NCT01807949

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 563 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older 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 3, randomized, double-blind, placebo-controlled, parallel-group multicenter study of orally administered lumacaftor in combination with ivacaftor in participants aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation. The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1 \[first dose of study drug\] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7 days after the Week 24 Visit).

Conditions

• Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Interventions

Type	Name	Description
DRUG	Placebo	Matching placebo tablet
DRUG	Lumacaftor Plus Ivacaftor Combination	Fixed dose combination tablet
DRUG	Ivacaftor	Film-coated tablet

Timeline

• Start date: 2013-04-01
• Primary completion: 2014-04-01
• Completion: 2014-04-01
• First posted: 2013-03-08
• Last updated: 2016-09-27
• Results posted: 2015-09-01

Locations

82 sites across 10 countries: United States, Australia, Austria, Belgium, Canada, Denmark, France, Germany, Spain, United Kingdom