Ignite Creation Date:
2025-12-24 @ 9:19 PM
Ignite Modification Date:
2025-12-25 @ 7:06 PM
Study NCT ID:
NCT00176904
Status:
COMPLETED
Last Update Posted:
2017-12-28
First Post:
2005-09-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Stem Cell Transplant for Inborn Errors of Metabolism
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000326', 'term': 'Adrenoleukodystrophy'}, {'id': 'D007966', 'term': 'Leukodystrophy, Metachromatic'}, {'id': 'D007965', 'term': 'Leukodystrophy, Globoid Cell'}, {'id': 'D005776', 'term': 'Gaucher Disease'}, {'id': 'D005645', 'term': 'Fucosidosis'}, {'id': 'D015223', 'term': 'Wolman Disease'}, {'id': 'D009542', 'term': 'Niemann-Pick Diseases'}, {'id': 'D009472', 'term': 'Neuronal Ceroid-Lipofuscinoses'}, {'id': 'D016537', 'term': 'Gangliosidosis, GM1'}, {'id': 'D013661', 'term': 'Tay-Sachs Disease'}, {'id': 'D012497', 'term': 'Sandhoff Disease'}], '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': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'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': 'D052516', 'term': 'Sulfatidosis'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}, {'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'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D015217', 'term': 'Cholesterol Ester Storage Disease'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D005733', 'term': 'Gangliosidoses'}, {'id': 'D020143', 'term': 'Gangliosidoses, GM2'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'D016026', 'term': 'Bone Marrow Transplantation'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D000961', 'term': 'Antilymphocyte Serum'}], 'ancestors': [{'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D014180', 'term': 'Transplantation'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D016378', 'term': 'Tissue Transplantation'}, {'id': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D007106', 'term': 'Immune Sera'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D001688', 'term': 'Biological Products'}, {'id': 'D045424', 'term': 'Complex Mixtures'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'orcha001@umn.edu', 'phone': '612-626-2313', 'title': 'Paul Orchard, M.D.', 'organization': 'Masonic Cancer Center, University of Minnesota'}, 'certainAgreement': {'piSponsorEmployee': True, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'Change in Neuropsychometric function data is not available. Data would have been noted as descriptive and patient specific, as well as disease specific; not table format. No formal analysis was intended.'}}, 'adverseEventsModule': {'timeFrame': 'Serious adverse experiences were collected during the first 100 days after transplant then 6 months and annually for 3 years.', 'description': 'Selected serious adverse experiences (graft failure/autologous recovery, severe acute GVHD (grades III and IV) and death were collected during the first 100 days after transplant then 6 months and annually for 3 years. After day 100, only death or an unexpected adverse event will be reported. Adverse events were not collected in this study.', 'eventGroups': [{'id': 'EG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).', 'otherNumAtRisk': 135, 'otherNumAffected': 0, 'seriousNumAtRisk': 135, 'seriousNumAffected': 62}], 'seriousEvents': [{'term': 'Auto recovery', 'notes': 'Delayed side effects that includes, loss of appetite, nausea and vomiting, changes in vision, mouth and throat soreness, diarrhea, difficulty sleeping and fatigue, infection, hair loss, skin reactions and graft-versus-host disease.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 135, 'numEvents': 9, 'numAffected': 9}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Death', 'stats': [{'groupId': 'EG000', 'numAtRisk': 135, 'numEvents': 54, 'numAffected': 54}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Primary graft failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 135, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '135', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'classes': [{'title': 'Day 100', 'categories': [{'measurements': [{'value': '120', 'groupId': 'OG000'}]}]}, {'title': '1 Year', 'categories': [{'measurements': [{'value': '92', 'groupId': 'OG000'}]}]}, {'title': '3 Years', 'categories': [{'measurements': [{'value': '81', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 Days, 1 Year and 3 Years', 'description': 'Number of patients alive at designated timepoints after transplant.