Eltrombopag Combined With Telitacicept Versus Eltrombopag Monotherapy for the Treatment of Immune Thrombocytopenia Refractory or Relapsed to Corticosteroid Therapy: A Randomized Exploratory, Controlled, Open-label, Phase II Clinical Study
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Sustained Response Comparison
研究概览
简要总结
Background: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Corticosteroids are first-line therapy, but about one-third of patients relapse or are refractory. Eltrombopag (a TPO-RA) promotes platelet production, yet some patients show no response or relapse upon discontinuation. Telitacicept, a TACI-Fc fusion protein, dual-targets BAFF and APRIL, inhibiting B cell and plasma cell function and reducing autoantibody production, potentially providing synergistic immunomodulatory benefit.
Objective: To evaluate the sustained response rate of eltrombopag plus telitacicept vs. eltrombopag alone in patients with steroid-refractory/relapsed ITP.
Design: Randomized, open-label, controlled, exploratory Phase II study. 40 patients planned (20 combination, 20 monotherapy). Combination group: eltrombopag + telitacicept . Monotherapy group: eltrombopag alone for 12 weeks. Monotherapy patients with no response after 4 weeks may cross over to the combination group. After 12 weeks, treatment is stopped and patients are followed until Week 24.
Primary endpoint: Proportion of patients maintaining platelet count ≥30×10⁹/L with no bleeding at 24 weeks post-treatment. Secondary endpoints include platelet response rates during 12 weeks, safety, bleeding events, etc.
Expected results: The combination group is expected to have a significantly higher proportion of patients achieving the primary endpoint without increased adverse events.
Population: Age ≥18, diagnosed ITP ≥3 months, baseline platelets <30×10⁹/L, prior corticosteroid failure.
Safety: Monitoring for bleeding, infection, thrombosis, cytopenia, etc., with dose adjustment/cessation as per protocol.
Conclusion: This study explores whether dual-targeting (platelet production + autoimmune suppression) with eltrombopag and telitacicept can provide more durable remission for steroid-refractory/relapsed ITP patients.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years, regardless of sex.
- •Clinically diagnosed with immune thrombocytopenia for at least 3 months prior to enrollment. Platelet count <30×10⁹/L within 48 hours before the first dose of study drug.
- •Previous failure (ineffective, unable to maintain response, or relapse) to first-line standard corticosteroid therapy for ITP as recommended by guidelines.
- •Any prior emergency treatment for ITP (e.g., corticosteroids, platelet transfusion, intravenous immunoglobulin) must have been completed at least 2 weeks before the first dose.
- •Patients receiving maintenance corticosteroid therapy must be on a stable dose for at least 2 weeks prior to the first dose; patients receiving immunosuppressants (e.g., azathioprine, danazol, cyclosporine A, tacrolimus, sirolimus, etc.) must be on a stable dose for at least 4 weeks prior to the first dose; anti-CD20 antibody therapy must have been completed >3 months prior.
- •Understand the study procedures and voluntarily provide written informed consent.
排除标准
- •Received anti-CD20 antibody therapy within 3 months.
- •Uncontrolled primary disease of vital organs, such as malignant tumors, liver failure, heart failure, renal failure, etc.
- •Positive for HIV.
- •Uncontrolled active viral or bacterial infections, including positive for hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus, or syphilis.
- •Extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.
- •Currently have cardiac disease requiring treatment, arrhythmia, or hypertension poorly controlled as judged by the investigator.
- •Patients with thrombotic diseases such as pulmonary embolism, thrombosis, atherosclerosis, etc.
- •Patients with mental disorders who are unable to give informed consent or undergo study procedures and follow-up normally.
- •Patients whose toxic symptoms from prior treatment before enrollment have not yet resolved.
- •Other serious diseases that may limit the patient's participation in this study (e.g., poorly controlled diabetes; severe cardiac insufficiency; myocardial infarction, unstable arrhythmia, or unstable angina within the past 6 months; gastric ulcer; active autoimmune disease, etc.).
- •Pregnant women, suspected pregnancy (positive urinary human chorionic gonadotropin pregnancy test at screening), or lactating patients.
结局指标
主要结局
Sustained Response Comparison
时间窗: Within 24weeks of first dose
To compare the sustained response rate of eltrombopag combined with telitacicept versus eltrombopag monotherapy in patients with immune thrombocytopenia who are refractory or relapsed to prior corticosteroid therapy.
次要结局
- Platelet Response Within 12 Weeks(Within 12 weeks of first dose)
- Platelet ≥50×10⁹/L at Week 12(Within 12 weeks of first dose)
- Platelet ≥100×10⁹/L at Week 12(Within 12 weeks of first dose)
- Time to First Platelet Response(Within 12 weeks of first dose)
- Proportion of Days with Platelet ≥30×10⁹/L(Within 12 weeks of first dose)
- Platelet Response After Crossover (Monotherapy Group)(With in 24 weeks of first dose)
- Safety and Tolerability(Within 24 weeks of first dose)
- Time to Rescue Therapy or Platelet Decline(Within 24 weeks of first dose)
- Proportion Requiring Rescue Therapy(Within 24 weeks of first dose)