drugset / Trial / NCT02934698
An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations
Phase 3
Completed
2 enrolled
Medical University of South Carolina
Vertex Pharmaceuticals Incorporated · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2016-11-01
- Primary completion
- 2018-05
- Completion
- 2018-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ivacaftor | Other / unclassified | — | — |