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
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  Data: 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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  Data: <searchLink fieldCode="AU" term="%22Sabino+LR%22">Sabino LR</searchLink>; School of Biomedical Sciences and Pharmacy, University of Newcastle, Newcastle, NSW, 2308, Australia.; Immune Health Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia.<br /><searchLink fieldCode="AU" term="%22Kaiko+GE%22">Kaiko GE</searchLink>; School of Biomedical Sciences and Pharmacy, University of Newcastle, Newcastle, NSW, 2308, Australia.; Immune Health Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia.<br /><searchLink fieldCode="AU" term="%22Dorahy+D%22">Dorahy D</searchLink>; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia.<br /><searchLink fieldCode="AU" term="%22Goodsall+T%22">Goodsall T</searchLink>; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia.; Department of Gastroenterology, John Hunter Hospital, Newcastle, NSW, 2305, Australia.<br /><searchLink fieldCode="AU" term="%22Jaffe+A%22">Jaffe A</searchLink>; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW, Sydney, Australia.; Department of Respiratory Medicine, Sydney Children's Hospital, Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Schultz+A%22">Schultz A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Waters+S%22">Waters S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Cook+D%22">Cook D</searchLink>; School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, 2308, Australia.; Department of Gastroenterology, John Hunter Hospital, Newcastle, NSW, 2305, Australia.<br /><searchLink fieldCode="AU" term="%22Kok+H%22">Kok H</searchLink>; Department of Respiratory & Sleep Medicine, John Hunter Hospital, Newcastle, NSW, Australia.; Asthma and Breathing Program, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia.<br /><searchLink fieldCode="AU" term="%22Tong+K%22">Tong K</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Totterdell+J%22">Totterdell J</searchLink>; Sydney School of Public Health, The University of Sydney, Sydney, NSW, 2006, Australia.<br /><searchLink fieldCode="AU" term="%22McGree+J%22">McGree J</searchLink>; School of Mathematical Sciences, Queensland University of Technology, Brisbane, QLD, 4000, Australia.<br /><searchLink fieldCode="AU" term="%22Snelling+T%22">Snelling T</searchLink>; Sydney School of Public Health, The University of Sydney, Sydney, NSW, 2006, Australia.<br /><searchLink fieldCode="AU" term="%22Wark+PAB%22">Wark PAB</searchLink>; 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.
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