Article
Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ⩾6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial.
American journal of respiratory and critical care medicine - 1 Jul 2023
Wainwright Claire, McColley Susanna A, McNally Paul, Powers Michael, Ratjen Felix, Rayment Jonathan H, Retsch-Bogart George, Roesch Erica, Ahluwalia Neil, Chin Anna, Chu Chenghao, Lu Mengdi, Menon Prema, Waltz David, Weinstock Tanya, Zelazoski Laura, Davies Jane C
Abstract excerpt
Rationale: A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children aged 6-11 years with cystic fibrosis (CF) and one or more F508del-CFTR alleles. Objectives: To assess long-term safety and efficacy of ELX/TEZ/IVA in children who completed the pivotal 24-week phase 3 trial. Methods: In this phase 3, two-part (part A and part B), open-label...
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