This clinical trial aims to compare the efficacy and safety of venetoclax-based consolidation therapy versus conventional consolidation chemotherapy (intermediate/high-dose cytarabine) in newly diagnosed adult patients with intermediate-risk acute myeloid leukemia (AML). Participants must have achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi) after induction therapy with venetoclax and azacitidine and are planned to undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT).
The goal of this prospective, randomized, open-label, multi-center study is to demonstrate that consolidation therapy with venetoclax + azacitidine (VA) is non-inferior to conventional intensive chemotherapy (cytarabine-based regimens) in this specific patient population, while offering a more favorable safety profile.Eligible patients will be randomized 1:1 to receive either 1-2 cycles of VA (Venetoclax 400mg d1-28 + Azacitidine 75mg/m² d1-7) or 1-2 cycles of intermediate/high-dose cytarabine (AraC) ± anthracycline. The primary endpoint is 2-year Leukemia-Free Survival (LFS). Secondary endpoints include pre-transplant MRD-negative rate, Overall Survival (OS), Cumulative Incidence of Relapse (CIR), Non-Relapse Mortality (NRM), and safety profile (CTCAE v5.0). This study will provide high-level evidence for consolidation therapy in intermediate-risk AML in the venetoclax era.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
226
Venetoclax: 400 mg, orally, once daily on Days 1-28. (Dose adjustment required per prescribing information/guidelines when combined with CYP3A4 inhibitors). Azacitidine: 75 mg/m²/day, subcutaneously or intravenously, on Days 1-7. Patients will receive 1-2 cycles of this regimen before proceeding to allo-HSCT.
Cytarabine (AraC): ≥6g/m² per cycle (e.g., 1-2g/m², every 12 hours on days 1-3), administered intravenously. May be combined with anthracycline/anthraquinone agents per standard practice. Patients will receive 1-2 cycles of this regimen before proceeding to allo-HSCT.
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
RECRUITINGLeukemia-Free Survival (LFS)
Time from randomization to the first occurrence of relapse or death, whichever comes first.
Time frame: From the date of randomization to the date of first documented hematologic relapse, extramedullary relapse, or death from any cause, assessed up to 2 years.
Pretransplantation MRD-Negative Rate
Proportion of patients who achieve minimal residual disease (MRD) negativity prior to hematopoietic stem cell transplantation, as assessed by multi-parameter flow cytometry.
Time frame: From the end of the last consolidation therapy to the initiation of conditioning regimen for allo-HSCT, approximately within 1 month.
Overall Survival (OS)
Time frame: From the first day of randomization to the date of death from any cause, assessed up to 2 years.
Cumulative Incidence of Relapse (CIR)
Time frame: From the date of randomization to the date of hematologic relapse, assessed up to 2 years.
Non-Relapse Mortality (NRM)
Time frame: From the date of randomization until the date of death without prior relapse or disease progression, assessed up to 2 years.
Incidence of Adverse Events (Safety Profile)
Graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
Time frame: Throughout the consolidation treatment period and up to 30 days post-treatment.
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