A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
- Registration Number
- NCT05444257
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 822
- Part A: Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period in the parent study
- Part B: Completed study drug treatment in Part A; or had study drug interruption(s) in Part A, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A
Key
- History of drug intolerance in a parent study
- Pregnant or breast-feeding females at the time of enrollment in Part A
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description VX-121/TEZ/D-IVA VX-121/TEZ/D-IVA Part A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks.
- Primary Outcome Measures
Name Time Method Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Baseline up to Week 148
- Secondary Outcome Measures
Name Time Method Absolute Change From Baseline in Sweat Chloride (SwCl) From Baseline up to Week 144 Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline up to Week 144 Number of Pulmonary Exacerbations (PEx) From Baseline up to Week 144
Trial Locations
- Locations (195)
Indiana University
🇺🇸Indianapolis, Indiana, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
St. Paul's Hospital
🇨🇦Vancouver, Canada
Erasmus Medical Center
🇳🇱Rotterdam, Netherlands
Nemours Children's Specialty Care
🇺🇸Pensacola, Florida, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
🇺🇸Boston, Massachusetts, United States
CF Therapeutics Development Center of Western New York
🇺🇸Buffalo, New York, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center
🇺🇸Hollywood, Florida, United States
The Emory Clinic at Chantilly
🇺🇸Atlanta, Georgia, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Columbia University Medical Center
🇺🇸New York, New York, 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
Cardinal Glennon Children's Hospital - St. Louis University
🇺🇸Saint Louis, Missouri, United States
SUNY Upstate Medical University
🇺🇸Syracuse, New York, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Augusta University
🇺🇸Augusta, Georgia, United States
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
🇺🇸Saint Petersburg, Florida, United States
McGill University Health Centre, Glen Site, Montreal Children's Hospital
🇨🇦Montreal, Canada
Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
🇨🇦Quebec, Canada
Texas Children's Hospital - Baylor College of Medicine
🇺🇸Houston, Texas, United States
Mater Adult Hospital
🇦🇺South Brisbane, Australia
Albany Medical College, Pulmonary and Critical Care Medicine
🇺🇸Albany, New York, United States
Aarhus University Hospital
🇩🇰Aarhus, Denmark
Centre Hospitalier Intercommunal Creteil
🇫🇷Creteil, France
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
🇺🇸Toledo, Ohio, United States
The Prince Charles Hospital
🇦🇺Chermside, Australia
The Royal Children's Hospital
🇦🇺Parkville, Australia
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
🇺🇸Spokane, Washington, United States
Hopital Cochin
🇫🇷Paris, France
Telethon Kids Institute
🇦🇺Nedlands, Australia
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Centre Hospitalier Lyon Sud
🇫🇷Pierre-Benite, France
Westmead Hospital
🇦🇺Westmead, Australia
Stollery Children's Hospital
🇨🇦Edmonton, Canada
Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
🇩🇪Gießen, Germany
Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin
🇩🇪Jena, Germany
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
🇨🇦Montreal, Canada
Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
🇩🇪Ulm, Germany
Klinika Detskych Infekcnich Nemoci
🇨🇿Brno, Czechia
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
CHU de Rouen - Hopital Charles Nicolle
🇫🇷Rouen, France
Hopital Robert Debre
🇫🇷Paris, France
University Hospital and UW Health Clinics
🇺🇸Madison, Wisconsin, United States
Alfred Hospital
🇦🇺Melbourne, Australia
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
🇩🇪Erlangen, Germany
St. Joseph's Health Care London
🇨🇦London, Canada
CHU Lyon - Hopital Femme Mere-Enfant
🇫🇷Bron Cedex, France
Institut Cœur Poumon, CHU de Lille
🇫🇷Lille, France
Johannes Gutenberg-Universitaet
🇩🇪Mainz, Germany
Hopital Foch (Suresnes), Hopital Foch, Adultes
🇫🇷Suresnes, France
Johann Wolfgang Goethe University
🇩🇪Frankfurt, Germany
Hopital Arnaud de Villeneuve
🇫🇷Montpellier Cedex 5, France
Hannover Medical School
🇩🇪Hannover, Germany
Hôpital de Hautepierre, AX5
🇫🇷Strasbourg, France
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
Pneumologisches Studienzentrum Muenchen-West
🇩🇪Muenchen, Germany
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Starship Children's Hospital
🇳🇿Grafton, New Zealand
Hospital Saint Joan de Deu
🇪🇸Barcelona, Spain
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden
Kinderspital Zuerich
🇨🇭Zurich, Switzerland
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Medizinische Universität Wien
🇦🇹Vienna, Austria
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
St. Luke's Cystic Fibrosis Center of Idaho
🇺🇸Boise, Idaho, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Maine Medical Partners
🇺🇸Portland, Maine, United States
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
Pulmonology Institute Torokbalint
🇭🇺Torokbalint, Hungary
Cork University Hospital
🇮🇪Cork, Ireland
Dartmouth Hitchcock
🇺🇸Manchester, New Hampshire, United States
Dayton Children's Hospital
🇺🇸Dayton, Ohio, United States
Billings Clinic
🇺🇸Billings, Montana, United States
University of Graz
🇦🇹Graz, Austria
General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki
🇬🇷Thessaloniki, Greece
St. Vincent's University Hospital
🇮🇪Dublin, Ireland
Pediatric Hospital Polanki named of Maciej Płażyński
🇵🇱Gdańsk, Poland
Hospital Sao Joao
🇵🇹Porto, Portugal
New York Medical College
🇺🇸Valhalla, New York, United States
National Koranyi Institute for TBC and Pulmonology
🇭🇺Budapest, Hungary
Vermont Lung Center
