Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 35
- 试验地点
- 6
- 主要终点
- Best Response
研究概览
简要总结
The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.
Funding Source- FDA OOPD
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients undergoing workup for suspected newly diagnosed sAA:
- •Patients with severe cytopenias and a hypocellular marrow concerning for sAA
- •Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:
- •Marrow Cellularity: <25%, or 25-50% with <30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): <500 x 10^9/L Platelets: <20 x 10^9/L Absolute Reticulocyte Count: <60 x 10^9/L
- •Patients that do not have evidence of leukemia or MDS
- •Patients < 25 years of age at time of diagnosis
- •Able to tolerate emapalumab and IST (with standard institutional organ function criteria)
排除标准
- •Uncontrolled infection at presentation.
- •Patients who have undergone previous treatment for sAA.
- •Patients with known inherited bone marrow failure
- •Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial
- •Patients with leukemia or MDS
- •Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.
研究组 & 干预措施
Emapalumab, then Standard IST
Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will receive standard IST with drugs called equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab
干预措施: Emapalumab (Biological)
Emapalumab, then HCT
Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).
干预措施: Emapalumab (Biological)
结局指标
主要结局
Best Response
时间窗: 6 weeks
The primary objective of the study is to assess the efficacy of early upfront emapalumab on hematologic recovery within 6 weeks of starting therapy after a new diagnosis of Aplastic Anemia. Response will be determined by blood count.
次要结局
未报告次要终点