Evaluation of Venetoclax-Based Combination Therapies in Childhood Acute Myeloid Leukemia: A Phase II Randomized Controlled Trial
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 74
- 试验地点
- 1
研究概览
简要总结
Acute Myeloid Leukemia (AML) is a serious type of blood cancer that affects children. Treating this disease requires strong chemotherapy, which can lead to side effects and even death during treatment, especially in the early phase. We need better treatments that are both effective and safer for children. This study will test whether adding a new medicine called venetoclax, in combination with other chemotherapy drugs, can be safely used in the early part of treatment. Venetoclax has shown promise in adult patients and in smaller studies of children, but it has not yet been widely tested in large groups of children with newly diagnosed AML. In this study, we will give venetoclax along with two chemotherapy drugs (either cytarabine or azacitidine) for the first 1–2 weeks of treatment. The main goal is to see if this new approach helps children go into remission while keeping side effects manageable. We will also closely monitor for any early complications like infections or low blood counts. Apart from treatment, we will also study the biology of the disease in Indian children using advanced laboratory techniques such as BH3 profiling, to understand how leukemia cells respond to venetoclax. These tests will help us understand which children are most likely to benefit from venetoclax and improve future treatment plans. If the results of this study are promising, we plan to take this research further into larger trials that could eventually change the way we treat childhood AML in India and similar settings. We hope this study will lead to better outcomes with fewer side effects for children with this serious illness.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- None
入排标准
- 年龄范围
- 1.00 Year(s) 至 15.00 Year(s)（—）
- 性别
- All
入选标准
- •1.Subject must have histological confirmation of AML by WHO criteria, with greater than 5 percentage bone marrow blasts.
- •2.Subject must have adequate renal function as demonstrated by a creatinine clearance greater than and equal to 30 mL/min, calculated by the Cockcroft Gault formula or measured by 24-hours urine collection.
- •3.Subject must have adequate liver function as demonstrated by: aspartate aminotransferase (AST) less than and equal to 5.0 × ULN and alanine aminotransferase (ALT) less than and equal to 5.0 × ULN and bilirubin less than and equal to 3 × ULN (Unless considered to be due to Leukemic organ involvement).
排除标准
- •1.Age less than 1 year or greater than 15 years.
- •2.Previous chemotherapy for AML, except for cytoreductive therapy (hydroxyurea or low dose cytarabine for up to 96 hours).
- •3.Relapsed or refractory AML 4.Secondary AML 5.Subject has known CNS involvement with AML 6.Diagnosis of myelodysplastic neoplasm 7.Diagnosis of acute promyelocytic leukemia (APL, AML-M3) 8.Core-binding factor AML 9.Acute megakaryocytic leukemia 10.Children with Down syndrome 11.Subject exhibits evidence of other clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial or fungal).
- •12.Subject has a white blood cell count greater than 25 × 10 to the power of 9 /L.
- •(Note: Hydroxyurea or low-dose cytarabine for up to 96 hours administration or leukapheresis is permitted to meet this criterion).
- •13.Documented hypersensitivity to any component of the chemotherapy regimen.
研究者
Dr Shyam Srinivasan
Tata Memorial Hospital