NCT01531673

Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation

A Phase 2, Multicenter, Double-Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 194 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del-CFTR mutation.

Detailed description

This is a Phase 2, randomized, multicenter, double-blinded, placebo-controlled, study of VX-661 monotherapy, and VX-661/ivacaftor co-therapy in participants with CF who are homozygous or heterozygous for the F508del CFTR mutation. This study is separated into seven groups: Group 1-7, respectively. Approximately 180 participants were randomized in a ratio of 4:1; active drug to matching placebo in each group.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	VX-661
DRUG	Ivacaftor
DRUG	Placebo matched to VX-661
DRUG	Placebo matched to ivacaftor

Timeline

• Start date: 2012-02-01
• Primary completion: 2014-03-01
• Completion: 2014-03-01
• First posted: 2012-02-13
• Last updated: 2018-04-13
• Results posted: 2018-04-13

Locations

36 sites across 4 countries: United States, Canada, Germany, United Kingdom