Please use this identifier to cite or link to this item:
https://dora.health.qld.gov.au/qldresearchjspui/handle/1/3583
Title: | Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for phe508del CFTR | Authors: | Munck, A. Wainwright, Claire Elborn, J. S. Ramsey, B. W. Marigowda, G. Huang, X. Cipolli, M. Colombo, C. Davies, J. C. De Boeck, K. Flume, P. A. Ratjen, F. Boyle, M. P. Waltz, D. Rowe, S. M. Konstan, M. W. McColley, S. A. McCoy, K. McKone, E. F. |
Issue Date: | 2015 | Source: | 373, (3), 2015, p. 220-231 | Pages: | 220-231 | Journal: | New England Journal of Medicine | Abstract: | BACKGROUND: Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the most common CFTR mutation. METHODS: We conducted two phase 3, randomized, double-blind, placebo-controlled studies that were designed to assess the effects of lumacaftor (VX-809), a CFTR corrector, in combination with ivacaftor (VX-770), a CFTR potentiator, in patients 12 years of age or older who had cystic fibrosis and were homozygous for the Phe508del CFTR mutation. In both studies, patients were randomly assigned to receive either lumacaftor (600 mg once daily or 400 mg every 12 hours) in combination with ivacaftor (250 mg every 12 hours) or matched placebo for 24 weeks. The primary end point was the absolute change from baseline in the percentage of predicted forced expiratory volume in 1 second (FEV1) at week 24. RESULTS: A total of 1108 patients underwent randomization and received study drug. The mean baseline FEV | DOI: | 10.1056/NEJMoa1409547 | Resources: | https://www.embase.com/search/results?subaction=viewrecord&id=L605207920&from=exporthttp://dx.doi.org/10.1056/NEJMoa1409547 | | Keywords: | hemoptysis;homozygosity;human;hypertransaminasemia;lung disease;male;middle aged;molecular pathology;nausea;nose obstruction;oropharynx pain;outcome assessment;phase 3 clinical trial;prediction;predictive value;priority journal;randomized controlled trial;rash;rhinopharyngitis;school child;upper respiratory tract infection;vx 770;vx 809;syndrome;NCT01807923NCT01807949;cystic fibrosis transmembrane conductance regulator;ivacaftor;lumacaftor;placebo;adult;article;bronchospasm;chest tightness;child;clinical effectiveness;controlled study;coughing;cystic fibrosis;cystic fibrosis transmembrane conductance regulator gene;diarrhea;disease exacerbation;distal intestinal obstruction syndrome;double blind procedure;drug efficacy;drug fatality;drug response;drug safety;drug tolerability;drug withdrawal;dyspnea;female;forced expiratory volume;gene;gene mutation;headache | Type: | Article |
Appears in Sites: | Children's Health Queensland Publications |
Show full item record
Items in DORA are protected by copyright, with all rights reserved, unless otherwise indicated.