A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
- Registration Number
- NCT05076149
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del muta...
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 597
-
Participant has one of the following genotypes:
- Homozygous for F508del;
- Heterozygous for F508del and a gating (F/G) mutation;
- Heterozygous for F508del and a residual function (F/RF) mutation;
- At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation
-
Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy
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 1second (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 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 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Trial Locations
- Locations (170)
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
Tulane Medical Center
🇺🇸New Orleans, Louisiana, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
Pediatric Hospital Polanki named of Maciej Płażyński
🇵🇱Gdańsk, Poland
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Abteilung fuer Pulmologie Klinikum Wels-Grieskirchen
🇦🇹Wels, Austria
St. Vincent's University Hospital
🇮🇪Dublin, Ireland
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Dayton Children's Hospital
🇺🇸Dayton, Ohio, United States
Cork University Hospital
🇮🇪Cork, Ireland
Medizinische Universität Wien
🇦🇹Vienna, Austria
The University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
University Hospital Limerick (Pediatrics)
🇮🇪Limerick, Ireland
Juliane Marie Center, Rigshospitalet
🇩🇰Copenhagen, Denmark
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
Harper University Hospital
🇺🇸Detroit, Michigan, United States
Santiago Reyes, M.D.
🇺🇸Oklahoma City, Oklahoma, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
Nemours Children's Specialty Care, Pensacola
🇺🇸Pensacola, Florida, United States
St. Luke's Cystic Fibrosis Center of Idaho
🇺🇸Boise, Idaho, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
🇺🇸Boston, Massachusetts, United States
CF Therapeutics Development Center of Western New York
🇺🇸Buffalo, New York, United States
Billings Clinic
🇺🇸Billings, Montana, United States
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
🇺🇸Toledo, Ohio, United States
SUNY Upstate Medical University
🇺🇸Syracuse, New York, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
University of Tennessee Medical Center
🇺🇸Knoxville, Tennessee, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
Royal Prince Alfred Hospital
🇦🇺Camperdown, Australia
Institute for Respiratory Health
🇦🇺Nedlands, Australia
Telethon Kids Institute
🇦🇺Nedlands, Australia
The Royal Children's Hospital
🇦🇺Parkville, VIC, Australia
Sydney Children's Hospital
🇦🇺Randwick, Australia
Mater Adult Hospital
🇦🇺South Brisbane, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Westmead Hospital
🇦🇺Westmead, Australia
Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde
🇦🇹Salzburg, Austria
University of Graz
🇦🇹Graz, Austria
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪Brussel, Belgium
Cliniques Universitaires de Bruxelles Hopital Erasme
🇧🇪Brussel, Belgium
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
Cliniques Universitaires Saint-Luc
🇧🇪Woluwe-Saint-Lambert, Belgium
Stollery Children's Hospital
🇨🇦Edmonton, Canada
University of Calgary Medical Clinic of the Foothills Medical Centre
🇨🇦Calgary, Canada
St. Joseph's Health Care London
🇨🇦London, Canada
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
🇨🇦Montreal, Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital
🇨🇦Montréal, Canada
Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
🇨🇦Québec, Canada
St. Michael's Hospital
🇨🇦Toronto, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
St. Paul's Hospital
🇨🇦Vancouver, Canada
Aarhus University Hospital
🇩🇰Aarhus, Denmark
Groupe Hospitaler Pellegrin, CHU De Bordeaux
🇫🇷Bordeaux cedex, France
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
🇫🇷Nice, France
CHU Lyon - Hopital Femme Mere-Enfant
🇫🇷Bron Cedex, France
Centre Hospitalier Intercommunal Creteil
🇫🇷Créteil, France
Institut Cœur Poumon, CHU de Lille
🇫🇷Lille, France
Hopital Cochin
🇫🇷Paris, France
Hopital Robert Debre
🇫🇷Paris, France
Hopital Necker, Enfants Malades
🇫🇷Paris Cedex 15, France
Centre Hospitalier Lyon Sud
🇫🇷Pierre-Bénite, France
Centre de Perharidy
🇫🇷Roscoff cedex, France
CHU de Rouen - Hopital Charles Nicolle
🇫🇷Rouen Cedex, Seine Maritime, France
Hôpital de Hautepierre, AX5
🇫🇷Strasbourg, France
Hopital Foch (Suresnes), Hopital Foch, Adultes
🇫🇷Suresnes, France
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Hopital Bretonneau
🇫🇷Tours, France
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
🇩🇪Gießen, Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki
🇬🇷Thessaloníki, Greece
