Clinical trial
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
NCT02171104 is registered in ClinicalTrials.gov with Masonic Cancer Center, University of Minnesota as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Phase
- Phase 2
- Overall status
- Active, not recruiting
- Start date
- July 10, 2014
- Completion date
- July 14, 2029
- Enrollment
- 149 (actual)
- Sites
- 1
Conditions
As the registry lists them; a condition with its own page is linked.
acyl-coa oxidase deficiency, adrenoleukodystrophy with cerebral involvement, alpha-mannosidosis, alpha-methylacyl-coa racmase deficiency, aspartylglucosaminuria, d-bifunctional enzyme deficiency, fucosidosis, globoid cell leukodystrophy, glycoprotein metabolic disorders, hereditary leukoencephalopathy with axonal spheroids (hdls; csf1r mutation), hunter syndrome, hurler syndrome, infantile refsum disease, inherited metabolic disorders, maroteaux lamy syndrome, metachromatic leukodystrophy, mitochondrial neurogastrointestingal encephalopathy, mucopolysaccharidosis disorders, multifunctional enzyme deficiency, neonatal adrenoleukodystrophy, niemann-pick b, niemann-pick c subtype 2, peroxisomal disorders, recessive leukodystrophies, severe osteopetrosis, sly syndrome, sphingolipidoses, sphingomyelin deficiency, zellweger syndrome