@article{Rosenfeld:2018:10.1016/S2213-2600(18)30202-9,
author = {Rosenfeld, M and Wainwright, CE and Higgins, M and Wang, LT and McKee, C and Campbell, D and Tian, S and Schneider, J and Cunningham, S and Davies, JC and ARRIVAL, study group},
doi = {10.1016/S2213-2600(18)30202-9},
journal = {Lancet Respiratory Medicine},
pages = {545--553},
title = {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},
url = {http://dx.doi.org/10.1016/S2213-2600(18)30202-9},
volume = {6},
year = {2018}
}

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TY  - JOUR
AB  - 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 <24 months. METHODS: The ARRIVAL study is a phase 3, single-arm, two-part, multicentre study. Eligible children were aged 12 to <24 months at enrolment and had a confirmed diagnosis of cystic fibrosis and a 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 <14 kg) or 75 mg (bodyweight ≥14 to <25 kg) ivacaftor orally every 12 h. In study part A, children received ivacaftor for 3 days plus one morning. In study part B, children received 24 weeks of treatment. Children were enrolled into part A at seven sites in Australia (one site), the UK (one), and the USA (five) and into part B at 13 sites in Australia (two sites), Canada (one), the UK (three), and the USA (seven). Primary endpoints were pharmacokinetics (part A) and safety (parts A and B) in children who received at least one dose of ivacaftor. Secondary endpoints in part B were pharmacokinetics in children who received at least one dose of ivacaftor and absolute change from baseline in sweat chloride concentration. We also explored changes in growth parameters and markers of pancreatic function. This study is registered with ClinicalTrials.gov, number NCT02725567. FINDINGS: Children aged 12 to <24 months were enrolled between Aug 25, 2016, and Nov 1, 2017. Seven children were enrolled in part A, of whom five received 50 mg and two received 75 mg ivacaftor. All completed treatment. Of 19 children enrolled in part B, including one from part A, all received 50 mg ivacaftor and 18 completed treatment (one withdrew because of difficulty with blood draws). All children received at least one dose of ivacaftor. Pharmacokinetics indicated exposure was similar to that in children aged 2 to <6 years and adults. No children discont
AU  - Rosenfeld,M
AU  - Wainwright,CE
AU  - Higgins,M
AU  - Wang,LT
AU  - McKee,C
AU  - Campbell,D
AU  - Tian,S
AU  - Schneider,J
AU  - Cunningham,S
AU  - Davies,JC
AU  - ARRIVAL,study group
DO  - 10.1016/S2213-2600(18)30202-9
EP  - 553
PY  - 2018///
SN  - 2213-2600
SP  - 545
TI  - 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
T2  - Lancet Respiratory Medicine
UR  - http://dx.doi.org/10.1016/S2213-2600(18)30202-9
UR  - https://www.ncbi.nlm.nih.gov/pubmed/29886024
UR  - http://hdl.handle.net/10044/1/60167
VL  - 6
ER  - 

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