A Phase 2 interventional study of Sodium Benzoate and Sodium Phenylacetate in Amino Acid Metabolism, Inborn Errors, sponsored by National Center for Research Resources (NCRR). Completed. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2005-06-24.
Sponsored by National Center for Research Resources (NCRR) · Phase 2, Interventional, and Treatment
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)
107 studies on the registry are indexed under Metabolism, Inborn Errors; 23 are open to participants now.
This study's enrollment of 20 is below the median of 30 across 62 interventional studies indexed under Metabolism, Inborn Errors.
Browse Metabolism, Inborn Errors studies →National Center for Research Resources (NCRR) is the lead sponsor of 265 studies on the registry; none are open to participants now.
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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)
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This study is completed, as verified in Dec 2001. You cannot join it, but the record below documents what was studied.
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National Center for Research Resources (NCRR)