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

Description

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).

Conditions

Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, 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, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders

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Study Overview

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Study Details

Study overview

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).

MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG

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

Condition
Mucopolysaccharidosis Disorders
Intervention / Treatment

-

Contacts and Locations

Minneapolis

Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States, 55455

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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)

Ages Eligible for Study

to 55 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Masonic Cancer Center, University of Minnesota,

Paul Orchard, M.D., PRINCIPAL_INVESTIGATOR, Masonic Cancer Center, University of Minnesota

Study Record Dates

2028-07-14