CF drug shows promise for toddlers in Final-Stage trial

NCT ID NCT05882357

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

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers aged 12 to 24 months with cystic fibrosis. The goal was to check how the drug moves through the body, its safety, and any side effects. The trial has finished, and results will help determine if this treatment is suitable for very young children.

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 (a triple-combination drug taken by mouth)
What this could lead to: If successful, this could provide a safe and effective treatment option for very young children with cystic fibrosis, potentially improving lung function and quality of life from an early age.
What could go wrong: This is a completed Phase 3 trial, but results are not yet published. The study is small (70 participants) and focused on safety and drug levels, so long-term benefits are still uncertain. Side effects are possible, as with any medication.

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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
Charite Paediatric Pulmonology Department

Berlin, Germany
Children and Young Adults Research Unit

Cardiff, United Kingdom
Erasmus Medical Center / Sophia Children's Hospital

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 Zuerich

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
Telethon Kids Institute

Nedlands, Australia
The Children's Hospital at Westmead

Westmead, Australia
The Hospital for Sick Children

Toronto, Canada
The Royal Children's Hospital

Parkville, Australia

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