Phase I/II: Umbilical Cord Derived Mesenchymal Stem Cells Transplantation For Active And Refractory Systemic Lupus Erythematosus
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- British Isles Lupus Assessment Group score (BILAG)
研究概览
简要总结
This study will explore safety and efficacy of allogeneic umbilical cord (UC) derived mesenchymal stem cells transplantation (MSCT) to treat patients with active and refractory systemic lupus erythematosus (SLE) who have been resistant to multiple standard treatments. The underlying hypothesis is that the active SLE condition is caused by an abnormal immune homeostasis that can be restored by MSCT.
详细描述
Inclusion Criteria:
- All patients fulfilled the American College of Rheumatology (ACR) criteria of SLE, man or woman aged from 15 to 70 years old, SLEDAI≥8;
- Lupus nephritis with 24h urine protein≥1g;
- Refractory disease as determined by failure of the following regimens:
Trial of corticosteroids (oral prednisone more than 20 mg/day); Trial of cyclophosphamide 0.4 ~ 0.6 / m2 every two weeks for six months, or other immunosuppressive drugs, such as mycophenolate mofetil 2 g / day, for three months; 4. Patients must sign an informed consent indicating that they are aware of the investigational nature of the study in keeping with the policy of the hospital; 5. Willing to use contraception throughout the study and for 12 months following treatment
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
- 15 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients fulfilled the American College of Rheumatology (ACR) criteria of SLE, man or woman aged from 15 to 60 years old, SLEDAI≥8;
- •Lupus nephritis with 24h urine protein≥1g;
- •Refractory disease as determined by failure of the following regimens:
- •Trial of corticosteroids (oral prednisone more than 20 mg/day); Trial of cyclophosphamide 0.4 ~ 0.6 / m2 every two weeks for six months, or other immunosuppressive drugs, such as mycophenolate mofetil 2 g / day, for three months;
- •Patients must sign an informed consent indicating that they are aware of the investigational nature of the study in keeping with the policy of the hospital;
- •Willing to use contraception throughout the study and for 12 months following treatment
排除标准
- •Abnormal liver function (ALT higher than 3 times the normal value);
- •End-stage renal failure;
- •Severe heart and pulmonary failure, or other important organs damage;
- •Uncontrolled infections
- •Pregnant or breast feeding women, male or female who intended to recent pregnancy
结局指标
主要结局
British Isles Lupus Assessment Group score (BILAG)
时间窗: up to 12 months
次要结局
- Renal function (GFR, Blood Urea Nitrogen, urinalysis)(pre-MSC transplantation, 1, 3, 6 and 12 months post MSC transplantation (for GFR assessed at baseline and 12 months after MSCT))
- Lupus serology (Alb, ANA, dsDNA, C3, C4)(pre-MSC transplantation, 1, 3, 6 and 12 months post MSC transplantation)
研究者
Lingyun Sun
Department of Rheumatology and Immunology
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School