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

NCT ID NCT05153317

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

First seen Mar 15, 2026 · Last updated Jun 22, 2026 · Updated 15 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 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.

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Contacts and locations

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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

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.

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 CYSTIC FIBROSIS