A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- To measure the incidence and quality of engraftment and hematopoietic reconstitution.
研究概览
简要总结
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
详细描述
We are currently recruiting patients.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia
- •Karnofsky or Lansy performance scale > or = to 70%.
- •Must have adequate cardiac, hepatic, renal and pulmonary function.
- •Must have 7/8 or 8/8 available unrelated donor.
排除标准
- •Pregnant or breastfeeding.
- •Active CNS leukemic involvement
- •Active uncontrolled viral, bacterial or fungal infection
- •Positive for HIV.
研究组 & 干预措施
Bone marrow processing
Bone Marrow processing using the CliniMACs device
干预措施: CliniMACs device (Device)
Bone marrow processing
Bone Marrow processing using the CliniMACs device
干预措施: Busulfan (Drug)
Bone marrow processing
Bone Marrow processing using the CliniMACs device
干预措施: Fludarabine (Drug)
Bone marrow processing
Bone Marrow processing using the CliniMACs device
干预措施: Cyclophosphamide (Drug)
Bone marrow processing
Bone Marrow processing using the CliniMACs device
干预措施: ATG (Drug)
结局指标
主要结局
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
时间窗: 1, 3, 6 and 12 months post transplant date
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
次要结局
- The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD(weekly for the first 30 days and then 3, 6, and 12 months post transplant date)
研究者
David A. Margolis
Professor of Pediatrics and BMT Program Director
Medical College of Wisconsin