A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation.

doi:10.1016/j.jcf.2020.07.023

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A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2021

Davies Jane C, Sermet-Gaudelus Isabelle, Naehrlich Lutz, Harris R Scott, Campbell Daniel, Ahluwalia Neil, Short Christopher, Haseltine Eric, Panorchan Paul, Saunders Clare, Owen Caroline A, Wainwright Claire E

Abstract excerpt

BACKGROUND: The CFTR modulator tezacaftor/ivacaftor was efficacious and generally safe and well tolerated in Phase 3 studies in participants ≥12 years of age with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation or heterozygous with a residual function-CFTR mutation (F/F or F/RF respectively). We evaluated tezacaftor/ivacaftor's efficacy and safety over 8 weeks in participants 6 through 11 years of...

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PMID: 32967799
DOI: 10.1016/j.jcf.2020.07.023

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