MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

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 ofsymptomatic neurologic disease but is expected to have a neurologicphenotype

• 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 whohave other life-threatening, rare lysosomal, peroxisomal or other similarinherited disorders characterized by white matter disease or other neurologicmanifestations for which there is rationale that transplantation would be ofbenefit, 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 testwithin 14 days of study treatment start

• Prior myeloablative chemotherapy exposure within 4 months of the start ofconditioning on this protocol (patients excluded for this reason may be eligible forother institutional protocols)

• Uncontrolled bacterial, fungal or viral infections including HIV (including activeinfection with Aspergillus or other mold within 30 days)