Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome
试验速览
- 阶段
- 2 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Event-free survival
研究概览
简要总结
The aim of the current study is to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in HSCT in Nijmegen breakage syndrome
详细描述
Nijmegen breakage syndrome (NBS) is a DNA repair disorder. The only curative option for combine immunodeficiency in NBS is allogeneic hematopoietic stem cell transplantation (HSCT). Standard myeloablative conditioning regimens in DNA repair disorders lead to increased morbidity and mortality after HSCT. Low doses of alkylators are used to reduce toxicity rates, which, however, increase the risks of mixed chimerism and graft failure. The data of treosulfan usage in NBS are sparse. To evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in NBS, treosulfan 21g/m2 in combination with fludarabine 150mg/mg, cyclophosphamide 40mg/kg, thymoglobulin (Genzyme) 5mg/kg and rituximab 100mg/m2 will be used from day -6 to -1 day, followed by stem cell infusion. The primary endpoint is event-free survival, where graft failure, death, and malignancies are considered as events.
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
- 3 Months 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged ≥ 3 months and < 21 years
- •Patients diagnosed with NBS eligible for an allogeneic HSCT
- •Signed written informed consent signed by a parent or legal guardian
排除标准
- 未提供
研究组 & 干预措施
intervention/treatment
Fludarabine 150mg/m2 (days -6, -5, -4, -3, -2) Treosulfan 21g/m2 (days -6, -5, -4) Cyclophosphamide 40mg/kg (days -3, -2) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Stem cell infusion - day 0
干预措施: Treosulfan (Drug)
结局指标
主要结局
Event-free survival
时间窗: 3 years after HSCT
Events: graft failure, death, malignancies
次要结局
- Cumulative incidence of chronic graft versus host disease(3 years)
- Incidence of early organ toxicity(100 days)
- Cumulative incidence of transplant related mortality(3 years)
- Cumulative incidence of acute graft versus host disease(1 year)
- Cumulative incidence of engraftment(100 days)
- Incidence of long-term toxicity(3 years)
- Overall survival(3 years after HSCT)
- Cumulative incidence of graft failure(3 years)
- Cumulative incidence of viral infections(1 year)