An Exploratory Study on the Efficacy and Safety of Lusutrombopag in the Treatment of Recurrent/Refractory/Intolerable NSAA
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Overall response rate at 3 months
研究概览
简要总结
In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.
详细描述
The enrolled patients: were given Lusutrombopag at 3mg/qd orally for 12 weeks (the starting dose of lusutrombopag was 3mg, taken once daily. After 2 weeks of continuous administration, the dose was increased by 3mg every 2 weeks based on the platelet count and safety of the subjects. The dose was gradually increased to 9mg/d over a total of 12 weeks). The treatment duration was at least 3 months. When the platelet increase was <20×10^9/L, the daily dose was increased by 3mg, up to a maximum of 9mg/day. When the platelet increase was ≥50×109/L and ≤200×10^9/L, the dose was maintained at the previous level. When the platelet count was ≥200×10^9/L and ≤400×10^9/L, the daily dose was reduced by 3mg. When the platelet count was >400×10^9/L, the drug could be suspended, and the dose was reduced by 3mg when the platelet count decreased to <200×10^9/L. In this case, if the lowest dose of 3mg/day was used, the drug could be suspended. Responders continued treatment for 6 months. Other TPO-RA therapies were not allowed during the study period.
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 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must be at least 18 years old, male or female.
- •Participants must be diagnosed with NSAA and have a refractory/relapsed/intolerable response to standard-dose cyclosporine (CsA). The definition of refractory/relapsed is patients who have been treated with sufficient doses of cyclosporine (3-5mg/kg) for at least 6 months without response or relapse. The definition of intolerable is patients who cannot tolerate CsA and have stopped treatment due to significant side effects.
- •Participants must meet the following criteria at enrollment: platelets <30×109/L.
- •Baseline liver and kidney function must be within 2 times of normal range.
- •No active infection; no pregnancy or breastfeeding.
- •Participants must agree to sign the informed consent form.
- •Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
排除标准
- •Other causes of pancytopenia, such as myelodysplastic syndrome (MDS).
- •Evidence of clonal hematopoietic system bone marrow disease (MDS, AML) with cytogenetics.
- •PNH clone ≥50%.
- •Received hematopoietic stem cell transplant (HSCT) prior to enrollment.
- •Received ATG treatment within 6 months prior to enrollment.
- •Infection or bleeding that cannot be controlled with standard therapy.
- •Allergic to ruxolitinib.
- •Active HIV, HCV, or HBV infection, cirrhosis, or portal hypertension.
- •Any malignant tumor within 5 years, or local basal cell carcinoma of the skin.
- •History of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid syndrome) and current use of anticoagulants.
- •Pregnant or breastfeeding (lactating) women.
- •Participated in another clinical trial within 3 months.
研究组 & 干预措施
Lusutrombopag
Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is <20×10^9/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10^9/L and ≤200×10^9/L, the dose can be maintained; when the platelet count is ≥200×10^9/L and ≤400×10^9/L, the daily dose can be reduced by 3mg; when the platelet count is >400×10^9/L, the drug can be suspended and resumed when the platelet count decreases to <200×10^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months.
干预措施: Lusutrombopag (Drug)
结局指标
主要结局
Overall response rate at 3 months
时间窗: 3 month
Proportion of patients who achieved complete response, partial response and hematological response
Overall response rate at 6 months
时间窗: 6 month
Proportion of patients who achieved complete response, partial response and hematological response
次要结局
- adverse event rate at 6 months(6 month)
- adverse event rate at 3 months(3 month)
研究者
Bing Han
Professor
Peking Union Medical College Hospital