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

This study has been completed.
Sponsor:
Collaborator:
Johns Hopkins University
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004767
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 2001

February 24, 2000
June 23, 2005
January 1985
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Complete list of historical versions of study NCT00004767 on ClinicalTrials.gov Archive Site
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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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OBJECTIVES:

I. Assess the safety and efficacy of sodium phenylbutyrate, sodium benzoate, sodium phenylacetate, and dietary intervention in patients with urea cycle disorders.

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)

Interventional
Phase 2
Primary Purpose: Treatment
Amino Acid Metabolism, Inborn Errors
  • Drug: Sodium Benzoate
  • Drug: Sodium Phenylacetate
  • Drug: Sodium Phenylbutyrate
  • Behavioral: Dietary Intervention
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
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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)

Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
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NCT00004767
199/11753, JHUSM-11753
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National Center for Research Resources (NCRR)
Johns Hopkins University
Study Chair: Saul W. Brusilow Johns Hopkins University
Office of Rare Diseases (ORD)
December 2001

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP