NCT00909532

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 167 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 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 AMP-dependent protein kinase A (PKA) activation.

Detailed description

This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%. Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation. This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Ivacaftor	150-mg tablets given orally q12h for up to 48 weeks
DRUG	Placebo	Tablet given orally q12h for up to 48 weeks

Timeline

• Start date: 2009-06-01
• Primary completion: 2010-07-01
• Completion: 2012-11-01
• First posted: 2009-05-28
• Last updated: 2013-01-18
• Results posted: 2012-08-21

Locations

65 sites across 8 countries: United States, Australia, Canada, Czechia, France, Germany, Ireland, United Kingdom