S63 Ivacaftor in 4- to <6-month-old infants with cystic fibrosis and a gating mutation: results of a 2-part, single-arm, Phase 3 study

Introduction and Objectives ARRIVAL, a single-arm Phase 3 study, characterises pharmacokinetics, safety and tolerability of ivacaftor in children aged Methods Infants received ivacaftor q12h for 4 days (Part A; 5 to Results Twelve infants, six each in A (mean [SD] age, 4.2 [0.98] months) and B (4.5 [0.55] months), received ivacaftor. Pharmacokinetics was consistent with older groups; most AEs were mild/moderate. Most common AE in B was cough (n=3; 50%). Two infants had SAEs (A: thrombocytopenia [suspected causal agent: omeprazole]; B: bronchiolitis), both assessed as not/unlikely related to ivacaftor. No deaths or AEs leading to treatment interruption/discontinuation occurred. No notable transaminase elevations or clinically relevant findings in laboratory tests (except one thrombocytopenia), vital signs or electrocardiogram parameters were reported. Improvements were seen in SwCl and FE-1 (Table). Baseline elevations of serum IRT and lipase, although not amylase, improved. All growth parameters increased on average. Conclusions This first CFTR modulation study in infants aged 4 to Please refer to page A239 for declarations of interest related to this abstract.