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

  • STATUS
    Recruiting
  • End date
    Dec 25, 2021
  • participants needed
    100
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 25 January 2021
fludarabine
deficiency
treatment regimen
encephalopathy
acetylcysteine
metabolic disorders
niemann pick disease
preparative regimen
glycoprotein
sandhoff disease
leukodystrophy
regimen b
mucopolysaccharidosis
hurler syndrome
sphingolipidoses
gm1 gangliosidosis
aspartylglucosaminuria
hdls
wolman's disease
cald
adrenoleukodystrophy
sly syndrome
mngie
peroxisomal disorder

Summary

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).

Details
Condition beta-Glucuronidase Deficiency, Hunter's Syndrome, Krabbe's Disease, Metachromatic leukodystrophy, Nephropathy, alpha-Mannosidosis, LIVER DISEASE, Maroteaux-Lamy Syndrome, Aspartylglucosaminuria, Neonatal adrenoleukodystrophy, Fucosidosis, Infantile Refsum disease, Zellweger Syndrome, Hurler's Syndrome, Sphingolipidosis, Kidney Disease (Pediatric), Mucopolysaccharidosis Disorders, Glycoprotein Metabolic Disorders, Recessive Leukodystrophies, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Adrenoleukodystrophy With Cerebral Involvement, 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, Hunter Syndrome (MPS II), Niemann-Pick B, Niemann-Pick C Subtype 2, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Metachromatic Leukodystrophy - MLD, Kidney Disease, Liver Disorders, Niemann-Pick B, Niemann-Pick C Subtype 2, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Niemann-Pick B, Niemann-Pick C Subtype 2, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), hunter syndrome, krabbe disease, sly syndrome, sphingolipidoses, hurler syndrome, dysostosis multiplex, peroxisomal disorder, peroxisomal disorders, Niemann-Pick B, Niemann-Pick C Subtype 2, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)
Treatment Stem cell transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Clinical Study IdentifierNCT02171104
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on25 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

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)
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