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Efficacy and Safety of Romiplostim N01 for Injection in Promoting Platelet Reconstitution After Allogeneic Hematopoietic Stem Cell Transplantation

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

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.

研究概览

研究类型

介入性

注册 (估计的)

87

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习联系方式

学习地点

    • Henan
      • Luoyang、Henan、中国、471023
        • The First Affiliated Hospital of Henan University of Science and Technology
        • 接触:

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

18年 及以上 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

  • 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. 3. Patients with hematological malignancies undergoing allogeneic hematopoietic stem-cell transplantation (allo-HSCT).

    4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. 5. 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. 6. 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.

Exclusion Criteria:

  • 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 N01.

    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. 10. 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.

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

  • 主要用途:治疗
  • 分配:随机化
  • 介入模型:并行分配
  • 屏蔽:无(打开标签)

武器和干预

参与者组/臂
干预/治疗
实验性的: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.

Subcutaneous injection once weekly, initiated on Day +1 after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The starting dose is 3.0 μg/kg. The dose will be titrated according to peripheral platelet counts up to a maximum weekly dose of 10 μg/kg. Treatment will be discontinued when any of the following criteria are met: platelet count ≥20×10⁹/L sustained for 7 consecutive days without platelet transfusion; inadequate platelet response after 4 consecutive weeks at the maximum dose of 10 μg/kg; or discontinuation at the investigator's clinical discretion.

无干预:Control Group

Participants in the control group receive identical standard allo-HSCT supportive care, with the exception of Romiplostim N01 for Injection

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
Platelet engraftment time
大体时间: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

From Day +1 after allogeneic-HSCT up to 1 year post-transplant

次要结果测量

结果测量
措施说明
大体时间
Neutrophil engraftment time
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

the first day when absolute neutrophil count (ANC) ≥0.5×10⁹/L sustained for 3 consecutive days in the absence of G-CSF support

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Median time to achieve complete remission (CR)
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Defined as the median time to achieve complete remission (CR), where complete remission is defined as platelet count ≥50×10⁹/L sustained for 7 consecutive days without platelet transfusion.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of primary poor graft function (PGF)
大体时间:Within 28 days after allo-HSCT

Defined as the incidence of primary poor graft function, a condition occurring within 28 days after transplantation, characterized by full donor chimerism, absence of severe GVHD or disease relapse, successful myeloid and erythroid engraftment (absolute neutrophil count >0.5×10⁹/L and hemoglobin >70 g/L), with sustained platelet count ≤20×10⁹/L.

Within 28 days after allo-HSCT
Incidence of secondary platelet recovery failure
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Incidence of secondary platelet recovery failure (defined as a decline in platelet count to <20,000/μL after achievement of initial platelet recovery, lasting for at least 7 days or requiring platelet transfusion within 7 days)

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of graft-versus-host disease
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Acute GVHD is graded according to the Glucksberg grading criteria; chronic GVHD is diagnosed and graded based on the National Institutes of Health (NIH) consensus criteria and Chinese expert consensus.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of primary graft failure
大体时间:At Day 30 or Day 42 after cell infusion

① For peripheral blood stem cells (PBS) or non-mobilized bone marrow cells: ANC < 0.5×10⁹/L accompanied by pancytopenia on Day 30 after infusion.

② For umbilical cord blood (UCB): ANC < 0.5×10⁹/L accompanied by pancytopenia on Day 42 after infusion.

At Day 30 or Day 42 after cell infusion
Incidence of secondary graft failure
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

deterioration of hematopoietic function (involving hemoglobin and/or platelets and/or neutrophils) after achievement of hematopoietic reconstitution (including hemoglobin, platelets and/or neutrophils), requiring blood product transfusion or growth factor support.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Comparison of treatment-related costs between Romiplostim N01 group and control group
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Comparison of treatment-related costs between the Romiplostim N01 group and the control group.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Platelet transfusion requirements during engraftment
大体时间:From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Total number of platelet transfusions administered during the platelet engraftment period.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence and severity of adverse events (AEs) and adverse drug reactions (ADRs)
大体时间:From treatment initiation to 28 days after treatment completion

Adverse events and adverse drug reactions will be recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

From treatment initiation to 28 days after treatment completion

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年11月1日

初级完成 (估计的)

2028年6月1日

研究完成 (估计的)

2028年12月31日

研究注册日期

首次提交

2026年9月11日

首先提交符合 QC 标准的

2026年9月17日

首次发布 (实际的)

2026年9月21日

研究记录更新

最后更新发布 (实际的)

2026年9月21日

上次提交的符合 QC 标准的更新

2026年9月17日

最后验证

2026年9月1日

更多信息

与本研究相关的术语

其他研究编号

  • HN-Blood-HSCT-107

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.

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