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The Efficacy and Safety of Selinisole Combined With Azacitidine and Venetoclax in the Treatment of Newly Diagnosed High-risk Myeloid Tumors With TP53 Mutations

2026年6月5日 更新者:Bing Han

The Efficacy and Safety of Selinisole Combined With Azacitidine and Venetoclax in the Treatment of Newly Diagnosed High-risk Myeloid Tumors With TP53 Mutations: A Multicenter, Single-arm, Prospective Study

Patients with high-risk myeloid tumors accompanied by TP53 mutations have a poor survival prognosis, and there are still many unmet treatment needs. The current treatment regimens have many limitations. Selinisol, as a novel export protein inhibitor, has good anti-tumor activity. The current preclinical and preliminary clinical research results abroad suggest its effectiveness and safety. This study aims to evaluate the efficacy and safety of AZA combined with selinisole (with or without venetoclax) in the treatment of patients with high-risk myeloid tumors with TP53 mutations through a multicenter, prospective clinical study.

研究概览

地位

尚未招聘

条件

干预/治疗

详细说明

At present, AZA combined with venetocla (" AV regimen ") has become the standard first-line regimen for elderly patients with AML who are intolerant to intensive chemotherapy, but its efficacy in TP53-mutated patients still needs to be improved (ORR approximately 30%-40%). In recent years, several early clinical studies have preliminarily verified the safety and efficacy of AZA combined with selinisole and AZA combined with selinisole + venetoclax regimens. A Phase Ib study presented at the 2023 ASH conference demonstrated that the ORR of patients with relapsed/refractory MDS/AML treated with AZA combined with selinisole reached 42%, among which the ORR of TP53-mutated patients was 38%, and the median OS was 8.5 months, significantly better than historical data. Another study explored the efficacy of the combination of AZA, selinisol and venetoclax in the treatment of R/R AML, involving 12 patients. The preliminary results showed that the ORR of this regimen could reach 91.7%, and the CRR was 42.7%, with good tolerance. The main adverse reactions were controllable gastrointestinal reactions and cytopenia. These clinical data further confirm the application potential of the AZA combined with selinisole (with or without venetoclax) regimen in high-risk myeloid tumors with TP53 mutations, laying a solid foundation for conducting large-scale clinical research.

研究类型

介入性

注册 (估计的)

30

阶段

  • 不适用

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习地点

    • Shuangfuyuan, NO I.
      • Beijing、Shuangfuyuan, NO I.、中国
        • Peking Union Medical College Hospital
        • 接触:

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

Inclusion Criteria:

  1. Age ≥18 years old; Gender is not limited.
  2. The presence of TP53 mutations (According to the 5th Edition of the WHO Classification of hematopoietic and lymphoid tumors and the International Consensus Classification (ICC 2022), the definition of myeloid tumors with TP53 mutations is: pathogenic/potentially pathogenic variations of the TP53 gene are detected through molecular technologies such as next-generation sequencing (NGS), and any of the following conditions are met: The frequency of variant alleles (VAF) is ≥10%, or there are multiple TP53 mutations (≥2 mutations), or both TP53 mutations and 17p deletion (del(17p)) are present.
  3. For the initial treatment of myeloid tumors, the diagnosis was made through peripheral blood and bone marrow examinations and exclusion tests (according to the fifth edition of the WHO and ICC consensus) :

    3.1 MDS (IPSS-R/IPSS-M at high risk or above; or complex karyotype/alone -5/-5q, -7/-7q, i (17q), inv (3)/t(3;3); Or bone marrow blasts ≥10%; 3.2 AML (bone marrow/peripheral blood blasts ≥20%; and high-risk cytogenetic or molecular abnormalities exist); 3.3 MPN (High risk score above threat; or presence of any one of ASXL1, SRSF2, EZH2, IDH1/2, or U2AF1 gene mutations; or bone marrow blasts ≥10%); 3.4 MDS/MPN (IPSS-R high-risk or above; or bone marrow blast granulocytes ≥10%).

  4. Voluntarily join this study, sign the informed consent form with good compliance, and be willing to cooperate with regular follow-ups for efficacy evaluation and side effect monitoring.
  5. Before treatment, the patient's total bilirubin (TBIL) was less than 1.5 times the upper limit of the normal value (ULN), and the alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were less than 3 times the ULN.
  6. ECOG score ≤2 points.

