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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05444257
Other study ID # VX20-121-104
Secondary ID 2021-000713-17
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date November 8, 2022
Est. completion date October 2025

Study information

Verified date December 2023
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 822
Est. completion date October 2025
Est. primary completion date October 2025
Accepts healthy volunteers No
Gender All
Age group 12 Years and older
Eligibility Key Inclusion Criteria: - 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 Key Exclusion Criteria: - History of drug intolerance in a parent study - Pregnant or breast-feeding females Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.

Locations

Country Name City State
Australia 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
Austria University of Graz Graz
Austria Medizinische Universität Innsbruck Innsbruck
Austria Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde Salzburg
Austria Medizinische Universität Wien Vienna
Belgium Cliniques Universitaires de Bruxelles Hopital Erasme Brussels
Belgium Universitair Ziekenhuis Brussel - Campus Jette Brussels
Belgium Universitair Ziekenhuis Gent Gent
Belgium Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg Leuven
Belgium Cliniques Universitaires Saint-Luc Woluwe-Saint-Lambert
Canada 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 Quebec
Canada British Columbia Children's Hospital Vancouver
Canada St. Paul's Hospital Vancouver
Czechia Klinika Detskych Infekcnich Nemoci Brno
Czechia Fakultni nemocnice v Motole Praha 5
Denmark Aarhus University Hospital Aarhus
Denmark Juliane Marie Center, Rigshospitalet Copenhagen
France Groupe Hospitaler Pellegrin, CHU De Bordeaux Bordeaux cedex
France CHU Lyon - Hopital Femme Mere-Enfant Bron Cedex
France Centre Hospitalier Intercommunal Creteil Creteil
France Institut Cœur Poumon, CHU de Lille Lille
France Hopital Arnaud de Villeneuve Montpellier Cedex 5
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-Benite
France Centre de Perharidy Roscoff cedex
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
Germany 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 Hannover
Germany Medizinische Hochschule Hannover Hannover
Germany Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin Jena
Germany Johannes Gutenberg-Universitaet Mainz
Germany Pneumologisches Studienzentrum Muenchen-West Muenchen
Germany Klinikum Westbrandenburg (CF) Potsdam
Germany Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF) Ulm
Germany Universitätsklinikum Würzburg Würzburg
Greece General Hospital of Thessaloniki "Papanikolaou" Thessaloniki
Greece General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki Thessaloniki
Hungary National Koranyi Institute for TBC and Pulmonology Budapest
Hungary Pulmonology Institute Torokbalint Torokbalint
Ireland 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
Israel 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 HaShomer
Italy 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 Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena Milan
Italy Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano
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
Netherlands Academisch Medisch Centrum (Academic Medical Centre) Amsterdam
Netherlands HagaZiekenhuis van den Haag Den Haag
Netherlands UMC St. Radboud Nijmegen
Netherlands Erasmus Medical Center Rotterdam
Netherlands University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis Utrecht
New Zealand 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
Norway Haukeland Universitetssjukehus (CF) Bergen
Norway Oslo University Hospital, Department of Paediatric Medicine Oslo
Poland Pediatric Hospital Polanki named of Maciej Plazynski Gdansk
Poland Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY Lomianki
Portugal Hospital de Santa Maria Lisbon
Portugal CHP - Hospital de Santo Antonio Porto
Portugal Hospital Sao Joao Porto
Spain 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 Sevilla
Spain Hospital Universitario y Politecnico La Fe Valencia
Sweden Sahlgrenska Universitetssjukhuset Göteborg
Sweden Lund University Skanes Universitetssjukhus Lund
Sweden Karolinska Universitetssjukhuset, Huddinge Stockholm
Switzerland Kinderspital Zuerich Zurich
United Kingdom 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 States Akron Children's Hospital Akron Ohio
United States Albany Medical College, Pulmonary and Critical Care Medicine Albany New York
United States Michigan Medicine Ann Arbor Michigan
United States The Emory Clinic at Chantilly Atlanta Georgia
United States Augusta University Augusta Georgia
United States Children's Hospital of Colorado Aurora Colorado
United States Billings Clinic Billings Montana
