Allogenic Stem Cell Transplantation for Children, Adolescents and Young Adults With Relapsed or Refractory AML; Multi Center Therapy Concept
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 154
- 试验地点
- 24
- 主要终点
- To evaluate whether stem cell transplantation (SCT) from a matched sibling donor (MSD) is equivalent to a matched unrelated donor (MUD) in second complete remission (CR2).
研究概览
简要总结
- To evaluate whether stem cell transplantation from a matched sibling donor is equivalent to a matched unrelated donor in in a second complete remission of acute myeloid leukemia (AML).
- To evaluate whether stem cell transplantation (SCT) after chemotherapy (FLAMSA-schema) increases survival compared to a threshold derived from historical data
- To evaluate whether SCT from haploidentical donors for children having no matched donor will result in better survival with acceptable toxicity.
详细描述
Target variables:
- Treatment response
- Event Free Survival
- Leukemia Free Survival
- Graft Versus Host Disease
- Regimen related toxicity
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
- •Aged between 0-21 years
- •Patients suffering from either refractory de novo AML or relapsed AML or patients with very high risk AML in CR1
- •In sexually active patients two reliable contraception methods are used. This includes every combination of a hormonal contraceptive (such as injection, transdermal patch, implant, cervical ring) or of an intrauterine device(IUD) with a barrier method (e.g. diaphragm, cervical cap, or condom) or with a spermicide.
- •Written informed consent of patient, parents or legal guardians
排除标准
- •Severe renal impairment (GFR < 30% predicted for age)
- •Pregnant or lactating females
- •Current participation in another clinical trial
- •Patients ≥ 12 years old for Group 1 ("BuCyMel") (patients younger that 12 years continue to be included)
研究组 & 干预措施
1
Group I: Relapsed AML-patients with blast cell reduction to <20% before the second course of induction therapy. These patients will receive conventional SCT.
Group II: Patients with non response to frontline treatment of AML, patients with blast cells <20% before the second course of induction therapy who do not achieve a second remission and relapsed AML-patients with blast cells >=20% before the second course of induction therapy. If these patients have a matched donor (MSD/MD) they will receive SCT with "FLAMSA".
Group III: Patients who are eligible for Group II but have no matched donor. These patients will receive SCT from a haploidentical donor.
干预措施: Hematopoietic stem cells from bone marrow or peripheral blood (Biological)
结局指标
主要结局
To evaluate whether stem cell transplantation (SCT) from a matched sibling donor (MSD) is equivalent to a matched unrelated donor (MUD) in second complete remission (CR2).
时间窗: day 100
To evaluate whether "FLAMSA" increases leukemia free survival (LFS) and overall survival (OS) as compared to a historic control group
时间窗: day 100
To evaluate whether SCT from haploidentical donors for children having no matched donor will result in an acceptable toxicity profile and a better LFS as compared to historic controls.
时间窗: day 100
次要结局
- Prospective evaluation of event free survival (EFS), LFS, and OS after SCT from either a MSD or a MUD(day 100)
- Prospective evaluation of late toxicities(day 100 and year 5)
- Decrease of transplantation associated mortality by standardized donor selection criteria(day 100)
- To further evaluate the contribution of immunomediated effects for the treatment of children suffering from very high risk AML(day 100)
- To evaluate whether it is feasible to standardize transplantation procedures in children with AML within the AML-Berlin/Frankfurt/Münster (BFM) study network(day 100)