drugset / Trial / 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

NCT03277196

Phase 3 Completed 86 enrolled Vertex Pharmaceuticals Incorporated
Non-randomizedParallel-groupOpen-labelTreatment

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.

Timeline

Start
2017-08-16
Primary completion
2023-10-02
Completion
2023-10-02

Drugs

EvaluationDrugModalityDoseRoute
Subject Ivacaftor Other / unclassified 25 mg Oral
Subject Ivacaftor Other / unclassified 50 mg Oral
Subject Ivacaftor Other / unclassified 75 mg Oral

Indications