Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 6
- 主要终点
- Conditioning-related toxicity
研究概览
简要总结
RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's bone marrow. The donated bone marrow stem cells may replace the patient's immune system and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before or after transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects and best dose of cyclophosphamide in treating patients who are undergoing a donor bone marrow transplant for Fanconi's anemia.
详细描述
OBJECTIVES:
- Decrease the conditioning-related toxicity of cyclophosphamide without decreasing the engraftment rate to < 90% in patients undergoing allogeneic bone marrow transplantation for Fanconi's anemia.
OUTLINE: This is a multicenter, dose-finding study of cyclophosphamide.
- Nonmyeloablative conditioning regimen: Patients receive cyclophosphamide IV on days -5 to -2.
Cohorts of 5-10 patients receive decreasing doses of cyclophosphamide until the optimal dose (OD) is determined. The OD is defined as the dose at which ≥ 4 of 5 patients achieve engraftment and < 1 of 10 patients experiences dose-limiting toxicity.
研究设计
- 研究类型
- Interventional
- 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
- •DISEASE CHARACTERISTICS:
- •Diagnosis of Fanconi's anemia by chromosome fragility with a diepoxybutane (DEB) or mitomycin C test
- •Hemoglobin ≤ 8.0 g/dL, absolute granulocyte count ≤ 1,000/mm^3, or platelet count ≤ 50,000/mm^3
- •No refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute leukemia
- •HLA-identical related donor available
- •PATIENT CHARACTERISTICS:
- •Glomerular filtration rate ≥ 30% predicted for age
- •No liver disease (e.g., active hepatitis or moderate to severe portal fibrosis/cirrhosis by biopsy)
- •No symptomatic cardiac insufficiency or symptomatic arrhythmia
- •No other diseases that would severely limit the probability of survival
- •No HIV seropositivity
- •Not pregnant or nursing
- •Fertile patients must use effective contraception
- •PRIOR CONCURRENT THERAPY:
- •Not specified
排除标准
- 未提供
结局指标
主要结局
Conditioning-related toxicity
时间窗: 100 days post-transplant
Graft rejection
时间窗: 100 days post-transplant
次要结局
未报告次要终点