Avapritinib in Relapsed Refractory or MRD-positive CBF-AML With KIT Mutations
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Composite complete remission (CRc)
研究概览
简要总结
AML with t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as CBF-AML. KIT mutations are common in CBF-AML, which have a worse prognosis.This study is aimed to evaluate the efficacy of Avapritinib, an highly specific inhibitor of the KIT gene, in CBF-AML with KIT mutations.
详细描述
Acute Myeloid Leukemia (AML) with the chromosomal abnormality of t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as the Core Binding Factor AML (CBF-AML). KIT mutation is a common mutation in CBF-AML, which is more likely to relapse and have a worse prognosis.
Avapritinib is an oral tyrosine kinase inhibitor (TKI) with selective inhibitory activity against KIT and PDGFRA. Avapritinib has been approved by FDA for the treatment of gastrointestinal stromal tumors(GIST) with PDGFRA mutations and Advanced systemic mastocytosis (AdvSM). However, the efficacy of avapritinib in AML with KIT mutations is uncertain.
This prospective, multicenter clinical study of the efficacy and safety of avapritinib in relapsed refractory or molecular minimal residual disease (MRD)-positive AML with KIT mutations.
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
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with acute myeloid leukemia accompanied by t(8; 21)/RUNX1-RUNX1T1, or inv(16)/t(16; 16)/CBFβ-MYH11;
- •Accompanied by KIT mutation
- •Disease recurrence after the first remission, or the mol-MRD remains positive after the morphologic remission of AML.
- •No active infection.
- •Liver function: TBIL≤ 2×ULN,ALT/AST≤ 3×ULN, CCr ≥ 50ml/min，NYHA grading ≤2; SaO2 >92%.
- •(11) Predicted survival > 12 weeks.
排除标准
- •Accept other AML targeted therapies, such as dasatinib, sorafenib, gilteritinib, etc. simultaneously;
- •The presence of uncontrolled and active infections (including bacterial, fungal or viral infections).
- •Underlying diseases such as myocardial infarction, chronic heart failure, decompensated liver dysfunction, renal failure, etc.
- •Pregnant or lactating women;
- •Accompanied by other malignant tumors requiring treatment;
- •Other interventional clinical studies have been enrolled.
研究组 & 干预措施
Treatment Group
The treatment group will receive avapritinib orally. The dosage is 100mg to 300mg qd, allowed to combine with other chemotheray drugs.
干预措施: Avapritinib (Drug)
结局指标
主要结局
Composite complete remission (CRc)
时间窗: Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.
The proportion of participants who achieve Composite complete remission (CRc)，which includes complete remission (CR)、CR with partial hematologic recovery (CRh)、CR with incomplete blood count recovery (CRi) and morphology leukemia free (MLFS) based on response criteria for AML.
次要结局
- Progression-free survival (PFS)(From the first day of treatment until any failure (resistant disease, relapse, or death), assessed up to 1 to 3 years.)
- Overall survival (OS)(From the first day of treatment to time of death from any cause, assessed up 1 to 3 years.)
- Incidence of adverse events (AEs)(Up to approximately 1 to 3 years.)
- MRD-negative rate(Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.)
研究者
Chen Suning
Principal Investigator
The First Affiliated Hospital of Soochow University