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Trikafta's long-term safety tested in toddlers with cystic fibrosis

NCT ID NCT06460506

Ongoing Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study is testing the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (known as Trikafta) in 50 people with cystic fibrosis who are at least 12 months old. Participants previously completed a related study and will continue taking the drug to see how well it works over time. The main goal is to track side effects and changes in sweat chloride levels, a key measure of cystic fibrosis severity.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
elexacaftor/tezacaftor/ivacaftor (Trikafta)
What this could lead to
If successful, this study could confirm that Trikafta is safe and effective for long-term use in young children with cystic fibrosis, helping them breathe easier and have fewer lung infections.
What could go wrong
This is an open-label extension study with no placebo group, so results may be less reliable. Side effects like liver problems or drug interactions are possible, and the small size (50 participants) limits how broadly the findings can be applied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CYSTIC FIBROSIS

Contacts and locations

Locations

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, United Kingdom

  • British Columbia Children's Hospital

    Vancouver, Canada

  • Charité Universitätsmedizin BerlinX

    Berlin, Germany

  • Children and Young Adults Research Unit

    Cardiff, United Kingdom

  • Erasmus Medical Center

    Rotterdam, Netherlands

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Inselspital - Universitaetsspital Bern

    Bern, Switzerland

  • Juliane Marie Center, Rigshospitalet

    Copenhagen, Denmark

  • Kinderklinik III, Abt. fur Pneumologie

    Essen, Germany

  • Kinderspital Zurich

    Zurich, Switzerland

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Queensland Children's Hospital

    South Brisbane, Australia

  • Royal Brompton Hospital

    London, United Kingdom

  • Southampton General Hospital

    Southampton, United Kingdom

  • The Hospital for Sick Children

    Toronto, Canada

  • The Kids Research Institute Australia

    Nedlands, Australia

  • The Royal Children's Hospital

    Parkville, Australia

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