Ignite Creation Date:
2025-12-25 @ 12:37 AM
Ignite Modification Date:
2025-12-25 @ 10:47 PM
Study NCT ID:
NCT00004767
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2000-02-24
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D035583', 'term': 'Rare Diseases'}, {'id': 'D056806', 'term': 'Urea Cycle Disorders, Inborn'}], 'ancestors': [{'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020160', 'term': 'Sodium Benzoate'}, {'id': 'C025136', 'term': 'phenylacetic acid'}, {'id': 'C075773', 'term': '4-phenylbutyric acid'}, {'id': 'D004035', 'term': 'Diet Therapy'}], 'ancestors': [{'id': 'D019817', 'term': 'Benzoic Acid'}, {'id': 'D001565', 'term': 'Benzoates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D001555', 'term': 'Benzene Derivatives'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D044623', 'term': 'Nutrition Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'primaryPurpose': 'TREATMENT'}, 'enrollmentInfo': {'count': 20}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1985-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2001-12', 'lastUpdateSubmitDate': '2005-06-23', 'studyFirstSubmitDate': '2000-02-24', 'studyFirstSubmitQcDate': '2000-02-24', 'lastUpdatePostDateStruct': {'date': '2005-06-24', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2000-02-25', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['inborn errors of metabolism', 'rare disease', 'urea cycle disorder'], 'conditions': ['Amino Acid Metabolism, Inborn Errors']}, 'descriptionModule': {'briefSummary': 'OBJECTIVES:\n\nI. Assess the safety and efficacy of sodium phenylbutyrate, sodium benzoate, sodium phenylacetate, and dietary intervention in patients with urea cycle disorders.', 'detailedDescription': 'PROTOCOL OUTLINE: This protocol describes several clinical studies of pharmacologic and dietary management in patients with urea cycle disorders.\n\nPatients 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.\n\nPatients 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.\n\nPatients 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.\n\nIf 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.\n\nConcurrent therapy with ondansetron, high caloric intake, and mannitol for elevated intracranial pressure is allowed. Dietary and intravenous nitrogen is prohibited. (Discontinued 11/97)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\nUrea cycle deficiency, i.e.: Carbamyl phosphate synthetase deficiency (CPSD) Ornithine transcarbamylase deficiency (OTCD) Argininosuccinic acid synthetase deficiency (ASD) Argininosuccinic aciduria (AA)'}, 'identificationModule': {'nctId': 'NCT00004767', 'briefTitle': 'Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders', 'organization': {'class': 'NIH', 'fullName': 'Office of Rare Diseases (ORD)'}, 'orgStudyIdInfo': {'id': '199/11753'}, 'secondaryIdInfos': [{'id': 'JHUSM-11753'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'Sodium Benzoate', 'type': 'DRUG'}, {'name': 'Sodium Phenylacetate', 'type': 'DRUG'}, {'name': 'Sodium Phenylbutyrate', 'type': 'DRUG'}, {'name': 'Dietary Intervention', 'type': 'BEHAVIORAL'}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Saul W. Brusilow', 'role': 'STUDY_CHAIR', 'affiliation': 'Johns Hopkins University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Center for Research Resources (NCRR)', 'class': 'NIH'}, 'collaborators': [{'name': 'Johns Hopkins University', 'class': 'OTHER'}]}}}