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

Last updated: May 16, 2024
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Completed

Phase

3

Condition

Cystic Fibrosis

Scar Tissue

Lung Disease

Treatment

Placebo (matched to VX-121/TEZ/D-IVA)

IVA

Placebo (matched to ELX/TEZ/IVA)

Clinical Study ID

NCT05076149
VX20-121-103
2021-000694-85
  • Ages > 12
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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/IVAand no F508del mutation

  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted meanfor age, sex, and height for participants currently receiving CFTR protein modulatortherapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR proteinmodulator therapy

Exclusion

Key Exclusion Criteria:

  • History of solid organ or hematological transplantation

  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child PughScore 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)

  • Lung infection with organisms associated with a more rapid decline in pulmonarystatus

  • Pregnant or breast-feeding females

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 597
Treatment Group(s): 6
Primary Treatment: Placebo (matched to VX-121/TEZ/D-IVA)
Phase: 3
Study Start date:
October 27, 2021
Estimated Completion Date:
November 30, 2023

Connect with a study center

  • Royal Prince Alfred Hospital

    Camperdown,
    Australia

    Site Not Available

  • The Prince Charles Hospital

    Chermside,
    Australia

    Site Not Available

  • Alfred Hospital

    Melbourne, VIC,
    Australia

    Site Not Available

  • Institute for Respiratory Health

    Nedlands,
    Australia

    Site Not Available

  • Telethon Kids Institute

    Nedlands,
    Australia

    Site Not Available

  • The Royal Children's Hospital

    Parkville, VIC,
    Australia

    Site Not Available

  • Sydney Children's Hospital

    Randwick,
    Australia

    Site Not Available

  • Mater Adult Hospital

    South Brisbane,
    Australia

    Site Not Available

  • Queensland Children's Hospital

    South Brisbane,
    Australia

    Site Not Available

  • Westmead Hospital

    Westmead,
    Australia

    Site Not Available

  • University of Graz

    Graz,
    Austria

    Site Not Available

  • Medizinische Universität Innsbruck

    Innsbruck,
    Austria

    Site Not Available

  • Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde

    Salzburg,
    Austria

    Site Not Available

  • Medizinische Universität Wien

    Vienna,
    Austria

    Site Not Available

  • Abteilung fuer Pulmologie Klinikum Wels-Grieskirchen

    Wels,
    Austria

    Site Not Available

  • Cliniques Universitaires de Bruxelles Hopital Erasme

    Brussel,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Brussel - Campus Jette