Exclusion Criteria:

  1. Patients who have transplant plans within three months;
  2. All laboratory or clinical records of HIV infection, previous clinical history of hepatitis C, previous hepatitis B infection, or evidence of active hepatitis during screening. Laboratory tests during the screening period suggest hepatitis C infection or hepatitis B infection. (Defined as a positive HBsAg test. Additionally, if the HBsAg test is negative but HBcAb is positive, regardless of the HBsAb status, HBV DNA testing is required. If it is positive, the subject should be excluded.)
  3. Suffering from mental disorders or other conditions and unable to cooperate with the requirements of research, treatment and monitoring;
  4. Pregnant patients or those who cannot take appropriate contraceptive measures during the treatment period;
  5. Those suspected of being allergic to the experimental drug or any of its excipients;
  6. Active heart disease is defined as one or more of the following:

    ① A history of uncontrolled or symptomatic angina pectoris;

    ② Myocardial infarction less than 6 months from the time of enrollment in the study;

    ③ There is a history of arrhythmia that requires drug treatment or has severe clinical symptoms;

    ④ Uncontrolled or symptomatic congestive heart failure (NYHA grade 2)

    ⑤ The ejection fraction is lower than the lower limit of the normal range.

  7. Patients who the researchers consider unsuitable to participate in this trial, such as those whose safety or compliance with the study procedures may be affected by any other medical, social or psychological factors.

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

  • 主要用途:治疗
  • 分配:不适用
  • 介入模型:单组作业
  • 屏蔽:无(打开标签)

武器和干预

参与者组/臂
干预/治疗
实验性的:Azacitidine +Selinexor ± venetoclax
Azacitidine +Selinexor ± venetoclax were administered (Selinexor: 60mg/ week, qw*4 weeks; azacitidine: 100mg/m2*d1-d7; venetoclax, 100mg d1-14). The specific medication duration can be adjusted by the researcher based on the patient's specific condition. A time window of 14 to 28 days is allowed. Each treatment cycle lasts for 30 days until disease progression or the occurrence of intolerable toxicity, whichever comes first.
Azacitidine +Selinexor ± venetoclax were administered (Selinexor: 60mg/ week, qw*4 weeks; azacitidine: 100mg/m2*d1-d7; venetoclax, 100mg d1-14). The specific medication duration can be adjusted by the researcher based on the patient's specific condition. A time window of 14 to 28 days is allowed. Each treatment cycle lasts for 30 days until disease progression or the occurrence of intolerable toxicity, whichever comes first.
其他名称:
  • 阿扎胞苷
  • 维奈托克

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
3 and 6 months overall response rate (ORR)
大体时间:3 and 6 months
The overall response rate (ORR) after the completion of 3 and 6 months. The overall response rate (ORR) is defined as the ratio of patients achieving complete response (CR) plus partial response (PR)
3 and 6 months
3 and 6 months complete response rate (CRR)
大体时间:3 and 6 months
The complete response rate (CRR) after the completion of 3 and 6 months.
3 and 6 months

次要结果测量

结果测量
大体时间
Progression-free survival (PFS);
大体时间:6 months
6 months
Overall survival (OS);
大体时间:6 months
6 months
The incidence of cumulative infection and significant bleeding;
大体时间:6 months
6 months
Incidence of adverse reaction events.
大体时间:6 months
6 months

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年11月1日

初级完成 (估计的)

2027年10月1日

研究完成 (估计的)

2027年10月1日

研究注册日期

首次提交

2025年9月28日

首先提交符合 QC 标准的

2026年6月5日

首次发布 (实际的)

2026年6月8日

研究记录更新

最后更新发布 (实际的)

2026年6月8日

上次提交的符合 QC 标准的更新

2026年6月5日

最后验证

2026年6月1日

更多信息

与本研究相关的术语

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

是的

IPD 共享时间框架

10 years

IPD 共享访问标准

email request

IPD 共享支持信息类型

  • 研究方案
  • 树液
  • 国际碳纤维联合会
  • 企业社会责任

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.

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