Ignite Creation Date:
2025-12-25 @ 1:27 AM
Ignite Modification Date:
2025-12-27 @ 11:31 PM
Study NCT ID:
NCT00004493
Status:
COMPLETED
Last Update Posted:
2015-03-25
First Post:
1999-10-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000140', 'term': 'Acidosis, Lactic'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D035583', 'term': 'Rare Diseases'}], 'ancestors': [{'id': 'D000138', 'term': 'Acidosis'}, {'id': 'D000137', 'term': 'Acid-Base Imbalance'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003999', 'term': 'Dichloroacetic Acid'}], 'ancestors': [{'id': 'D062845', 'term': 'Chloroacetates'}, {'id': 'D000085', 'term': 'Acetates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D006843', 'term': 'Hydrocarbons, Chlorinated'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'primaryPurpose': 'TREATMENT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 75}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1998-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2000-05', 'completionDateStruct': {'date': '2003-09'}, 'lastUpdateSubmitDate': '2015-03-24', 'studyFirstSubmitDate': '1999-10-18', 'studyFirstSubmitQcDate': '1999-10-18', 'lastUpdatePostDateStruct': {'date': '2015-03-25', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '1999-10-19', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['inborn errors of metabolism', 'lactic acidosis', 'rare disease'], 'conditions': ['Lactic Acidosis']}, 'descriptionModule': {'briefSummary': 'OBJECTIVES: I. Determine the pharmacokinetics of sodium dichloroacetate (DCA) in patients with congenital lactic acidemia.\n\nII. Determine the efficacy of DCA in decreasing the frequency and/or severity of acute episodes of acidotic illness, improving linear growth, improving neurological or developmental function, or slowing neurological or developmental deterioration in these patients.', 'detailedDescription': 'PROTOCOL OUTLINE: This is a randomized, double blind, cross over study. Patients are randomized to start with one of two different doses of sodium dichloroacetate (DCA).\n\nPatients receive one of two doses of oral DCA for 6 months, then switch to the alternate dose for 6 months. This course is repeated once.\n\nPatients are followed for up to 2 years.\n\nCompletion date provided represents the completion date of the grant per OOPD records'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\n--Disease Characteristics--\n\n* Diagnosis of stable, persistent lactic acidemia Venous lactate at least 3 mM under basal conditions defined as: At least 4 hours postprandial No concurrent illness\n\nOR\n\n* Diagnosis of cerebral lactic acidemia with elevated lactic acid in CSF but not in the blood\n* No organic acidemias or defective gluconeogenesis\n\n--Patient Characteristics--\n\n* Hematopoietic: Hemoglobin at least 7 mg/dL\n* Hepatic: Bilirubin no greater than 3 times upper limit of normal (ULN) AST, ALT, or GGT no greater than 10 times ULN\n* Renal: Creatinine no greater than 2 mg/dL\n* Cardiovascular: Ejection fraction at least 25%\n* Other: No hypoglycemia (blood sugar less than 50 mg/dL at no greater than 12 hours fasting) No severe peripheral neuropathy interfering with normal activities of living'}, 'identificationModule': {'nctId': 'NCT00004493', 'briefTitle': 'Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia', 'organization': {'class': 'FED', 'fullName': 'FDA Office of Orphan Products Development'}, 'orgStudyIdInfo': {'id': '199/14274'}, 'secondaryIdInfos': [{'id': 'UCSD-FDR001481'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'sodium dichloroacetate', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'zip': '92093-0652', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'University of California San Diego School of Medicine', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}], 'overallOfficials': [{'name': 'Bruce Barshop', 'role': 'STUDY_CHAIR', 'affiliation': 'University of California, San Diego'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of California, San Diego', 'class': 'OTHER'}}}}