Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
Primary Purpose
Amino Acid Metabolism, Inborn Errors
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Sodium Benzoate
Sodium Phenylacetate
Sodium Phenylbutyrate
Dietary Intervention
Sponsored by
About this trial
This is an interventional treatment trial for Amino Acid Metabolism, Inborn Errors focused on measuring inborn errors of metabolism, rare disease, urea cycle disorder
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: Urea cycle deficiency, i.e.: Carbamyl phosphate synthetase deficiency (CPSD) Ornithine transcarbamylase deficiency (OTCD) Argininosuccinic acid synthetase deficiency (ASD) Argininosuccinic aciduria (AA)
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004767
First Posted
February 24, 2000
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
Johns Hopkins University
1. Study Identification
Unique Protocol Identification Number
NCT00004767
Brief Title
Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
Study Type
Interventional
2. Study Status
Record Verification Date
December 2001
Overall Recruitment Status
Completed
Study Start Date
January 1985 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
Johns Hopkins University
4. Oversight
5. Study Description
Brief Summary
OBJECTIVES:
I. Assess the safety and efficacy of sodium phenylbutyrate, sodium benzoate, sodium phenylacetate, and dietary intervention in patients with urea cycle disorders.
Detailed Description
PROTOCOL OUTLINE: This protocol describes several clinical studies of pharmacologic and dietary management in patients with urea cycle disorders.
Patients with carbamyl phosphate synthetase and ornithine transcarbamylase deficiency are treated with a low-protein diet, essential amino acids (for neonatal onset disease), caloric supplementation, oral sodium phenylbutyrate (now approved as a prescription drug 11/97), and citrulline or arginine free base.
Patients with argininosuccinic acid synthetase deficiency are treated with a low-protein diet, caloric supplementation, oral sodium phenylbutyrate (now approved as a prescription drug 11/97), and arginine free base.
Patients with argininosuccinic aciduria (AA) are treated with a low-protein diet, caloric supplementation, and arginine free base. (Discontinued 11/97) Any patient who develops hyperammonemia is treated with intravenous sodium benzoate, sodium phenylbutyrate, and arginine hydrochloride; benzoate and phenylbutyrate are not given to patients with AA.
If ammonium stabilizes at normal or near normal levels, intravenous medications are gradually replaced by oral medications. If there is no significant decrease in ammonium within 8 hours, patients begin hemodialysis.
Concurrent therapy with ondansetron, high caloric intake, and mannitol for elevated intracranial pressure is allowed. Dietary and intravenous nitrogen is prohibited. (Discontinued 11/97)
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amino Acid Metabolism, Inborn Errors
Keywords
inborn errors of metabolism, rare disease, urea cycle disorder
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Enrollment
20 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Sodium Benzoate
Intervention Type
Drug
Intervention Name(s)
Sodium Phenylacetate
Intervention Type
Drug
Intervention Name(s)
Sodium Phenylbutyrate
Intervention Type
Behavioral
Intervention Name(s)
Dietary Intervention
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
Urea cycle deficiency, i.e.: Carbamyl phosphate synthetase deficiency (CPSD) Ornithine transcarbamylase deficiency (OTCD) Argininosuccinic acid synthetase deficiency (ASD) Argininosuccinic aciduria (AA)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Saul W. Brusilow
Organizational Affiliation
Johns Hopkins University
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
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