Prospective randomized study on the feasibility of allogeneic stem cell transplantation in higher-risk-myelodysplastic syndromes, performed upfront or preceded by azacitidine or conventional chemotherapy, according to the BM-blast proportion (ACROBAT trial). MDS0519
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 274
- 试验地点
- 46
- 主要终点
- Feasibility of HSCT- Non-inferiority design. The feasibility of HSCT will be estimated in terms of proportion of patients who receive HSCT of the total number of randomized patients. For the primary endpoint of feasibility of HSCT, all patients who perform HSCT will be considered as "successes", and all others, as "failures". For the primary efficacy endpoint, sensitivity analyses will be performed adjusting the treatment comparison by factors which appeared to be of prognotic importance
研究概览
简要总结
The primary objective of this study has been split in two categories: the feasibility of HSCT (ITT) in patients with HR-MDS with a proportion of bone marrow blasts below 10% and in patients with a proportion of BM blasts equal or greater than 10%.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high
- •Age 18-70 years
- •Previously untreated for HR-MDS
- •HSCT – eligible
- •Life expectancy greater than or equal to 3 months
- •Signed written informed consent according to ICH/EU/GCP and national local laws
- •Eastern Cooperative Oncology Group Performance Status Grade of 0-2
排除标准
- •Acute myeloid leukaemia with >20% blasts in BM or peripheral blood (PB)
- •concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma)
- •severe renal, cardiac, liver or lung impairment
- •pregnant or lactating or potentially fertile (both males and females), who have not agreed to avoid pregnancy during the trial period; Women of childbearing potential and men must agree to use effective contraception during and up to 3 months after treatment with azacitidine.
- •HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis
- •clinically relevant neurological or psychiatric diseases
- •hypersensitivity (known or suspected) to AZA
- •prior Treatments: a) prior investigational drugs (within 30 days); b) radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months; c) growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days; d) androgenic hormones during the previous 14 days; e) prior transplantation or cytotoxic therapy, including azacitidine, AZA or chemotherapy, administered to treat MDS.
结局指标
主要结局
Feasibility of HSCT- Non-inferiority design. The feasibility of HSCT will be estimated in terms of proportion of patients who receive HSCT of the total number of randomized patients. For the primary endpoint of feasibility of HSCT, all patients who perform HSCT will be considered as "successes", and all others, as "failures". For the primary efficacy endpoint, sensitivity analyses will be performed adjusting the treatment comparison by factors which appeared to be of prognotic importance
Feasibility of HSCT- Non-inferiority design. The feasibility of HSCT will be estimated in terms of proportion of patients who receive HSCT of the total number of randomized patients. For the primary endpoint of feasibility of HSCT, all patients who perform HSCT will be considered as "successes", and all others, as "failures". For the primary efficacy endpoint, sensitivity analyses will be performed adjusting the treatment comparison by factors which appeared to be of prognotic importance
次要结局
- Overall survival ITT
- Event-free survival ITT (including relapse, progression, or death from any cause)
- Safety in terms of AE/SAEs
- Pattern of relapse/progression after HSCT
- Translational studies with mutational, and cytofluorimetric analysis (patient BMsampling at enrollment, before HSCT and at 6 months after HSCT)
- Quality of life assessment at enrollment, before HSCT and at 6 months after HSCT
- Pharmacoeconomic evaluation (i.e. duration of hospitalization, RBC transfusions, etc)
研究者
Data center
Scientific
Fondazione Gimema Franco Mandelli Onlus