CF drug shows promise for toddlers in Final-Stage trial
NCT ID NCT05882357
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom
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British Columbia Children's Hospital
Vancouver, Canada
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Charite Paediatric Pulmonology Department
Berlin, Germany
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Children and Young Adults Research Unit
Cardiff, United Kingdom
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Erasmus Medical Center / Sophia Children's Hospital
Rotterdam, Netherlands
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Great Ormond Street Hospital for Children
London, United Kingdom
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Inselspital - Universitaetsspital Bern
Bern, Switzerland
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Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
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Kinderklinik III, Abt. fur Pneumologie
Essen, Germany
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Kinderspital Zuerich
Zurich, Switzerland
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Leeds General Infirmary
Leeds, United Kingdom
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Medizinische Hochschule Hannover
Hanover, Germany
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Queensland Children's Hospital
South Brisbane, Australia
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Royal Brompton Hospital
London, United Kingdom
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Southampton General Hospital
Southampton, United Kingdom
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Telethon Kids Institute
Nedlands, Australia
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The Children's Hospital at Westmead
Westmead, Australia
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The Hospital for Sick Children
Toronto, Canada
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The Royal Children's Hospital
Parkville, Australia