NCT01238328Unknown2 期
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Mucopolysaccharidosis type 1, 6 and 7 confirm by leukocyte enzyme assay
- •Age up to 8 year old
- •Have suitable donor
排除标准
- •Creatinine clearance ≤ 40ml/min/1.73m^2
- •Bilirubin ≥ 3mg/dL
- •SGPT ≥ 500 U/L
- •Current severe infection
- •Evidence of CNS involvement
- •Morbidity such as blindness or deafness
研究组 & 干预措施
Transplantation
Experimental
干预措施: Conditioning regimen (Drug)
Transplantation
Experimental
干预措施: Graft-versus-host disease (GVHD) prophylaxis (Drug)
Transplantation
Experimental
干预措施: Stem Cell Transplantation (Procedure)
结局指标
主要结局
Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)
时间窗: 1 year
次要结局
- One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT)(1 year)
- One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT)(1 year)
- Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT)(1 year)
- Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT)(1 year)
研究者
研究点 (1)
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