Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia: a Prospective Single Arm Study
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- ORR at 6 Months
研究概览
简要总结
This is a prospective one arm study to explore the efficacy and safety of Hetrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited. Treatment of Hetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day. All patients would receive treatment for at least 6 months except that the platelet <20×10e9/L at the dosage of 15mg/day for 4 weeks or the platelet ≥200×10e9/L at the dosage of 5mg/week for 2 weeks. The hematological response rate and safety will be recorded and compared at D15, 1month, 1.5month, 2month, 3month, 4month, 5month, 6month, 8month, 10month and 1year.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed to be non-severe aplastic anemia
- •Patients with platelet count < 30×109/L and have at least one of the followings: ①absolute neutrophil count < 1.5×109/L, ②platelet count < 50×109/L, ③ hemoglobin level < 100g/L
- •Patients have no response or relapsed following at least one treatment course in a period time of > 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
- •Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
- •Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.
排除标准
- •Congenital aplastic anemia;
- •Presence of chromosomal aberration;
- •Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
- •Presence with PNH clone ≥50%;
- •Patients received HSCT before;
- •Uncontrolled infection or bleeding with standard treatment;
- •Allergic to Hetrombopag or accessories;
- •HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
- •Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site, unstable angina pectoris, uncontrolled hypertension(>180/100mmHg)，pulmonary artery hypertension;
- •Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
- •Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
- •Pregnant or nursing (lactating) woman;
- •Have attended other clinical trials within 3 months;
研究组 & 干预措施
treatment group
Hetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day.
干预措施: Hetrombopag (Drug)
结局指标
主要结局
ORR at 6 Months
时间窗: 6 month
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at 6 months
次要结局
- ORR at 3 Months(3 month)
- Volume of Platelet Transfusions(12 months)
- percentage of side effects at 12 months(12 months)
- Percentage of patients with clonal evolution at 12 months(12 months)
研究者
Bing Han
Docter
Peking Union Medical College Hospital