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 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)