National Koranyi Institute for TBC and Pulmonology
🇭🇺Budapest, Hungary
General Hospital of Thessaloniki "Papanikolaou"
🇬🇷Pilea Chortiatis, Greece
Children's Health Ireland at Crumlin
🇮🇪Dublin, Ireland
Pulmonology Institute Torokbalint
🇭🇺Torokbalint, Hungary
Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
🇮🇱Tel HaShomer, Israel
Schneider Children's Medical Center of Israel
🇮🇱Petach Tikvah, Israel
Children's University Hospital, Dublin
🇮🇪Dublin, Ireland
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Azienda Ospedaliero Universitaria Ospedale Riuniti
🇮🇹Ancona, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
🇮🇹Genova, Italy
Academisch Medisch Centrum (Academic Medical Centre)
🇳🇱Amsterdam, Netherlands
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
🇮🇹Firenze, Italy
UMC St. Radboud
🇳🇱Nijmegen, Netherlands
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
🇮🇹Potenza, Italy
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
🇮🇹Verona, Italy
Canterbury Respiratory Research Group
🇳🇿Christchurch Hospital, New Zealand
Oslo University Hospital, Department of Paediatric Medicine
🇳🇴Oslo, Norway
Waikato Hospital
🇳🇿Hamilton, New Zealand
Institute of Tuberculosis and Lung Diseases
🇵🇱Rabka Zdrój, Poland
Haukeland Universitetssjukehus (CF)
🇳🇴Bergen, Norway
Birmingham Children's Hospital
🇬🇧Birmingham, United Kingdom
St. James University Hospital
🇬🇧Leeds, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
Leeds General Infirmary
🇬🇧Leeds, United Kingdom
King's College Hospital
🇬🇧London, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
🇬🇧Penarth, United Kingdom
Cook Children's Health Care System
🇺🇸Fort Worth, Texas, United States
University Hospital and UW Health Clinics
🇺🇸Madison, Wisconsin, United States
West Virginia University
🇺🇸Morgantown, West Virginia, United States
UMass Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
🇺🇸Saint Petersburg, Florida, United States
The Emory Clinic at Chantilly
🇺🇸Atlanta, Georgia, United States
Maine Medical Partners
🇺🇸Portland, Maine, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Sanford Children's Speciality Clinic
🇺🇸Sioux Falls, South Dakota, United States
University of Utah - Primary Children's Medical Center
🇺🇸Salt Lake City, Utah, United States
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
🇺🇸Spokane, Washington, United States
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
Hopital Arnaud de Villeneuve
🇫🇷Montpellier Cedex 5, France
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
🇩🇪Erlangen, Germany
Children's Health Ireland at Tallaght
🇮🇪Dublin, Ireland
Hadassah Medical Organization
🇮🇱Jerusalem, Israel
University Hospital Limerick (Adults)
🇮🇪Limerick, Ireland
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
🇮🇹Milan, Italy
Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
🇮🇹Orbassano, Italy
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
🇳🇱Utrecht, Netherlands
HagaZiekenhuis van den Haag
🇳🇱Den Haag, Netherlands
Erasmus Medical Center
🇳🇱Rotterdam, Netherlands
Greenlane Clinical Centre
🇳🇿Auckland, New Zealand
Karolinska Universitetssjukhuset, Huddinge
🇸🇪Stockholm, Sweden
Lund University Skanes Universitetssjukhus
🇸🇪Malmoe, Sweden
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden
Réseau Hospitalier Neuchâtelois
🇨🇭Neuchâtel, Switzerland
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
🇵🇱Łomianki, Poland
Kinderspital Zuerich
🇨🇭Zürich, Switzerland
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
Lindenhofspital - Quartier Bleu
🇨🇭Bern, Switzerland
Nemours Children's Specialty Care
🇺🇸Jacksonville, Florida, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
🇺🇸Grand Rapids, Michigan, United States
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
🇫🇷Nantes, France
St. Bartholomew's Hospital
🇬🇧London, United Kingdom
Prisma Health Richland Campus
🇺🇸Columbia, South Carolina, United States
Northwell Health- Long Island Jewish Medical Center
🇺🇸New Hyde Park, New York, United States
Alfred Hospital
🇦🇺Melbourne, VIC, Australia
Centre hospitalier universitaire de Nancy, Hôpital de Braboi
🇫🇷Vandoeuvre Les Nancy, France
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Vermont Lung Center
🇺🇸Colchester, Vermont, United States
The Prince Charles Hospital
🇦🇺Chermside, Australia
Pneumologisches Studienzentrum Muenchen-West
🇩🇪Muenchen, Germany
Royal Papworth Hospital NHS Foundation Trust
🇬🇧Cambridge, United Kingdom
Starship Children's Hospital
🇳🇿Auckland, New Zealand
Wythenshawe Hospital
🇬🇧Manchester, United Kingdom
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Wake Forest Baptist Health
🇺🇸Winston-Salem, North Carolina, United States
Royal Brompton Hospital
🇬🇧London, United Kingdom
Medizinische Universität Innsbruck
🇦🇹Innsbruck, Austria
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