Clareo Health | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Amgen
Study acquired from Horizon in 2024.
Age
0–2
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Male and female subjects up to 2 years of age
Signed informed consent by subject's parent/legal guardian
UCD diagnosis or suspected diagnosis of any subtype, except N-acetyl glutamate synthetase deficiency. If UCD has not been previously confirmed by genetic testing, consent must be obtained from parent/legal guardian prior to perform genetic testing. If genetic testing is inconsistent with or excludes a UCD diagnosis, the subject will be withdrawn from the study.
You may not be if
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
