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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 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

30

Fase

  • Ikke anvendelig

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Andre navne:
  • Azacitidin
  • venetoklax

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
3 and 6 months overall response rate (ORR)
Tidsramme: 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)
Tidsramme: 3 and 6 months
The complete response rate (CRR) after the completion of 3 and 6 months.
3 and 6 months

Sekundære resultatmål

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

Samarbejdspartnere og efterforskere

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Sponsor

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. november 2026

Primær færdiggørelse (Anslået)

1. oktober 2027

Studieafslutning (Anslået)

1. oktober 2027

Datoer for studieregistrering

Først indsendt

28. september 2025

Først indsendt, der opfyldte QC-kriterier

5. juni 2026

Først opslået (Faktiske)

8. juni 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

8. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

5. juni 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-delingstidsramme

10 years

IPD-delingsadgangskriterier

email request

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

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Kliniske forsøg med Myeloide tumorer

Kliniske forsøg med Selinexor

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