NCT01946412

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will further explore the efficacy of long-term ivacaftor treatment in this population of patients with CF.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Ivacaftor

Timeline

• Start date: 2013-12-01
• Primary completion: 2015-12-01
• Completion: 2015-12-01
• First posted: 2013-09-19
• Last updated: 2017-02-01
• Results posted: 2017-02-01

Locations

16 sites across 3 countries: United States, Canada, United Kingdom