Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F15%3A10316251" target="_blank" >RIV/00216208:11130/15:10316251 - isvavai.cz</a>
Alternative codes found
RIV/00064203:_____/15:10316251
Result on the web
<a href="http://dx.doi.org/10.1056/NEJMoa1409547" target="_blank" >http://dx.doi.org/10.1056/NEJMoa1409547</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1056/NEJMoa1409547" target="_blank" >10.1056/NEJMoa1409547</a>
Alternative languages
Result language
angličtina
Original language name
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Original language description
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 24weeks. The primary end point was the absolute change from baseline in the percentage of predicted forced expiratory volume in 1 second (FEV 1) at week 24. RESULTS A total of 1108 patients underwent randomization and received study drug.
Czech name
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Czech description
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Classification
Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FC - Pneumology
OECD FORD branch
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Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2015
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
New England Journal of Medicine
ISSN
0028-4793
e-ISSN
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Volume of the periodical
373
Issue of the periodical within the volume
3
Country of publishing house
US - UNITED STATES
Number of pages
12
Pages from-to
220-231
UT code for WoS article
000357956200005
EID of the result in the Scopus database
2-s2.0-84937035647