A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant.

doi:10.1016/j.jcf.2022.02.003

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A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jul 2022

Sawicki Gregory S, Chilvers Mark, McNamara John, Naehrlich Lutz, Saunders Clare, Sermet-Gaudelus Isabelle, Wainwright Claire E, Ahluwalia Neil, Campbell Daniel, Harris R Scott, Paz-Diaz Hildegarde, Shih Judy L, Davies Jane C

Abstract excerpt

BACKGROUND: Two previous Phase 3 studies ("parent studies") showed that tezacaftor/ivacaftor was generally safe and efficacious for up to 24 weeks in children 6 through 11 years of age with cystic fibrosis (CF) and F508del/F508del (F/F) or F508del/residual function (F/RF) genotypes. We assessed the safety and efficacy of tezacaftor/ivacaftor in an open-label, 96-week extension study. METHODS: This was a Phase 3,...

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PMID: 35190292
DOI: 10.1016/j.jcf.2022.02.003

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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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