Overview

A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

Status:
Enrolling by invitation

Trial end date:
2024-07-03

Target enrollment:

Participant gender:

Summary

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation

Phase:

Phase 3

Details

Lead Sponsor:

Vertex Pharmaceuticals Incorporated

Treatments:

Ivacaftor