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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01257737
Recruitment Status : Completed
First Posted : December 10, 2010
Last Update Posted : February 27, 2017
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland ( Horizon Therapeutics, LLC )

Brief Summary:
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, (NCT00947544), HPN-100-007, (NCT00947297), or HPN-100-012, (NCT01347073). Under this protocol, long-term safety will continue to be assessed.

Condition or disease Intervention/treatment Phase
Urea Cycle Disorders Drug: HPN-100 Phase 4

Detailed Description:
The present protocol will allow subjects continued use of HPN-100 following their treatment and study completion in Study HPN-100-005, HPN-100-007, or HPN-100-012. Subjects will be able to continue in this study until approval and commercial availability of HPN-100 in each country participating in this study. Subjects will be required to return to the clinic every 6 months in which physical exam, safety labs, dietary assessment and neuropsychological testing will occur. Subjects will be dispensed sufficient HPN-100 to last until the next clinic visit.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 88 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long Term Use of HPN-100 in Urea Cycle Disorders
Study Start Date : October 2010
Primary Completion Date : February 16, 2017
Study Completion Date : February 16, 2017

Arm Intervention/treatment
Experimental: HPN-100
Subjects will continue treatment with HPN-100 after having completed HPN-100 treatment in HPN-100-005, HPN-100-007, or HPN-100-012.
Drug: HPN-100
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.
Other Name: GT4P, Glyceryl tri-(4-phenylbutyrate)

Primary Outcome Measures :
  1. Rate of Adverse Events [ Time Frame: Every six months ]

Secondary Outcome Measures :
  1. Venous ammonia levels [ Time Frame: Every six months ]
  2. Number of hyperammonemic crises [ Time Frame: Every six months ]
  3. Neuropsychological testing [ Time Frame: Every twelve months ]
    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®)

  4. Causes of hyperammonemic crises [ Time Frame: Every six month ]

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male and female subjects who completed HPN-100-005,(NCT00947544), HPN-100-007,(NCT00947297), or HPN-100-012,(NCT01347073).
  • 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,(phenylacetate) or PBA,(phenylbutyrate).
  • Liver transplant, including hepatocellular transplant.
  • Pregnant, breastfeeding or lactating females.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01257737

United States, California
UCLA Pediatrics/Genetics
Los Angeles, California, United States, 90095
Stanford University School of Medicine
Palo Alto, California, United States, 94305
United States, Colorado
Denver Children's Hospital
Aurora, Colorado, United States, 80045
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Maine
Maine Medical Center
Portland, Maine, United States, 04102
United States, Minnesota
University of Minnesota Medical Center
Minneapolis, Minnesota, United States, 55454
United States, New York
Mount Sinai School of Medicine
New York, New York, United States, 10029
United States, Ohio
University Hospitals Case Medical Center
Cleveland, Ohio, United States, 44106
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Pittsburg of UPMC
Pittsburg, Pennsylvania, United States, 15224
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
Horizon Therapeutics, LLC
Study Chair: Colleen Canavan, BS Horizon Pharma

Responsible Party: Horizon Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT01257737     History of Changes
Other Study ID Numbers: HPN-100-011
First Posted: December 10, 2010    Key Record Dates
Last Update Posted: February 27, 2017
Last Verified: February 2017

Keywords provided by Horizon Pharma Ireland, Ltd., Dublin Ireland ( Horizon Therapeutics, LLC ):
Urea Cycle Disorders
Sodium Phenylbutyrate
Glyceryl tri-(4-phenylbutyrate)

Additional relevant MeSH terms:
Urea Cycle Disorders, Inborn
Pathologic Processes
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
4-phenylbutyric acid
Antineoplastic Agents
Cryoprotective Agents
Protective Agents
Physiological Effects of Drugs