ORIGIN-1 trial: study protocol for an organoid-guided N-of-1 trial evaluating CFTR modulator response in cystic fibrosis with rare non-F508del mutations.

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Title: ORIGIN-1 trial: study protocol for an organoid-guided N-of-1 trial evaluating CFTR modulator response in cystic fibrosis with rare non-F508del mutations.
Authors: Sabino LR; School of Biomedical Sciences and Pharmacy, University of Newcastle, Newcastle, NSW, 2308, Australia.; Immune Health Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia., Kaiko GE; School of Biomedical Sciences and Pharmacy, University of Newcastle, Newcastle, NSW, 2308, Australia.; Immune Health Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia., Dorahy D; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia., Goodsall T; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia.; Department of Gastroenterology, John Hunter Hospital, Newcastle, NSW, 2305, Australia., Jaffe A; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW, Sydney, Australia.; Department of Respiratory Medicine, Sydney Children's Hospital, Sydney, NSW, Australia., Schultz A; Wal-Yan Respiratory Research Centre, The Kids Research Institute, University of Western Australia, Perth, Australia.; Department of Respiratory and Sleep Medicine, Perth Children's Hospital, Perth, Australia., Waters S; Department of Respiratory Medicine, Sydney Children's Hospital, Sydney, NSW, Australia.; School of Biomedical Sciences, Faculty of Medicine and Health, UNSW Sydney, Sydney, NSW, Australia., Cook D; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia.; Department of Gastroenterology, John Hunter Hospital, Newcastle, NSW, 2305, Australia., Kok H; Department of Respiratory & Sleep Medicine, John Hunter Hospital, Newcastle, NSW, Australia.; Asthma and Breathing Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia., Tong K; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia.; Department of Respiratory & Sleep Medicine, John Hunter Hospital, Newcastle, NSW, Australia.; Asthma and Breathing Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia., Totterdell J; Sydney School of Public Health, The University of Sydney, Sydney, NSW, 2006, Australia., McGree J; School of Mathematical Sciences, Queensland University of Technology, Brisbane, QLD, 4000, Australia., Snelling T; Sydney School of Public Health, The University of Sydney, Sydney, NSW, 2006, Australia., Wark PAB; Department of Respiratory Medicine, Alfred Health, Melbourne, VIC, 3181, Australia. P.Wark@alfred.org.au.; School of Translational Medicine, Monash University, Melbourne, Australia. P.Wark@alfred.org.au.
Source: Trials [Trials] 2026 Jun 15. Date of Electronic Publication: 2026 Jun 15.
Publication Type: Journal Article
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101263253 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1745-6215 (Electronic) Linking ISSN: 17456215 NLM ISO Abbreviation: Trials Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1745-6215
DOI:10.1186/s13063-026-09789-y