Ignite Creation Date:
2025-12-24 @ 8:03 PM
Ignite Modification Date:
2026-02-19 @ 12:26 AM
Study NCT ID:
NCT02171104
Status:
RECRUITING
Last Update Posted:
2025-08-03
First Post:
2014-06-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008059', 'term': 'Mucopolysaccharidosis I'}, {'id': 'D016532', 'term': 'Mucopolysaccharidosis II'}, {'id': 'D009087', 'term': 'Mucopolysaccharidosis VI'}, {'id': 'D016538', 'term': 'Mucopolysaccharidosis VII'}, {'id': 'D008363', 'term': 'alpha-Mannosidosis'}, {'id': 'D005645', 'term': 'Fucosidosis'}, {'id': 'D054880', 'term': 'Aspartylglucosaminuria'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D007965', 'term': 'Leukodystrophy, Globoid Cell'}, {'id': 'D007966', 'term': 'Leukodystrophy, Metachromatic'}, {'id': 'D018901', 'term': 'Peroxisomal Disorders'}, {'id': 'D015211', 'term': 'Zellweger Syndrome'}, {'id': 'D052919', 'term': 'Refsum Disease, Infantile'}, {'id': 'C536662', 'term': 'Peroxisomal ACYL-COA oxidase deficiency'}, {'id': 'C535818', 'term': 'Pseudo-Zellweger syndrome'}, {'id': 'D010022', 'term': 'Osteopetrosis'}, {'id': 'C580150', 'term': 'Hereditary Diffuse Leukoencephalopathy with Spheroids'}, {'id': 'C565768', 'term': 'Alpha-Methylacyl-CoA Racemase Deficiency'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D044904', 'term': 'Mannosidase Deficiency Diseases'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'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': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D052516', 'term': 'Sulfatidosis'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D010026', 'term': 'Osteosclerosis'}, {'id': 'D010009', 'term': 'Osteochondrodysplasias'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'C072254', 'term': 'Regimen B'}], '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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2014-07-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2029-07-14', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-01', 'studyFirstSubmitDate': '2014-06-20', 'studyFirstSubmitQcDate': '2014-06-20', 'lastUpdatePostDateStruct': {'date': '2025-08-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-06-23', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2026-07-14', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent of subjects who achieve high-level donor hematopoietic engraftment', 'timeFrame': 'Day +42 post-transplant', 'description': 'Defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant'}, {'measure': 'Percent of subjects who achieve high-level donor hematopoietic engraftment', 'timeFrame': 'Day +100 post-transplant', 'description': 'Defined as ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant'}], 'secondaryOutcomes': [{'measure': 'Graft-versus-host disease', 'timeFrame': 'Day +100 post-transplant', 'description': 'Incidence and severity of GvHD'}, {'measure': 'Transplant-related mortality', 'timeFrame': 'Day +100 post-transplant', 'description': 'Incidence of TRM'}, {'measure': 'Regimen-related toxicity', 'timeFrame': 'Day +100 post-transplant', 'description': 'Defined as infection, acute renal failure, respiratory failure, cardiac failure, and veno-occlusive disease'}, {'measure': 'Post-HSCT changes in disease', 'timeFrame': '1 year', 'description': 'Incidence of radiographic, physiologic, neuro-psychologic, and/or biochemical aspects of the disease as assessed on a disease-specific basis'}, {'measure': 'Post-HSCT changes in disease', 'timeFrame': '2 years', 'description': 'Incidence of radiographic, physiologic, neuro-psychologic, and/or biochemical aspects of the disease as assessed on a disease-specific basis'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['allogeneic