BibTex format
@article{Ratjen:2017:10.1016/S2213-2600(17)30215-1,
author = {Ratjen, F and Hug, C and Marigowda, G and Tian, S and Huang, X and Stanojevic, S and Milla, CE and Robinson, PD and Waltz, D and Davies, JC},
doi = {10.1016/S2213-2600(17)30215-1},
journal = {Lancet Respiratory Medicine},
pages = {557--567},
title = {Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled, phase 3 trial},
url = {http://dx.doi.org/10.1016/S2213-2600(17)30215-1},
volume = {5},
year = {2017}
}
RIS format (EndNote, RefMan)
TY - JOUR
AB - BackgroundLumacaftor and ivacaftor combination treatment showed efficacy in patients aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis transmembrane conductance regulator (CFTR) in placebo-controlled studies and patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR in an open-label study. We report efficacy and safety of lumacaftor and ivacaftor in patients with cystic fibrosis aged 6–11 years homozygous for F508del-CFTR.MethodsIn this phase 3, randomised, double-blind, placebo-controlled, multicentre study, patients were enrolled at 54 hospitals and medical centres in nine countries (the USA, Australia, Belgium, Canada, Denmark, France, Germany, Sweden, and the UK). Eligible patients weighed at least 15 kg, with a confirmed diagnosis of cystic fibrosis, percent predicted forced expiratory volume in 1 s (FEV1) of 70 or more, and lung clearance index2·5 (LCI2·5) of 7·5 or more at screening (values less than these thresholds were permitted at day 1). All patients were tested for CFTR genotype at screening; eligible patients had to have the F508del-CFTR mutation on both alleles. Exclusion criteria included any comorbidity or laboratory abnormality that might confound the study results or pose additional risk to the patient. Patients were stratified by weight (<25 kg vs ≥25 kg) and ppFEV1 severity (<90 vs ≥90) determined at the screening visit, and randomly assigned 1:1 to treatment using an interactive web response system to receive 200 mg lumacaftor and 250 mg ivacaftor every 12 hours or placebo for 24 weeks. Patients, all site personnel including the investigator and the site monitor, and the study team were blinded, with the exception of site personnel needing this information in the event of medical emergency or pregnancy and patient safety and regulatory affairs personnel to meet serious adverse event reporting requirements. The primary endpoint was the mean absolute
AU - Ratjen,F
AU - Hug,C
AU - Marigowda,G
AU - Tian,S
AU - Huang,X
AU - Stanojevic,S
AU - Milla,CE
AU - Robinson,PD
AU - Waltz,D
AU - Davies,JC
DO - 10.1016/S2213-2600(17)30215-1
EP - 567
PY - 2017///
SN - 2213-2600
SP - 557
TI - Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled, phase 3 trial
T2 - Lancet Respiratory Medicine
UR - http://dx.doi.org/10.1016/S2213-2600(17)30215-1
UR - http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000404047900020&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
UR - https://www.sciencedirect.com/science/article/pii/S2213260017302151?via%3Dihub
UR - http://hdl.handle.net/10044/1/50570
VL - 5
ER -
