Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 43
- 主要终点
- ORR
研究概览
简要总结
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of relapsed/refractory chemoradiotherapy-induced aplastic anemia
详细描述
Currently, there are few studies on the use of TPO-RAs after tumor radiotherapy and chemotherapy. Our center has published a study on the efficacy and safety of avatrombopag (AVA) in AA secondary to radiotherapy and chemotherapy. The study demonstrated that the ORR at 1, 3, and 6 months were 32.4%, 55.9%, and 58.8%, respectively. In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27, which showed that romiplostim seemed to be effective and safe in patients with refractory aplastic anemia, with a recommended starting dose of 20 μg/kg once a week.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years old;
- •Diagnosed with relapsed/refractory chemoradiotherapy-induced aplastic anemia (AA). Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;
- •At least one of the following conditions must be met: hemoglobin < 90 g/L, platelet count < 30×109/L, neutrophil count < 1.0×109/L;
- •With baseline liver and kidney functions <2 ULN;
- •Without active infection that cannot be controlled by standard treatment;
- •Signed the informed consent;
- •ECOG score ≤ 2;
排除标准
- •Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
- •With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
- •PNH clone ≥ 50%;
- •Received HSCT before enrollment;
- •Received ATG treatment before enrollment;
- •Severe bleeding or infection that cannot be controlled by standard treatment;
- •Allergic or intolerant to romiplostim N01;
- •Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension
- •History of malignant tumors within 5 years;
- •History of arterial or venous thrombosis;
- •Pregnant or lactating patients;
- •Participated in other clinical trials within 3 months.
研究组 & 干预措施
Romiplostim N01
Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months
干预措施: Romiplostim N01 (Drug)
结局指标
主要结局
ORR
时间窗: 3-month, 6-month
Overall response rate (ORR) = PRR+CRR
次要结局
- CRR(3-month, 6-month, 12-month)
- AE rate(through study completion, an average of 1 year)
- Predictive factors of efficacy(3-month)
- Clonal evolution rate(through study completion, an average of 1 year)
研究者
Bing Han
Professor
Peking Union Medical College Hospital