NCT02034630招募中1 期
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- To evaluate engraftment rate
研究概览
简要总结
To evaluate the outcome of hematopoietic stem cell transplantation using targeted busulfan, fludarabine conditioning regimen in genetic rare disease
详细描述
Busulfan is a highly toxic drug with narrow therapeutic window used for the conditioning of hematopoietic stem cell transplantation. High exposure is associated with systemic toxicity such as veno-occlusive disease (VOD) and underexposure is associated with graft failure or relapse. In this study the investigators plan to improve the outcome of hematopoietic stem cell transplantation by using optimal busulfan dose through pharmacokinetic study.
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 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who are diagnosed as genetic rare disease.
- •Patients who need hematopoietic stem cell transplantation
- •Age: up to 21 years
- •Performance status: ECOG 0-
- •Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.
- •Heart: a shortening fraction > 30% and ejection fraction > 45%.
- •Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.
- •Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m
- •Patients must lack any active viral infections or active fungal infection.
- •Appropriate donor is available: Matched in 6/6 of A, B, DR loci.
- •Patients (or one of parents if patients age < 19) should sign informed consent.
排除标准
- •Pregnant or nursing women.
- •Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
- •Psychiatric disorder that would preclude compliance.
研究组 & 干预措施
Busulfan
Experimental
First dose: busulfan (120 mg/m2 ivs once daily) (if age<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study
干预措施: Busulfan (Drug)
结局指标
主要结局
To evaluate engraftment rate
时间窗: 1 year
To evaluate engraftment rate
次要结局
- To evaluate treatment related mortality(1 year)
研究者
研究点 (1)
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