A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
- Registration Number
- NCT05033080
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 435
- Heterozygous for F508del and a minimal function mutation (F/MF genotype)
- Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 >=40% and <=80% for participants not currently receiving ELX/TEZ/IVA
Key
- History of solid organ or hematological transplantation
- Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Pregnant or breast-feeding females
Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ELX/TEZ/IVA ELX/TEZ/IVA Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks. ELX/TEZ/IVA IVA Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks. ELX/TEZ/IVA Placebo (matched to VX-121/TEZ/D-IVA) Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks. VX-121/TEZ/D-IVA VX-121/TEZ/D-IVA Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks. VX-121/TEZ/D-IVA Placebo (matched to ELX/TEZ/IVA) Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks. VX-121/TEZ/D-IVA Placebo (matched to IVA) Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
- Primary Outcome Measures
Name Time Method 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.
- Secondary Outcome Measures
Name Time Method Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Trial Locations
- Locations (139)
Childrens Hospital University of Missouri Health Sciences Center
🇺🇸Columbia, Missouri, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center
🇺🇸Hollywood, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Cleveland Clinic CF
🇺🇸Cleveland, Ohio, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
🇺🇸Boston, Massachusetts, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
Texas Children's Hospital - Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of California San Francisco, Lung Transplant Program
🇺🇸San Francisco, California, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
University of Utah - Primary Children's Medical Center
🇺🇸Salt Lake City, Utah, United States
Dayton Children's Hospital
🇺🇸Dayton, Ohio, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Billings Clinic
🇺🇸Billings, Montana, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
New York Medical College
🇺🇸Valhalla, New York, United States
Sanford Children's Speciality Clinic
🇺🇸Sioux Falls, South Dakota, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
Tulane Medical Center
🇺🇸New Orleans, Louisiana, United States
University of Florida, Shands Hospital
🇺🇸Gainesville, Florida, United States
Driscoll Children's Hospital
🇺🇸Corpus Christi, Texas, United States
The University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Cook Children's Health Care System
🇺🇸Fort Worth, Texas, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Kaiser Permanente
🇺🇸Oakland, California, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
🇺🇸Saint Petersburg, Florida, United States
St. Luke's Cystic Fibrosis Center of Idaho
🇺🇸Boise, Idaho, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Helen DeVos Children's Hospital CF Center
🇺🇸Grand Rapids, Michigan, United States
UMass Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
Dartmouth Hitchcock, Manchester
🇺🇸Manchester, New Hampshire, United States
CF Therapeutics Development Center of Western New York
🇺🇸Buffalo, New York, United States
Atrium Health Levine Children's Hospital
🇺🇸Charlotte, North Carolina, United States
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
🇺🇸Toledo, Ohio, United States
University of Tennessee Medical Center
🇺🇸Knoxville, Tennessee, United States
Children's Foundation Research Center / Le Bonheur Children's Hospital
🇺🇸Memphis, Tennessee, United States
Inova Fairfax
🇺🇸Falls Church, Virginia, United States
Royal Prince Alfred Hospital
🇦🇺Camperdown, Australia
Alfred Hospital
🇦🇺Melbourne, VIC, Australia
The Prince Charles Hospital
🇦🇺Chermside, Australia
Mater Adult Hospital
🇦🇺South Brisbane, Australia
Westmead Hospital
🇦🇺Westmead, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Klinika Detskych Infekcnich Nemoci
🇨🇿Brno, Czechia
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
St. Josef-Hospital
🇩🇪Bochum, Germany
Johann Wolfgang Goethe University
🇩🇪Frankfurt, Germany
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
