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

Completed · Phase 4

Conditions studied: Urea Cycle Disorder

In brief

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 enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children. Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to \< 2 years of age with either a diagnosed or clinically suspected UCD.

Key facts

Study ID: NCT02246218
Run by: Amgen
People needed: 27
Starts: 2014-12-31
Expected to finish: 2017-07-17
Last updated by the study team: 2024-07-01

Who can join

Age: 0 and older, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify 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 If genetic testing is inconsistent with or excludes a UCD diagnosis, the subject will be withdrawn from the study.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 qualify 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
Subjects on RAVICTI for UCD management
Currently treated with Carbaglu® (carglumic acid)

Where it is running

Kaiser Permanente Regional Metabolic Center — Los Angeles, California, United States
Stanford Center for Clinical & Translational Research & Education — Palo Alto, California, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
Shands at University of Florida — Gainesville, Florida, United States
Emory University — Decatur, Georgia, United States
Indiana University — Indianapolis, Indiana, United States
Maine Medical Center — Portland, Maine, United States
Children's Hospital of Michigan — Detroit, Michigan, United States
University of Minnesota — Minneapolis, Minnesota, United States
Mount Sinai School of Medicine — New York, New York, United States
University Hospitals Case Medical Center — Cleveland, Ohio, United States
Oregon Health & Science University — Portland, Oregon, United States
Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
University of Utah — Salt Lake City, Utah, United States
Seattle Children's Hospital — Seattle, Washington, United States
The Hospital for Sick Children — Toronto, Canada

Full record on ClinicalTrials.gov

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