Overview

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 an Ivacaftor-Responsive CFTR Mutation

Status:
Recruiting

Trial end date:
2022-07-01

Target enrollment:

Participant gender:

Summary

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 an ivacaftor-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Phase:

Phase 3

Details

Lead Sponsor:

Vertex Pharmaceuticals Incorporated

Treatments:

Ivacaftor