This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
307
Fixed-dose combination (FDC) tablets for oral administration.
Tablet for oral administration.
Placebo matched to ELX/TEZ/IVA for oral administration.
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
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 (RD) 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
Absolute Change in Weight
Time frame: From Baseline at 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
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Placebo matched to IVA for oral administration.
Medizinische Universität Innsbruck
Innsbruck, Austria
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, Belgium
Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
Edegem, Belgium
Universitair Ziekenhuis Gent
Ghent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
Stollery Children's Hospital
Edmonton, Canada
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
Montreal, Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Canada
...and 80 more locations
Time frame: Day 1 up to Week 28