@article{Rosenfeld:2019:10.1016/j.jcf.2019.03.009,
author = {Rosenfeld, M and Cunningham, S and Harris, WT and Lapey, A and Regelmann, WE and Sawicki, GS and Southern, KW and Chilvers, M and Higgins, M and Tian, S and Cooke, J and Davies, JC and KLIMB, study group},
doi = {10.1016/j.jcf.2019.03.009},
journal = {Journal of Cystic Fibrosis},
pages = {838--843},
title = {An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5years (KLIMB).},
url = {http://dx.doi.org/10.1016/j.jcf.2019.03.009},
volume = {18},
year = {2019}
}

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TY  - JOUR
AB  - BACKGROUND: KIWI (NCT01705145) was a 24-week, single-arm, pharmacokinetics, safety, and efficacy study of ivacaftor in children aged 2 to 5years with cystic fibrosis (CF) and a CFTR gating mutation. Here, we report the results of KLIMB (NCT01946412), an 84-week, open-label extension of KIWI. METHODS: Children received age- and weight-based ivacaftor dosages for 84weeks. The primary outcome was safety. Other outcomes included sweat chloride, growth parameters, and measures of pancreatic function. RESULTS: All 33 children who completed KIWI enrolled in KLIMB; 28 completed 84weeks of treatment. Most adverse events were consistent with those reported during KIWI. Ten (30%) children had transaminase elevations >3×upper limit of normal (ULN), leading to 1 discontinuation in a child with alanine aminotransferase >8×ULN. Improvements in sweat chloride, weight, and body mass index z scores and fecal elastase-1 observed during KIWI were maintained during KLIMB; there was no further improvement in these parameters. CONCLUSIONS: Ivacaftor was generally well tolerated for up to 108weeks in children aged 2 to 5years with CF and a gating mutation, with safety consistent with the KIWI study. Improvements in sweat chloride and growth parameters during the initial 24weeks of treatment were maintained for up to an additional 84weeks of treatment. Prevalence of raised transaminases remained stable and did not increase with duration of exposure during the open-label extension.
AU  - Rosenfeld,M
AU  - Cunningham,S
AU  - Harris,WT
AU  - Lapey,A
AU  - Regelmann,WE
AU  - Sawicki,GS
AU  - Southern,KW
AU  - Chilvers,M
AU  - Higgins,M
AU  - Tian,S
AU  - Cooke,J
AU  - Davies,JC
AU  - KLIMB,study group
DO  - 10.1016/j.jcf.2019.03.009
EP  - 843
PY  - 2019///
SN  - 1569-1993
SP  - 838
TI  - An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5years (KLIMB).
T2  - Journal of Cystic Fibrosis
UR  - http://dx.doi.org/10.1016/j.jcf.2019.03.009
UR  - https://www.ncbi.nlm.nih.gov/pubmed/31053538
UR  - https://www.sciencedirect.com/science/article/pii/S156919931930061X?via%3Dihub
UR  - http://hdl.handle.net/10044/1/71513
VL  - 18
ER  - 

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