Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele.

doi:10.1164/rccm.202301-0084OC

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Article

Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele.

American journal of respiratory and critical care medicine - 1 Jul 2023

Goralski Jennifer L, Hoppe Jordana E, Mall Marcus A, McColley Susanna A, McKone Edward, Ramsey Bonnie, Rayment Jonathan H, Robinson Phil, Stehling Florian, Taylor-Cousar Jennifer L, Tullis Elizabeth, Ahluwalia Neil, Chin Anna, Chu Chenghao, Lu Mengdi, Niu Tao, Weinstock Tanya, Ratjen Felix, Rosenfeld Margaret

Abstract excerpt

Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and effective in people with cystic fibrosis (CF) aged ⩾6 years with at least one F508del-CFTR allele but has not been studied in younger children. Objectives: To evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of ELX/TEZ/IVA in children with CF aged 2-5 years. Methods: In this phase 3, open-label, two-part...

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PMID: 36921081
DOI: 10.1164/rccm.202301-0084OC
PMCID: PMC10870849

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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