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Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: A double-blind, randomized, phase 3 trial

The Lancet Nov 28, 2019

Heijerman HGM, McKone EF, Downey DG, et al. - In this phase 3, multicentre, randomized, double-blind, active-controlled trial of elexacaftor in combination with tezacaftor plus ivacaftor conducted at 44 sites in four countries of 113 individuals with cystic fibrosis homozygous for the F508del mutation, aged 12 years or older with stable disease, and with a percentage prognosticated forced expiratory volume in 1 of 40–90%, inclusive, experts ascertained the efficiency and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in individuals with cystic fibrosis homozygous for the F508del mutation. In comparison with tezacaftor plus ivacaftor alone, in the lives of persons with cystic fibrosis who are homozygous for the F508del mutation, a clinically strong advantage and a favorable safety profile by elexacaftor plus tezacaftor plus ivacaftor along with the potential to result in transformative developments was given.
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