    Brussel,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Gent

    Gent,
    Belgium

    Site Not Available

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven,
    Belgium

    Site Not Available

  • Cliniques Universitaires Saint-Luc

    Woluwe-Saint-Lambert,
    Belgium

    Site Not Available

  • University of Calgary Medical Clinic of the Foothills Medical Centre

    Calgary,
    Canada

    Site Not Available

  • Stollery Children's Hospital

    Edmonton,
    Canada

    Site Not Available

  • St. Joseph's Health Care London

    London,
    Canada

    Site Not Available

  • Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu

    Montreal,
    Canada

    Site Not Available

  • McGill University Health Centre, Glen Site, Montreal Children's Hospital

    Montréal,
    Canada

    Site Not Available

  • Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval

    Québec,
    Canada

    Site Not Available

  • St. Michael's Hospital

    Toronto,
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver,
    Canada

    Site Not Available

  • St. Paul's Hospital

    Vancouver,
    Canada

    Site Not Available

  • Aarhus University Hospital

    Aarhus,
    Denmark

    Site Not Available

  • Juliane Marie Center, Rigshospitalet

    Copenhagen,
    Denmark

    Site Not Available

  • Groupe Hospitaler Pellegrin, CHU De Bordeaux

    Bordeaux cedex,
    France

    Site Not Available

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron Cedex,
    France

    Site Not Available

  • Centre Hospitalier Intercommunal Creteil

    Créteil,
    France

    Site Not Available

  • Institut Cœur Poumon, CHU de Lille

    Lille,
    France

    Site Not Available

  • Hopital Arnaud de Villeneuve

    Montpellier Cedex 5,
    France

    Site Not Available

  • Centre Hospitalier Universitaire De Nantes - G. R. Laennec

    Nantes,
    France

    Site Not Available

  • Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur

    Nice,
    France

    Site Not Available

  • Hopital Cochin

    Paris,
    France

    Site Not Available

  • Hopital Robert Debre

    Paris,
    France

    Site Not Available

  • Hopital Necker, Enfants Malades

    Paris Cedex 15,
    France

    Site Not Available

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite,
    France

    Site Not Available

  • Centre de Perharidy

    Roscoff cedex,
    France

    Site Not Available

  • CHU de Rouen - Hopital Charles Nicolle

    Rouen Cedex, Seine Maritime,
    France

    Site Not Available

  • Hôpital de Hautepierre, AX5

    Strasbourg,
    France

    Site Not Available

  • Hopital Foch (Suresnes), Hopital Foch, Adultes

    Suresnes,
    France

    Site Not Available

  • Centre Hospitalier Universitaire De Nantes - G. R. Laennec

    Tissot, Adrien,
    France

    Site Not Available

  • Hopital Bretonneau

    Tours,
    France

    Site Not Available

  • Centre hospitalier universitaire de Nancy, Hôpital de Braboi

    Vandoeuvre Les Nancy,
    France

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin,
    Germany

    Site Not Available

  • Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital

    Erlangen,
    Germany

    Site Not Available

  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

    Essen,
    Germany

    Site Not Available

  • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie

    Essen,
    Germany

    Site Not Available

  • Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin

    Gießen,
    Germany

    Site Not Available

  • Pneumologisches Studienzentrum Muenchen-West

    Muenchen,
    Germany

    Site Not Available

  • General Hospital of Thessaloniki "Papanikolaou"

    Pilea Chortiatis,
    Greece

    Site Not Available

  • General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki

    Thessaloníki,
    Greece

    Site Not Available

  • National Koranyi Institute for TBC and Pulmonology

    Budapest,
    Hungary

    Site Not Available

  • Pulmonology Institute Torokbalint

    Torokbalint,
    Hungary

    Site Not Available

  • Cork University Hospital

    Cork,
    Ireland

    Site Not Available

  • Children's Health Ireland at Crumlin

    Dublin,
    Ireland

    Site Not Available

  • Children's Health Ireland at Tallaght

    Dublin,
    Ireland

    Site Not Available

  • Children's University Hospital, Dublin

    Dublin,
    Ireland

    Site Not Available

  • St. Vincent's University Hospital

    Dublin,
    Ireland

    Site Not Available

  • University Hospital Limerick (Adults)

    Limerick,
    Ireland

    Site Not Available

  • University Hospital Limerick (Pediatrics)

    Limerick,
    Ireland

    Site Not Available

  • Hadassah Medical Organization

    Jerusalem,
    Israel

    Site Not Available

  • Schneider Children's Medical Center of Israel

    Petach Tikvah,
    Israel

    Site Not Available

  • Sheba Medical Center - The Edmond and Lili Safra Children's Hospital

    Tel HaShomer,
    Israel

    Site Not Available

  • Azienda Ospedaliero Universitaria Ospedale Riuniti

    Ancona,
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

    Firenze,
    Italy

    Site Not Available

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova,
    Italy

    Site Not Available

  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

    Milan,
    Italy

    Site Not Available

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milano,
    Italy

    Site Not Available

  • Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica

    Orbassano,
    Italy

    Site Not Available

  • Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo

    Potenza,
    Italy

    Site Not Available

  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

    Verona,
    Italy

    Site Not Available

  • Academisch Medisch Centrum (Academic Medical Centre)

