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Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders

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ClinicalTrials.gov Identifier: NCT00004767
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
Sponsor:
Collaborator:
Johns Hopkins University
Information provided by:
Office of Rare Diseases (ORD)

Study Description
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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.


Condition or disease Intervention/treatment Phase
Amino Acid Metabolism, Inborn Errors Drug: Sodium Benzoate Drug: Sodium Phenylacetate Drug: Sodium Phenylbutyrate Behavioral: Dietary Intervention Phase 2

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)


Study Design
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Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Primary Purpose: Treatment
Study Start Date : January 1985

Resource links provided by the National Library of Medicine


Arms and Interventions
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Outcome Measures
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Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)

Contacts and Locations
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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): NCT00004767


Sponsors and Collaborators
National Center for Research Resources (NCRR)
Johns Hopkins University
Investigators
Study Chair: Saul W. Brusilow Johns Hopkins University
More Information
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ClinicalTrials.gov Identifier: NCT00004767     History of Changes
Other Study ID Numbers: 199/11753
JHUSM-11753
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2001

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
rare disease
urea cycle disorder

Additional relevant MeSH terms:
Urea Cycle Disorders, Inborn
Metabolism, Inborn Errors
Amino Acid Metabolism, Inborn Errors
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
 4-phenylbutyric acid
Phenylacetic acid
Sodium Benzoate
Antineoplastic Agents
Antifungal Agents
Anti-Infective Agents
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action