A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121/TEZ/D-IVA Combination Therapy in Subjects With Cystic Fibrosis
3 other identifiers
interventional
822
22 countries
186
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Nov 2022
Typical duration for phase_3
186 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 29, 2022
CompletedFirst Posted
Study publicly available on registry
July 5, 2022
CompletedStudy Start
First participant enrolled
November 8, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 30, 2026
May 5, 2026
April 1, 2026
4 years
June 29, 2022
April 28, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (3)
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline up to Week 144
Absolute Change From Baseline in Sweat Chloride (SwCl)
From Baseline up to Week 144
Number of Pulmonary Exacerbations (PEx)
From Baseline up to Week 144
Study Arms (1)
VX-121/TEZ/D-IVA
EXPERIMENTALPart 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.
Interventions
Fixed-dose combination tablets for oral administration.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- History of drug intolerance in a parent study
- Pregnant or breast-feeding females at the time of enrollment in Part A
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (195)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Banner University of Arizona Medical Center
Tucson, Arizona, 85724, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, 90806, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Kaiser Permanente
Oakland, California, 94611, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
University of California San Francisco, Lung Transplant Program
San Francisco, California, 94143, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Florida, Shands Hospital
Gainesville, Florida, 32610, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center
Hollywood, Florida, 33021, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Central Florida Pulmonary Group,PA
Orlando, Florida, 32803, United States
Nemours Children's Specialty Care
Pensacola, Florida, 32514, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, 33701, United States
The Emory Clinic at Chantilly
Atlanta, Georgia, 30324, United States
Augusta University
Augusta, Georgia, 30912, United States
St. Luke's Cystic Fibrosis Center of Idaho
Boise, Idaho, 83702, United States
Northwestern Memorial Hospital
Chicago, Illinois, 60611, United States
Indiana University
Indianapolis, Indiana, 46202, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Kentucky
Lexington, Kentucky, 40508, United States
Tulane Medical Center
New Orleans, Louisiana, 70112, United States
Maine Medical Partners
Portland, Maine, 04102, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
UMass Memorial Medical Center
Worcester, Massachusetts, 01655, United States
Michigan Medicine
Ann Arbor, Michigan, 48109, United States
Harper University Hospital
Detroit, Michigan, 48201, United States
Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
Grand Rapids, Michigan, 49546, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Childrens Hospital University of Missouri Health Sciences Center
Columbia, Missouri, 65212, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, 63104, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Billings Clinic
Billings, Montana, 59101, United States
Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Dartmouth Hitchcock
Manchester, New Hampshire, 03104, United States
Morristown Medical Center
Morristown, New Jersey, 07960, United States
Albany Medical College, Pulmonary and Critical Care Medicine
Albany, New York, 12208, United States
CF Therapeutics Development Center of Western New York
Buffalo, New York, 14203, United States
Long Island Jewish Medical Center
New Hyde Park, New York, 11040, United States
Columbia University Medical Center
New York, New York, 10032, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
New York Medical College
Valhalla, New York, 10595, United States
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, 28207, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27104, United States
Akron Children's Hospital
Akron, Ohio, 44308, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Dayton Children's Hospital
Dayton, Ohio, 45404, United States
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio, 43606, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Santiago Reyes, M.D.
Oklahoma City, Oklahoma, 73112, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Prisma Health Richland Campus
Columbia, South Carolina, 29203, United States
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, 57105, United States
University of Tennessee Medical Center
Knoxville, Tennessee, 37920, United States
Vanderbilt University Medical Center, Nephrology Clinical Trials Center
Nashville, Tennessee, 37232, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Cook Children's Health Care System
Fort Worth, Texas, 76104, United States
Texas Children's Hospital - Baylor College of Medicine
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Vermont Lung Center
Colchester, Vermont, 05446, United States
University of Virginia Health System
Charlottesville, Virginia, 22908, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
Spokane, Washington, 99204, United States
West Virginia University
Morgantown, West Virginia, 26506, United States
University Hospital and UW Health Clinics
Madison, Wisconsin, 53792, United States
Royal Prince Alfred Hospital
Camperdown, Australia
The Prince Charles Hospital
Chermside, Australia
Alfred Hospital
Melbourne, Australia
Institute for Respiratory Health
Nedlands, Australia
Telethon Kids Institute
Nedlands, Australia
The Royal Children's Hospital
Parkville, Australia
