Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
OBJECTIVES:
I. Assess the safety and efficacy of sodium phenylbutyrate, sodium benzoate, sodium phenylacetate, and dietary intervention in patients with urea cycle disorders.
| Condition | Intervention | Phase |
|---|---|---|
|
Amino Acid Metabolism, Inborn Errors |
Drug: Sodium Benzoate Drug: Sodium Phenylacetate Drug: Sodium Phenylbutyrate Behavioral: Dietary Intervention |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Primary Purpose: Treatment |
| Estimated Enrollment: | 20 |
| Study Start Date: | January 1985 |
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)
Eligibility| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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 More Information
No publications provided
| ClinicalTrials.gov Identifier: | NCT00004767 History of Changes |
| Other Study ID Numbers: | 199/11753, JHUSM-11753 |
| Study First Received: | February 24, 2000 |
| Last Updated: | June 23, 2005 |
| Health Authority: | United States: Federal Government |
Keywords provided by Office of Rare Diseases (ORD):
|
inborn errors of metabolism rare disease urea cycle disorder |
Additional relevant MeSH terms:
|
Amino Acid Metabolism, Inborn Errors Metabolism, Inborn Errors Urea Cycle Disorders, Inborn Genetic Diseases, Inborn Metabolic Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Benzoates |
Sodium Benzoate Phenylacetic acid 4-phenylbutyric acid Antifungal Agents Anti-Infective Agents Therapeutic Uses Pharmacologic Actions Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Antineoplastic Agents |
ClinicalTrials.gov processed this record on June 13, 2013