    Amsterdam,
    Netherlands

    Site Not Available

  • HagaZiekenhuis van den Haag

    Den Haag,
    Netherlands

    Site Not Available

  • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

    Heidelberglaan,
    Netherlands

    Site Not Available

  • UMC St. Radboud

    Nijmegen,
    Netherlands

    Site Not Available

  • Erasmus Medical Center

    Rotterdam,
    Netherlands

    Site Not Available

  • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

    Utrecht,
    Netherlands

    Site Not Available

  • Greenlane Clinical Centre

    Auckland,
    New Zealand

    Site Not Available

  • Starship Children's Hospital

    Auckland,
    New Zealand

    Site Not Available

  • Canterbury Respiratory Research Group

    Christchurch Hospital,
    New Zealand

    Site Not Available

  • Waikato Hospital

    Hamilton,
    New Zealand

    Site Not Available

  • Haukeland Universitetssjukehus (CF)

    Bergen,
    Norway

    Site Not Available

  • Oslo University Hospital, Department of Paediatric Medicine

    Oslo,
    Norway

    Site Not Available

  • Pediatric Hospital Polanki named of Maciej Płażyński

    Gdańsk,
    Poland

    Site Not Available

  • Institute of Tuberculosis and Lung Diseases

    Rabka Zdrój,
    Poland

    Site Not Available

  • Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY

    Łomianki,
    Poland

    Site Not Available

  • Sahlgrenska Universitetssjukhuset

    Göteborg,
    Sweden

    Site Not Available

  • Lund University Skanes Universitetssjukhus

    Malmoe,
    Sweden

    Site Not Available

  • Karolinska Universitetssjukhuset, Huddinge

    Stockholm,
    Sweden

    Site Not Available

  • Lindenhofspital - Quartier Bleu

    Bern,
    Switzerland

    Site Not Available

  • Réseau Hospitalier Neuchâtelois

    Neuchâtel,
    Switzerland

    Site Not Available

  • Kinderspital Zuerich

    Zurich,
    Switzerland

    Site Not Available

  • Kinderspital Zuerich

    Zürich,
    Switzerland

    Site Not Available

  • Birmingham Children's Hospital

    Birmingham,
    United Kingdom

    Site Not Available

  • Royal Papworth Hospital NHS Foundation Trust

    Cambridge,
    United Kingdom

    Site Not Available

  • Clinical Research Facility, Queen Elizabeth University Hospital

    Glasgow,
    United Kingdom

    Site Not Available

  • Leeds General Infirmary

    Leeds,
    United Kingdom

    Site Not Available

  • St. James University Hospital

    Leeds,
    United Kingdom

    Site Not Available

  • King's College Hospital

    London,
    United Kingdom

    Site Not Available

  • Royal Brompton Hospital

    London,
    United Kingdom

    Site Not Available

  • St. Bartholomew's Hospital

    London,
    United Kingdom

    Site Not Available

  • Royal Manchester Children's Hospital

    Manchester,
    United Kingdom

    Site Not Available

  • Wythenshawe Hospital

    Manchester,
    United Kingdom

    Site Not Available

  • All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

    Penarth,
    United Kingdom

    Site Not Available

  • Southampton General Hospital

    Southampton,
    United Kingdom

    Site Not Available

  • University of Alabama at Birmingham

    Birmingham, Alabama 35233
    United States

    Site Not Available

  • Banner University of Arizona Medical Center

    Tucson, Arizona 85724
    United States

    Site Not Available

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas 72205
    United States

    Site Not Available

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Site Not Available

  • Children's Hospital of Orange County

    Orange, California 92868
    United States

    Site Not Available

  • University of California Davis Medical Center

    Sacramento, California 95817
    United States

    Site Not Available

  • Children's Hospital of Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • National Jewish Health

