학술논문
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508delmutation: a double-blind, randomised, phase 3 trial
Document Type
Article
Author
Heijerman, Harry G M; McKone, Edward F; Downey, Damian G; Van Braeckel, Eva; Rowe, Steven M; Tullis, Elizabeth; Mall, Marcus A; Welter, John J; Ramsey, Bonnie W; McKee, Charlotte M; Marigowda, Gautham; Moskowitz, Samuel M; Waltz, David; Sosnay, Patrick R; Simard, Christopher; Ahluwalia, Neil; Xuan, Fengjuan; Zhang, Yaohua; Taylor-Cousar, Jennifer L; McCoy, Karen S; McCoy, Karen; Donaldson, Scott; Walker, Seth; Chmiel, James; Rubenstein, Ronald; Froh, Deborah K.; Neuringer, Isabel; Jain, Manu; Moffett, Kathryn; Taylor-Cousar, Jennifer L.; Barnett, Bruce; Mueller, Gary; Flume, Patrick; Livingston, Floyd; Mehdi, Nighat; Teneback, Charlotte; Welter, John; Jain, Raksha; Kissner, Dana; Patel, Kapilkumar; Calimano, Francisco J.; Johannes, Jimmy; Daines, Cori; Keens, Thomas; Scher, Herschel; Chittivelu, Subramanyam; Reddivalam, Sudhakar; Klingsberg, Ross Carl; Johnson, Larry G.; Verhulst, Stijn; Macedo, Patricia; Downey, Damien; Connett, Gary; Nash, Edward; Withers, Nicholas; Lee, Timothy; Bakker, Marleen; Heijerman, Harry; Vermeulen, Francois; Van Braeckel, Eva; Knoop, Christiane; De Wachter, Elke; van der Meer, Renske; Merkus, Petrus; Majoor, Christof
Source
The Lancet; November 2019, Vol. 394 Issue: 10212 p1940-1948, 9p
Subject
Language
ISSN
01406736; 1474547X
Abstract
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved with the combination of a CFTR corrector and potentiator in people with cystic fibrosis homozygous for the F508delmutation. The addition of elexacaftor (VX-445), a next-generation CFTR corrector, to tezacaftor plus ivacaftor further improved F508del-CFTR function and clinical outcomes in a phase 2 study in people with cystic fibrosis homozygous for the F508delmutation.