Life-Changing CF drug tested in babies for first time
NCT ID NCT05882357
Summary
This study tested a three-drug combination therapy (elexacaftor/tezacaftor/ivacaftor) in very young children with cystic fibrosis, aged 12 to 24 months. The main goals were to see how the medicine behaves in the body and to check if it is safe and well-tolerated in this age group. The study included 70 children who have specific genetic mutations that respond to this treatment.
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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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