HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- To assess the engraftment rate and survival of CD3±CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin.
研究概览
简要总结
Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion CD3±CD19 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.
Purpose: This phase I/II trial is to evaluate the safety and efficacy of fludarabine, cyclophosphamide and antithymocyte globulin with CD3±CD19 depleted graft from haploidentical donors in treating patients with aplastic anemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Diagnosis of life-threatening marrow failure (severe aplastic anemia) of nonmalignant etiology meeting 2 of the following criteria:
- •Granulocyte count < 500/mm3,
- •Corrected reticulocyte count < 1%,
- •Platelet count < 20,000/mm3
- •No HLA-identical family member or closely matched (8 of 8 HLA-locus match) unrelated marrow donor available
- •HLA-haploidentical related donor available
排除标准
- •Paroxysmal nocturnal hemoglobinuria or Fanconi anemia
- •Clonal cytogenetic abnormalities or myelodysplastic syndromes
- •Active fungal infections
- •HIV positive
- •Severe disease other than aplastic anemia that would severely limit the probability of survival during the graft procedure
- •Pregnant or nursing
研究组 & 干预措施
HAPLO
干预措施: anti-thymocyte globulin (Biological)
HAPLO
干预措施: filgrastim (Biological)
HAPLO
干预措施: Fludarabine (Drug)
HAPLO
干预措施: Cyclophosphamide (Drug)
HAPLO
干预措施: CD3±CD19 depleted hematopoietic stem cell transplantation (Procedure)
结局指标
主要结局
To assess the engraftment rate and survival of CD3±CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin.
时间窗: 2 years post-transplant
次要结局
- To assess engraftment and graft failure(28 days post-transplant)
- To estimate the risk of acute GVHD(100 days post-transplant)
- To assess treatment related mortality(100 days post-transplant)
- To estimate overall survival(1 year after transplantation)
研究者
Ho Joon Im
Professor
Asan Medical Center