A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Status: | Completed |
---|---|
Healthy: | No |
Age Range: | Any - 2 |
Updated: | 3/7/2019 |
Start Date: | December 31, 2014 |
End Date: | July 17, 2017 |
An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)
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.
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.
Inclusion Criteria:
- 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be withdrawn from the study.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 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)
We found this trial at
17
sites
Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Susan Berry, MD
Phone: 612-626-5275
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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425 University Blvd.
Indianapolis, Indiana 46202
Indianapolis, Indiana 46202
(317) 274-4591
Principal Investigator: Bryan Hainline, MD, PhD
Phone: 317-278-6650
Indiana University INDIANA UNIVERSITY is a major multi-campus public research institution, grounded in the liberal...
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South 34th Street
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
215-590-1000
Principal Investigator: Can Ficicioglu, MD, PhD
Phone: 267-425-2111
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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22 Bramhall St
Portland, Maine 04102
Portland, Maine 04102
(207) 662-0111
Principal Investigator: Wendy E Smith, MD
Phone: 207-661-7646
Maine Medical Center One of the country's consistently highest rated hospitals is right in your...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
Portland, Oregon 97239
503 494-8311
Principal Investigator: Cary Harding, MD
Phone: 503-494-4290
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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201 Presidents Circle
Salt Lake City, Utah 84108
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Nicola Longo, MD, PhD
Phone: 801-587-3605
University of Utah Research is a major component in the life of the U benefiting...
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Cleveland, Ohio 44194
Principal Investigator: Shawn McCandless, MD
Phone: 216-844-7124
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Decatur, Georgia 30033
Principal Investigator: Michael J Gambello, MD,PhD
Phone: 404-778-8552
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3901 Beaubien St
Detroit, Michigan 48201
Detroit, Michigan 48201
(313) 745-5437
Principal Investigator: Robert Conway, MD
Phone: 303-832-9261
Children's Hospital of Michigan Since 1886, the Children's Hospital of Michigan has been dedicated to...
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Gainesville, Florida 32610
Principal Investigator: Roberto Zori, MD
Phone: 352-294-5511
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Los Angeles, California 90027
Principal Investigator: Rebecca Mardach, MD
Phone: 323-783-3253
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New York, New York 10029
Principal Investigator: George Diaz, MD
Phone: 212-659-1477
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Palo Alto, California 94304
Principal Investigator: William Berquist, MD
Phone: 650-736-0644
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3414 Fifth Avenue
Pittsburgh, Pennsylvania 15213
Pittsburgh, Pennsylvania 15213
Principal Investigator: Gerard Vockley, MD, PhD
Phone: 412-692-6378
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4800 Sand Point Way NE
Seattle, Washington 98105
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Lawrence Merritt, MD
Phone: 206-884-1264
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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555 University Avenue
Toronto, M5G 1X8
Toronto, M5G 1X8
Principal Investigator: Andreas Schulze, MD,PhD
Phone: 416-813-7654
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111 Michigan Ave NW
Washington, District of Columbia
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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