This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
251
Fixed-dose combination (FDC) tablet for oral administration.
Tablet for oral administration.
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Baseline up to Week 100
Part A: Absolute Change From Parent Study Baseline 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 96
Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline 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 96
Part A: Absolute Change From Parent Study Baseline in BMI Z-score
BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Time frame: From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline in Body Weight
Time frame: From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
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Banner University of Arizona Medical Center
Tucson, Arizona, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, United States
Stanford University
Palo Alto, California, United States
University of California Davis Medical Center
Sacramento, California, United States
University of California San Francisco, Lung Transplant Program
San Francisco, California, United States
National Jewish Health
Denver, Colorado, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, United States
Indiana University
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
...and 74 more locations
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 96