This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
385
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Participants received IVA orally once daily in the evening.
Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to 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 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 through Week 24
Number of Pulmonary Exacerbations (PEx)
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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
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 indicating fewer symptoms and better health-related quality of life.
Time frame: From Baseline through Week 24
Absolute Change in Body Mass Index (BMI)
BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
Time frame: From Baseline at Week 24
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Stanford University
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Hartford Health
Hartford, Connecticut, United States
Yale New Haven Medical Center
New Haven, Connecticut, United States
University of Miami/ Miller School of Medicine
Miami, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Arnold Palmer Hospital
Orlando, Florida, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, United States
St. Luke's CF Center of Idaho
Boise, Idaho, United States
...and 91 more locations
Absolute Change in Sweat Chloride
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 indicating fewer symptoms and better health-related quality of life.
Time frame: From Baseline at Week 4
Time-to-first Pulmonary Exacerbation (PEx)
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Time frame: From Baseline through Week 24
Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline
BMI was defined as weight in kg divided by height in m\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI.
Time frame: From Baseline at Week 24
Absolute Change in Body Weight
Time frame: From Baseline at Week 24
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 28 weeks)
Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA
Time frame: Pre-dose on Week 4, 8, 12, and 16