The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
597
Fixed-dose combination tablets for oral administration.
Fixed-dose combination tablets for oral administration.
Tablet for oral administration.
Placebo matched to VX-121/TEZ/D-IVA for oral administration.
Placebo matched to ELX/TEZ/IVA for oral administration.
Placebo matched to IVA for oral administration.
University of Alabama at Birmingham
Birmingham, Alabama, United States
Banner University of Arizona Medical Center
Tucson, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline Through Week 24
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline Through Week 24
Percentage of Participants With SwCl <60 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline Through Week 24
Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline Through Week 24
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University of California Davis Medical Center
Sacramento, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
National Jewish Health
Denver, Colorado, United States
Yale New Haven Hospital
New Haven, Connecticut, United States
Nemours Children's Specialty Care
Jacksonville, Florida, United States
...and 160 more locations