A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
- Registration Number
- NCT04105972
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 176
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- 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.
Trial Locations
- Locations (35)
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪Brussels, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
Perth Children's Hospital
🇦🇺Nedlands, Australia
Belfast City Hospital
🇬🇧Belfast, United Kingdom
St. James University Hospital
🇬🇧Leeds, United Kingdom
Leeds General Infirmary
🇬🇧Leeds, West Yorkshire, United Kingdom
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
🇦🇺New Lambton, Australia
The Prince Charles Hospital
🇦🇺Chermside, Australia
Institute for Respiratory Health
🇦🇺Nedlands, Australia
The Royal Children's Hospital
🇦🇺Parkville, VIC, Australia
UZ Antwerpen
🇧🇪Edegem, Belgium
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Johann Wolfgang Goethe University
🇩🇪Frankfurt, Germany
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
🇩🇪Jena, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
🇩🇪Koeln, Germany
Klinikum Innenstadt, University of Munich
🇩🇪München, Germany
University Hospitals Birmingham NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
University Hospitals Bristol NHS Foundation Trust, Bristol Royal Hospital
🇬🇧Bristol, United Kingdom
Papworth Hospital NHS Foundation Trust, Papworth Everard
🇬🇧Cambridge, United Kingdom
Western General Hospital
🇬🇧Edinburgh, United Kingdom
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
🇬🇧Exeter, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
Great Ormond Street Hospital for Sick Children
🇬🇧London, United Kingdom
Alder Hey Children's Alder Hey Children's NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
London and St Bartholomew's Hospital
🇬🇧London, United Kingdom
The University Hospital of South Manchester
🇬🇧Manchester, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
🇬🇧Newcastle Upon Tyne, United Kingdom
Nottingham University Hospitals NHS Trust, Queens Medical Center
🇬🇧Nottingham, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
🇬🇧Penarth, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Pneumologisches Studienzentrum Muenchen-West
🇩🇪Muenchen, Germany
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium