A Pilot Study of Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamide (Immunochemotherapy) and Allogeneic Stem Cell Transplantation in Patients With High Risk Acute Myelogenous Leukemia and Myelodysplastic Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Response rate
研究概览
简要总结
Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with chemotherapy followed by allogeneic stem cell transplantation will be given to patients with high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).
详细描述
Gemtuzumab ozogamicin in combination with busulfan and cyclophosphamide (immunochemotherapy) conditioning followed by allogeneic stem cell transplantation + anti-thymocyte globulin (unrelated donors only) in patients with high risk CD33+AML or MDS meeting eligibility criteria.
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
- — 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Disease Status:
- •History of AML Induction/Reinduction Failure
- •AML 1st Complete Remission (CR) with poor cytogenetics
- •AML 2nd CR with minimal residual disease (MRD)
- •AML 3rd CR
- •AML in refractory relapse but ≤25% bone marrow leukemia blasts
- •MDS with >6% bone marrow blasts at diagnosis
- •Secondary MDS with ≤5% bone marrow myeloblasts at diagnosis
- •Disease Immunophenotype:
- •Disease must express a minimum of >/= 10% CD33+ for patients with AML
- •Organ Function:
- •Adequate renal function, adequate liver function, adequate cardiac function, adequate pulmonary function Age: ≤25 years of age Donor: matched family donor, unrelated cord blood donor, unrelated adult donor
排除标准
- •Patients with active central nervous system (CNS) AML/MDS disease at time of conditioning therapy
- •Female patients who are pregnant
- •Karnofsky <50% or Lansky <50% if 10 years or less
- •Age >25 years
- •Has received gemtuzumab in the previous 30 days or has not recovered from prior gemtuzumab therapy.
研究组 & 干预措施
Gemtuzumab Ozogamicin
Conditioning therapy with Gemtuzumab Ozogamicin in combination with busulfan and cyclophosphamide chemotherapy followed by allogeneic stem cell transplantation.
干预措施: Gemtuzumab Ozogamicin (Drug)
结局指标
主要结局
Response rate
时间窗: 1 year
overall response rate (CR + PR) in patients receiving GO, busulfan and cyclophosphamide and AlloSCT in patients with measurable disease (relapse/refractory) with high risk CD33+ AML/MDS
次要结局
未报告次要终点
研究者
Mitchell Cairo
Principal Investigator
New York Medical College