Article
Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.
Thorax - 1 Jan 2012
Clancy J P, Rowe Steven M, Accurso Frank J, Aitken Moira L, Amin Raouf S, Ashlock Melissa A, Ballmann Manfred, Boyle Michael P, Bronsveld Inez, Campbell Preston W, De Boeck Kris, Donaldson Scott H, Dorkin Henry L, Dunitz Jordan M, Durie Peter R, Jain Manu, Leonard Anissa, McCoy Karen S, Moss Richard B, Pilewski Joseph M, Rosenbluth Daniel B, Rubenstein Ronald C, Schechter Michael S, Botfield Martyn, Ordoñez Claudia L, Spencer-Green George T, Vernillet Laurent, Wisseh Steve, Yen Karl, Konstan Michael W
Abstract excerpt
BACKGROUND: VX-809, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, has been shown to increase the cell surface density of functional F508del-CFTR in vitro. METHODS: A randomised, double-blind, placebo-controlled study evaluated the safety, tolerability and pharmacodynamics of VX-809 in adult patients with cystic fibrosis (n=89) who were homozygous for the F508del-CFTR mutation. Subjects...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.