Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders
Status:
Recruiting
Trial end date:
2023-03-01
Target enrollment:
Participant gender:
Summary
This is a randomized, controlled, open-label parallel arm study to assess the safety,
tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to NaPBA in urea
cycle disorder subjects not currently or previously chronically treated with phenylacetic
acid (phenylacetate; PAA) prodrugs. The study design will include: 1) Baseline Period; 2)
Initial Treatment Period; 3) a RAVICTI only Transition Period 4) a RAVICTI only Maintenance
Period; and 5) a RAVICTI only Safety Extension Period. The study will run for approximately
25 weeks.