A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 18
- 主要终点
- Primary Objective: Event-free Survival at 1 Year.
研究概览
简要总结
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.
This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.
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
- 1 Year 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1-16.00 years old
- •Have transfusion dependent thalassemia major
- •Shall not have an HLA-matched family donor
- •Must have a suitably matched unrelated marrow donor or UCB product
- •Lansky score >/= 70
- •Adequate pulmonary, renal, liver, and other organ function as defined in protocol
- •Negative pregnancy test
- •Adequate total nucleated cell or CD34+ dose of product as defined in protocol
- •Iron chelation must be discontinued >/= 48 hours prior to conditioning regimen
排除标准
- •Pregnant or breastfeeding
- •HIV positive
- •Prior allogeneic marrow or stem cell transplantation
研究组 & 干预措施
Conditioning regimen
Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0
干预措施: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan (Drug)
结局指标
主要结局
Primary Objective: Event-free Survival at 1 Year.
时间窗: 1 year
次要结局
- Median Time to ANC Engraftment(100 days)
- Median Time to Platelet Engraftment(100 days)
- Development of Graft Versus Host Disease (GVHD)(2 years)
- Incidence of Disease Recurrence(2 years)