NCT00865904

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ∆F508-CFTR Gene Mutation

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 93 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Detailed description

This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	VX-809	Capsules
DRUG	Placebo	Placebo matched to VX-809 capsules.

Timeline

• Start date: 2009-03-01
• Primary completion: 2009-12-01
• Completion: 2009-12-01
• First posted: 2009-03-19
• Last updated: 2015-08-28
• Results posted: 2015-08-28

Locations

25 sites across 5 countries: United States, Belgium, Canada, Germany, Netherlands