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.

@article{Clancy2012ResultsOA,
  title={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.},
  author={John P Clancy and Steven M Rowe and Frank Accurso and Moira L. Aitken and Raouf S. Amin and Melissa A. Ashlock and Manfred Ballmann and Michael P Boyle and Inez Bronsveld and Preston W. Campbell and Kris de Boeck and Scott Donaldson and Henry L. Dorkin and Jordan M Dunitz and Peter R. Durie and Manu Jain and Anissa Leonard and Karen S Mccoy and Richard B. Moss and Joseph Pilewski and Daniel B Rosenbluth and Ronald C Rubenstein and Michael S Schechter and Martyn C Botfield and Claudia Luc{\'i}a Ord{\'o}{\~n}ez and George T Spencer-Green and Laurent Vernillet and Steve Wisseh and Karl Yen and Michael W Konstan},
  journal={Thorax},
  year={2012},
  volume={67 1},
  pages={12-8}
}
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 were randomised to one of four VX-809 28 day dose groups (25, 50, 100 and 200 mg) or… CONTINUE READING
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VX - 809 , a CFTR corrector , increases the cell surface density of functional F 508 del - CFTR in pre - clinical models of cystic fibrosis [ abstract ]

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