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

Descriptive Information
Brief Title † MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Official Title † MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
Brief 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).
Detailed Description
Study Phase Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Percent of subjects who achieve high-level donor hematopoietic engraftment
Secondary Outcome Measure † Graft-versus-host disease
Condition † 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
Intervention † BiologicalStem Cell Transplantation
Study Arms / Comparison Groups IMD - Except Haplo-identical Inherited Metabolic Disease (IMD) - Except Haplo-Identical See intervention descriptions. OP - Except Haplo-Identical Severe Osteoperosis (OP) - Except Haplo-Identical See intervention descriptions. OP and IMD -Haplo-Identical Only Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD) -Haplo-Identical Only See intervention descriptions. cALD SR-A (Standard-Risk, Regimen A) See intervention descriptions. cALD SR-B (Standard-Risk, Regimen B) See intervention descriptions. cALD HR-C (High-Risk, Regimen C) See intervention descriptions. cALD HR-D (High-Risk, Regimen D) See intervention descriptions.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Biological
Estimated Enrollment † 100
Start Date † July 10, 2014
Completion Date September 2019
Primary Completion Date September 2019
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 lifethreatening, 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)
Gender All
Ages N/A - 55 Years
Accepts Healthy Volunteers No
Contacts †† Kim Nelson, 612-273-2925,
Location Countries † United States
Administrative Information
NCT ID † NCT02171104
Organization ID 2013LS104
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Masonic Cancer Center, University of Minnesota
Collaborators ††
Investigators † Principal Investigator: Weston Miller, MD, Masonic Cancer Center, University of Minnesota
Information Provided By
Verification Date June 2017
First Received Date † June 20, 2014
Last Updated Date June 15, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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