Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 46
- 试验地点
- 1
- 主要终点
- Number of Patients With Donor Derived Engraftment
研究概览
简要总结
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells. When healthy stem cells from a donor are infused into the patient, the donor white blood cells can provide the missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving a monoclonal antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening. This may be an effective treatment for inherited metabolic disorders.
Purpose: The design of this study is to achieve donor cell engraftment in patients with standard-risk inherited metabolic diseases with limited peri-transplant morbidity and mortality. This will be achieved through the administration of the chemotherapy regimen described. The intention is to follow transplanted patient for years after transplant monitoring them for complications of their disease and assisting families with a multifaceted interdisciplinary approach.
详细描述
Primary Objective:
- To estimate the proportion of patients with donor derived engraftment at day 100 post transplant as defined by 80% or greater donor cells in the CD3 (T cell) fraction
Secondary Objectives:
- To determine the incidence and severity of graft-versus-host disease (GVHD) by day 100
- To determine the incidence of peri-transplant mortality (death by day 100)
- To monitor donor cell chimerism at various time points following allogeneic transplantation with this transplant regimen as determined at day 28, 42, 100, 6 months and yearly for 5 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have diagnosis of one of the following: mucopolysaccharidosis disorder, glycoprotein metabolic disorder, sphingolipidoses or inherited leukodystrophy, peroxisomal disorder or other inherited diseases of metabolism
- •Must have an acceptable graft source as defined by University of Minnesota criteria
- •Adequate organ function
排除标准
- •Pregnant - menstruating females must have a negative serum pregnancy test within 14 days of treatment start
- •Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology
研究组 & 干预措施
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
干预措施: Campath-1H (Drug)
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
干预措施: Cyclophosphamide (Drug)
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
干预措施: Busulfan (Drug)
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
干预措施: Allogeneic stem cell transplantation (Procedure)
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
干预措施: Cyclosporine A (Drug)
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
干预措施: Mycophenolate Mofetil (Drug)
结局指标
主要结局
Number of Patients With Donor Derived Engraftment
时间窗: Day 100 Post Transplant
Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells.
次要结局
- Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)(Day 100 Post Transplant)
- Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)(Day 100 Post Transplant)
- Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)(Day 100 Post Transplant)
- Donor Cell Chimerism Following Transplant(One year)
- Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)(Day 100 Post Transplant)
- Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)(Day 100 Post Transplant)
- Number of Patients Who Died Peri-Transplant(By Day 100 Post Transplant)