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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.

調査の概要

状態

まだ募集していません

介入・治療

詳細な説明

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.

研究の種類

介入

入学 (推定)

30

段階

  • 適用できない

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

    • Shuangfuyuan, NO I.
      • Beijing、Shuangfuyuan, NO I.、中国
        • Peking Union Medical College Hospital
        • コンタクト:

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

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.

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:処理
  • 割り当て:なし
  • 介入モデル:単一グループの割り当て
  • マスキング:なし(オープンラベル)

武器と介入

参加者グループ / アーム
介入・治療
実験的: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.
他の名前:
  • アザシチジン
  • ベネトクラクス

この研究は何を測定していますか?

主要な結果の測定

結果測定
メジャーの説明
時間枠
3 and 6 months overall response rate (ORR)
時間枠: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)
時間枠:3 and 6 months
The complete response rate (CRR) after the completion of 3 and 6 months.
3 and 6 months

二次結果の測定

結果測定
時間枠
Progression-free survival (PFS);
時間枠:6 months
6 months
Overall survival (OS);
時間枠:6 months
6 months
The incidence of cumulative infection and significant bleeding;
時間枠:6 months
6 months
Incidence of adverse reaction events.
時間枠:6 months
6 months

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研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年11月1日

一次修了 (推定)

2027年10月1日

研究の完了 (推定)

2027年10月1日

試験登録日

最初に提出

2025年9月28日

QC基準を満たした最初の提出物

2026年6月5日

最初の投稿 (実際)

2026年6月8日

学習記録の更新

投稿された最後の更新 (実際)

2026年6月8日

QC基準を満たした最後の更新が送信されました

2026年6月5日

最終確認日

2026年6月1日

詳しくは

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個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

はい

IPD 共有時間枠

10 years

IPD 共有アクセス基準

email request

IPD 共有サポート情報タイプ

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

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