Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
- Registration Number
- NCT04058366
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study evaluated the long-term safety, efficacy, 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 heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 251
- Completed study drug treatment in parent study (VX18-445-104); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
Key
- History of study drug intolerance in parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX/TEZ/IVA ELX/TEZ/IVA Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. ELX/TEZ/IVA IVA Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
- Primary Outcome Measures
Name Time Method Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) From Baseline up to Week 100
- Secondary Outcome Measures
Name Time Method Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at Week 96 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.
Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) From Baseline at Week 96 Sweat samples were collected using an approved collection device.
Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) From Baseline at Week 96 BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
Part A: Absolute Change From Parent Study Baseline in BMI Z-score From Baseline at Week 96 BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Part A: Absolute Change From Parent Study Baseline in Body Weight From Baseline at Week 96 Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline at Week 96 FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Trial Locations
- Locations (84)
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
The University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
University Hospital Limerick (Adults)
🇮🇪Limerick, Ireland
University of North Carolina Hospitals
🇺🇸Chapel Hill, North Carolina, United States
Children's Health Ireland at Crumlin
🇮🇪Dublin, Ireland
St. Vincent's University Hospital
🇮🇪Dublin, Ireland
Mount Sinai Beth Israel
🇺🇸New York, New York, United States
Centre Hospitalier Lyon Sud
🇫🇷Benite Cedex, France
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
🇩🇪Giessen, Germany
The Prince Charles Hospital
🇦🇺Chermside, Australia
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
🇩🇪Halle, Germany
St. Paul's Hospital
🇨🇦Vancouver, Canada
Telethon Kids Institute
🇦🇺Nedlands, Australia
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
UC Health Holmes
🇺🇸Cincinnati, Ohio, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Dell Children's Medical Group
🇺🇸Austin, Texas, United States
CHU de Montpellier - Hopital Arnaud de Villeneuve
🇫🇷Montpellier Cedex 5, France
The Royal Children's Hospital
🇦🇺Parkville, Australia
Hopital Necker, Enfants Malades
🇫🇷Paris Cedex 15, France
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Alfred Hospital
🇦🇺Melbourne, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Beaumont Hospital
🇮🇪Dublin, Ireland
Groupe Hospitaler Pellegrin, CHU De Bordeaux
🇫🇷Bordeaux cedex, France
Azienda Ospedaliero Universitaria Ospedale Riuniti
🇮🇹Ancona, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Hospital Universitario y Politecnico La Fe
🇪🇸Valencia, Spain
Hospital Virgen de la Arrixaca
🇪🇸Murcia, Spain
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
🇬🇧London, United Kingdom
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
CHU Marseille - Hopital Nord
🇫🇷Marseille, France
Pneumologisches Studienzentrum Muenchen-West
🇩🇪Muenchen, Germany
University Hospital Wuerzburg
🇩🇪Würzburg, Germany
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
🇮🇹Genova, Italy
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
🇬🇧Exeter, United Kingdom
Academic Medical Center
🇳🇱Amsterdam, Netherlands
St. James University Hospital
🇬🇧Leeds, United Kingdom
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Juliane Marie Center, Rigshospitalet
🇩🇰Copenhagen, Denmark
McGill University Health Center
🇨🇦Montréal, Canada
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
Wythenshawe Hospital
🇬🇧Manchester, United Kingdom
Hopital Pontchaillou CHU de Rennes
🇫🇷Rennes Cedex, France
Stanford University
🇺🇸Palo Alto, California, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
🇺🇸Boston, Massachusetts, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Westmead Hospital
🇦🇺Westmead, Australia
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
CHRU de Lille - Hopital Albert Calmette
🇫🇷Lille, France
Ospedale Pediatrico Bambino Gesu
🇮🇹Rome, Italy
Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
🇮🇹Verona, Italy
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
🇳🇱Heidelberglaan, Netherlands
Papworth Hospital NHS Foundation Trust, Papworth Everard
🇬🇧Cambridge, United Kingdom
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
🇪🇸Barcelona, Spain
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Mater Adult Hospital
🇦🇺South Brisbane, Australia
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
🇺🇸Toledo, Ohio, United States
Hopital Cochin
🇫🇷Paris, France
National Jewish Health
🇺🇸Denver, Colorado, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
University of California San Francisco, Lung Transplant Program
🇺🇸San Francisco, California, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
University of Utah / Primary Children's Medical Center
🇺🇸Salt Lake City, Utah, United States
Cliniques Universitaires de Bruxelles Hopital Erasme
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪Brussels, Belgium
University of Kentucky
🇺🇸Lexington, Kentucky, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States