04Jul 2023
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

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

A 24-week phase 3 study evaluated the safety and effectiveness of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in children aged 6-11 with cystic fibrosis (CF) and specific genetic mutations. The study aimed to assess the long-term safety and efficacy of ELX/TEZ/IVA in children who completed the initial 24-week trial. The open-label extension study included children with CF who received ELX/TEZ/IVA based on their weight. The results showed that ELX/TEZ/IVA remained safe and well-tolerated for up to 96 weeks of treatment. The treatment led to improvements in lung function, respiratory symptoms, and CFTR function, demonstrating the sustained clinical benefits of ELX/TEZ/IVA in this pediatric population.

  • #pulmonology

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