A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Last updated: June 11, 2021
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Completed

Phase

3

Condition

Cystic Fibrosis

Lung Disease

Scar Tissue

Treatment

N/A

Clinical Study ID

NCT04058353
VX18-445-104
2018-002835-76
  • Ages > 12
  • All Genders

Study Summary

This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either agating or residual function mutation (F/G and F/RF genotypes)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean forage, sex, and height

Exclusion

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Solid organ or hematological transplantation Other protocol defined Inclusion/Exclusion criteria may apply

Study Design

Total Participants: 271
Study Start date:
August 28, 2019
Estimated Completion Date:
June 12, 2020

Connect with a study center

  • The Prince Charles Hospital

    Chermside,
    Australia

    Site Not Available

  • Alfred Hospital

    Melbourne, VIC,
    Australia

    Site Not Available

  • Perth Children's Hospital

    Nedlands,
    Australia

    Site Not Available

  • The Royal Children's Hospital

    Parkville, 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

  • Cliniques Universitaires de Bruxelles Hopital Erasme

    Brussels,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Brussel - Campus Jette

    Brussels,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Gent

    Gent,
    Belgium

    Site Not Available

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven,
    Belgium

    Site Not Available

  • Stollery Children's Hospital

    Edmonton,
    Canada

    Site Not Available

  • McGill University Health Center

    Québec,
    Canada

    Site Not Available

  • St. Michael's Hospital

    Toronto,
    Canada

    Site Not Available

  • St. Paul's Hospital

    Vancouver,
    Canada

    Site Not Available

  • Juliane Marie Center, Rigshospitalet

    Copenhagen,
    Denmark

    Site Not Available

  • Centre Hospitalier Lyon Sud

    Benite Cedex,
    France

    Site Not Available

  • Groupe Hospitaler Pellegrin, CHU De Bordeaux

    Bordeaux cedex,
    France

    Site Not Available

  • CHRU de Lille - Hopital Albert Calmette

    Lille,
    France

    Site Not Available

  • CHU Marseille - Hopital Nord

    Marseille,
    France

    Site Not Available

  • CHU de Montpellier - Hopital Arnaud de Villeneuve

    Montpellier Cedex 5,
    France

    Site Not Available

  • Hopital Cochin

    Paris,
    France

    Site Not Available

  • Hopital Necker, Enfants Malades

    Paris Cedex 15,
    France

    Site Not Available

  • Hopital Pontchaillou CHU de Rennes

    Rennes Cedex,
    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

  • Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin

    Giessen,
    Germany

    Site Not Available

  • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie

    Halle,
    Germany

    Site Not Available

  • Pneumologisches Studienzentrum Muenchen-West

    Muenchen,
    Germany

    Site Not Available

  • University Hospital Wuerzburg

    Würzburg,
    Germany

    Site Not Available

  • Cork University Hospital

    Cork,
    Ireland

    Site Not Available

  • Beaumont Hospital

    Dublin,
    Ireland

    Site Not Available

  • St. Vincent's University Hospital

    Dublin,
    Ireland

    Site Not Available

  • University Hospital Limerick (Adults)

    Limerick,
    Israel

    Site Not Available

  • Azienda Ospedaliero Universitaria Ospedale Riuniti

    Ancona,
    Italy

    Site Not Available

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova,
    Italy

    Site Not Available

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milano,
    Italy

    Site Not Available

  • Malattie Apparato Respiratorio 2 - Fibrosi Cistica

    Orbassano,
    Italy

    Site Not Available

  • Ospedale Pediatrico Bambino Gesu

    Rome,
    Italy

    Site Not Available

  • Azienda Ospedaliera di Verona-Ospedale Civile Maggiore

    Verona,
    Italy

    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

  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

    Barcelona,
    Spain

    Site Not Available

  • Hospital Virgen de la Arrixaca

    Murcia,
    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

  • Papworth Hospital NHS Foundation Trust, Papworth Everard

    Cambridge,
    United Kingdom

    Site Not Available

  • Royal Devon and Exeter NHS Foundation Trust, 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

  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

    London,
    United Kingdom

    Site Not Available

  • Wythenshawe Hospital

    Manchester,
    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

  • Miller Children's Hospital / Long Beach Memorial

    Long Beach, California 90806
    United States

    Site Not Available

  • Stanford University

    Palo Alto, California 94304
    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

  • National Jewish Health

    Denver, Colorado 80206
    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

  • Indiana University

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • The University of Iowa Hospitals and Clinics

    Iowa City, Iowa 52242
    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

  • 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

  • Michigan Medicine

    Ann Arbor, Michigan 48109-5212
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

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

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Dartmouth Hitchcock Medical Center

    Lebanon, New Hampshire 03756
    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

  • Mount Sinai Beth Israel

    New York, New York 10003
    United States

    Site Not Available

  • University of North Carolina Hospitals

    Chapel Hill, North Carolina 27514
    United States

    Site Not Available

  • UC Health Holmes

    Cincinnati, Ohio 45220
    United States

    Site Not Available

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio 44106
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    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

  • 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

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania 15224
    United States

    Site Not Available

  • Dell Children's Medical Group

    Austin, Texas 78723
    United States

    Site Not Available

  • The University of Texas Southwestern Medical Center

    Dallas, Texas 75390
    United States

    Site Not Available

  • Texas Children's Hospital

    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

  • 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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