Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- SAA clinical symptoms
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg in subject for the therapy of severe aplastic anemia (SAA).
详细描述
Severe aplastic anemia (SAA) is a condition that involves a low level of red blood cells, white blood cells, and platelets without evidence of another bone marrow disease. Patients with severe aplastic anemia produce too few blood cells, causing fatigue, easy bruising and bleeding, and susceptibility to infections. In many cases, the very low blood counts result from an autoimmune process. The patient's own immune system damages their stem cells in bone marrow.
Although immune-suppressing drugs, such as corticosteroids, CsA and ATG, have been used in the treatment of SAA, however, many studies have indicated that the overall response rate to these drugs is less than 60%. Addition, the severe side effects of these immune-suppressing drugs have also been observed. The management of SAA patients therefore remains unsatisfactory and targeted therapies are needed. Human MSCs isolated from human umbilical cord/placenta have been shown to have immunosuppressive, stimulating hematopoiesis and tissue repairing properties. This study will evaluate the safety and effectiveness of MSC transplantation in the SAA patients.
This study will last 2 to 3 years. Participants will be randomly assigned to receive either MSC transplant and CsA therapy (experimental group) or CsA therapy alone (control group). Patients will undergo MSC transplant at the start of the study on Day 0. After 3 months, patients will receive the second MSC transplantation. After six and twelve months from the first transplantation, patients will be evaluated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient age 18~80 years old with plan to infuse MSCs.
- •Standard of diagnosis of aplastic anemia is according to Chinese domestic classification of AA for
- •Patients must have an ECOG 0~
- •No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 umol/L.
- •No active severe viral or fungus infection.
- •Each patient must sign written informed consent.
排除标准
- •Psychiatric condition that would limit informed consent.
- •HIV positive
- •Positive Pregnancy Test
- •Patient has enrolled another clinical trial study within last 4 weeks.
结局指标
主要结局
SAA clinical symptoms
时间窗: 1 year
Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.
The number of blood cells
时间窗: 1 year
The number of blood cells, which contains WBC, Neu, RBC, Hb,PLT and reticulocyte, will be mainly tested monthly after transplantion of MSCs for one year
Bone borrow hemocytology
时间窗: 1 year
Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.
次要结局
- Percentage of systemic T regulatory cell population and T lymphocyte subsets(1 year)