NCT03277196

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

A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics 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: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 86 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 0 Months – 24 Months
• Healthy volunteers: Not accepted

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 participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	IVA	Granules for oral administration.

Timeline

• Start date: 2017-08-16
• Primary completion: 2023-10-02
• Completion: 2023-10-02
• First posted: 2017-09-08
• Last updated: 2024-10-23
• Results posted: 2024-10-23

Locations

29 sites across 6 countries: United States, Australia, Canada, Germany, Ireland, United Kingdom

Regulatory

• FDA-regulated drug study