Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Last updated: May 29, 2025
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Active - Recruiting

Phase

3

Condition

Lung Disease

Cystic Fibrosis

Scar Tissue

Treatment

VX-121/TEZ/D-IVA

Clinical Study ID

NCT05422222
VX21-121-105
2021-005930-40
2024-513754-29-00
  • Ages 1-11
  • All Genders

Study Summary

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Participants with stable CF and at least 1 TCR mutation (including F508del) in theCFTR gene

Exclusion

Key Exclusion Criteria:

  • History of solid organ, hematological transplantation, or cancer

  • 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

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 210
Treatment Group(s): 1
Primary Treatment: VX-121/TEZ/D-IVA
Phase: 3
Study Start date:
June 21, 2022
Estimated Completion Date:
June 30, 2030

Connect with a study center

  • Telethon Kids Institute

    Nedlands,
    Australia

    Site Not Available

  • The Kids Research Institute Australia

    Nedlands,
    Australia

    Active - Recruiting

  • Women & Children's Hospital

    North Adelaide,
    Australia

    Site Not Available

  • Women's and Children's Hospital

    North Adelaide,
    Australia

    Active - Recruiting

  • The Royal Children's Hospital

    Parkville,
    Australia

    Active - Recruiting

  • Queensland Children's Hospital

    South Brisbane,
    Australia

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto,
    Canada

    Active - Recruiting

  • British Columbia Children's Hospital

    Vancouver,
    Canada

    Active - Recruiting

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron Cedex,
    France

    Site Not Available

  • Hopital Necker, Enfants Malades

    Paris Cedex 15,
    France

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin,
    Germany

    Site Not Available

  • Charité Universitätsmedizin BerlinX

    Berlin,
    Germany

    Active - Recruiting

  • Kinderklinik III, Abt. fur Pneumologie

    Essen,
    Germany

    Active - Recruiting

  • Medizinische Hochschule Hannover

    Hannover,
    Germany

    Active - Recruiting

  • Erasmus Medical Center / Sophia Children's Hospital

    Rotterdam,
    Netherlands

    Active - Recruiting

  • Starship Children's Hospital

    Grafton,
    New Zealand

    Active - Recruiting

  • Sahlgrenska Universitetssjukhuset

    Göteborg,
    Sweden

    Completed

  • Inselspital - Universitaetsspital Bern

    Bern,
    Switzerland

    Completed

  • Kinderspital Zurich

    Zurich,
    Switzerland

    Active - Recruiting

  • Kinderspital Zuerich

    Zürich,
    Switzerland

    Site Not Available

  • Children and Young Adults Research Unit

    Cardiff,
    United Kingdom

    Active - Recruiting

  • Great Ormond Street Hospital for Children

    London,
    United Kingdom

    Active - Recruiting

  • Children's Hospital of Orange County

    Orange, California 92868
    United States

    Active - Recruiting

  • Stanford University

    Palo Alto, California 94304
    United States

    Site Not Available

  • Stanford University Clinical and Translational Research Unit

    Palo Alto, California 94304
    United States

    Active - Recruiting

  • Children's Hospital of Colorado

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston

    Atlanta, Georgia 30322
    United States

    Active - Recruiting

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

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana 46202
    United States

    Active - Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota 55404
    United States

    Active - Recruiting

  • The Children's Mercy Hospital

    Kansas City, Missouri 64108
    United States

    Active - Recruiting

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

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Cohen Children's Medical Center

    Lake Success, New York 11042
    United States

    Completed

  • Cohen Children's Medical Center

    New York, New York 11042
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

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

    Cleveland, Ohio 44106
    United States

    Active - Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Active - Recruiting

  • Oregon Health & Science University

    Portland, Oregon 97239
    United States

    Completed

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania 15224
    United States

    Active - Recruiting

  • Texas Children's Hospital - Baylor College of Medicine

    Houston, Texas 77030
    United States

    Site Not Available

  • Texas Children's Hospital - Wallace Tower

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Vermont Lung Center

    Colchester, Vermont 05446
    United States

    Active - Recruiting

  • American Family Childrens Hospital

    Madison, Wisconsin 53792
    United States

    Site Not Available

  • University of Wisconsin Hospital and Clinics

    Madison, Wisconsin 53792
    United States

    Site Not Available

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