Organoid-Guided drug trial offers hope for cystic fibrosis patients with rare mutations
NCT ID NCT06468527
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a triple-drug combination (dirocaftor/posenacaftor/nesolicaftor) in 41 adults with cystic fibrosis who have rare CFTR gene mutations not covered by existing treatments. Participants were chosen based on lab tests using their own intestinal organoids to predict who might benefit. The goal was to see if the drugs improve lung function and other measures like sweat chloride and weight.
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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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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
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Other studies related to the condition(s) this trial covers.
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