Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

Summary

The purpose of this study was to evaluate the safety and efficacy of ivacaftor in participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. Ivacaftor is a potent and selective CFTR 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

2009-09

Primary completion

2010-07

Completion

2013-05

Outcome

Missed primary endpoint

Stopped: “Following review of results obtained from a pre-specified 6-month analysis of Part B data the study was terminated on the basis of futility.”

registry p = 0.1509; Mean Difference (Final Values) 1.7 (95% CI -0.6 to 4.1) NCT00953706 ↗

paper between the ivacaftor and placebo groups was 1.7% (P = .15). PMID 22383668 ↗

Publications

• Flume PA, Liou TG, Borowitz DS, Li H, Yen K, Ordonez CL, Geller DE; VX 08-770-104 Study Group. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. Chest. 2012 Sep;142(3):718-724. doi: 10.1378/chest.11-2672.

Drugs

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

Indications