A Phase 1a/b Study to Evaluate the Safety & Tolerability of JSP191 in Combination With a Regimen of Low Dose Radiation & Fludarabine in Subjects With MDS or AML Undergoing Hematopoietic Cell Transplantation (HCT)
试验速览
- 阶段
- 1 期
- 入组人数
- 40
- 试验地点
- 12
- 主要终点
- The number of subjects experiencing adverse events and serious adverse events will be assessed.
研究概览
简要总结
This is a Phase 1a/b study to evaluate the safety and tolerability of an antibody conditioning regimen known as JSP191, in combination with low dose radiation and fludarabine, in subjects with Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML) undergoing allogenic blood stem cell transplantation.
详细描述
This is a Phase 1a/b study to evaluate the safety and tolerability of an antibody conditioning regimen known as JSP191, in combination with low dose radiation and fludarabine in subjects with Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML) undergoing allogenic blood stem cell transplantation.
Blood Stem Cell transplantation offers the only potentially curative therapy for many forms of Acute Myeloid Leukemia (AML) and for Myelodysplastic Syndrome (MDS). While standard of care conditioning regimens given prior to blood Stem Cell transplantation, such as standard TBI/Flu conditioning are well tolerated, they are associated with increased rates of relapse due to persistence of disease causing Hematopoietic Stem Cells and insufficient graft versus leukemia effect.
The biological conditioning regimen JSP191 is an antibody that binds to CD117. CD117 is the receptor for Stem Cell Factor on blood forming cells. CD117 binding to Stem Cell Factor is critical for survival and maintenance of blood forming stem cells.
The binding of JSP191 to CD117 blocks CD117 from binding to Stem Cell Factor on blood forming stem cells. In the absence of CD117/Stem Cell Factor binding, hematopoietic stem cells that are currently occupying the bone marrow niches in MDS/AML patients are depleted.
This study will investigate the safety and tolerability of adding JSP191 (an anti-CD117 monoclonal antibody therapy) to standard TBI/Flu conditioning regimen in adults with AML and MDS undergoing hematopoietic stem cell transplant.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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
- •AML/MDS as defined by specific criteria, including but not limited to the following subtypes:
- •MDS < 5% BM blasts
- •MDS 5 - 10% BM blasts
- •AML not in CR or MDS > 10% BM blasts
- •Patients with human leukocyte antigen (HLA) matched related or unrelated donors
- •Adequate end organ function as defined in study protocol
排除标准
- •Patients with any acute or uncontrolled infections
- •Patients receiving any other investigational agents
- •Patients with active non-hematologic malignancy
- •Prior allogeneic hematologic cell transplantation
结局指标
主要结局
The number of subjects experiencing adverse events and serious adverse events will be assessed.
时间窗: Up to 1 year post Donor Cell Transplant (28 days dose limiting toxicity period)
The number of subjects experiencing adverse events and serious adverse events will be assessed.
The number of dose limiting toxicities will be assessed.
时间窗: Up to 1 year post Donor Cell Transplant (28 days dose limiting toxicity period)
The number of dose limiting toxicities will be assessed.
The type of dose limiting toxicities will be assessed.
时间窗: Up to 1 year post Donor Cell Transplant (28 days dose limiting toxicity period)
The type of dose limiting toxicities will be assessed.
次要结局
未报告次要终点