A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- Proportion of Subjects Achieving a Platelet Response at Week 9
研究概览
简要总结
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.
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
- 19 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient who is diagnosed as AA and refractory to immunosuppressive therapy
- •Platelet ≤ 30,000/μL
排除标准
- •Concurrent active infection not adequately responding to appropriate therapy
- •HIV positivity
- •Bone marrow reticulin grade of > 1
- •Clinically significant cardiac disease
- •Arterial or venous thrombosis within the last 1 year before enrollment
- •Other cause of thrombocytopenia
- •AA with hemolytic predominant paroxysmal nocturnal hemoglobinuria (PNH)
- •Uncontrolled diabetes
- •Receiving any agent used to treat AA, including antithymocyte globulin (ATG) or ATG + cyclosporine within 6 months before starting study treatment and/or cyclosporine or anabolic hormone within 6 weeks before starting the study treatment
- •History of PEG-rHuMGDF, recombinant human thrombopoietin, AMG531, and other thrombopoietin (TPO)-receptor agonist
- •Who plans to conduct hematopoietic stem cell transplantation within 1 year
研究组 & 干预措施
AMG531 (Dose 1)
干预措施: AMG531 (Drug)
AMG531 (Dose 2)
干预措施: AMG531 (Drug)
AMG531 (Dose 3)
干预措施: AMG531 (Drug)
AMG531 (Dose 4)
干预措施: AMG531 (Drug)
结局指标
主要结局
Proportion of Subjects Achieving a Platelet Response at Week 9
时间窗: At week 9
The proportion of subjects achieving a platelet response at Week 9, and two-sided 95% confidence interval will be calculated. Platelet response is defined as 1) achieving absolute platelet increase of ≥ 20x10\^9/L above baseline or 2) increase to ≥ 10x10\^9/L and by at least 100% from baseline.
次要结局
- Proportion of Subjects Achieving Tri-lineage Responses(Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157)
- Duration of Study Drug Discontinuation While Maintaining Stable Response(Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156)
- Proportion of Subjects Achieving a Platelet Response(Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156)
- Proportion of Subjects Achieving Platelet Transfusion Independency(Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156)
- Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response(Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156)
- Duration of Platelet Response and Time to Platelet Response(Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156)