A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

Phase 3

Completed

Brief Summary: This study will evaluate the efficacy, safety, 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 homozygous for F508del.

Recruitment & Eligibility

Inclusion Criteria

Homozygous for the F508del mutation (F/F)
Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key

Exclusion Criteria

Clinically significant cirrhosis with or without portal hypertension
Lung infection with organisms associated with a more rapid decline in pulmonary status
Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Arm && Interventions

Group	Intervention	Description
ELX/TEZ/IVA	IVA	Following TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.
TEZ/IVA	TEZ/IVA	Following TEZ/IVA run-in period of 4 weeks, participants received TEZ 100 milligrams (mg) once daily (qd)/IVA 150 mg every 12 hours (q12h) in the treatment period for 24 weeks.
TEZ/IVA	IVA	Following TEZ/IVA run-in period of 4 weeks, participants received TEZ 100 milligrams (mg) once daily (qd)/IVA 150 mg every 12 hours (q12h) in the treatment period for 24 weeks.
ELX/TEZ/IVA	ELX/TEZ/IVA	Following TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.

Primary Outcome Measures

Name	Time	Method
Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score	From Baseline Through Week 24	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.

Secondary Outcome Measures

Name	Time	Method
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	From Day 1 in the 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 Week 28)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)	From Baseline Through Week 24	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Absolute Change in Sweat Chloride (SwCl)	From Baseline Through Week 24	Sweat samples were collected using an approved collection device.

Locations (35): The Prince Charles Hospital
🇦🇺
Chermside, Australia
Institute for Respiratory Health
🇦🇺
Nedlands, Australia
Perth Children's Hospital
🇦🇺
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
🇦🇺
New Lambton, Australia
The Royal Children's Hospital
🇦🇺
Parkville, VIC, Australia
Queensland Children's Hospital
🇦🇺
South Brisbane, Australia
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪
Brussels, Belgium
UZ Antwerpen
🇧🇪
Edegem, Belgium
Universitair Ziekenhuis Gent
🇧🇪
Gent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪
Leuven, Belgium
Scroll for more (25 remaining)
The Prince Charles Hospital
🇦🇺Chermside, Australia