MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis Disorders focused on measuring allogeneic hematopoietic cell transplantation, bone marrow transplantation, IMD, AMACRD, MNGIE, HDLS, OP, ALD
Eligibility Criteria
Inclusion Criteria:
- 0 through 55 years of age
- 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:
- Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
- 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)
Sites / Locations
- Masonic Cancer Center, University of MinnesotaRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
IMD - Except Haplo-identical
OP - Except Haplo-Identical
OP and IMD -Haplo-Identical Only
cALD SR-A (Standard-Risk, Regimen A)
cALD SR-B (Standard-Risk, Regimen B)
cALD HR-C (High-Risk, Regimen C)
cALD HR-D (High-Risk, Regimen D)
Inherited Metabolic Disease (IMD) - Except Haplo-Identical See intervention descriptions.
Severe Osteoperosis (OP) - Except Haplo-Identical See intervention descriptions.
Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD) -Haplo-Identical Only See intervention descriptions.
See intervention descriptions.
See intervention descriptions.
See intervention descriptions.
See intervention descriptions.