Haploidentical Stem Cell Transplantation With Fixed Dose of T Cells After in Vitro T Cell Depletion Using CD3 Monoclonal Antibody for Children With Acquired Severe Aplastic Anemia
试验速览
- 阶段
- 2 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- To assess engraftment rate and survival of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia
研究概览
简要总结
Rationale: Fludarabine, cyclophosphamide, anti-thymocyte globulin and low-dose total body irradiation (LD-TBI) may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion of CD3 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.
Purpose: Phase II trials to evaluate the efficacy of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia
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
- •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: Fludarabine (Drug)
HAPLO
干预措施: Cyclophosphamide (Drug)
HAPLO
干预措施: anti-thymocyte globulin (Biological)
HAPLO
干预措施: filgrastim (Biological)
HAPLO
干预措施: Total body irradiation (Radiation)
HAPLO
干预措施: CD3-depleted hematopoietic cell transplantation (Procedure)
结局指标
主要结局
To assess engraftment rate and survival of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia
时间窗: 2 years posttransplant
次要结局
- To assess engraftment and graft failure(28 days posttransplant)
- To estimate the risk of acute GVHD(100 days posttransplant)
- To assess treatment related mortality(100 days posttransplant)
- To estimate overall survival and failure free survival(1 year posttransplant)
研究者
Ho Joon Im
Professor
Asan Medical Center