Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study

Summary Background Ivacaftor is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. We assessed its use in children aged 12 to Methods The ARRIVAL study is a phase 3, single-arm, two-part, multicentre study. Eligible children were aged 12 to CFTR gating mutation on at least one allele and could participate in one or both parts of the study. Children received 50 mg (bodyweight 7 to Findings Children aged 12 to Interpretation Ivacaftor was generally safe and well tolerated in children aged 12 to Funding Vertex Pharmaceuticals Incorporated.