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2011-04'}, {'type': 'SECONDARY', 'title': 'Overall Donor Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '134', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'classes': [{'categories': [{'measurements': [{'value': '123', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': 'Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '1 Patient not included due to early death (before day 40).', 'anticipatedPostingDate': '2011-04'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '135', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'classes': [{'categories': [{'measurements': [{'value': '34', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': "Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2011-04'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '135', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'classes': [{'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': "Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2011-04'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Chronic Graft-Versus-Host Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '135', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'classes': [{'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 Year Post Transplant', 'description': "Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2011-04'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '135'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '135'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '135', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Patients Treated With Stem Cell Transplant', 'description': 'All patients treated with protocol regimen (chemotherapy and stem cell transplant).'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '117', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '8.7', 'spread': '8.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '44', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '91', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '135', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 135}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1995-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2010-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-03', 'studyFirstSubmitDate': '2005-09-12', 'resultsFirstSubmitDate': '2011-05-17', 'studyFirstSubmitQcDate': '2005-09-12', 'lastUpdatePostDateStruct': {'date': '2017-12-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2011-05-17', 'studyFirstPostDateStruct': {'date': '2005-09-15', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2011-06-10', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Survival', 'timeFrame': '100 Days, 1 Year and 3 Years', 'description': 'Number of patients alive at designated timepoints after transplant.'}], 'secondaryOutcomes': [{'measure': 'Overall Donor Engraftment', 'timeFrame': 'Day 100', 'description': 'Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%.'}, {'measure': 'Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease', 'timeFrame': 'Day 100', 'description': "Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening."}, {'measure': 'Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease', 'timeFrame': 'Day 100', 'description': "Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening."}, {'measure': 'Number of Patients With Chronic Graft-Versus-Host Disease', 'timeFrame': '1 Year Post Transplant', 'description': "Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome."}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Inborn errors', 'Storage disease', 'errors of metabolism', 'stem cell transplant'], 'conditions': ['Adrenoleukodystrophy', 'Metachromatic Leukodystrophy', 'Globoid Cell Leukodystrophy', "Gaucher's Disease", 'Fucosidosis', 'Wolman Disease', 'Niemann-Pick Disease', 'Batten Disease', 'GM1 Gangliosidosis', 'Tay Sachs Disease', 'Sandhoff Disease']}, 'referencesModule': {'references': [{'pmid': '21586746', 'type': 'DERIVED', 'citation': 'Miller WP, Rothman SM, Nascene D, Kivisto T, DeFor TE, Ziegler RS, Eisengart J, Leiser K, Raymond G, Lund TC, Tolar J, Orchard PJ. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report. Blood. 2011 Aug 18;118(7):1971-8. doi: 10.1182/blood-2011-01-329235. Epub 2011 May 17.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic storage disease.', 'detailedDescription': 'Prior to transplantation, subjects will receive Busulfan intravenously (IV) via the Hickman line four times daily for 4 days, Cyclophosphamide intravenously via the Hickman line once a day for 4 days, and Anti-Thymocyte Globulin (ATG) intravenously (IV) via the Hickman line twice daily for three days before the transplant. These three drugs are being given to subjects to help the new marrow "take" and grow.\n\nOn the day of transplantation, the donor\'s hematopoietic cells will be transfused via central venous catheter.\n\nAfter hematopoietic cell transplant, subjects will then receive two drugs, cyclosporin and either methylprednisolone or Mycophenolate Mofetil (MMF). Cyclosporin and methylprednisolone or MMF are given to help prevent the complication of graft-versus-host disease and to decrease the chance that the new donor cells will be rejected.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patients with adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy, Gaucher's disease, Fucosidosis, Wolman disease, Niemann-Pick disease and Batten disease (CLN3) who have a human leukocyte antigen (HLA)-identical or haplotype mismatched (at 1-3 antigens) related marrow, or umbilical cord blood donor. One or two umbilical cord blood (UCB) units may be used.