A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

Last updated: September 18, 2024
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Completed

Phase

3

Condition

Cystic Fibrosis

Lung Disease

Scar Tissue

Treatment

ELX/TEZ/IVA

IVA

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

Clinical Study ID

NCT05033080
VX20-121-102
2021-000712-31
  • Ages > 12
  • All Genders

Study Summary

The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Heterozygous for F508del and a minimal function mutation (F/MF genotype)

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

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: 435
Treatment Group(s): 6
Primary Treatment: ELX/TEZ/IVA
Phase: 3
Study Start date:
September 14, 2021
Estimated Completion Date:
November 21, 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

  • 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

  • Klinika Detskych Infekcnich Nemoci

    Brno,
    Czechia

    Site Not Available

  • Fakultni nemocnice v Motole

    Praha 5,
    Czechia

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin,
    Germany

    Site Not Available

  • St. Josef-Hospital

    Bochum,
    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

  • Johann Wolfgang Goethe University

    Frankfurt,
    Germany

    Site Not Available

  • Hannover Medical School

    Hannover,
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover

    Hannover,
    Germany

    Site Not Available

  • Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin

    Jena,
    Germany

    Site Not Available

  • Johannes Gutenberg-Universitaet

    Mainz,
    Germany

    Site Not Available

  • Pneumologisches Studienzentrum Muenchen-West

    Muenchen,
    Germany

    Site Not Available

  • Klinikum Innenstadt, University of Munich

    München,
    Germany

    Site Not Available

  • Klinikum Westbrandenburg (CF)

    Potsdam,
    Germany

    Site Not Available

  • Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)

    Ulm,
    Germany

    Site Not Available

  • Universitätsklinikum Würzburg

    Würzburg,
    Germany

    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 Health Ireland at Temple Street

    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 University Hospital Mount Scopus

    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

  • 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

  • Hospital de Santa Maria

    Lisbon,
    Portugal

    Site Not Available

  • CHP - Hospital de Santo Antonio

    Porto,
    Portugal

    Site Not Available

  • Hospital Sao Joao

    Porto,
    Portugal

    Site Not Available

  • Hospital Saint Joan de Deu

    Barcelona,
    Spain

    Site Not Available

  • Hospital Universitari Vall d Hebron

    Barcelona,
    Spain

    Site Not Available

  • Hospital Infantil Universitario Nino Jesus

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario Ramon y Cajal

    Madrid,
    Spain

    Site Not Available

  • Hospital Virgen de la Arrixaca

    Murcia,
    Spain

    Site Not Available

  • Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

    Sabadell,
    Spain

    Site Not Available

  • Hospital Universitario Virgen del Rocio

    Sevilla,
    Spain

    Site Not Available

  • Hospital Universitario y Politecnico La Fe

    Valencia,
    Spain

    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

  • Birmingham Heartlands Hospital

    Birmingham,
    United Kingdom

    Site Not Available

  • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital

    Bristol,
    United Kingdom

    Site Not Available

  • Royal Papworth Hospital NHS Foundation Trust

    Cambridge,
    United Kingdom

    Site Not Available

  • Royal Hospital for Sick Children

    Edinburgh,
    United Kingdom

    Site Not Available

  • Western General Hospital

    Edinburgh,
    United Kingdom

    Site Not Available

  • Royal Devon and Exeter Hospital

    Exeter,
    United Kingdom

    Site Not Available

  • Clinical Research Facility, Queen Elizabeth University Hospital

    Glasgow,
    United Kingdom

    Site Not Available

  • St. James University Hospital

    Leeds,
    United Kingdom

    Site Not Available

  • Liverpool Heart and Chest Hospital

    Liverpool,
    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

  • Wythenshawe Hospital

    Manchester,
    United Kingdom

    Site Not Available

  • Clinical Research Facility

    Newcastle Upon Tyne,
    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

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Site Not Available

  • Miller Children's Hospital / Long Beach Memorial

    Long Beach, California 90806
    United States

    Site Not Available

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Site Not Available

  • Kaiser Permanente

    Oakland, California 94611
    United States

    Site Not Available

  • University of California Davis Medical Center

    Sacramento, California 95817
    United States

    Site Not Available

  • University of California San Francisco, Lung Transplant Program

    San Francisco, California 94143
    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

  • University of Florida, Shands Hospital

    Gainesville, Florida 32610
    United States

    Site Not Available

  • Joe DiMaggio Cystic Fibrosis & Pulmonary Center

    Hollywood, Florida 33021
    United States

    Site Not Available

  • Central Florida Pulmonary Group, PA

    Orlando, Florida 32803
    United States

    Site Not Available

  • Nemours Children's Hospital

    Orlando, Florida 32827
    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

  • Augusta University

    Augusta, Georgia 30912
    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 Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • University of Kentucky

    Lexington, Kentucky 40536
    United States

    Site Not Available

  • Kosair Charities Pediatric Clinical Research Unit

    Louisville, Kentucky 40202
    United States

    Site Not Available

  • Tulane Medical Center

    New Orleans, Louisiana 70112
    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

  • Helen DeVos Children's Hospital CF Center

    Grand Rapids, Michigan 49503
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • University of Mississippi Medical Center

    Jackson, Mississippi 39216
    United States

    Site Not Available

  • Childrens Hospital University of Missouri Health Sciences Center

    Columbia, Missouri 65212
    United States

    Site Not Available

  • The Children's Mercy Hospital

    Kansas City, Missouri 64108
    United States

    Site Not Available

  • Cardinal Glennon Children's Hospital - St. Louis University

    Saint Louis, Missouri 63104
    United States

    Site Not Available

  • Washington University School of Medicine / St. Louis Children's Hospital

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Billings Clinic

    Billings, Montana 59101
    United States

    Site Not Available

  • Nebraska Medical Center

    Omaha, Nebraska 68198
    United States

    Site Not Available

  • Dartmouth Hitchcock, Manchester

    Manchester, New Hampshire 03104
    United States

    Site Not Available

  • Morristown Medical Center

    Morristown, New Jersey 07960
    United States

    Site Not Available

  • Albany Medical College

    Albany, New York 12208
    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

  • New York Medical College

    Valhalla, New York 10595
    United States

    Site Not Available

  • Atrium Health Levine Children's Hospital

    Charlotte, North Carolina 28203
    United States

    Site Not Available

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina 27104
    United States

    Site Not Available

  • Cleveland Clinic CF

    Cleveland, Ohio 44195
    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

  • 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

  • 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

  • Children's Foundation Research Center / Le Bonheur Children's Hospital

    Memphis, Tennessee 38105
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • Driscoll Children's Hospital

    Corpus Christi, Texas 78411
    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

  • Texas Children's Hospital - Baylor College of Medicine

    Houston, Texas 77030
    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

  • Inova Fairfax

    Falls Church, Virginia 22042
    United States

    Site Not Available

  • University of Washington Medical Center

    Seattle, Washington 98195
    United States

    Site Not Available

  • University of Wisconsin Hospital and Clinics

    Madison, Wisconsin 53792
    United States

    Site Not Available

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