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2 Study Matches

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

Recruiting

Troy Lund, M.D.Ph.D. - lundx072@umn.edu

All
up to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02171104
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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)
Biological: Stem Cell Transplantation, Drug: cALD SR-B (Standard-Risk, Regimen B), Drug: cALD HR-D (High-Risk, Regimen C), Drug: Osteopetrosis Haploidentical Only Preparative Regimen, Drug: cALD SR-A (Standard-Risk, Regimen A), Drug: cALD HR-D (High-Risk, Regimen D), Drug: IMD Preparative Regimen, Drug: Osteopetrosis Only Preparative Regimen
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
allogeneic hematopoietic cell transplantation, bone marrow transplantation, IMD, AMACRD, MNGIE, HDLS, OP, ALD
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Longitudinal Studies of Brain Structure and Function in MPS Disorders

Recruiting

Ashley Schneider - amwiesen@umn.edu

All
6 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT01870375
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Inclusion Criteria:

• Any MPS I, II, IV, VI or VII child or adult aged 6 years of age or older
Exclusion Criteria:

• Exclusion Criteria for Neuroimaging:
• Participants with:
• Pacemakers
• Any indwelling electronic device including programmable shunts
• Orthodontic braces unless they are not made of metal
• Other implanted metal in the body other than titanium
• Unable to stay still during MRI because of low cognitive function, behavioral dysregulation, or young age, if the patient is not a clinical patient having sedation/anesthesia
• Pregnancy
• Exclusion Criteria for Neuropsychological and Neurobehavioral Testing
• Participants who:
• Are too functionally impaired for testing
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Mucopolysaccharidosis, Longitudinal, Brain, Cognition, Quality-of-Life, Hurler syndrome, Hunter syndrome, Hurler-Scheie syndrome, Scheie syndrome, Maroteaux-Lamy syndrome, MPS I, MPS II, MPS VI, Mucopolysaccharidosis type I, Mucopolysaccharidosis type II, Mucopolysaccharidosis type VI, MPS IV, MPS VII, Mucopolysaccharidosis type IV, Mucopolysaccharidosis type VII, Morquio syndrome, Sly syndrome
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