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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT05076149
Other study ID # VX20-121-103
Secondary ID 2021-000694-85
Status Completed
Phase Phase 3
First received
Last updated
Start date October 27, 2021
Est. completion date November 30, 2023

Study information

Verified date May 2024
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 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 mutation.


Recruitment information / eligibility

Status Completed
Enrollment 597
Est. completion date November 30, 2023
Est. primary completion date May 18, 2023
Accepts healthy volunteers No
Gender All
Age group 12 Years and older
Eligibility Key Inclusion Criteria: - 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 Exclusion Criteria: - 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


Related Conditions & MeSH terms


Intervention

Drug:
VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.
ELX/TEZ/IVA
Fixed-dose combination tablets for oral administration.
IVA
Tablet for oral administration.
Placebo (matched to VX-121/TEZ/D-IVA)
Placebo matched to VX-121/TEZ/D-IVA for oral administration.
Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration.
Placebo (matched to IVA)
Placebo matched to IVA for oral administration.

Locations

Country Name City State
Australia Royal Prince Alfred Hospital Camperdown
Australia The Prince Charles Hospital Chermside
Australia Alfred Hospital Melbourne, VIC
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
Austria University of Graz Graz
Austria Medizinische Universität Innsbruck Innsbruck
Austria Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde Salzburg
Austria Medizinische Universität Wien Vienna
Austria Abteilung fuer Pulmologie Klinikum Wels-Grieskirchen Wels
Belgium Cliniques Universitaires de Bruxelles Hopital Erasme Brussel
Belgium Universitair Ziekenhuis Brussel - Campus Jette Brussel
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 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 St. Paul's Hospital Vancouver
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 Créteil
France Institut Cœur Poumon, CHU de Lille Lille
France Hopital Arnaud de Villeneuve Montpellier Cedex 5
France Centre Hospitalier Universitaire De Nantes - G. R. Laennec Nantes
France Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur Nice
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 Hopital Bretonneau Tours
France Centre hospitalier universitaire de Nancy, Hôpital de Braboi Vandoeuvre Les Nancy
Germany Charite Paediatric Pulmonology Department Berlin
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 Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Gießen
Germany Pneumologisches Studienzentrum Muenchen-West Muenchen
Greece General Hospital of Thessaloniki "Papanikolaou" Pilea Chortiatis
Greece General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki Thessaloníki
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 Tallaght Dublin
Ireland Children's University Hospital, Dublin Dublin
Ireland St. Vincent's University Hospital Dublin
Ireland University Hospital Limerick (Adults) Limerick
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 Greenlane Clinical Centre Auckland
New Zealand Starship Children's Hospital Auckland
New Zealand Canterbury Respiratory Research Group Christchurch Hospital
New Zealand Waikato Hospital Hamilton
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
Poland Institute of Tuberculosis and Lung Diseases Rabka Zdrój
Sweden Sahlgrenska Universitetssjukhuset Göteborg
Sweden Lund University Skanes Universitetssjukhus Malmoe
Sweden Karolinska Universitetssjukhuset, Huddinge Stockholm
Switzerland Lindenhofspital - Quartier Bleu Bern
Switzerland Réseau Hospitalier Neuchâtelois Neuchâtel
Switzerland Kinderspital Zuerich Zürich
United Kingdom Birmingham Children's Hospital Birmingham
United Kingdom Royal Papworth Hospital NHS Foundation Trust Cambridge
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 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 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 Michigan Medicine Ann Arbor Michigan
United States The Emory Clinic at Chantilly Atlanta 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 Rsearch Center Boston Massachusetts
United States CF Therapeutics Development Center of Western New York Buffalo New York
United States University of Virginia Health System Charlottesville Virginia
United States Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States Northwestern Memorial Hospital Chicago Illinois
United States Vermont Lung Center Colchester Vermont
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 Spectrum Health Medical Group Adult Cystic Fibrosis Care Center Grand Rapids Michigan
United States Indiana University Indianapolis Indiana
United States Riley Hospital for Children at Indiana University Health Indianapolis Indiana
United States University of Mississippi Medical Center Jackson Mississippi
United States Nemours Children's Specialty Care Jacksonville Florida
United States The Children's Mercy Hospital Kansas City Missouri
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 Children's Hospital Los Angeles Los Angeles California
United States University Hospital and UW Health Clinics Madison Wisconsin
United States University of Miami Miller School of Medicine Miami Florida
United States West Virginia University Morgantown West Virginia
United States Morristown Medical Center Morristown New Jersey
United States Vanderbilt University Medical Center Nashville Tennessee
United States Yale New Haven Hospital New Haven Connecticut
United States Northwell Health- 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 Children's Hospital of the King's Daughters Norfolk Virginia
United States Santiago Reyes, M.D. Oklahoma City Oklahoma
United States University of Oklahoma Health Sciences Center Oklahoma City Oklahoma
United States Children's Hospital of Orange County Orange California
United States Central Florida Pulmonary Group, PA Orlando Florida
United States Nemours Children's Specialty Care, Pensacola 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 Washington University School of Medicine Saint Louis Missouri
United States Johns Hopkins All Children's Hospital Outpatient Care Center Saint Petersburg Florida
United States University of Utah - Primary Children's Medical Center Salt Lake City Utah
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 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,  Denmark,  France,  Germany,  Greece,  Hungary,  Ireland,  Israel,  Italy,  Netherlands,  New Zealand,  Norway,  Poland,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1) FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. From Baseline Through Week 24
Secondary Absolute Change in Sweat Chloride (SwCl) Sweat samples were collected using an approved collection device. From Baseline Through Week 24
Secondary Percentage of Participants With SwCl <60 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102) Sweat samples were collected using an approved collection device. From Baseline Through Week 24
Secondary Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102) Sweat samples were collected using an approved collection device. From Baseline Through Week 24
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