MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT ID: NCT02171104
Last Updated: 2026-01-07
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
149 participants
INTERVENTIONAL
2014-07-10
2029-07-14
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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IMD - Except Haplo-identical
Inherited Metabolic Disease (IMD) - Except Haplo-Identical
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
OP - Except Haplo-Identical
Severe Osteoperosis (OP) - Except Haplo-Identical
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
Osteopetrosis Only Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
* Thiotepa
OP and IMD -Haplo-Identical Only
Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD)
-Haplo-Identical Only
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
Osteopetrosis Haploidentical Only Preparative Regimen
* Rituximab
* Alemtuzumab
* Busulfan
* Fludarabine
cALD SR-A (Standard-Risk, Regimen A)
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
cALD SR-A (Standard-Risk, Regimen A)
N-acetylcysteine start day +1 through day +28
cALD SR-B (Standard-Risk, Regimen B)
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
cALD SR-B (Standard-Risk, Regimen B)
N-acetylcysteine start day +1through day +56
cALD HR-C (High-Risk, Regimen C)
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
cALD HR-D (High-Risk, Regimen C)
N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100
cALD HR-D (High-Risk, Regimen D)
See intervention descriptions.
Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
cALD HR-D (High-Risk, Regimen D)
N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100
Interventions
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Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
Osteopetrosis Only Preparative Regimen
* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
* Thiotepa
Osteopetrosis Haploidentical Only Preparative Regimen
* Rituximab
* Alemtuzumab
* Busulfan
* Fludarabine
cALD SR-A (Standard-Risk, Regimen A)
N-acetylcysteine start day +1 through day +28
cALD SR-B (Standard-Risk, Regimen B)
N-acetylcysteine start day +1through day +56
cALD HR-D (High-Risk, Regimen C)
N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100
cALD HR-D (High-Risk, Regimen D)
N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100
Eligibility Criteria
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Inclusion Criteria
* Adequate graft available
* Adequate organ function
* Eligible Diseases:
* Mucopolysaccharidosis Disorders:
* MPS IH (Hurler syndrome)
* MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
* MPS VI (Maroteaux-Lamy syndrome)
* MPS VII (Sly syndrome)
* Glycoprotein Metabolic Disorders:
* Alpha mannosidosis
* Fucosidosis
* Aspartylglucosaminuria
* Sphingolipidoses and Recessive Leukodystrophies:
* Globoid cell leukodystrophy
* Metachromatic leukodystrophy
* Niemann-Pick B patients (sphingomyelin deficiency)
* Niemann-Pick C subtype 2
* 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 (AMACRD)
* Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
* Severe Osteopetrosis (OP)
* Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
* 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.
* Voluntary written consent
Exclusion Criteria
* 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)
* Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)
55 Years
ALL
No
Sponsors
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Masonic Cancer Center, University of Minnesota
OTHER
Responsible Party
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Principal Investigators
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Paul Orchard, M.D.
Role: PRINCIPAL_INVESTIGATOR
Masonic Cancer Center, University of Minnesota
Locations
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Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
Countries
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Other Identifiers
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2013LS104
Identifier Type: -
Identifier Source: org_study_id
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