The Third Affiliated Hospital of Guangzhou Medical University
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 2
- 主要终点
- The check of granulocyte transplantation-platelet plant living standards
研究概览
简要总结
In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.
详细描述
- On a Good Manufacturing Practice(GMP) condition, we establish non-exotic of different mutation types of beta-thalassemia -induced pluripotent stem cells（iPS） and make a comparisons of the stability and the differentiation of efficiency of these iPS cells inducing from different sources with foreign gene integration.
- Using the technique of artificial nuclease and in situ repairmen, we establish efficient system for different beta-thalassemia mutation site and in view of the security of these system.
- Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology system.
- Build a functional gene therapy self-limiting slow viruses, optimizing the preparation system and to establish a virus preparation of infection of hematopoietic stem cell technology system under the GMP condition.
- Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy and efficiency before the clinical experiment.
- Improve the existing hematopoietic stem cell transplant(HSCT) clinical application solutions, detect rate of graft rejection, rate of transplantation and other indicators, finish the evaluation of application by clinical cases.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •The genetic diagnosis confirmed homozygous for beta to poverty or double heterozygote, clinical severe anemia;
- •Aged 1 ~ 18 years old, no obvious iron overload cause organ damage;
- •Has a suitable donor HLA high resolution;
- •The informed consent
排除标准
- •There has been a significant increase in the level of tumor markers (AFP/CEA/CA199 / CA125) in the past five years.
- •Serious primary diseases such as cardiovascular, liver and hematopoietic systems; Those who have major organs with serious function; An adrenal disease or other disease that causes the organ failure of the organ;
- •An autoimmune disease, a family history of genetic disease, and an abnormal thyroid function;
- •Peripheral blood chromosome checking for nuclear aliens;
- •HIV, hepatitis b or hepatitis c;
- •A person with a history of severe drug allergies or an allergic person;
- •Those who do not expect to live for more than one year;
- •The researchers suggest that the patient may have a potential or have a disorder (such as an uncontrolled infection, right heart failure, pulmonary hypertension) that is interfering with this study.
- •In the first six months, alcohol and other substance abuse were not allowed;
- •Subjects who participated in other clinical trials or participated in other clinical trials within 3 months.
结局指标
主要结局
The check of granulocyte transplantation-platelet plant living standards
时间窗: seven consecutive days
Platelet plant living standard for seven consecutive days after transplantation the platelet should be greater than 20 x 109 / L and infusion.
Effect of cell transplantation
时间窗: seven days
The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.
The check of granulocyte transplantation-granulocyte plant living standards
时间窗: 3 days
Granulocyte plant living standards for three days in a row after transplantation granulocyte should be greater than 0.5 x 109 / L,
次要结局
未报告次要终点
研究者
Xiaofang Sun
Principal Investigator
The Third Affiliated Hospital of Guangzhou Medical University