Mater Adult Hospital
South Brisbane, Australia
Queensland Children's Hospital
South Brisbane, Australia
Westmead Hospital
Westmead, Australia
University of Graz
Graz, Austria
Medizinische Universität Innsbruck
Innsbruck, Austria
Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde
Salzburg, Austria
Medizinische Universität Wien
Vienna, Austria
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, Belgium
Universitair Ziekenhuis Gent
Ghent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary, Canada
Stollery Children's Hospital
Edmonton, 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
Montreal, Canada
Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
Québec, Canada
British Columbia Children's Hospital
Vancouver, Canada
St. Paul's Hospital
Vancouver, Canada
Klinika Detskych Infekcnich Nemoci
Brno, Czechia
Fakultni nemocnice v Motole
Prague, Czechia
Aarhus University Hospital
Aarhus, Denmark
Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux, France
CHU Lyon - Hopital Femme Mere-Enfant
Bron, France
Centre Hospitalier Intercommunal Creteil
Créteil, France
Institut Cœur Poumon, CHU de Lille
Lille, France
Hopital Arnaud de Villeneuve
Montpellier, France
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
Nice, France
Hopital Cochin
Paris, France
Hopital Robert Debre
Paris, France
Centre Hospitalier Lyon Sud
Pierre-Bénite, France
Centre de Perharidy
Roscoff, France
CHU de Rouen - Hopital Charles Nicolle
Rouen, France
Hôpital de Hautepierre, AX5
Strasbourg, France
Hopital Foch (Suresnes), Hopital Foch, Adultes
Suresnes, France
Hopital Bretonneau
Tours, France
Charite Paediatric Pulmonology Department
Berlin, Germany
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen, Germany
Kinderklinik III, Abt. fur Pneumologie
Essen, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Johann Wolfgang Goethe University
Frankfurt, Germany
Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
Giessen, Germany
Hannover Medical School
Hanover, Germany
Medizinische Hochschule Hannover
Hanover, Germany
Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin
Jena, Germany
Johannes Gutenberg-Universitaet
Mainz, Germany
Pneumologisches Studienzentrum Muenchen-West
München, Germany
Klinikum Westbrandenburg (CF)
Potsdam, Germany
Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
Ulm, Germany
Universitätsklinikum Würzburg
Würzburg, Germany
General Hospital of Thessaloniki "Papanikolaou"
Thessaloniki, Greece
General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki
Thessaloniki, Greece
National Koranyi Institute for TBC and Pulmonology
Budapest, Hungary
Pulmonology Institute Torokbalint
Törökbálint, Hungary
Cork University Hospital
Cork, Ireland
Children's Health Ireland at Crumlin
Dublin, Ireland
Children's Health Ireland at Temple Street
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
University Hospital Limerick (Pediatrics)
Limerick, Ireland
Hadassah Medical Organization
Jerusalem, Israel
Schneider Children's Medical Center of Israel
Petach Tikvah, Israel
Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
Tel Litwinsky, Israel
Azienda Ospedaliero Universitaria Ospedale Riuniti
Ancona, Italy
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Florence, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova, Italy
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
Milan, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
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
Academisch Medisch Centrum (Academic Medical Centre)
Amsterdam, Netherlands
UMC St. Radboud
Nijmegen, Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
HagaZiekenhuis van den Haag
The Hague, Netherlands
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
Utrecht, Netherlands
Canterbury District Health Board
Christchurch, New Zealand
Starship Children's Hospital
Grafton, New Zealand
Waikato Hospital
Hamilton, New Zealand
Greenlane Clinical Centre
One Tree Hill, New Zealand
Haukeland Universitetssjukehus (CF)
Bergen, Norway
Oslo University Hospital, Department of Paediatric Medicine
Oslo, Norway
Pediatric Hospital Polanki named of Maciej Płażyński
Gdansk, Poland
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Łomianki, Poland
Hospital de Santa Maria
Lisbon, Portugal
CHP - Hospital de Santo Antonio
Porto, Portugal
Hospital Sao Joao
Porto, Portugal
Hospital Saint Joan de Deu
Barcelona, Spain
Hospital Universitari Vall d'Hebron
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitario Ramon y Cajal
Madrid, Spain
Hospital Virgen de la Arrixaca
Murcia, Spain
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
Sabadell, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Hospital Universitario y Politecnico La Fe
Valencia, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden
Lund University Skanes Universitetssjukhus
Lund, Sweden
Karolinska Universitetssjukhuset, Huddinge
Stockholm, Sweden
Kinderspital Zuerich
Zurich, Switzerland
Birmingham Heartlands Hospital
Birmingham, 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
Royal Devon University Healthcare NHS Foundation Trust
Exeter, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
Leeds General Infirmary
Leeds, United Kingdom
St. James University Hospital
Leeds, United Kingdom
Liverpool Heart and Chest Hospital
Liverpool, United Kingdom
King's College Hospital
London, United Kingdom
Royal Brompton Hospital
London, United Kingdom
St. Bartholomew's 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
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 29, 2022
First Posted
July 5, 2022
Study Start
November 8, 2022
Primary Completion (Estimated)
October 30, 2026
Study Completion (Estimated)
October 30, 2026
Last Updated
May 5, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing