An Open-label, Single-arm, Single-center Study to Evaluate the Efficacy and Safety of Romiplostim N01 in Combination With Rituximab in Patients With Primary Immune Thrombocytopenia Refractory to Oral TPO-RAs
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Efficacy evaluation
研究概览
简要总结
The primary objective of this trial is to assess the efficacy and safety of combining Romiplostim N01 with Rituximab for the treatment of adult patients with primary immune thrombocytopenia (ITP) whose disease is refractory to oral TPO-RAs.
All participants in this study will receive the same combination treatment:
Rituximab: Given once a week through an intravenous infusion for 4 weeks. Romiplostim N01: Given as a weekly injection，the dose may be adjusted each week based on the patient's platelet count.
Participants will be asked to:
Visit the clinic regularly for check-ups, blood tests (to monitor platelet counts), and safety assessments.
Report their bleeding symptoms for evaluation. This is an open-label, single-arm trial, meaning that all participants will receive the investigational treatment, and both the research team and participants will be aware of the treatment assigned. The study aims to enroll approximately 30 adult patients aged 14 years or older who have been diagnosed with ITP and have not responded adequately to prior oral TPO-RAs (eltrombopag/hetrombopag).
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
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of primary immune thrombocytopenia (ITP).
- •Age ≥14 years.
- •Prior exposure to at least one ITP-directed therapy (e.g., corticosteroids, intravenous immunoglobulin, recombinant human thrombopoietin, TPO-RAs, immunosuppressants, or splenectomy), including a minimum 4-week course of an oral TPO-RA (hetrombopag or eltrombopag) that was discontinued due to insufficient response.
- •Platelet count < 30 × 10⁹/L at screening, OR platelet count < 50 × 10⁹/L with concurrent clinically significant bleeding.
- •Stable glucocorticoid dose (e.g., prednisone or methylprednisolone, not exceeding 4 tablets daily) for at least 2 weeks, and stable dosing of any other immunosuppressants for at least 4 weeks prior to enrollment.
- •No receipt of intravenous immunoglobulin within 2 weeks before the first dose of study treatment.
- •No platelet transfusion within 1 week before the first dose of study treatment.
排除标准
- •Secondary ITP due to underlying conditions such as other autoimmune disorders, viral infections, or drug exposure.
- •Presence of active malignancy, pregnancy, significant cardiovascular or cerebrovascular disease, or history of arterial/venous thrombosis.
研究组 & 干预措施
Romiplostim N01 + Rituximab Combination Therapy
干预措施: Romiplostim N01 (Drug)
Romiplostim N01 + Rituximab Combination Therapy
干预措施: Rituximab (Drug)
结局指标
主要结局
Efficacy evaluation
时间窗: from week 2 to month 12
Efficacy assessments (platelet count) are performed weekly from Week 2 to Week 8, and then monthly until Month 12. Efficacy evaluation relies on time to platelet response, overall response rate, complete response rate and maintained response rate
Incidence of severe Adverse Events
时间窗: up to 12 months
Adverse events include the monitoring of the incidence of thrombotic events, myelofibrosis, and severe infections.
次要结局
- Long-term Sustained Response Rate versus Relapse Rate(at 6 months and 12 months)
- Proportion of Participants Achieving a Rapid Platelet Response(within the first 8 weeks of treatment)
- Change in Bleeding Score Over Time(up to 12 months)
- Incidence of other adverse events(up to 12 months)