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Trikafta passes long-term safety check in toddlers with cystic fibrosis

NCT ID NCT05153317

First seen Mar 15, 2026 · Last updated May 22, 2026 · Updated 12 times

Summary

This study looked at the long-term safety and effectiveness of the triple-combination drug Trikafta (elexacaftor/tezacaftor/ivacaftor) in 71 children aged 2 and older with cystic fibrosis. All participants had already taken the drug in an earlier study. The main goal was to track side effects, and secondary goals included changes in sweat chloride and lung function. Because cystic fibrosis requires lifelong medication, this treatment controls the disease but is not a cure.

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Contacts and locations

Locations

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, United Kingdom

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

    Chicago, Illinois, 60611, United States

  • Banner University of Arizona Medical Center

    Tucson, Arizona, 85724, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, Canada

  • Charite Paediatric Pulmonology Department

    Berlin, Germany

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

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

    Minneapolis, Minnesota, 55404, United States

  • NC TraCS Institute - CTRC University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Queensland Children's Hospital

    South Brisbane, Australia

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana, 46202, United States

  • Royal Brompton Hospital

    London, United Kingdom

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • Telethon Kids Institute

    Nedlands, Australia

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • The Royal Children's Hospital

    Parkville, Australia

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

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

    Essen, Germany

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

    St Louis, Missouri, 63110, United States

Conditions

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