The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
64
Fixed-dose combination (FDC) tablet for oral administration
Mono tablet for oral administration.
Children's Hospital of Orange County
Orange, California, United States
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 196
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 up to Week 192
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline up to Week 192
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 up to Week 192
Absolute Change in Body Mass Index (BMI)
BMI was defined as weight in kg divided by squared height in meters (m\^2).
Time frame: From Baseline up to Week 192
Absolute Change in BMI-for-age Z-score
BMI was defined as weight in kg divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a mean was above or below the standard. 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.
Time frame: From Baseline up to Week 192
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Children's Hospital Colorado
Aurora, Colorado, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Northwell Health- Long Island Jewish Medical Center
New Hyde Park, New York, United States
Clinical Research of Charlotte
Charlotte, North Carolina, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
...and 11 more locations
Number of Participants With Pulmonary Exacerbations (PEx) for 106/107
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 up to Week 192
Number of CF-related Hospitalizations for 106/107
The total number of CF related hospitalization (Planned + Unplanned) events across all participants were reported.
Time frame: From Baseline up to Week 192
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Time frame: From Baseline up to Week 192
Absolute Change in Weight
Time frame: From Baseline up to Week 192
Absolute Change in Weight-for-age Z-score
The z-score is a statistical measure to describe whether a mean was above or below the standard. 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.
Time frame: From Baseline up to Week 192
Absolute Change in Height
Time frame: From Baseline up to Week 192
Absolute Change in Height-for-age Z-score
The z-score is a statistical measure to describe whether a mean was above or below the standard. 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.
Time frame: From Baseline up to Week 192