Phase Ⅱ/Ⅲ Clinical Trial, Multicenter, Randomized, Controlled, for the Evaluation of Efficacy and Safety of Therapy With Allogenic Mesenchymal Stem Cells in Patients With Chronic Graft Versus Host Disease
试验速览
- 阶段
- 2 期
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- The total Response rate defined as patients with complete and partial response
研究概览
简要总结
The primary purpose of the study is to evaluate the safety and efficacy of mesenchymal stem cells (MSC) for the treatment of patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to first-line therapy.
The secondary purpose of the study is to evaluate the effect of mesenchymal stem cells (MSC) on one-year survival rate, long-term survival rate, life quality and recurrence of patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to hormone treatment.
详细描述
Chronic Graft-versus-host disease (GVHD)， with the incidence of 30%-60%, is a serious late complication of allogeneic hematopoietic stem cell transplantation (HSCT) and is the major cause of death in the late stage of transplantation. According to targeted organs, cGVHD is divided into two types, limited cGVHD and extensive cGVHD. Extensive cGVHD needs systemic immunosuppressant treatment. However, currently standard first-line regimen including cyclophosphamide and prednisolone is only effective for some patients. Novel treatment is urgently needed. Our previous study has shown that mesenchymal stem cells (MSCs) are effective for cGVHD patients with multiple skin damage. To further explore the therapeutic effect of MSCs for extensive cGVHD, we plan to conduct a multi-center clinical trial. Patients who developed an extensive cGVHD (with skin and/or liver damage) after HSCs transplantation and do not respond to first-line therapy are enrolled. They will be randomly divided into two groups which will receive MSCs and routine second-line drugs respectively. We will evaluate the efficacy and safety of MSCs for extensive cGVHD by comparison of symptom improvement, survival rate, recurrence as well as side effects in the two groups.
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
- None
入排标准
- 年龄范围
- 2 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Extensive cGVHD with skin and/or liver damage developed after allogeneic hematopoietic stem cell transplantation
- •cGVHD that do not response to conventional immunosuppressant treatment for two months
- •KPS>= 30
- •informed consent from the patient
排除标准
- •Extensive cGVHD without skin or liver damage
- •With other acute severe complications
- •In pregnancy or lactation
- •Disease relapses
- •With non-hematological malignancy
- •Have a history of mental disorder, drug or alcohol abuse over the past five years
- •Participate in other clinical trial within three months before the start of this trial
- •With bone marrow fibrosis
- •Have undergone hematopoietic stem cell transplantation to treat solid tumor
研究组 & 干预措施
Control group
patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to standard first-line regimen including cyclophosphamide and prednisolone.
干预措施: Cyclosporine and Glucocorticoid (Drug)
结局指标
主要结局
The total Response rate defined as patients with complete and partial response
时间窗: 1 year after MSCs administration.
次要结局
- one-year survival rate(1 year after MSCs administration)
- disease relapse(2 years after MSCs administration)
- quality of life(2 years after MSCs administration)
研究者
Robert Chunhua Zhao, MD, PhD
MD, PhD，Professor of medicine
Chinese Academy of Medical Sciences