This is a Phase 2, randomized, double blind, placebo and active-controlled, parallel group, multicenter study designed to evaluate the safety and tolerability of VX-152 in Triple Combination (TC) with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy (F508del/MF), or who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
80
Tablet for oral administration.
Fixed-dose combination tablet for oral administration.
Tablet for oral administration.
Placebo matched to VX-152.
Placebo matched to VX-152/TEZ/IVA triple combination (TC).
Unnamed facility
Birmingham, Alabama, United States
Unnamed facility
La Jolla, California, United States
Unnamed facility
Los Angeles, California, United States
Unnamed facility
Orlando, Florida, United States
Unnamed facility
Chicago, Illinois, United States
Unnamed facility
Peoria, Illinois, United States
Unnamed facility
St Louis, Missouri, United States
Unnamed facility
Chapel Hill, North Carolina, United States
Unnamed facility
Akron, Ohio, United States
Unnamed facility
Cleveland, Ohio, United States
...and 6 more locations
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 Through Safety Follow-up Visit (Up to Day 43 for Part 1 and Day 71 for Part 2)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 15 for Part 1 and Part 2 Cohort 2A
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline at Day 15
Absolute Change in ppFEV1 Through Day 29 for Part 2 Cohort 2B
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline Through Day 29
Absolute Change in Sweat Chloride Concentrations at Day 15 for Part 1 and Part 2 Cohort 2A
Sweat samples were collected using an approved collection device.
Time frame: From Baseline at Day 15
Absolute Change in Sweat Chloride Concentrations Through Day 29 for Part 2 Cohort 2B
Sweat samples were collected using an approved collection device.
Time frame: From Baseline Through Day 29
Relative Change in ppFEV1 at Day 15 for Part 1 and Part 2 Cohort 2A
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline at Day 15
Relative Change in ppFEV1 Through Day 29 for Part 2 Cohort 2B
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline Through Day 29
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 15 for Part 1 and Part 2 Cohort 2A
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time frame: From Baseline at Day 15
Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 29 for Part 2 Cohort 2B
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time frame: From Baseline at Day 29
Pre-dose Plasma Concentration (Ctrough) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA
Time frame: Pre-dose at Day 8, Day 15 and Day 29
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