The Efficacy and Safety Study of Avatrombopag Combined With IST as First-line Treatment for Severe Aplastic Anemia, A Single-arm, Phase II Clinical Study
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 53
- 试验地点
- 1
- 主要终点
- Treatment response
研究概览
简要总结
This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).
详细描述
This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled.
Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight<50 kg, for a total of 12 weeks.
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
- 12 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with newly diagnosed severe aplastic anemia.
- •Men and women aged between 12 and
- •Subjects must complete all screening assessments as outlined in the test protocol.
- •Able to swallow or administer orally.
- •Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form.
排除标准
- •Congenital bone marrow failure (eg. Fanconi anemia).
- •Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) .
- •ATG or middle/high-dose cyclophosphamide was used in the past.
- •Previous treatment with cyclosporine or tacrolimus > 6 months.
- •The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month.
- •Serious infectious diseases (tuberculosis without effective control, pulmonary aspergillosis, viral infections).
- •AIDS patients.
- •Pregnant or breastfeeding, fertile but unwilling to take effective contraceptive measures.
- •Patients with malignant tumors who are not suitable for ATG treatment.
- •A newly diagnosed history of cardio/cerebral vascular thrombosis within 12 months.
- •Those who are assessed as unsuitable for inclusion by the investigator.
研究组 & 干预措施
Treatment group
53 subjects will be enrolled with the indicated treatment dose of avatrombopag.
干预措施: avatrombopag (Drug)
结局指标
主要结局
Treatment response
时间窗: From the start of study treatment (Day1) to end of week 12.
Percentage of patients who achieves complete response(CR) at 12 weeks.
次要结局
- Treatment response(From the start of study treatment (Day1) to end of week 24.)
- Incidence of Treatment-Emergent Adverse Events by CTCAE(From the start study treatment (Day1) up to week 12.)
- Dose-effect relationship(From the start study treatment(Day1) up to week 24.)
- Change of CD34+ cell(From the start study treatment(Day1) up to end of week 12 and 24.)
- Supportive treatment(From the start study treatment (Day1) up to transfusion independence.)