Two Arm Bridging Study to Evaluate the Efficacy of Romiplostim in the Treatment of Adult Severe Aplastic Anemia Participants Who Are Either Previously Untreated With IST or Refractory to IST
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- Amgen
- 主要终点
- Arms 1 and 2: proportion of participants achieving any hematologic response at week 14
研究概览
简要总结
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. This study will evaluate the efficacy of romiplostim in the treatment of participants with SAA/vSAA.
The primary objectives of this study are to:
Arm 1: Evaluate the efficacy of romiplostim and IST in adult SAA/vSAA participants who are previously untreated with IST (1L)
Arm 2: Evaluate the efficacy of romiplostim treatment in adult SAA/vSAA participants who are refractory to IST (2L+)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years at time of enrollment
- •Diagnosis of SAA/vSAA confirmed by blood, bone marrow, and cytogenetic studies
- •An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening
- •Arm 1 only: participant requires initial treatment for SAA/vSAA, no matched related donor is available for allogenic hematopoietic cell transplantation (HCT) and will begin IST with antithymocyte globulin and CsA
- •Arm 2 only: refractory to at least one course of immunosuppressive therapy including horse or rabbit ATG; or ineligible for ATG treatment and refractory to CsA
排除标准
- •Diagnosed as having congenital aplastic anemia (AA) (Fanconi anemia, congenital dyskeratosis, etc)
- •History of other malignancy within the past 5 years, with exceptions.
- •Aplastic anemia with hemolytic paroxysmal nocturnal hemoglobinuria (PNH) (hemolytic predominant is defined as lactate dehydrogenase (LDH) > 1.5 x the upper limit of site normal
- •Arm 1 only: Previously treated with ATG, CsA, or Alemtuzumab
- •Previously treated with PEGylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin protein (TPO), romiplostim and other TPO-receptor agonist (eltrombopag, etc)
- •Patients who are eligible for allogenic HCT and have an available matched related donor
研究组 & 干预措施
Arm 1: Previously Untreated IST
Participants with SAA/vSAA that are previously untreated with IST.
干预措施: Romiplostim (Drug)
Arm 1: Previously Untreated IST
Participants with SAA/vSAA that are previously untreated with IST.
干预措施: Antithymocyte Globulin (Drug)
Arm 1: Previously Untreated IST
Participants with SAA/vSAA that are previously untreated with IST.
干预措施: Cyclosporine A (Drug)
Arm 2: Refractory IST
Participants with SAA/vSAA that are refractory to IST.
干预措施: Romiplostim (Drug)
结局指标
主要结局
Arms 1 and 2: proportion of participants achieving any hematologic response at week 14
时间窗: Week 14
Proportion of participants achieving any hematologic response at week 14 based on response criteria: * Platelet response * Erythroid response * Red blood cell count * Hemoglobin concentration * Neutrophil response
次要结局
- Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14(Week 14)
- Arms 1 and 2: number of participants with clinically significant changes in laboratory values(24 Weeks)
- Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14(Baseline and Week 14)
- Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim(Weeks 1, 2, 4, 5, 9, 13, and 24)
- Arms 1 and 2: area under the curve (AUC) of romiplostim(Weeks 1, 2, 4, 5, 9, 13, and 24)
- Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim(Weeks 1, 2, 4, 5, 9, 13, and 24)
- Arms 1 and 2: half-life (t1/2) of romiplostim(Weeks 1, 2, 4, 5, 9, 13, and 24)
- Arms 1 and 2: number of participants with antibodies to thrombopoietin(Prior to romiplostim administration on Weeks 1 and 13)
- Arms 1 and 2: number of participants who have a decrease in frequency of platelet and/or red blood cell (RBC) transfusions, or become platelet and/or RBC transfusion independent at week 14(Week 14)
- Arms 1 and 2: number of participants with serious adverse events(24 Weeks)
- Arms 1 and 2: serum romiplostim trough concentrations(Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24)
- Arms 1 and 2: number of participant with anti-romiplostim antibodies(Prior to romiplostim administration on Weeks 1 and 13)