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

Masonic Cancer Center, University of Minnesota1 个研究点分布在 1 个国家目标入组 149 人开始时间: 2014年7月10日最近更新: 2026年1月7日

适应症

干预措施

试验速览

阶段: 2 期
状态: 进行中（未招募）
发起方: Masonic Cancer Center, University of Minnesota
入组人数: 149
试验地点: 1
主要终点: Percent of subjects who achieve high-level donor hematopoietic engraftment

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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).

研究设计

研究类型: Interventional
分配方式: Randomized
干预模型: Single Group
主要目的: Treatment
盲法: None

入排标准

年龄范围: — 至 55 Years（Child, Adult）
性别: All
接受健康志愿者: 否

入选标准

•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

排除标准

•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)