    Denver, Colorado 80206
    United States

    Site Not Available

  • Yale New Haven Hospital

    New Haven, Connecticut 06510
    United States

    Site Not Available

  • Nemours Children's Specialty Care

    Jacksonville, Florida 32207
    United States

    Site Not Available

  • University of Miami Miller School of Medicine

    Miami, Florida 33136
    United States

    Site Not Available

  • Central Florida Pulmonary Group, PA

    Orlando, Florida 32803
    United States

    Site Not Available

  • Nemours Children's Specialty Care, Pensacola

    Pensacola, Florida 32514
    United States

    Site Not Available

  • Johns Hopkins All Children's Hospital Outpatient Care Center

    Saint Petersburg, Florida 33701
    United States

    Site Not Available

  • The Emory Clinic at Chantilly

    Atlanta, Georgia 30324
    United States

    Site Not Available

  • St. Luke's Cystic Fibrosis Center of Idaho

    Boise, Idaho 83702
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Northwestern Memorial Hospital

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Indiana University

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • University of Kentucky

    Lexington, Kentucky 40536
    United States

    Site Not Available

  • Tulane Medical Center

    New Orleans, Louisiana 70112
    United States

    Site Not Available

  • Maine Medical Partners

    Portland, Maine 04102
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • UMass Memorial Medical Center

    Worcester, Massachusetts 01655
    United States

    Site Not Available

  • Michigan Medicine

    Ann Arbor, Michigan 48109-5212
    United States

    Site Not Available

  • Harper University Hospital

    Detroit, Michigan 48201
    United States

    Site Not Available

  • Spectrum Health Medical Group Adult Cystic Fibrosis Care Center

    Grand Rapids, Michigan 49546
    United States

    Site Not Available

  • University of Mississippi Medical Center

    Jackson, Mississippi 39216
    United States

    Site Not Available

  • The Children's Mercy Hospital

    Kansas City, Missouri 64108
    United States

    Site Not Available

  • Washington University School of Medicine

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Billings Clinic

    Billings, Montana 59101
    United States

    Site Not Available

  • Morristown Medical Center

    Morristown, New Jersey 07960
    United States

    Site Not Available

  • CF Therapeutics Development Center of Western New York

    Buffalo, New York 14203
    United States

    Site Not Available

  • Northwell Health- Long Island Jewish Medical Center

    New Hyde Park, New York 11040
    United States

    Site Not Available

  • Columbia University Medical Center

    New York, New York 10032
    United States

    Site Not Available

  • SUNY Upstate Medical University

    Syracuse, New York 13210
    United States

    Site Not Available

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina 27104
    United States

    Site Not Available

  • Akron Children's Hospital

    Akron, Ohio 44308
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • Dayton Children's Hospital

    Dayton, Ohio 45404
    United States

    Site Not Available

  • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center

    Toledo, Ohio 43606
    United States

    Site Not Available

  • Santiago Reyes, M.D.

    Oklahoma City, Oklahoma 73112
    United States

    Site Not Available

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma 73104
    United States

    Site Not Available

  • Oregon Health & Science University

    Portland, Oregon 97239
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania 15224
    United States

    Site Not Available

  • Prisma Health Richland Campus

    Columbia, South Carolina 29203
    United States

    Site Not Available

  • Sanford Children's Speciality Clinic

    Sioux Falls, South Dakota 57105
    United States

    Site Not Available

  • University of Tennessee Medical Center

    Knoxville, Tennessee 37920
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • The University of Texas Southwestern Medical Center

    Dallas, Texas 75390-8558
    United States

    Site Not Available

  • Cook Children's Health Care System

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • University of Utah - Primary Children's Medical Center

    Salt Lake City, Utah 84132
    United States

    Site Not Available

  • Vermont Lung Center

    Colchester, Vermont 05446
    United States

    Site Not Available

  • University of Virginia Health System

    Charlottesville, Virginia 22908
    United States

    Site Not Available

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia 23507
    United States

    Site Not Available

  • University of Washington Medical Center

    Seattle, Washington 98195
    United States

    Site Not Available

  • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

    Spokane, Washington 99204
    United States

    Site Not Available

  • West Virginia University

    Morgantown, West Virginia 26506
    United States

    Site Not Available

  • University Hospital and UW Health Clinics

    Madison, Wisconsin 53792
    United States

    Site Not Available

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