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NCT Number: NCT07078591

Venetoclax-containing Therapy Combined With Microtransplant for Newly Diagnosed AML

The purpose of this study is to evaluate whether HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cell (GPBMC) infusion with venetoclax-containing regimens (microtransplant, MST) could improve survival in adult patients with newly diagnosed acute myeloid leukemia (AML).

Recruiting

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age >=18 years, male or female, non-limited by race or ethnicity.
  • No prior anti-acute leukemia treatment (including hypomethylators for leukemia or MDS) with the exception that prior hydroxyurea and/or leukapheresis are permitted.
  • Confirmed acute myeloid leukemia in accordance with WHO criteria with a WBC count < 25 × 109/L.
  • Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )<= 3 × upper limit of normal(ULN), and total bilirubin <= 1.5 × ULN.
  • Adequate renal function including serum creatinine <= 2 × ULN or CrCl>= 40mL/min.
  • LVEF measured by echocardiogram is within the normal range (LVEF > 50%).
  • The subject must have one donor who is >= 18 years old and HLA matched at 0-7/10 loci (i.e., at least 3 HLA loci must be mismatched). In addition, the donor voluntarily donates hematopoietic stem cells and signs the consent form.
  • Each subject (or his/her legal representatives) must sign the Informed Consent Form (ICF), indicating that he/she understands the purpose and procedures of research, and is willing to participate in research.
  • Donor inclusion criteria: The donor meets the institution's criteria for related peripheral blood hematopoietic stem cell donors. The donor must be able to tolerate the cell separation and collection process, and sign the Informed Consent Form.

Exclusion criteria

  • Acute promyelocytic leukemia, myeloid sarcoma, chronic myeloid leukemia accelerated phase and blast crisis.
  • Uncontrolled infection or hemorrhage.
  • Cardiovascular disease with clinical significance, such as uncontrolled or highly symptomatic cardiac arrhythmias, congestive heart failure, or myocardial infarction within 6 months prior to screening, or New York Heart Association (NYHA) function class 3 (moderate) or class 4 (severe) heart disease.
  • Uncontrolled autoimmune disease or requiring immunosuppression treatment.
  • History of severe blood infusion reaction.
  • Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception.
  • Psychiatric disorder or cognitive impairment that in the researcher's judgment would make the subject not likely to adhere to the protocol requirements.
  • Any major surgery within 4 weeks before enrollment.
  • Life-threatening illness other than AML or uncontrolled intercurrent illness.

Treatment and study plan

Venetoclax

Drug

Given PO

Azacitidine (AZA)

Drug

Given SC

Daunorubicin

Drug

Given IV

Idarubicin(IDA)

Drug

Given IV

Cytarabine (ARA-C)

Drug

Given IV

GPBMC infusion

Biological

HLA-mismatched donor GPBMC infusion

Primary outcomes

  1. Overall survival (OS)

    Time frame: Measured up to 4 years after the last participant is enrolled

    OS is defined as the number of days from the date of initiation of treatment to the date of death.

  2. Event-free Survival (EFS)

    Time frame: Measured up to 4 years after the last participant is enrolled

    EFS is defined as the number of days from initiation of treatment to the date of progressive disease, relapse from CR or CRi, treatment failure or death from any cause.

Secondary outcomes

  1. Complete remission (CR) and complete remission with incomplete marrow recovery (CRi)

    Time frame: Measured up to 2 years after the last participant is enrolled

    CR is defined as absolute neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, red cell transfusion independence, and bone marrow with < 5% blasts. CRi is defined as bone marrow with less than 5% blasts, and absolute neutrophil count <= 1 × 109/L or platelet count <= 100 × 109/L.

  2. Partial remission (PR)

    Time frame: Measured up to 2 years after the last participant is enrolled

    PR is defined as decrease of at least 50% in the percentage of blasts to 5-25% in the bone marrow, peripheral blood neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, red cell transfusion independence.

  3. Non-relapse Mortality (NRM)

    Time frame: Measured up to 4 years after the last participant is enrolled

    NRM is defined as the death without relapse.

Study contacts

Contact information is provided by the study sponsor or research team.

Bo Cai, MD

CONTACT

[email protected]

+861066947168

Yangyang Lei, MD

CONTACT

[email protected]

+861066947180

Sponsors and collaborators

Lead sponsor

Beijing 302 Hospital

Other

Registry information

Official study title

Phase 2 Study of Venetoclax-containing Therapy in Combination With HLA-mismatched Mobilized Peripheral Blood Mononuclear Cell Infusion for Newly Diagnosed Acute Myeloid Leukemia

Important dates

Study start
2025
Primary completion
2030
Study completion
2031
First posted
Jul 22, 2025
Registry last updated
Jul 28, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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