Overview

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

Status:
Completed
Trial end date:
2013-03-01
Target enrollment:
Participant gender:
Summary
This non-randomized, open-label study was approximately one year in duration and consisted of a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a 12-month long term treatment period involving monthly visits.
Phase:
Phase 3
Details
Lead Sponsor:
Horizon Pharma Ireland, Ltd., Dublin Ireland
Treatments:
4-phenylbutyric acid
Glycerol