New triple therapy for kids with cystic fibrosis shows promise in phase 3 trial

NCT ID NCT05422222

First seen Nov 06, 2025 · Last updated Jun 11, 2026 · Updated 35 times

Summary

This study tests a triple-combination drug (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific genetic mutation. The goal is to check how the medicine works in the body, its safety, and how well children tolerate it. Around 210 children will take part, and the study also looks at changes in sweat chloride levels, a key marker of CF.

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Contacts and locations

Locations

  • American Family Childrens Hospital

    Madison, Wisconsin, 53792, United States

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

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, Canada

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron, France

  • Charité - Paediatric Pulmonology Department

    Berlin, Germany

  • Children and Young Adults Research Unit

    Cardiff, United Kingdom

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

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

    Minneapolis, Minnesota, 55404, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Cohen Children's Medical Center

    Lake Success, New York, 11042, United States

  • Erasmus Medical Center / Sophia Children's Hospital

    Rotterdam, Netherlands

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Hopital Necker, Enfants Malades

    Paris, France

  • Inselspital - Universitaetsspital Bern

    Bern, Switzerland

  • Karolinska University Hospital - Pulmonology

    Stockholm, Sweden

  • Kinderspital Zurich

    Zurich, Switzerland

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Queensland Children's Hospital

    South Brisbane, Australia

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

    Cleveland, Ohio, 44106, United States

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana, 46202, United States

  • Sahlgrenska Universitetssjukhuset

    Gothenburg, Sweden

  • Stanford University Clinical and Translational Research Unit

    Palo Alto, California, 94304, United States

  • Starship Children's Hospital

    Grafton, New Zealand

  • Texas Children's Hospital - Wallace Tower

    Houston, Texas, 77030, United States

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

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

    Atlanta, Georgia, 30322, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • The Kids Research Institute Australia

    Nedlands, Australia

  • The Royal Children's Hospital

    Parkville, Australia

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Universitatsklinikum Essen

    Essen, Germany

  • Vermont Lung Center

    Colchester, Vermont, 05446, United States

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

    St Louis, Missouri, 63110, United States

  • Women's and Children's Hospital

    North Adelaide, Australia

Conditions

Explore the condition pages connected to this study.