Ignite Creation Date:
2025-12-25 @ 4:42 AM
Ignite Modification Date:
2025-12-26 @ 3:44 AM
Study NCT ID:
NCT00004418
Status:
TERMINATED
Last Update Posted:
2020-10-23
First Post:
1999-10-18
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000326', 'term': 'Adrenoleukodystrophy'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D035583', 'term': 'Rare Diseases'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}], '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': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D018901', 'term': 'Peroxisomal Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000309', 'term': 'Adrenal Insufficiency'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C030774', 'term': 'trierucate'}, {'id': 'D014304', 'term': 'Triolein'}, {'id': 'C079420', 'term': "Lorenzo's oil"}], 'ancestors': [{'id': 'D005224', 'term': 'Fats, Unsaturated'}, {'id': 'D005223', 'term': 'Fats'}, {'id': 'D008055', 'term': 'Lipids'}, {'id': 'D014280', 'term': 'Triglycerides'}, {'id': 'D005989', 'term': 'Glycerides'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'raymond@kennedykrieger.org', 'phone': '443-923-2750', 'title': 'Gerald Raymond', 'organization': 'Kennedy Krieger Institute'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '10 years (throughout the study)', 'eventGroups': [{'id': 'EG000', 'title': 'GTO/GTE Treatment', 'description': 'Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats.\n\nGTO/GTE orally; 30-60 ml daily for study period\n\nglyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids\n\nglyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.', 'otherNumAtRisk': 126, 'deathsNumAtRisk': 126, 'otherNumAffected': 1, 'seriousNumAtRisk': 126, 'deathsNumAffected': 1, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Thrombocytopenia', 'notes': 'Low platelet count defined as \\<80, 000', 'stats': [{'groupId': 'EG000', 'numAtRisk': 126, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Elevation in liver function tests', 'stats': [{'groupId': 'EG000', 'numAtRisk': 126, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Death', 'notes': 'Subject had profound adrenal crisis and died', 'stats': [{'groupId': 'EG000', 'numAtRisk': 126, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'adrenal crisis', 'notes': 'Two events of adrenal crisis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 126, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '126', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GTO/GTE Treatment', 'description': 'Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats.\n\nGTO/GTE orally; 30-60 ml daily for study period\n\nglyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids\n\nglyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.41', 'groupId': 'OG000', 'lowerLimit': '0.05', 'upperLimit': '0.41'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, an average of 10 years, up to age 13', 'description': 'we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.', 'unitOfMeasure': 'mcg/mL', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With T2 MRI Abnormality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '126', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GTO/GTE Treatment', 'description': 'Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats.\n\nGTO/GTE orally; 30-60 ml daily for study period\n\nglyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids\n\nglyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '10 years', 'description': 'We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'GTO/GTE Treatment', 'description': 'Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats.\n\nGTO/GTE orally; 30-60 ml daily for study period\n\nglyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids\n\nglyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '126'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'The definition of completed is until the subject wished to stop taking the oil.', 'groupId': 'FG000', 'numSubjects': '126'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': "An open trial of Lorenzo's oil spanning decades (1998-2013). Centered at the Kennedy Krieger."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '126', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'GTO/GTE Treatment', 'description': 'Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats.\n\nGTO/GTE orally; 30-60 ml daily for study period\n\nglyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids\n\nglyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '126', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '3.25', 'groupId': 'BG000', 'lowerLimit': '1.5', 'upperLimit': '8'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '126', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Male children only were eligible for this study.', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '126', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 126}}, 'statusModule': {'whyStopped': 'No funding, move to expanded access', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '1998-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-10', 'completionDateStruct': {'date': '2014-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-10-22', 'studyFirstSubmitDate': '1999-10-18', 'resultsFirstSubmitDate': '2016-04-13', 'studyFirstSubmitQcDate': '1999-10-18', 'lastUpdatePostDateStruct': {'date': '2020-10-23', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-10-22', 'studyFirstPostDateStruct': {'date': '1999-10-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-10-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2014-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels', 'timeFrame': 'Baseline, an average of 10 years, up to age 13', 'description': 'we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With T2 MRI Abnormality', 'timeFrame': '10 years', 'description': 'We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['adrenoleukodystrophy', 'inborn errors of metabolism', 'rare disease', 'sphingolipidoses'], 'conditions': ['Adrenoleukodystrophy']}, 'descriptionModule': {'briefSummary': "OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.\n\nII. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.", 'detailedDescription': 'PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.\n\nPatients are followed monthly for 6 months, then every 3 months until they reach the age of 13 years or death.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '8 Years', 'minimumAge': '18 Months', 'genderBased': True, 'genderDescription': 'males only', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria.\n\n1. Boys between the age of 18 months and 8 years of age\n2. Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.\n3. Platelet count in normal range\n\nExclusion criteria.\n\n1. Abnormal MRI consistent with childhood cerebral disease\n2. Boys who have undergone bone marrow transplantation\n3. Other medical condition which in the opinion of the investigator prevents evaluation or treatment'}, 'identificationModule': {'nctId': 'NCT00004418', 'briefTitle': 'Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy', 'organization': {'class': 'OTHER', 'fullName': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.'}, 'officialTitle': "Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy", 'orgStudyIdInfo': {'id': '199/13312'}, 'secondaryIdInfos': [{'id': 'Nutricia-Loma Linda', 'type': 'OTHER_GRANT', 'domain': 'Nutricia-Loma Linda'}, {'id': 'KKI-FDR000685', 'type': 'OTHER', 'domain': 'FDA'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Glyceryl trierucate/glyceryl trioleate', 'description': 'Treatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial', 'interventionNames': ['Drug: glyceryl trierucate/glyceryl trioleate']}], 'interventions': [{'name': 'glyceryl trierucate/glyceryl trioleate', 'type': 'DRUG', 'otherNames': ["Lorenzo's oil"], 'description': 'Administration of glyceryl trierucate/glyceryl trioleate', 'armGroupLabels': ['Glyceryl trierucate/glyceryl trioleate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '21287-6681', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins Hospital', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'Gerald V Raymond, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}