Status:
COMPLETED
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Lead Sponsor:
Amgen
Conditions:
Urea Cycle Disorder
Eligibility:
All Genders
1-2 years
Phase:
PHASE4
Brief Summary
This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at...
Detailed Description
Study acquired from Horizon in 2024.
Eligibility Criteria
Inclusion
- 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 If genetic testing is inconsistent with or excludes a UCD diagnosis, the subject will be withdrawn from the study.
Exclusion
- 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)
Key Trial Info
Start Date :
December 31 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 17 2017
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT02246218
Start Date
December 31 2014
End Date
July 17 2017
Last Update
July 1 2024
Active Locations (17)
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1
Kaiser Permanente Regional Metabolic Center
Los Angeles, California, United States, 90027
2
Stanford Center for Clinical & Translational Research & Education
Palo Alto, California, United States, 94034
3
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
4
Shands at University of Florida
Gainesville, Florida, United States, 32610