Organoid-Guided drug trial offers hope for cystic fibrosis patients with rare mutations
NCT ID NCT06468527
First seen Jan 04, 2026 · Last updated May 15, 2026 · Updated 25 times
Summary
This study tested a combination of three drugs (Dirocaftor, Posenacaftor, Nesolicaftor) in 41 adults with cystic fibrosis who have rare gene mutations not covered by existing treatments. Participants were selected based on lab tests using their own intestinal organoids to predict if they would respond. The goal was to see if the drugs improve lung function and other measures over 8 weeks, with the aim of expanding treatment options for this underserved group.
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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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Azienda Ospedaliera Universitaria Integrata
Verona, Italy
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CHU de Nice
Nice, France
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Charité Universitätsmedizin Berlin
Berlin, Germany
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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
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Hospital Vall d'Hebron
Barcelona, Spain
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Hospital de Santa Maria
Lisbon, Portugal
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Hôpital Larrey CHU Toulouse
Toulouse, France
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Instituto Giannina Gaslini
Genova, Italy
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Medizinische Hochschule Hannover
Hanover, Germany
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Ospedale Pediatrico Bambino Gesù
Rome, Italy
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Royal Brompton Hospital
London, United Kingdom
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Sahlgrenska University Hospital, Gothenburg CF center
Gothenburg, Sweden
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UMC Utrecht
Utrecht, Utrecht, 3584 CX, Netherlands
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UZ Leuven
Leuven, Vlaams-Brabant, Belgium
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University Hospital Southampton
Southampton, United Kingdom
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University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
Conditions
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