Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells.
试验速览
- 阶段
- 4 期
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA
研究概览
简要总结
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment. The response rate of early treatment on Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG treatment) is associated with long-term effect. The injection of Umbilical Cord Derived Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.
详细描述
- Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from healthy human umbilical cord tissue; The " injection of mesenchymal stem cells (umbilical) manufacturing and verification regulation " was also formulated.
- The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 * 106 cells /kg, based on the previous human studies; And the maximum tolerated dose was 1 * 107 cells /kg
- The response and complete remission rate, relapse rate of the injection of Umbilical Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were determined.
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
- 1 Month 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Non-Severe Aplastic Anemia (NSAA)atients
排除标准
- •Severe Aplastic Anemia (SAA) patients
研究组 & 干预措施
MSC+ATG
The first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment.
干预措施: MSC+ATG (Drug)
结局指标
主要结局
The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA
时间窗: 1 year
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count \>100\*109/L, and absolute neutrophil count (ANC) \>1.5\*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count\>30\*109/L, platelet count \>20\*109/L, and ANC\>0.5\*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).
次要结局
- The relapse rate with different doses of MSC to treat child with SAA(3-10 year)
研究者
Xiaofan Zhu
chief physician
Chinese Academy of Medical Sciences