Phase II Open-label Clinical Trial Evaluating Efficacy of Romiplostim Added to Standard of Care for Children and Young Adults With Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24
研究概览
简要总结
This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage hematopoiesis in children and young adults.
详细描述
The study is designed as a Phase II, multicenter, investigator-initiated, open label, interventional study that will recruit children (age: >2 to <21 years) with SAA. The primary objective of the study is to evaluate the efficacy of romiplostim (a TPO-RA with an orphan drug designation) for the treatment of SAA in children and young adults with newly diagnosed and relapsed or refractory SAA. Hematologic complete response (HCR) will be used to assess the therapy response.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)
Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).
干预措施: Romiplostim (Drug)
Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)
Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).
干预措施: Immunosuppressive therapy (IST) (Drug)
Relapsed or refractory SAA (Cohort B): Romiplostim
Relapsed or refractory SAA (Cohort B) will be treated with romiplostim alone.
干预措施: Romiplostim (Drug)
结局指标
主要结局
To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24
时间窗: During 24 weeks of therapy
Proportion of participants with hematopoietic complete response at 24 weeks defined as hemoglobin ≥10 g/dL, ANC ≥1 x 10\^9/L, and platelet count ≥100 x 10\^9/L without having received transfusion of packed red blood cells within the last 6 weeks or platelets or G-CSF/GM-CSF within the last 2 weeks.
次要结局
- Incidence of adverse events as assessed by CTCAE v5.0(One year following completion of treatment)
- Proportion of participants with a new cytogenetic abnormality(One year following completion of treatment)
研究者
Anjali Sharathkumar
Clinical Professor
University of Iowa