\n* Patients with GM1 gangliosidosis, Tay Sachs disease or Sandhoff disease who have a HLA-identical or 1 antigen mismatched related or unrelated donor, or suitably matched umbilical cord blood unit(s). One or two UCB units may be used.\n* Patients with adrenoleukodystrophy must have magnetic resonance imaging (MRI) findings, neurological and neuropsychometric function consistent with the diagnosis, and for boys with parietal-occipital dysmyelination a performance intelligence quotient (IQ) ≥80. In cases, when the performance IQ is not ≥80, the protocol committee may recommend transplant if the patient's clinical condition and neuropsychometric status are deemed to be acceptable based upon consideration of such factors as age at onset of cerebral disease, magnitude of change in performance IQ and neurologic deficits.\n* Patients with arylsulfatase A deficiency (Metachromatic Leukodystrophy) must have either the presymptomatic late infantile, juvenile or adult form of the disease and must have acceptable neurological and neuropsychometric function.\n* Patients with galactocerebrosidase deficiency (Globoid Cell Leukodystrophy) must have acceptable neurological and neuropsychometric function.\n* Patients with acid lipase deficiency (Wolman disease) must have a liver biopsy that documents no evidence of hepatic cirrhosis, and acceptable neurological and neuropsychometric function.\n* Patients with fucosidase deficiency (Fucosidosis) must have acceptable neurological and neuropsychometric function.\n* Patients with glucocerebrosidase deficiency (Gaucher's Disease) must have acceptable neurologic and neuropsychometric function.\n* Patients with Batten's disease (CLN3) must have acceptable Neurological and neuropsychometric function.\n* Absence of major organ dysfunction. Organ evaluation results as follows:\n* Cardiac: ejection fraction \\>30%\n* Renal: serum creatinine \\<2x normal or creatinine clearance 60 mL/min.\n* Hepatic: total bilirubin \\<2x normal and Aspartate aminotransferase (AST) \\<2x normal\n* Signed consent.\n\nExclusion Criteria:\n\n* Patients with symptomatic late infantile form of metachromatic leukodystrophy.\n* Patients with symptomatic infantile globoid leukodystrophy.\n* Note: Patients with Hurler syndrome, mucopolysaccharidosis (MPS) VI, or Mannosidosis disease are no longer eligible for this protocol, but can be transplanted under protocol MT 9907 (NCT00176917 - Hematopoietic Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)).\n* Pregnancy\n* Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology\n* Patients or parents are psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance.\n* Patients ≥ 50 kg may be at risk for having cell doses below the goal of ≥ 10 x 10\\^6 CD34 cells/kg and therefore will not be eligible to receive unrelated peripheral blood stem cells (PBSCs)"}, 'identificationModule': {'nctId': 'NCT00176904', 'briefTitle': 'Stem Cell Transplant for Inborn Errors of Metabolism', 'nctIdAliases': ['NCT00005894'], 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation', 'orgStudyIdInfo': {'id': 'MT1995-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Treated Patients', 'description': 'All patients treated with protocol regimen (chemotherapy and surgery).', 'interventionNames': ['Procedure: Stem Cell Transplant', 'Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin']}], 'interventions': [{'name': 'Stem Cell Transplant', 'type': 'PROCEDURE', 'otherNames': ['Bone marrow transplant'], 'description': "The purpose of hematopoietic cell transplantation is to introduce hematopoietic cells from a normal donor that contains an enzyme able to get rid of the substances that have accumulated in the body of patients with storage diseases. Hematopoietic cells can come from bone marrow, peripheral blood (i.e., the blood circulating in our body's blood vessels) or umbilical cord blood (i.e., blood taken from the umbilical cord after a baby is born and umbilical cord is cut).", 'armGroupLabels': ['Treated Patients']}, {'name': 'Busulfan, Cyclophosphamide, Antithymocyte Globulin', 'type': 'DRUG', 'otherNames': ['Busulfex, Cytoxan, ATG'], 'description': 'Subjects will receive BUSULFAN intravenously (IV)- patients \\< or= 12 kg 1.1 mg/kd/dose IV every 6 hours for 16 doses; patients \\> 12kg 0.8 mg/kg/dose IV every 6 hours for 16 doses - via the Hickman line four times daily for 4 days, CYCLOPHOSPHAMIDE intravenously (50 mg/kg/day IV over 2 hours) via the Hickman line once a day for 4 days, and ANTI-THYMOCYTE GLOBULIN IV (15 mg/kg/day over 2 hours) via the Hickman line twice daily for three days before the transplant. These three drugs are being given to help the new marrow "take" and grow. METHYLPREDNISOLONE will be given as a pre-medication for the ATG.', 'armGroupLabels': ['Treated Patients']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Masonic Cancer Center, University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'overallOfficials': [{'name': 'Paul Orchard, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Masonic Cancer Center, University of Minnesota'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}