Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation

Phase 2 Completed 21 enrolled Vertex Pharmaceuticals Incorporated Cystic Fibrosis Foundation · collab

RandomizedCrossoverQuadruple-blindTreatment

Summary

The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.

Timeline

Start: 2011-01
Primary completion: 2011-11
Completion: 2011-11

Outcome

Met primary endpoint

paper Treatment with ivacaftor led to significant improvements compared with placebo in LCI PMID 24461666 ↗

paper difference between groups in the average of mean changes from baseline at days 15 and 29 was -2·16 [95% CI -2·88 to -1·44]; p<0·0001 PMID 24461666 ↗

Publications

Davies J, Sheridan H, Bell N, Cunningham S, Davis SD, Elborn JS, Milla CE, Starner TD, Weiner DJ, Lee PS, Ratjen F. Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med. 2013 Oct;1(8):630-638. doi: 10.1016/S2213-2600(13)70182-6. Epub 2013 Sep 10.

Drugs

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