Efficacy and Safety of Romiplostim N01 for Injection in Promoting Platelet Reconstitution After Allogeneic Hematopoietic Stem Cell Transplantation: A Prospective, Single-Center, Randomized Controlled Clinical Study
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 87
- 试验地点
- 1
- 主要终点
- Platelet engraftment time
研究概览
简要总结
This is a prospective, single-center, randomized controlled, investigator-initiated clinical study. The purpose of this study is to evaluate the efficacy and safety of Romiplostim N01 for Injection in promoting platelet reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible participants will be randomly assigned in a 2:1 ratio to receive either Romiplostim N01 for Injection (experimental group) or the same treatment with the exception of Romiplostim N01 for Injection (control group).In the experimental group, Romiplostim N01 for Injection will be administered subcutaneously once weekly starting from Day 1 after allo-HSCT, with an initial dose of 3.0 μg/kg and dose titration based on platelet counts, up to a maximum of 10 μg/kg once weekly. Treatment will continue until platelet count exceeds 20×10⁹/L for 7 consecutive days without platelet transfusion, or until lack of efficacy after 4 weeks at 10 μg/kg, or at the investigator's discretion. Participants will enter a safety follow-up period for 28 days after treatment completion, with visits as frequently as weekly to collect adverse events, concomitant medications, and supportive care information.
The primary efficacy endpoint is the time to platelet engraftment, defined as the first day of platelet count ≥20×10⁹/L for 7 consecutive days without platelet transfusion.
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
- •1.Subjects must understand study procedures, be willing to comply with the study protocol, and provide written informed consent prior to any study-related procedures.
- •2. Age ≥18 years, male or female.
- •Patients with hematological malignancies undergoing allogeneic hematopoietic stem-cell transplantation (allo-HSCT).
- •4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or
- •Adequate organ function defined as follows:
- •Serum bilirubin ≤ 2 mg/dL, unless due to benign congenital hyperbilirubinemia;
- •AST, ALT and alkaline phosphatase < 3 × upper limit of normal (ULN);
- •Creatinine clearance ≥ 40 mL/min (calculated by the Cockcroft-Gault formula);
- •Left ventricular ejection fraction (LVEF) ≥ 45% by MUGA scan or resting echocardiogram;
- •Pulmonary function: FEV1 and corrected DLCO ≥45% of predicted value.
- •Participants of reproductive potential must use reliable contraceptive methods throughout the study period (including male or female condoms, contraceptive foam, contraceptive gel, contraceptive diaphragm, contraceptive cream, contraceptive suppositories, abstinence, intrauterine device, etc.). Exceptions include female participants who have undergone hysterectomy, bilateral salpingectomy, bilateral tubal ligation, or are postmenopausal for more than 1 year; and male participants with bilateral vasectomy.
排除标准
- •1. Subjects who have received romiplostim, eltrombopag, or other TPO-RA thrombopoietic agents within 1 month prior to enrollment.
- •2. History of symptomatic or incidental venous thromboembolic events (e.g., deep vein thrombosis or pulmonary embolism), except patients who received and tolerated anticoagulation within the preceding 6 months, completed anticoagulation >6 months ago, or are receiving ongoing anticoagulation. Patients with central venous catheter-related venous thromboembolic events are excluded.
- •3. Patients with a history of symptomatic arterial thrombotic events including myocardial infarction, ischemic cerebrovascular accident, or transient ischemic attack within the preceding 6 months.
- •4. Patients with severe underlying diseases that may interfere with the conduct of the study, such as unstable angina, renal failure requiring hemodialysis, or active infection requiring intravenous antibiotic therapy.
- •5. Abnormal hepatic or renal function: total bilirubin > 2 × upper limit of normal (ULN); AST or ALT > 1.5 × ULN; creatinine > 1.5 × ULN; estimated glomerular filtration rate < 30 mL/min/1.73 m².
- •6. Patients with positive hepatitis C antibody plus elevated HCV-RNA; patients with positive hepatitis B surface antigen plus elevated HBV-DNA; patients with severe liver cirrhosis; patients with positive HIV antibody or positive syphilis antibody.
- •7. Subjects with known hypersensitivity or intolerance to the active ingredient or excipients of injectable Romiplostim N
- •8.Participation in any other interventional clinical study of investigational drugs or devices within 1 month prior to screening.
- •9. Female subjects who are pregnant or breastfeeding.
- •Subjects who, in the investigator's judgment, are unable to comply with study procedures, or for whom study participation is otherwise deemed inappropriate by the investigator.
研究组 & 干预措施
Control Group
Participants in the control group receive identical standard allo-HSCT supportive care, with the exception of Romiplostim N01 for Injection
Romiplostim N01
Participants receive Romiplostim N01 for Injection subcutaneously once weekly starting from Day 1 after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The initial dose is 3.0 μg/kg, with dose titration based on platelet counts, up to a maximum of 10 μg/kg once weekly. Treatment continues until platelet count exceeds 20×10⁹/L for 7 consecutive days without platelet transfusion, or until lack of efficacy after 4 weeks at 10 μg/kg, or at the investigator's discretion.
干预措施: Romiplostim N01 (Drug)
结局指标
主要结局
Platelet engraftment time
时间窗: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
the first day of platelet count ≥20×10⁹/L sustained for 7 consecutive days in the absence of platelet transfusion
次要结局
- Incidence and severity of adverse events (AEs) and adverse drug reactions (ADRs)(From treatment initiation to 28 days after treatment completion)
- Neutrophil engraftment time(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)
- Median time to achieve complete remission (CR)(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)
- Incidence of primary poor graft function (PGF)(Within 28 days after allo-HSCT)
- Incidence of secondary platelet recovery failure(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)
- Incidence of graft-versus-host disease(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)
- Incidence of primary graft failure(At Day 30 or Day 42 after cell infusion)
- Incidence of secondary graft failure(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)
- Comparison of treatment-related costs between Romiplostim N01 group and control group(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)
- Platelet transfusion requirements during engraftment(From Day +1 after allogeneic-HSCT up to 1 year post-transplant)