Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation

Phase 3 Completed 52 enrolled Vertex Pharmaceuticals Incorporated Cystic Fibrosis Foundation · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic adenosine monophosphate (AMP)-dependent protein kinase A (PKA) activation.

Timeline

Start: 2009-08
Primary completion: 2010-11
Completion: 2011-04

Outcome

Met primary endpoint

registry analysis (superiority or other test); Placebo vs 150 mg Ivacaftor q12h; p <0.0001; Mean Difference (Final Values) 12.5 (95% CI 6.6 to 18.3) NCT00909727 ↗

paper patients receiving ivacaftor had a significant increase in percent predicted FEV1 from baseline through Week 24 versus placebo group PMID 23590265 ↗

release “Results from ENVISION showed that children who received KALYDECO (kuh-LYE-deh-koh) experienced rapid and sustained improvements in lung function” vrtx.com ↗

Publications

Davies JC, Wainwright CE, Canny GJ, Chilvers MA, Howenstine MS, Munck A, Mainz JG, Rodriguez S, Li H, Yen K, Ordonez CL, Ahrens R; VX08-770-103 (ENVISION) Study Group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25. doi: 10.1164/rccm.201301-0153OC.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ivacaftor	Other / unclassified	150 mg	Oral