Efficacy and Safety of Romiplostim N01 in Promoting Platelet Reconstruction After Haploidentical Allogeneic Stem Cell Transplantation in Patients With Hematologic Malignancies
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 130
- 试验地点
- 1
- 主要终点
- Cumulative Platelet Engraftment Rate by Day +21 after Haploidentical HSCT
研究概览
简要总结
This is a prospective, randomized, controlled clinical study designed to evaluate the efficacy and safety of Romiplostim N01 in promoting platelet engraftment after haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) in patients with hematologic malignancies.
A total of 130 patients who undergo haplo-HSCT for acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or other hematologic malignancies will be enrolled and randomized 1:1 into a treatment group and a control group. The treatment group will receive Romiplostim N01 subcutaneously once weekly at a starting dose of 5 µg/kg, with dose adjustments based on platelet counts (maximum 10 µg/kg), for up to 4 weeks or until platelet counts reach ≥100 × 10⁹/L. The control group will not receive rh-TPO or any thrombopoietin receptor agonist (TPO-RA) therapy. Supportive care including transfusions and growth factors (G-CSF, ESA) is allowed in both groups.
The primary endpoint is the cumulative platelet engraftment rate by day +21 post-transplant, defined as sustained platelet counts > 20 × 10⁹/L for at least 7 consecutive days without transfusion. Secondary endpoints include median time to platelet engraftment, median time to achieve platelet counts ≥ 50 × 10⁹/L and ≥ 100 × 10⁹/L, total platelet transfusion volume, erythroid and neutrophil responses within 4 weeks, and overall hematopoietic recovery. Safety endpoints include the incidence of adverse events, thromboembolic events, and treatment-related serious adverse events.
The study aims to determine whether early administration of Romiplostim N01 can accelerate platelet recovery and reduce bleeding risk in patients undergoing haplo-HSCT, thereby improving post-transplant outcomes.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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
- •Patients with malignant hematologic diseases scheduled to undergo haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) Age ≥18 years, male or female. ECOG performance status 0-
- •Estimated life expectancy >6 months. Adequate renal function, defined as:serum creatinine ≤1.5 × upper limit of normal (ULN);Blood urea nitrogen (BUN) ≤1.5 × ULN.
- •Adequate hepatic function, defined as:Alanine aminotransferase (ALT) ≤2 × ULN.Aspartate aminotransferase (AST) ≤2 × ULN.Total bilirubin ≤1.5 × ULN.
- •Ability to understand and sign informed consent, and willingness to comply with all study requirements.
排除标准
- •Uncontrolled active infection or other active malignancy that could interfere with study participation.
- •Severe cardiovascular disease, including:New York Heart Association (NYHA) Class III-IV heart failure;Uncontrolled hypertension or hypotension;History of or high risk for thromboembolic events.
- •Receiving anticoagulation therapy for thrombotic events. Known hypersensitivity to romiplostim or similar agents. Use of rh-TPO or any thrombopoietin receptor agonist (TPO-RA) within 30 days prior to enrollment.
- •Participation in another interventional clinical study within 30 days prior to enrollment.
- •Any other condition that, in the investigator's judgment, makes the patient unsuitable for the study.
研究组 & 干预措施
Romiplostim N01 Treatment Arm
干预措施: Romiplostim N01 (Drug)
Romiplostim N01 Treatment Arm
干预措施: Standard Supportive Care (Other)
Standard Care Control Arm
干预措施: Standard Supportive Care (Other)
结局指标
主要结局
Cumulative Platelet Engraftment Rate by Day +21 after Haploidentical HSCT
时间窗: Within 21 days after transplantation
Platelet engraftment is defined as achieving a sustained platelet count \>20 × 10⁹/L for at least 7 consecutive days without platelet transfusion. The cumulative engraftment rate by day +21 post-transplant will be compared between the Romiplostim N01 treatment arm and the standard care control arm.
次要结局
- Time to Platelet Engraftment(Up to 60 days after transplantation)
- Proportion of Patients Achieving Platelet Counts ≥50 × 10⁹/L and ≥100 × 10⁹/L(Up to 60 days after transplantation)
- Median Time to Achieve Platelet Count ≥100 × 10⁹/L during the 4-Week Treatment Period(Within 4 weeks after initiation of Romiplostim N01 treatment)
- Total Platelet Transfusion Volume(Up to 60 days after transplantation)
- Proportion of Participants with Erythroid Response during the 4-Week Treatment Period(Within 4 weeks after initiation of Romiplostim N01 treatment)
- Proportion of Participants with Neutrophil Response during the 4-Week Treatment Period(Within 4 weeks after initiation of Romiplostim N01 treatment)