The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
507
Fixed-dose combination (FDC) tablet for oral administration.
Tablet for oral administration.
Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 196
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set
Sweat samples were collected using an approved collection device. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set
Sweat samples were collected using an approved collection device. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set
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.
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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
Kaiser Permanente
Oakland, California, United States
University of California Davis Medical Center
Sacramento, California, United States
UCSF Gateway Medical Center
San Francisco, California, United States
National Jewish Health
Denver, Colorado, United States
University of Florida, Shands Hospital
Gainesville, Florida, United States
...and 100 more locations
Time frame: From Baseline at Week 192
Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set
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 at Week 192
Time to First PEx for 102/105 Efficacy Set
Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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 at Week 192
Time to First PEx for 103/105 Efficacy Set
Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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 at Week 192
Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set
BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in Body Mass Index (BMI) for 103/105 Efficacy Set
BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in BMI Z-score for 102/105 Efficacy Set
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. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in BMI Z-score for 103/105 Efficacy Set
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. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in Body Weight for 102/105 Efficacy Set
This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change in Body Weight for 103/105 Efficacy Set
This analysis set included study 103 parent study participants who received TEZ/IVA and ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 102/105 Efficacy Set
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. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA.
Time frame: From Baseline at Week 192
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/105 Efficacy Set
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. This analysis set included study 103 parent study participants who received TEZ/IVA and ELX/TEZ/IVA.
Time frame: From Baseline at Week 192