To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs) - Full Text View

Purpose

The present protocol is a treatment protocol to allow continued use of HPN-100 for the treatment of UCD subjects who complete 12 months of treatment in Study HPN-100-005, HPN-100-007, or HPN-100-012. Under this protocol, long-term safety will continue to be assessed.

Condition	Intervention	Phase
Urea Cycle Disorders	Drug: HPN-100	Phase 4


Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Long Term Use of HPN-100 in Urea Cycle Disorders

Resource links provided by NLM:

Genetics Home Reference related topics: argininosuccinic aciduria citrullinemia N-acetylglutamate synthase deficiency ornithine translocase deficiency succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Carbamide Carbamide peroxide Glycerol phenylbutyrate

Genetic and Rare Diseases Information Center resources: Inborn Amino Acid Metabolism Disorder Urea Cycle Disorders

U.S. FDA Resources

Further study details as provided by Hyperion Therapeutics, Inc.:

Primary Outcome Measures:

Rate of Adverse Events [ Time Frame: Every six months for 2 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Venous ammonia levels [ Time Frame: Every six months for two years ] [ Designated as safety issue: Yes ]
Number of hyperammonemic crises [ Time Frame: Every six months for two years ] [ Designated as safety issue: Yes ]
Neuropsychological testing [ Time Frame: Every twelve months for two years ] [ Designated as safety issue: Yes ]
Wechsler Abbreviated Scale of Intelligence (WASI) The California Verbal Learning Test (CVLT) Digit Span The Child Behavior Checklist (CBCL) Behavior Rating Inventory of Executive Function® (BRIEF®)
Causes of hyperammonemic crises [ Time Frame: Every six month for two years ] [ Designated as safety issue: Yes ]


Enrollment:	88
Study Start Date:	October 2010
Estimated Study Completion Date:	June 2014
Primary Completion Date:	June 2013 (Final data collection date for primary outcome measure)

Intervention Details:

HPN-100 will be administered orally three times daily (TID) with meals. The maximum recommended dose of HPN-100 in subjects weighing less than 20 kg is 0.53 mL/kg/day (equivalent to 600 mg/kg/day of NaPBA), and is 11.48 mL/m2/day in heavier subjects (equivalent to 13g/m2/day of NaPBA). The maximum HPN-100 dose should be 17.4 mL/day, which is equivalent to 20 g/day of NaPBA.

Eligibility


Ages Eligible for Study:	1 Year and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male and female subjects who completed HPN-100-005, HPN-100-007, or HPN-100-012
Signed informed consent by subject and/or subject's legally authorized representative
Negative pregnancy test for all females of childbearing potential

Exclusion Criteria:

Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the subject at increased risk when participating
Known hypersensitivity to PAA or PBA
Liver transplant, including hepatocellular transplant
Pregnant, breastfeeding or lactating females

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01257737

Locations


United States, California
UCLA Pediatrics/Genetics
Los Angeles, California, United States
Stanford University School of Medicine
Palo Alto, California, United States
United States, Colorado
Denver Children's Hospital
Aurora, Colorado, United States
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States
United States, Maine
Maine Medical Center
Portland, Maine, United States
United States, Minnesota
University of Minnesota Medical Center
Minneapolis, Minnesota, United States
United States, New York
Mount Sinai School of Medicine
New York, New York, United States
United States, Ohio
University Hospitals Case Medical Center
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States
United States, Pennsylvania
Children's Hospital of Pittsburg of UPMC
Pittsburg, Pennsylvania, United States
United States, Texas
Baylor College of Medicine
Houston, Texas, United States
United States, Utah
University of Utah
Salt Lake City, Utah, United States
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8

Sponsors and Collaborators

Hyperion Therapeutics, Inc.

More Information

No publications provided


Responsible Party:	Hyperion Therapeutics, Inc.
ClinicalTrials.gov Identifier:	NCT01257737 History of Changes
Other Study ID Numbers:	HPN-100-011
Study First Received:	December 2, 2010
Last Updated:	July 12, 2013
Health Authority:	United States: Food and Drug Administration United States: Institutional Review Board Canada: Health Canada

Keywords provided by Hyperion Therapeutics, Inc.:


Urea Cycle Disorders GT4P HPN-100 Sodium Phenylbutyrate Glyceryl tri-(4-phenylbutyrate)	Phenylbutyrate Hyperion Buphenyl hyperammonemia

Additional relevant MeSH terms:


Urea Cycle Disorders, Inborn Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn	Central Nervous System Diseases Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases

ClinicalTrials.gov processed this record on March 03, 2015