Azacytidine-venetoclax Combination as Leukemia Debulking Treatment Followed by Reduced Toxicity Conditioning Regimen (Melphalan Busulfan and Fludarabine, MBF) as Salvage Therapy for Refractory Acute Myeloid Leukemia (AML).
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 23
- 试验地点
- 3
- 主要终点
- relapse-free survival
研究概览
简要总结
In this phase clinical trail, we evaluate the efficacy and feasibility of azacytidine and venetoclax as leukemia debulking treatment followed by reduced intensity conditioning regimen consisting of Fludarabine + Busulfan + Melphalan as salvage treatment in patients with refractory AML .
详细描述
In refractory AML, allogeneic hematopoietic stem cell transplantation (allo-HCST) is considered as the only curative regimen. In this phase II clinical trial, we plan to evaluate the efficacy and feasibility of new sequential transplantation protocol. All patients receive azacytidine and venetoclax as leukemia debulking treatment which is followed by reduced intensity conditioning regimen consisting of Fludarabine (150mg/m2) + Busulfan (6.4mg/kg) + Melphalan (70mg/m2). The graft-versus host disease (GVHD) prophylaxis regimen is based on reduced dose of post-transplantation cyclophosphamide (PT-CY) 40mg/kg day+3~+4, tacrolimus and low-dose anti-thymoglobulin (ATG, 2.5mg/kg) in case of HLA-matched unrelated donor or halo-donor transplantation.
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
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •refractory AML (no remission after 2 induction therapy, relapsed AML within 6 months of first complete remission (CR), relapse AML no CR after reinduction therapy), multiple relapse and refractory relapse AML
- •patients with HLA matched related or unrelated donor (9~10/10) or haplo-identical related donor
排除标准
- •Patients with poor liver function (enzyme >2N or bilirubin >2N)
- •poor renal function (Scr >1.5N)
- •poor cardiac function （EF<45%）
- •inform consent not provided
研究组 & 干预措施
aza-ven +MBF
treatment with aza-ven debulking therapy followed by allo-HSCT with MBF conditioning
干预措施: Aza-Ven-allo-HSCT (Drug)
结局指标
主要结局
relapse-free survival
时间窗: 12 months
event defined as relapse or death of any causes
次要结局
- overall survival(12 months)
- non-relapse mortality(12 months)
- relapse(12 months)
- GVHD and relapse free survival (GRFS)(12 months)
研究者
Jiong HU
Head BMT program, Rui Jin Hospital
Shanghai Jiao Tong University School of Medicine