single and multiple doses of VX-117 given alone and multiple doses of VX-117 given in triple combination with tezacaftor and ivacaftor. \n\nPart A will be a single dose, sequential group study. 48 subjects will be studied as 6 groups of 8 subjects (6 VX-117: 2 placebo). Subjects will reside at the Clinical Research Unit (CRU) from Day 1 (the day before dosing) to Day 7 and will return for a post-study visit 7 to 10 days post-dose.\n\nPart B will be a multiple dose, sequential group study. 48 subjects will be studied as 6 groups of 8 subjects (6 VX-117: 2 placebo). Subjects will reside at the CRU from Day 1 (the day before dosing) to Day 14 and will return for a post-study visit 7 to 10 days post-dose. In select Part B cohorts subjects will also receive a single dose of ivacaftor on Days 1 and 11 to evaluate the single dose pharmacokinetics of ivacaftor administered with and without VX 117.\n\nPart C will be a multiple dose, sequential group study. 16 subjects will be studied as 2 groups of 8 subjects (6 VX-117/ivacaftor/tezacaftor: 2 triple placebo). Subjects will reside at the CRU from Day 1 (the day before dosing) to Day 18 and will return for a post-study visit 7 to 10 days post-dose.\n
REC name
London - Brent Research Ethics Committee
REC reference
19/LO/0380
Date of REC Opinion
7 May 2019
REC opinion
Further Information Favourable Opinion