A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
NCT ID: NCT02246218
Last Updated: 2024-07-01
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE4
27 participants
INTERVENTIONAL
2014-12-31
2017-07-17
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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RAVICTI
RAVICTI Oral Liquid should be administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
RAVICTI
Interventions
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RAVICTI
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 If genetic testing is inconsistent with or excludes a UCD diagnosis, the subject will be withdrawn from the study.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 Criteria
* 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)
1 Day
2 Years
ALL
No
Sponsors
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Amgen
INDUSTRY
Responsible Party
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Principal Investigators
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MD
Role: STUDY_DIRECTOR
Amgen
Locations
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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
Countries
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References
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Berry SA, Longo N, Diaz GA, McCandless SE, Smith WE, Harding CO, Zori R, Ficicioglu C, Lichter-Konecki U, Robinson B, Vockley J. Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years. Mol Genet Metab. 2017 Nov;122(3):46-53. doi: 10.1016/j.ymgme.2017.09.002. Epub 2017 Sep 8.
Berry SA, Vockley J, Vinks AA, Dong M, Diaz GA, McCandless SE, Smith WE, Harding CO, Zori R, Ficicioglu C, Lichter-Konecki U, Perdok R, Robinson B, Holt RJ, Longo N. Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders. Mol Genet Metab. 2018 Nov;125(3):251-257. doi: 10.1016/j.ymgme.2018.09.001. Epub 2018 Sep 4.
Related Links
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Related Info
Other Identifiers
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2016-003460-38
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
HPN-100-009
Identifier Type: -
Identifier Source: org_study_id
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