A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

Exclusion Criteria

• •Use of any investigational drug within 30 days of Day 1
• •Uncontrolled infection (viral or bacterial) or any other condition known to precipitate hyperammonemic crises. Once these precipitating factors are medically controlled, patients presenting in crisis are eligible.
• •Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.03, except Grade 3 elevations in ammonia and liver enzymes, defined as levels 5-20 times the upper limit of normal in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable subject
• •Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the subject at increased risk by participating in this study
• •Known hypersensitivity to phenylacetate (PAA) or phenylbutyrate (PBA)
• •Liver transplantation, including hepatocellular transplant
• •Subjects on hemodialysis at time of initiating RAVICTI
• •Subjects on RAVICTI for UCD management
• •Currently treated with Carbaglu® (carglumic acid)