This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
113
Participants received VX-445/TEZ/IVA orally once daily in the morning.
Participants received TEZ/IVA orally once daily in the morning.
Participants received IVA orally once daily in the evening.
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline at Week 4
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline at Week 4
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
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 indicate fewer symptoms and better health-related quality of life.
Time frame: From Baseline at Week 4
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8)
Observed Pre-Dose Concentration (Ctrough) of VX-445, TEZ, TEZ Metabolite (M1-TEZ), and IVA
Time frame: Day 1 and Week 4
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Participants received placebo matched to VX-445/TEZ/IVA orally once daily in the morning.
Participants received placebo matched TEZ/IVA orally once daily in the morning.
Banner University of Arizona Medical Center
Tucson, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Miller Children's Hospital/ Long Beach Memorial
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Valley Children's Hospital
Madera, California, United States
National Jewish Health
Denver, Colorado, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center/ Joe DiMaggio Children's Hospital/ Memorial Regional Hospital
Hollywood, Florida, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Tampa General Hospital Cardiac and Lung Transplant Clinic
Tampa, Florida, United States
...and 34 more locations