An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

• Status: Completed
• Trial end date: 2018-05-01
• Target enrollment: 0
• Participant gender: Female

Summary

This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Medical University of South Carolina
• Collaborator: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Inclusion Criteria:

• Subjects are >18 years of age and able to provide informed consent.

• Subjects reside in the US and are willing to be treated with ivacaftor.

• Subjects have the splicing mutation of interest.

• Subjects are willing and able to perform requirements of the study.

Exclusion Criteria:

• There are no relevant exclusion criteria for this n-of-2 study.