hematopoietic cell transplantation', 'bone marrow transplantation', 'IMD', 'AMACRD', 'MNGIE', 'HDLS', 'OP', 'ALD'], 'conditions': ['Mucopolysaccharidosis Disorders', 'Hurler Syndrome', 'Hunter Syndrome', 'Maroteaux Lamy Syndrome', 'Sly Syndrome', 'Alpha-Mannosidosis', 'Fucosidosis', 'Aspartylglucosaminuria', 'Glycoprotein Metabolic Disorders', 'Sphingolipidoses', 'Recessive Leukodystrophies', 'Globoid Cell Leukodystrophy', 'Metachromatic Leukodystrophy', 'Niemann-Pick B', 'Niemann-Pick C Subtype 2', 'Sphingomyelin Deficiency', 'Peroxisomal Disorders', 'Adrenoleukodystrophy With Cerebral Involvement', 'Zellweger Syndrome', 'Neonatal Adrenoleukodystrophy', 'Infantile Refsum Disease', 'Acyl-CoA Oxidase Deficiency', 'D-Bifunctional Enzyme Deficiency', 'Multifunctional Enzyme Deficiency', 'Alpha-methylacyl-CoA Racmase Deficiency', 'Mitochondrial Neurogastrointestingal Encephalopathy', 'Severe Osteopetrosis', 'Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)', 'Inherited Metabolic Disorders']}, 'descriptionModule': {'briefSummary': 'This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '55 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* 0 through 55 years of age\n* Adequate graft available\n* Adequate organ function\n* Eligible Diseases:\n\n * Mucopolysaccharidosis Disorders:\n\n * MPS IH (Hurler syndrome)\n * MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype\n * MPS VI (Maroteaux-Lamy syndrome)\n * MPS VII (Sly syndrome)\n * Glycoprotein Metabolic Disorders:\n\n * Alpha mannosidosis\n * Fucosidosis\n * Aspartylglucosaminuria\n * Sphingolipidoses and Recessive Leukodystrophies:\n\n * Globoid cell leukodystrophy\n * Metachromatic leukodystrophy\n * Niemann-Pick B patients (sphingomyelin deficiency)\n * Niemann-Pick C subtype 2\n * Peroxisomal Disorders:\n\n * Adrenoleukodystrophy with cerebral involvement\n * Zellweger syndrome\n * Neonatal Adrenoleukodystrophy\n * Infantile Refsum disease\n * Acyl-CoA-Oxidase Deficiency\n * D-Bifunctional enzyme deficiency\n * Multifunctional enzyme deficiency\n * Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)\n * Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)\n * Severe Osteopetrosis (OP)\n * Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)\n * Other Inherited Metabolic Disorders (IMD): Patients will also be considered who have other life-threatening, rare lysosomal, peroxisomal or other similar inherited disorders characterized by white matter disease or other neurologic manifestations for which there is rationale that transplantation would be of benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis, I-cell disease, Tay-Sachs disease, Sandhoff disease or others.\n * Voluntary written consent\n\nExclusion Criteria:\n\n* Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start\n* Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)\n* Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)"}, 'identificationModule': {'nctId': 'NCT02171104', 'briefTitle': 'MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis', 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG', 'orgStudyIdInfo': {'id': '2013LS104'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'IMD - Except Haplo-identical', 'description': 'Inherited Metabolic Disease (IMD) - Except Haplo-Identical\n\nSee intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: IMD Preparative Regimen']}, {'type': 'EXPERIMENTAL', 'label': 'OP - Except Haplo-Identical', 'description': 'Severe Osteoperosis (OP) - Except Haplo-Identical\n\nSee intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: Osteopetrosis Only Preparative Regimen']}, {'type': 'EXPERIMENTAL', 'label': 'OP and IMD -Haplo-Identical Only', 