🇺🇸Colchester, Vermont, United States
Sanford Children's Speciality Clinic
🇺🇸Sioux Falls, South Dakota, United States
Prisma Health Richland Campus
🇺🇸Columbia, South Carolina, United States
The University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
West Virginia University
🇺🇸Morgantown, West Virginia, United States
Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde
🇦🇹Salzburg, Austria
Cliniques Universitaires de Bruxelles Hopital Erasme
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
Cliniques Universitaires Saint-Luc
🇧🇪Woluwe-Saint-Lambert, Belgium
Oslo University Hospital, Department of Paediatric Medicine
🇳🇴Oslo, Norway
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
🇵🇱Lomianki, Poland
Hospital de Santa Maria
🇵🇹Lisbon, Portugal
CHP - Hospital de Santo Antonio
🇵🇹Porto, Portugal
Schneider Children's Medical Center of Israel
🇮🇱Petach Tikvah, Israel
Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
🇮🇱Tel HaShomer, Israel
Academisch Medisch Centrum (Academic Medical Centre)
🇳🇱Amsterdam, Netherlands
Haukeland Universitetssjukehus (CF)
🇳🇴Bergen, Norway
General Hospital of Thessaloniki "Papanikolaou"
🇬🇷Thessaloniki, Greece
Children's Health Ireland at Crumlin
🇮🇪Dublin, Ireland
Children's Health Ireland at Temple Street
🇮🇪Dublin, Ireland
University Hospital Limerick (Pediatrics)
🇮🇪Limerick, Ireland
Hadassah Medical Organization
🇮🇱Jerusalem, Israel
Juliane Marie Center, Rigshospitalet
🇩🇰Copenhagen, Denmark
Azienda Ospedaliero Universitaria Ospedale Riuniti
🇮🇹Ancona, Italy
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
🇮🇹Firenze, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
🇮🇹Genova, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
🇮🇹Milan, Italy
Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
🇮🇹Orbassano, Italy
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
🇮🇹Potenza, Italy
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
🇮🇹Verona, Italy
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of California San Francisco, Lung Transplant Program
🇺🇸San Francisco, California, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
National Jewish Health
🇺🇸Denver, Colorado, 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
Vanderbilt University Medical Center, Nephrology Clinical Trials Center
🇺🇸Nashville, Tennessee, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Centre de Perharidy
🇫🇷Roscoff cedex, France
Hopital Bretonneau
🇫🇷Tours, France
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, United Kingdom
Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Cook Children's Health Care System
🇺🇸Fort Worth, Texas, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
Wake Forest Baptist Health
🇺🇸Winston-Salem, North Carolina, United States
Central Florida Pulmonary Group,PA
🇺🇸Orlando, Florida, United States
St. Bartholomew's Hospital
🇬🇧London, United Kingdom
University of Florida, Shands Hospital
🇺🇸Gainesville, Florida, United States
Kaiser Permanente
🇺🇸Oakland, California, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
UMass Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
Childrens Hospital University of Missouri Health Sciences Center
🇺🇸Columbia, Missouri, United States
Long Island Jewish Medical Center
🇺🇸New Hyde Park, New York, United States
Atrium Health Levine Children's Hospital
🇺🇸Charlotte, North Carolina, United States
University of Tennessee Medical Center
🇺🇸Knoxville, Tennessee, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
Royal Prince Alfred Hospital
🇦🇺Camperdown, Australia
Institute for Respiratory Health
🇦🇺Nedlands, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
University of Calgary Medical Clinic of the Foothills Medical Centre
🇨🇦Calgary, Canada
Groupe Hospitaler Pellegrin, CHU De Bordeaux
🇫🇷Bordeaux cedex, France
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
🇫🇷Nice, France
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Klinikum Westbrandenburg (CF)
🇩🇪Potsdam, Germany
Universitätsklinikum Würzburg
🇩🇪Würzburg, Germany
UMC St. Radboud
🇳🇱Nijmegen, Netherlands
HagaZiekenhuis van den Haag
🇳🇱Den Haag, Netherlands
Waikato Hospital
🇳🇿Hamilton, New Zealand
Canterbury District Health Board
🇳🇿Christchurch, New Zealand
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
🇳🇱Utrecht, Netherlands
Greenlane Clinical Centre
🇳🇿One Tree Hill, New Zealand
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Virgen de la Arrixaca
🇪🇸Murcia, Spain
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
🇪🇸Sabadell, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Lund University Skanes Universitetssjukhus
🇸🇪Lund, Sweden
Hospital Universitario y Politecnico La Fe
🇪🇸Valencia, Spain
Karolinska Universitetssjukhuset, Huddinge
🇸🇪Stockholm, Sweden
Royal Devon University Healthcare NHS Foundation Trust
🇬🇧Exeter, United Kingdom
Birmingham Heartlands Hospital
🇬🇧Birmingham, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
Leeds General Infirmary
🇬🇧Leeds, United Kingdom
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
🇬🇧Bristol, United Kingdom
Royal Papworth Hospital NHS Foundation Trust
🇬🇧Cambridge, United Kingdom
Western General Hospital
🇬🇧Edinburgh, United Kingdom
St. James University Hospital
🇬🇧Leeds, United Kingdom
King's College Hospital
🇬🇧London, United Kingdom
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
Wythenshawe Hospital
🇬🇧Manchester, United Kingdom
Clinical Research Facility
🇬🇧Newcastle Upon Tyne, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
🇬🇧Penarth, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Hospital Universitario Ramon y Cajal
🇪🇸Madrid, Spain
Tulane Medical Center
🇺🇸New Orleans, Louisiana, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
Royal Brompton Hospital
🇬🇧London, United Kingdom
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Medizinische Universität Innsbruck
🇦🇹Innsbruck, Austria