Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia: A Multicenter, Prospective Study
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 13
- 主要终点
- 3-years Overall survival(OS) rate
研究概览
简要总结
This clinical trial is an open-label, multicenter, prospective phase 2 clinical trial targeting pediatric leukemia patients of infant age. The goal is to improve survival rates by varying the presence or absence of chemotherapy and hematopoietic stem cell transplantation based on genetic characteristics at the time of diagnosis and minimal residual disease (MRD) values measured by various methods after treatment.
In addition, by clearly defining the patient group that requires hematopoietic stem cell transplantation, it is expected that the role of hematopoietic stem cell transplantation in infantile leukemia, for which there have been various guidelines for hematopoietic stem cell transplantation, can be confirmed. Additionally, due to the characteristics of infants, this study aim to identify long-term sequelae or prognosis related to treatment by prospectively collecting side effect data related to treatment during and after treatment.
详细描述
Infant leukemia patients are classified into low/intermediate/high risk groups and hematopoietic stem cell transplantation is performed after chemotherapy or chemotherapy as shown in the schema below.
- Low risk group : Induction chemotherapy-Low Risk Consolidation chemotherapy 1~4 - Maintenance chemotherapy
- Intermediate risk group : Induction chemotherapy-High Risk Consolidation chemotherapy 1~4 - Maintenance chemotherapy
- High risk group : Induction chemotherapy-High Risk Consolidation chemotherapy 1~4 - hematopoietic stem cell transplantation
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 2 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The age of diagnosis is less than 1 year old
- •The disgnosisi of ALL or ALAL(lymphoid predominant)
- •Informed consent of the parents(guardians) before participation in this study
排除标准
- •Burkitt leukemia/lymphoma or mature B-cell leukemia
- •Down syndrome, Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome or other bone marrow failure syndrome, hematopoietic stem cell transplantation
- •Relapsed infant leukemia
- •Participants with contraindication to medication
- •Administered systemic steroid therapy within 4 weeks prior to this study (However, steroid administration is allowable in oncologic emergencies only after the subject's disease diagnosis and risk group classification are completed.)
- •Participants in other interventional studies other than this protocol
研究组 & 干预措施
Low risk group
KMT2A wild type & minimal residual disease(MRD) (-) after consolidation 1
干预措施: Consolidation #4(without daunorubicin) (Drug)
Intermediate risk group
ntermediate risk (If one of the two cases below applies)
-
KMT2A: MLL mutation (+) & minimal residual disease (MRD) (-) after consolidation 1
-
KMT2A: wild type & minimal residual disease (MRD) (+) after consolidation 1
干预措施: Consolidation #4(with daunorubicin) (Drug)
High risk group
Somatic KMT2A mutation (+) & minimal residual disease (MRD) (+) after consolidation 1
干预措施: Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin) (Drug)
结局指标
主要结局
3-years Overall survival(OS) rate
时间窗: 3-years
The 3-years overall survival rate defined as the percentage of subject in a treatment group who are alive three years after the start of treatm
次要结局
- recurred rate(Up to 5years)
- Overall survival (OS)(Up to 5years)
- Event Free Survial(EFS)(3-years and 5-years)
- Death rate related to infusion(Up to 5years)
- The rate of hematopoietic stem cell transplant patients by risk group(Up to 5years)