MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis (NCT02171104) | Clinical Trial Compass
Active — Not RecruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
United States149 participantsStarted 2014-07-10
Plain-language 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).
Who can participate
Age range55 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 manifestat…
What they're measuring
1
Percent of subjects who achieve high-level donor hematopoietic engraftment
Timeframe: Day +42 post-transplant
2
Percent of subjects who achieve high-level donor hematopoietic engraftment
Timeframe: Day +100 post-transplant
Trial details
NCT IDNCT02171104
SponsorMasonic Cancer Center, University of Minnesota