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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

5. Juni 2026 aktualisiert von: 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.

Studienübersicht

Status

Noch keine Rekrutierung

Bedingungen

Intervention / Behandlung

Detaillierte Beschreibung

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.

Studientyp

Interventionell

Einschreibung (Geschätzt)

30

Phase

  • Unzutreffend

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

    • Shuangfuyuan, NO I.
      • Beijing, Shuangfuyuan, NO I., China
        • Peking Union Medical College Hospital
        • Kontakt:

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Beschreibung

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.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: N / A
  • Interventionsmodell: Einzelgruppenzuweisung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: 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.
Andere Namen:
  • Azacitidin
  • Venetoclax

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
3 and 6 months overall response rate (ORR)
Zeitfenster: 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)
Zeitfenster: 3 and 6 months
The complete response rate (CRR) after the completion of 3 and 6 months.
3 and 6 months

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Zeitfenster
Progression-free survival (PFS);
Zeitfenster: 6 months
6 months
Overall survival (OS);
Zeitfenster: 6 months
6 months
The incidence of cumulative infection and significant bleeding;
Zeitfenster: 6 months
6 months
Incidence of adverse reaction events.
Zeitfenster: 6 months
6 months

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Geschätzt)

1. November 2026

Primärer Abschluss (Geschätzt)

1. Oktober 2027

Studienabschluss (Geschätzt)

1. Oktober 2027

Studienanmeldedaten

Zuerst eingereicht

28. September 2025

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

5. Juni 2026

Zuerst gepostet (Tatsächlich)

8. Juni 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

8. Juni 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

5. Juni 2026

Zuletzt verifiziert

1. Juni 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Plan für individuelle Teilnehmerdaten (IPD)

Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?

JA

IPD-Sharing-Zeitrahmen

10 years

IPD-Sharing-Zugriffskriterien

email request

Art der unterstützenden IPD-Freigabeinformationen

  • STUDIENPROTOKOLL
  • SAFT
  • ICF
  • CSR

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .

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