A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 35 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation
Actual Study Start Date: March 2016
Estimated Primary Completion Date: June 2020
Estimated Study Completion Date: June 2020

Arms:
- Experimental: Part A
- Experimental: Part B