New hope for youngest CF patients: drug study in toddlers shows promise
NCT ID NCT05882357
First seen Oct 31, 2025 · Last updated Apr 30, 2026 · Updated 29 times
Summary
This study tested the safety and how the body processes the drug combination elexacaftor/tezacaftor/ivacaftor in 70 toddlers with cystic fibrosis who are 12 to 24 months old. The drug is already used in older patients to help the lungs work better. Researchers measured drug levels in the blood and watched for side effects. The goal is to see if this treatment is safe and works well in very young children.
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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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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
Conditions
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