'description': 'Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD)\n\n-Haplo-Identical Only\n\nSee intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: Osteopetrosis Haploidentical Only Preparative Regimen']}, {'type': 'EXPERIMENTAL', 'label': 'cALD SR-A (Standard-Risk, Regimen A)', 'description': 'See intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: IMD Preparative Regimen', 'Drug: cALD SR-A (Standard-Risk, Regimen A)']}, {'type': 'EXPERIMENTAL', 'label': 'cALD SR-B (Standard-Risk, Regimen B)', 'description': 'See intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: IMD Preparative Regimen', 'Drug: cALD SR-B (Standard-Risk, Regimen B)']}, {'type': 'EXPERIMENTAL', 'label': 'cALD HR-C (High-Risk, Regimen C)', 'description': 'See intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: IMD Preparative Regimen', 'Drug: cALD HR-D (High-Risk, Regimen C)']}, {'type': 'EXPERIMENTAL', 'label': 'cALD HR-D (High-Risk, Regimen D)', 'description': 'See intervention descriptions.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: IMD Preparative Regimen', 'Drug: cALD HR-D (High-Risk, Regimen D)']}], 'interventions': [{'name': 'Stem Cell Transplantation', 'type': 'BIOLOGICAL', 'description': 'Infusion given on Day 0', 'armGroupLabels': ['IMD - Except Haplo-identical', 'OP - Except Haplo-Identical', 'OP and IMD -Haplo-Identical Only', 'cALD HR-C (High-Risk, Regimen C)', 'cALD HR-D (High-Risk, Regimen D)', 'cALD SR-A (Standard-Risk, Regimen A)', 'cALD SR-B (Standard-Risk, Regimen B)']}, {'name': 'IMD Preparative Regimen', 'type': 'DRUG', 'description': '* Anti-thymocyte Globulin (ATG)\n* Fludarabine\n* Busulfan', 'armGroupLabels': ['IMD - Except Haplo-identical', 'cALD HR-C (High-Risk, Regimen C)', 'cALD HR-D (High-Risk, Regimen D)', 'cALD SR-A (Standard-Risk, Regimen A)', 'cALD SR-B (Standard-Risk, Regimen B)']}, {'name': 'Osteopetrosis Only Preparative Regimen', 'type': 'DRUG', 'description': '* Anti-thymocyte Globulin (ATG)\n* Fludarabine\n* Busulfan\n* Thiotepa', 'armGroupLabels': ['OP - Except Haplo-Identical']}, {'name': 'Osteopetrosis Haploidentical Only Preparative Regimen', 'type': 'DRUG', 'description': '* Rituximab\n* Alemtuzumab\n* Busulfan\n* Fludarabine', 'armGroupLabels': ['OP and IMD -Haplo-Identical Only']}, {'name': 'cALD SR-A (Standard-Risk, Regimen A)', 'type': 'DRUG', 'description': 'N-acetylcysteine start day +1 through day +28', 'armGroupLabels': ['cALD SR-A (Standard-Risk, Regimen A)']}, {'name': 'cALD SR-B (Standard-Risk, Regimen B)', 'type': 'DRUG', 'description': 'N-acetylcysteine start day +1through day +56', 'armGroupLabels': ['cALD SR-B (Standard-Risk, Regimen B)']}, {'name': 'cALD HR-D (High-Risk, Regimen C)', 'type': 'DRUG', 'description': 'N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100', 'armGroupLabels': ['cALD HR-C (High-Risk, Regimen C)']}, {'name': 'cALD HR-D (High-Risk, Regimen D)', 'type': 'DRUG', 'description': 'N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100', 'armGroupLabels': ['cALD HR-D (High-Risk, Regimen D)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Lisa Burke', 'role': 'CONTACT', 'email': 'lburke3@Fairview.org', 'phone': '612-273-8482'}], 'facility': 'Masonic Cancer Center, University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'centralContacts': [{'name': 'Lisa Burke', 'role': 'CONTACT', 'email': 'lburke3@Fairview.org', 'phone': '612-273-8482'}, {'name': 'Troy Lund, M.D.Ph.D.', 'role': 'CONTACT', 'email': 'lundx072@umn.edu', 'phone': '612-625-4185'}], 'overallOfficials': [{'name': 'Paul Orchard, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Masonic Cancer Center, University of Minnesota'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}