Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- 1. Engraftment at 6 months, 1 year and 2 years 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years
研究概览
简要总结
For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be :
- Patient survival probability at 100 days, 1 year and 2 years.
- Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
- Engraftment at 6 months, 1 year and 2 years
详细描述
The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results. For this initial study we plan to enroll up to 24 patients.
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 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of SAA based on bone marrow aspirate and biopsy results. Failure to respond to immunosuppressive therapy and/or lack of an HLA identical family member.
- •A 10/10 or 9/10 HLA matched unrelated donor or a 9/10 matched related donor available after high resolution typing.
排除标准
- •Patients with Aplastic anemia and active infection must be treated to maximally resolve this problem before beginning the conditioning regimen.
- •HIV seropositive patients
- •Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.
- •Patient greater than 60 years of age.
- •Women who are pregnant or nursing.
- •Patients with active hepatitis
- •Patients with severe cardiac dysfunction defined as shortening fraction <25%.
- •Patients with severe renal dysfunction defined as creatinine clearance <40ml/mim/1.73m
- •Patient with severe pulmonary dysfunction with FEV1, FVC and DLCO 40% of predicted or 3 SD below normal.
研究组 & 干预措施
No Arms
干预措施: Cyclophosphamide,Campath IH and TBI (Drug)
结局指标
主要结局
1. Engraftment at 6 months, 1 year and 2 years 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years
时间窗: 2 years
次要结局
- Patient survival probability at 100 days, 1 year and 2 years.(2 years)