Article

Organoid Guided N-of-1 (ORIGIN-1) Clinical Trial: A phase 4 study to investigate if people with cystic fibrosis with rare non-F508del CFTR mutations with an in-vitro response to Elexacaftor/ Tezacaftor/ Ivacaftor have a clinically meaningful response to Elexacaftor/ Tezacaftor/ Ivacaftor versus placebo.

2026-01-21

Abstract excerpt

<title>Abstract</title> <p> <bold>Background</bold> : Cystic Fibrosis (CF) is a genetic condition caused by mutation of the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. Recently licensed modulator therapies target the defective CFTR protein and have transformed the formerly life-limiting trajectory of people with CF (pwCF). elexacaftor/tezacaftor/ivacaftor (ETI) has shown outstanding clinical efficacy i...

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Literature Corpus work: 49cfb500-621c-5c1c-a243-082f1a9097a1
DOI: 10.21203/rs.3.rs-7787221/v1

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Organoid Guided N-of-1 (ORIGIN-1) Clinical Trial: A phase 4 study to investigate if people with cystic fibrosis with rare non-F508del CFTR mutations with an in-vitro response to Elexacaftor/ Tezacaftor/ Ivacaftor have a clinically meaningful response to Elexacaftor/ Tezacaftor/ Ivacaftor versus placebo.DOI 10.21203/rs.3.rs-7787221/v1Read

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