Prospective Non-randomized Multicenter Trial: the Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- overall survival (OS)
研究概览
简要总结
This prospective non-randomized multicenter trial created based on protocol ML DS 2006 and aimed at standardization of current therapy approaches and creating a national network for diagnostic, treatment and monitoring of children (0-18 years) with AML and Down syndrome in Russia. Based on the results the investigators expect to increase long-term overall and event-free survival in children with AML and DS and reduce the immediate and remote toxicity of chemotherapy by reducing the dose load of chemotherapeutic drugs.
The study protocol therapy for all patients includes four chemotherapy blocks:
Course 1 AIE (cytarabine/idarubicin/etoposide) Course 2 AI (cytarabine/idarubicin) Course 3 HAD (high -dose cytarabine (1g)/daunorubicin) Course 4 HA (high-dose cytarabine) Safety to be monitored based on CTCAE v5.0
详细描述
This prospective non-randomized multicenter trial created based on protocol ML DS 2006 and aimed at standardization of current therapy approaches and creating a national network for diagnostic, treatment and monitoring of children (0-18 years) with AML and Down syndrome in Russia. Based on the results the investigators expect to increase long-term overall and event-free survival in children with AML and DS and reduce the immediate and remote toxicity of chemotherapy by reducing the dose load of chemotherapeutic drugs.
Patients to be included: Age 0-18 years
- Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)
- Signed informed consent
Patients not be included:
- Children with Down syndrome and acute lymphoblastic leukemia (ALL)
- Severe comorbidities with contraindications to the treatment according to the protocol
- Pre-treatment >14 days with intensive induction therapy
- Refusal of all therapy or important elements of therapy
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 Day 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 0-18 years
- •Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)
- •Signed informed consent
排除标准
- •Children with Down syndrome and acute lymphoblastic leukemia (ALL)
- •Severe comorbidities with contraindications to the treatment according to the protocol
- •Pre-treatment >14 days with intensive induction therapy
- •Refusal of all therapy or important elements of therapy
结局指标
主要结局
overall survival (OS)
时间窗: up to 2 years
event-free survival
时间窗: up to 2 years
Response rate; event-free survival (EFS)
relapse-free survival (RFS)
时间窗: up to 2 years
treatment-related mortality
时间窗: up to 2 years
次要结局
- CTCAE(up to 2 years)