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Trikafta passes long-term safety test in kids with cystic fibrosis

NCT ID NCT05153317

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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 previously taken the drug in an earlier study. Researchers tracked side effects, sweat chloride levels, and lung function over time. The goal was to see if the drug remains safe and helpful when used for extended periods in young patients.

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) triple combination therapy
What this could lead to
If successful, this 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 study with no placebo group, so results may be less reliable. Side effects like liver problems or drug interactions are possible. The small size (71 participants) limits how broadly the findings apply.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

71 people

The number who actually took part.

Started

Jan 2022

Finished

Jan 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

key Inclusion Criteria: * Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study Key Exclusion Criteria: * History of study drug intolerance in the parent study * History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study Other protocol defined Inclusion/Exclusion criteria may apply

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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

  • 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

More trials for these conditions

Other studies related to the condition(s) this trial covers.