Ignite Creation Date:
2025-12-25 @ 3:03 AM
Ignite Modification Date:
2025-12-31 @ 5:54 AM
Study NCT ID:
NCT00004333
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020176', 'term': 'Tyrosinemias'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D035583', 'term': 'Rare Diseases'}], 'ancestors': [{'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'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'primaryPurpose': 'TREATMENT'}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1994-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2000-07', 'lastUpdateSubmitDate': '2005-06-23', 'studyFirstSubmitDate': '1999-10-18', 'studyFirstSubmitQcDate': '1999-10-18', 'lastUpdatePostDateStruct': {'date': '2005-06-24', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '1999-10-19', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['inborn errors of metabolism', 'rare disease', 'tyrosinemia I'], 'conditions': ['Tyrosinemia, Type I']}, 'descriptionModule': {'briefSummary': 'OBJECTIVES:\n\nAssess whether 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) at 0.6 mg/kg per day prevents liver failure in at least 1 patient with tyrosinemia type I.', 'detailedDescription': 'PROTOCOL OUTLINE: Only 2 patients with tyrosinemia type I are known to this research team; others will be treated if found and if clinical conditions permit.\n\nThe enzyme inhibitor 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) is administered orally, in 2 divided doses daily with meals.\n\nPatients will be followed closely for side effects attributable to NTBC.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '0 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\nTyrosinemia type I No cirrhosis No hepatocellular carcinoma'}, 'identificationModule': {'nctId': 'NCT00004333', 'briefTitle': 'Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I', 'organization': {'class': 'NIH', 'fullName': 'Office of Rare Diseases (ORD)'}, 'orgStudyIdInfo': {'id': '199/11892'}, 'secondaryIdInfos': [{'id': 'UMMC-1319'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'NTBC', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Jess G. Thoene', 'role': 'STUDY_CHAIR', 'affiliation': 'University of Michigan'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Center for Research Resources (NCRR)', 'class': 'NIH'}, 'collaborators': [{'name': 'University of Michigan', 'class': 'OTHER'}]}}}