🇩🇪Erlangen, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Hannover Medical School
🇩🇪Hannover, Germany
Pneumologisches Studienzentrum Muenchen-West
🇩🇪Muenchen, Germany
Klinikum Westbrandenburg (CF)
🇩🇪Potsdam, Germany
Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin
🇩🇪Jena, Germany
Johannes Gutenberg-Universitaet
🇩🇪Mainz, Germany
National Koranyi Institute for TBC and Pulmonology
🇭🇺Budapest, Hungary
Klinikum Innenstadt, University of Munich
🇩🇪München, Germany
Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
🇩🇪Ulm, Germany
Universitätsklinikum Würzburg
🇩🇪Würzburg, Germany
Cork University Hospital
🇮🇪Cork, Ireland
Children's Health Ireland at Tallaght
🇮🇪Dublin, Ireland
Pulmonology Institute Torokbalint
🇭🇺Torokbalint, Hungary
Children's Health Ireland at Temple Street
🇮🇪Dublin, Ireland
Children's Health Ireland at Crumlin
🇮🇪Dublin, Ireland
Hadassah University Hospital Mount Scopus
🇮🇱Jerusalem, Israel
St. Vincent's University Hospital
🇮🇪Dublin, Ireland
University Hospital Limerick (Adults)
🇮🇪Limerick, Ireland
University Hospital Limerick (Pediatrics)
🇮🇪Limerick, Ireland
Schneider Children's Medical Center of Israel
🇮🇱Petach Tikvah, Israel
Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
🇮🇱Tel HaShomer, Israel
Starship Children's Hospital
🇳🇿Auckland, New Zealand
Canterbury Respiratory Research Group
🇳🇿Christchurch Hospital, New Zealand
Greenlane Clinical Centre
🇳🇿Auckland, New Zealand
CHP - Hospital de Santo Antonio
🇵🇹Porto, Portugal
Waikato Hospital
🇳🇿Hamilton, New Zealand
Hospital Sao Joao
🇵🇹Porto, Portugal
Hospital Saint Joan de Deu
🇪🇸Barcelona, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital de Santa Maria
🇵🇹Lisbon, Portugal
Hospital Universitari Vall d Hebron
🇪🇸Barcelona, Spain
Hospital Universitario Ramon y Cajal
🇪🇸Madrid, Spain
Karolinska Universitetssjukhuset, Huddinge
🇸🇪Stockholm, Sweden
Birmingham Heartlands Hospital
🇬🇧Birmingham, United Kingdom
Hospital Virgen de la Arrixaca
🇪🇸Murcia, Spain
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
🇬🇧Bristol, United Kingdom
Lund University Skanes Universitetssjukhus
🇸🇪Malmoe, Sweden
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
🇪🇸Sabadell, Spain
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden
Hospital Universitario y Politecnico La Fe
🇪🇸Valencia, Spain
Royal Devon and Exeter Hospital
🇬🇧Exeter, United Kingdom
St. James University Hospital
🇬🇧Leeds, United Kingdom
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
Wythenshawe Hospital
🇬🇧Manchester, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
🇬🇧Penarth, United Kingdom
St. Bartholomew's Hospital
🇬🇧London, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Vermont Lung Center
🇺🇸Colchester, Vermont, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Augusta University
🇺🇸Augusta, Georgia, United States
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Albany Medical College
🇺🇸Albany, New York, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Royal Papworth Hospital NHS Foundation Trust
🇬🇧Cambridge, United Kingdom
Royal Hospital for Sick Children
🇬🇧Edinburgh, United Kingdom
Clinical Research Facility
🇬🇧Newcastle Upon Tyne, United Kingdom
The Emory Clinic at Chantilly
🇺🇸Atlanta, Georgia, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Northwell Health- Long Island Jewish Medical Center
🇺🇸New Hyde Park, New York, United States
Cardinal Glennon Children's Hospital - St. Louis University
🇺🇸Saint Louis, Missouri, United States
King's College Hospital
🇬🇧London, United Kingdom
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
Western General Hospital
🇬🇧Edinburgh, United Kingdom
Hospital Infantil Universitario Nino Jesus
🇪🇸Madrid, Spain
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
Nemours Children's Hospital
🇺🇸Orlando, Florida, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
Kosair Charities Pediatric Clinical Research Unit
🇺🇸Louisville, Kentucky, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Wake Forest Baptist Health
🇺🇸Winston-Salem, North Carolina, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Royal Brompton Hospital
🇬🇧London, United Kingdom
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
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