Efficacy and Safety of a Long-acting Thrombopoietin Receptor Agonist for Platelet Recovery in Adult Hematologic Malignancies Patients Undergoing Umbilical Cord Blood Transplantation
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Time of platelet engraftment
研究概览
简要总结
To evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after umbilical cord blood transplantation (UCBT) in patients with hematological malignancies.
详细描述
Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is an important means of curing a wide range of hematologic diseases, and good hematopoietic and immune reconstitution after transplantation is one of the most important factors determining the success of the transplantation.Persistent thrombocytopenia after Allo-HSCT is one of the common post-transplantation complications.This is an investigator-initiated, single-center, single-arm, Phase II clinical trial to evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after UCBT in patients with hematologic tumors. The study was conducted in patients ≥18 years of age with a diagnosis of hematological neoplasia who underwent UCBT. Subjects will be screened and receive roprostin N01 from +1 to +28 days after UCBT after signing an informed consent form, and the study will focus on the cumulative PLT implantation rate at 28 days after transplantation as the primary efficacy measure. The study is planned to enroll 34 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
- •Age ≥12 years old, male or female;
- •Patients diagnosed with hematological malignancies and undergoing UCBT;
- •ECOG score ≤2;
- •Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent
排除标准
- •Pregnant or lactating women；
- •Known allergy to Romiplostim N01;
- •A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
- •A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
- •Chronic active hepatitis B and C;
- •Repeat or multiple transplantation or multiple organ transplantation;
- •HIV positive, EBV-DNA positive, CMV-DNA positive;
- •Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
- •A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
- •Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
- •Subjects with cognitive impairment or uncontrolled mental illness;
- •Subjects and/or authorized family members to refuse treatment with Romiplostim N01;
- •Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).
研究组 & 干预措施
Romiplostim N01
干预措施: Romiplostim N01 (Drug)
结局指标
主要结局
Time of platelet engraftment
时间窗: 28 days
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.
The cumulative incidence of platelet engraftment at 28 days after transplantation
时间窗: 28 days
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.
次要结局
- The cumulative incidence of platelet recovery rate at 28 days after transplantation(28 days)
- Time of platelet recovery(28 days)
- Total platelet transfusion during 4 weeks of treatment(28 days)
- Cumulative implantation rate of the granulocyte lineage at 28 days post-transplantation(28 days)
- Megakaryocyte levels in bone marrow smears 4 weeks after treatment(4 weeks)
研究者
Xiaoyu Zhu
ph.D
Anhui Provincial Hospital