United States University of Alabama at Birmingham Birmingham Alabama
United States St. Luke's Cystic Fibrosis Center of Idaho Boise Idaho
United States Boston Children's Hospital Boston Massachusetts
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 Atrium Health Levine Children's Hospital Charlotte North Carolina
United States University of Virginia Health System Charlottesville Virginia
United States Northwestern Memorial Hospital Chicago Illinois
United States Vermont Lung Center Colchester Vermont
United States Childrens Hospital University of Missouri Health Sciences Center Columbia Missouri
United States Prisma Health Richland Campus Columbia South Carolina
United States Nationwide Children's Hospital Columbus Ohio
United States The University of Texas Southwestern Medical Center Dallas Texas
United States Dayton Children's Hospital Dayton Ohio
United States National Jewish Health Denver Colorado
United States Harper University Hospital Detroit Michigan
United States Cook Children's Health Care System Fort Worth Texas
United States University of Florida, Shands Hospital Gainesville Florida
United States Spectrum Health Medical Group Adult Cystic Fibrosis Care Center Grand Rapids Michigan
United States Joe DiMaggio Cystic Fibrosis & Pulmonary Center Hollywood Florida
United States Texas Children's Hospital - Baylor College of Medicine Houston Texas
United States Indiana University Indianapolis Indiana
United States Riley Hospital for Children at Indiana University Health Indianapolis Indiana
United States The Children's Mercy Hospital Kansas City Missouri
United States University of Kansas Medical Center Kansas City Kansas
United States University of Tennessee Medical Center Knoxville Tennessee
United States University of Kentucky Lexington Kentucky
United States University of Arkansas for Medical Sciences Little Rock Arkansas
United States Miller Children's Hospital / Long Beach Memorial Long Beach California
United States Children's Hospital Los Angeles Los Angeles California
United States University Hospital and UW Health Clinics Madison Wisconsin
United States Dartmouth Hitchcock Manchester New Hampshire
United States University of Miami Miller School of Medicine Miami Florida
United States University of Minnesota Minneapolis Minnesota
United States West Virginia University Morgantown West Virginia
United States Morristown Medical Center Morristown New Jersey
United States Vanderbilt University Medical Center, Nephrology Clinical Trials Center Nashville Tennessee
United States Long Island Jewish Medical Center New Hyde Park New York
United States Tulane Medical Center New Orleans Louisiana
United States Columbia University Medical Center New York New York
United States Kaiser Permanente Oakland California
United States Santiago Reyes, M.D. Oklahoma City Oklahoma
United States University of Oklahoma Health Sciences Center Oklahoma City Oklahoma
United States Nebraska Medical Center Omaha Nebraska
United States Central Florida Pulmonary Group,PA Orlando Florida
United States Nemours Children's Specialty Care Pensacola Florida
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
United States Maine Medical Partners Portland Maine
United States Oregon Health & Science University Portland Oregon
United States University of California Davis Medical Center Sacramento California
United States Cardinal Glennon Children's Hospital - St. Louis University Saint Louis Missouri
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 University of Utah Salt Lake City Utah
United States University of California San Francisco, Lung Transplant Program San Francisco California
United States University of Washington Medical Center Seattle Washington
United States Sanford Children's Speciality Clinic Sioux Falls South Dakota
United States Providence Pediatric Pulmonary & Cystic Fibrosis Clinic Spokane Washington
United States SUNY Upstate Medical University Syracuse New York
United States ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center Toledo Ohio
United States Banner University of Arizona Medical Center Tucson Arizona
United States New York Medical College Valhalla New York
United States Wake Forest Baptist Health Winston-Salem North Carolina
United States UMass Memorial Medical Center Worcester Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Belgium,  Canada,  Czechia,  Denmark,  France,  Germany,  Greece,  Hungary,  Ireland,  Israel,  Italy,  Netherlands,  New Zealand,  Norway,  Poland,  Portugal,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Day 1 up to Week 100
Secondary Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline up to Week 96
Secondary Absolute Change From Baseline in Sweat Chloride (SwCl) From Baseline up to Week 96
Secondary Number of Pulmonary Exacerbations (PEx) From Baseline up to Week 96
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