A Safety and Efficacy Study of N-acetylcysteine in Patients With Transplant-Associated Thrombotic Microangiopathy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 170
- 试验地点
- 1
- 主要终点
- The Incidence of TA-TMA.
研究概览
简要总结
HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.
详细描述
Hematopoietic stem cell transplantation (HSCT) has been commonly used as a potentially curative option in the treatment of various hematological malignancies. However, it may end up with serious complications in various systems, including the hemostatic system. HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. Since the pathophysiology has not been clarified, there are no established treatments for TA-TMA several agents seem to have successful results. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. Studies showed NAC reduce plasma VWF multimers and VWF multimeric size without an effect on the bleeding time in vitro and in vivo, thus proposed as a possible supplementary treatment for TTP. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Double (Participant, Investigator)
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients be scheduled to undergo HSCT;
- •Not received decitabine 6 month ago;
- •Without severe organ damage;
- •Informed consent were obtained.
排除标准
- •Be sensitive to NAC;
- •Bronchial asthma；
- •Peptic ulcer；
- •Severe organ damage；
- •Pregnancy and breastfeeding women；
研究组 & 干预措施
Experimental Group
N-Acetylcysteine
干预措施: N-Acetylcysteine (Drug)
Control Group
Placebo Oral Tablet
干预措施: Placebo Oral Tablet (Drug)
结局指标
主要结局
The Incidence of TA-TMA.
时间窗: 100 days
The incidence of TMA after HSCT.
次要结局
- The Level of Endothelial Micro Particle(100 days)
- The Level of TNF-α(40 days)
- The Level of ROS(100 days)
- The Level of VWF Multimers(100 days)