VEN+IDAC vs IDAC in AML

Official title

Venetoclax Combined With Intermediate-dose Cytarabine Versus Intermediate-dose Cytarabine for Consolidation Therapy of Acute Myeloid Leukemia (AML): a Multicenter, Prospective, Randomized Controlled, Phase 2 Study

Overview

This study intends to use a randomized controlled design to compare the MRD-negativw rate and related efficacy and safety of venetoclax combined with intermediate-dose cytarabine (IDAC) versus IDAC in the consolidation treatment of patients with AML after remission, providing high-quality evidence-based medical evidence for optimizing post-remission consolidation therapy for AML and improving patients' prognosis.

Detailed description

Studies have shown that venetoclax can significantly enhance the efficacy of intensive chemotherapy (IC) in the induction and salvage treatment of AML. However, there is still a lack of direct comparison of the efficacy and safety of venetoclax combined with intermediate-dose cytarabine (IDAC) with IDAC for consolidation in AML. In order to evaluate the benefit of venetoclax combined with IDAC, we plan to conduct a multicenter, prospective randomized controlled, phase 2 study to compare the efficacy and safety of venetoclax (d1-7, 400mg QD) combined with cytarabine (d1-3, 1.5g/㎡, q12h) versus cytarabine only for consolidation therapy in AML, aiming to fill the current evidence gap and provide scientific basis for improving deeper remission of AML and finally improve patients' long-term survival.

Interventions

• Drug Ventoclax
  Venetoclax 400mg per day from day 1 to day 7
• Drug Cytarabine
  Cytarabine 1.5g/m\^2 q12h from day 1 to day 3.

Primary outcome measures

• Rate of Measurable residual disease (MRD) negative [Time frame: At the end of cycle 2 (28 days for a cycle)]

Secondary outcome measures (5)

• Cumulative Incidence of Recurrence (CIR) [Time frame: 2 years]
• Overall survival (OS) [Time frame: 2 years]
• Disease Free Survival (DFS) [Time frame: 2 years]
• Aderse events [Time frame: At the end of cycle 2 (28 days for a cycle)]
• The rate of being bridged to allogeneic hematopoietic stem cell transplantation [Time frame: 2 years]

Eligibility criteria

Inclusion criteria

• Age between 18 and 65 years, any gender;
• Meet the WHO 2022 diagnostic criteria for acute myeloid leukemia (AML), and achieve complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) after induction therapy. CR is defined as bone marrow blasts ≤5%, absolute neutrophil count ≥1.0×10⁹/L, and platelets ≥100×10⁹/L; CRi is defined as bone marrow blasts ≤5%, absolute neutrophil count ≥0.5×10⁹/L, and platelets ≥50×10⁹/L;
• Baseline MRD testing (using multiparameter flow cytometry, MFC) must be completed at enrollment.
• Eastern Cooperative Oncology Group (ECOG) performance status score ≤2;
• Major organ function is basically normal and meets the following requirements: ① Liver function: total bilirubin ≤1.5×ULN (except patients with liver metastasis or Gilbert's syndrome), ALT and AST ≤2.5×ULN; ② Kidney function: serum creatinine ≤1.5×ULN or creatinine clearance ≥60 ml/min; ③ Cardiac function: left ventricular ejection fraction (LVEF) ≥50%;
• The patient or their legal representative signs a written informed consent form, agrees to comply with the study protocol, and completes the specified follow-up procedures.

Exclusion criteria

• Presence of other active malignant tumors (excluding indolent tumors that have been cured, such as basal cell carcinoma of the skin and carcinoma in situ of the cervix);
• Central nervous system leukemia;
• Existence of uncontrolled severe infection (such as sepsis, fungal pneumonia, active tuberculosis, etc.);
• History of severe heart disease, including but not limited to: severe arrhythmias (such as ventricular tachycardia, atrial fibrillation with rapid ventricular rate, etc.), history of myocardial infarction (within the past 6 months), severe heart failure (NYHA functional class ≥3), etc.;
• Women who are pregnant or breastfeeding; women planning pregnancy or men of reproductive potential who have not used effective contraception during the study and within 6 months after treatment;
• Known allergy to venetoclax, cytarabine, or other components of the combination regimen in the study;
• Having mental illness (such as schizophrenia, major depressive disorder, etc.) or cognitive dysfunction that prevents cooperation with study follow-up and treatment;
• Active hepatitis B virus infection (HBV DNA positive), active hepatitis C virus infection (HCV RNA positive), or human immunodeficiency virus (HIV) infection;
• Undergoing major surgery, radiation therapy, other chemotherapy drugs, or investigational drug treatments within 3 weeks prior to study drug treatment.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

China · 1 center

• Department of Hematology, