NCT05660785已完成2 期
The Efficacy and Safety of Herombopag Combined With Cyclosporine for Patients With Non Severe Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China4 个研究点 分布在 1 个国家目标入组 54 人开始时间: 2022年12月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 54
- 试验地点
- 4
- 主要终点
- Overall response rate
研究概览
简要总结
This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).
详细描述
This study aims to improve the 24 weeks response rate. The sample size is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 15 patients. If after 24 weeks at least 9 patients achieved a response, then enrollment was expanded to a total of 43 patients. The null hypothesis was unaccepted if more than 26 of 43 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 54 patients.
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
- •Willing and able to comply with the requirements for this study and written informed consent.
- •Male or female age ≥ 18 years
- •Diagnosis of untreated non severe aplastic anemia.
- •Platelet counts < 50 x 10^9/L at least 2 times consecutively (time interval ≥ 1 week)
排除标准
- •Receive immunosuppressive therapy more than 4 weeks before enrollment
- •Treatment with TPO-RA within 1 week before enrollment
- •Inherited bone marrow failure syndromes
- •Bone marrow fibrosis grade ≥ 2
- •The presence of hemolytic PNH clone
- •The presence of clonal karyotypic abnormalities (del(20q), +8 and -Y are not included in this category)
- •Previously treated with TPO-RA ≥ 4 weeks
- •Previously received immunosuppressive therapy ≥ 12 weeks
- •Ferritin > 1000 ng/ml (The increased level of Ferritin led by infection is not included in this category)
- •Have an allergy to eltrombopag or any other part of this medicine.
- •History of radiotherapy and chemotherapy for malignant solid tumors
- •Cytopenia caused by other non-hematologic diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc
- •Abnormal liver function: ALT or AST > 3 ULN, or TBil > 1.5 ULN after treatment.
- •Abnormal kidney function: Creatinine clearance < 30 ml/min, or serum creatinine (sCr) >1.5 ULN
- •Patients with diabetic nephropathy, neuropathy, or eye disease
- •Patients with poorly controlled hypertension or cardiac arrhythmia
- •Patients with congestive heart failure and the NYHA grade ≥ 3 historically or currently, and LVEF < 45% within 4 weeks before enrollment
- •History of arteriovenous thrombosis within 1 year before enrollment
- •Participation in another clinical trial within 4 months before the start of this trial
- •Pregnant or breast-feeding patients
- •Patients considered to be ineligible for the study by the investigator for reasons other than the above
研究组 & 干预措施
CsA + Herombopag
Experimental
Herombopag combined with cyclosporine
干预措施: Herombopag (Drug)
结局指标
主要结局
Overall response rate
时间窗: 24 weeks
Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin, platelet and neutrophil count in the routine blood test.
次要结局
- Proportion of patients with abnormal karyotype changes(Baseline and 24 weeks)
- Time duration for patients achieving hematological response(A minimum of 2 years of planned follow-up)
- Robust response rate(24 weeks)
- Change of the health-related quality of life(Baseline and 24 weeks)
- Severity of the adverse event(24 weeks)
- Incidence of the adverse event(24 weeks)
研究者
研究点 (4)
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