Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR : a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial

Autor: Sivagurunathan Sutharsan, Edward F McKone, Damian G Downey, Jamie Duckers, Gordon MacGregor, Elizabeth Tullis, Eva Van Braeckel, Claire E Wainwright, Danie Watson, Neil Ahluwalia, Bote G Bruinsma, Christopher Harris, Anna P Lam, Yiyue Lou, Samuel M Moskowitz, Simon Tian, Jason Yuan, David Waltz, Marcus A Mall, Paul Aurora, Stijn Verhulst, Michael Lorenz, Jobst Roehmel, Wolfgang Gleiber, Susanne Naehrig, Florian Stehling, Silke van Koningsbruggen-Rietschel, Rainald Fischer, Damian Downey, Charles Haworth, Julian Legg, Peter Barry, Rebecca Thursfield, Simon James Doe, Tom Hilliard, Edward F Nash, Nicholas John Withers, Daniel Peckham, Helen Louise Barr, Timothy Lee, Robert Gray, Francois Vermeulen, Eef Vanderhelst, Philip J Robinson, Daniel J Smith, Siobhain A Mulrennan, Barry S Clements, Peter Wark

Publikováno v: VX18-445-109 study group 2021, ' Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial ', The Lancet. Respiratory medicine . https://doi.org/10.1016/S2213-2600(21)00454-9

BackgroundElexacaftor plus tezacaftor plus ivacaftor is a triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be generally safe and efficacious in people with cystic fibrosis aged 12 years or older

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