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:
Participant gender:
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.