NCT01087398Unknown2 期
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
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 infantile Osteopetrosis who receive allogeneic 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 Malignant infantile osteopetrosis. 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 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Osteopetrosis confirm by bone biopsy and radiographic imaging
- •Age up to 5 year old
排除标准
- •Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant
- •Creatinine clearance ≤ 40ml/min/1.73m^2 or RTA
- •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
Intervention
Experimental
干预措施: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen) (Drug)
Intervention
Experimental
干预措施: Stem Cell Transplantation (Procedure)
Intervention
Experimental
干预措施: Cyclosporin, Methotrexate (GVHD prophylaxis) (Drug)
结局指标
主要结局
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
时间窗: 1 year
次要结局
- One year overall survival after allogeneic HSCT(1 year)
- One year Progressive Free Survival after allogeneic HSCT(1 year)
- Transplantation Related Mortality (TRM) after allogeneic HSCT(1 year)
- Acute and chronic GVHD rate after allogeneic HSCT(1 year)
研究者
研究点 (1)
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