Comparison of Etamsylate Versus Placebo to Prevent Bleeding in Hematopoietic Stem Cell Transplantation Recipients：a Randomized, Double-blind, Phase 3, Clinical Study
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 404
- 试验地点
- 1
- 主要终点
- WHO grade 2 or higher bleeding events or death.
研究概览
简要总结
This study employs a prospective, randomized, double-blind, placebo-controlled design. It aims to compare the efficacy of etamsylate versus placebo in preventing bleeding complications in patients with thrombocytopenia following hematopoietic stem cell transplantation.
详细描述
This study employs a prospective, randomized, double-blind, placebo-controlled design. It aims to compare the efficacy of etamsylate versus placebo in preventing bleeding complications in patients with thrombocytopenia following hematopoietic stem cell transplantation. The primary endpoint is the incidence of death or bleeding events (WHO grade ≥2) within 30 days. Secondary endpoints focus on the safety profile of etamsylate versus placebo.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age between 18 and 70 years (inclusive), regardless of gender;
- •Patients diagnosed with a hematological disease requiring hematopoietic stem cell transplantation;
- •Expected platelet count ≤ 10 x 10⁹/L persisting for 5 days or more;
- •Normal coagulation function;
- •Adequate organ function: Aspartate Aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 3 times the Upper Limit of Normal (ULN), Total Bilirubin ≤ 2 x ULN; Serum Creatinine ≤ 2 x ULN; Creatinine Clearance ≥ 50 mL/min (calculated using the Cockcroft-Gault formula); Left Ventricular Ejection Fraction (LVEF) ≥ 50% as measured by Echocardiography (ECHO);
- •Voluntary provision of signed informed consent, with the ability to understand and comply with all study requirements.
排除标准
- •Diagnosis of acute promyelocytic leukemia confirmed according to WHO diagnostic criteria;
- •Pregnant or breastfeeding women, and women of childbearing potential unwilling to use effective contraception;
- •Presence of active bleeding or infection;
- •History of diagnosed primary immune thrombocytopenia or hemolytic uremic syndrome;
- •Patients with known hereditary or acquired hemorrhagic disorders;
- •Patients receiving anticoagulant or antiplatelet therapy;
- •Patients with severe cardiac insufficiency (left ventricular ejection fraction [EF] < 60%); or severe arrhythmias: history of clinically significant QTc prolongation (male > 450 ms; female > 470 ms), ventricular tachycardia, atrial fibrillation, second-degree heart block; myocardial infarction within 1 year prior to enrollment; or symptomatic coronary artery disease requiring medication; patients with severe liver impairment (liver function indices [ALT, TBIL] > 3 times the upper limit of normal [ULN]);
- •Patients with severe pulmonary insufficiency (obstructive and/or restrictive ventilatory defects);
- •Patients with severe renal insufficiency (renal function index [Cr] > 2 times ULN); or 24-hour urinary creatinine clearance rate (Ccr) < 50 ml/min;
- •Patients with mental disorders or other conditions preventing provision of informed consent and compliance with study treatment and procedural requirements;
- •Other reasons deemed by the investigator to make the patient ineligible for inclusion.
研究组 & 干预措施
etamsylate
Etamsylate Injection: Administer by intravenous infusion, diluted with 100ml of 0.9% Sodium Chloride Injection, twice daily.
干预措施: etamsylate (Drug)
placebo
0.9% Sodium Chloride Injection, 100 ml, twice daily, intravenous drip
干预措施: Placebo (Drug)
结局指标
主要结局
WHO grade 2 or higher bleeding events or death.
时间窗: 30 days
The incidence of WHO grade 2 or higher bleeding events or death within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the incidence in the experimental group is 14.2% lower than that in the placebo group.
次要结局
- The number of platelet transfusions(The number of platelet transfusions within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the number of platelet transfusions in the experimental group is 2.5 fewer than in the placebo group.)
- The number of days without WHO grade 2 or higher bleeding events(30 days)
研究者
Yi Luo
Professor
First Affiliated Hospital of Zhejiang University