NCT01705145

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are 2 Through 5 Years of Age and Have a CFTR Gating Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 2 Years – 5 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in at least 1 allele. Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through 5 years of age.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Ivacaftor

Timeline

• Start date: 2013-01-01
• Primary completion: 2014-03-01
• Completion: 2014-03-01
• First posted: 2012-10-12
• Last updated: 2016-04-05
• Results posted: 2015-04-24

Locations

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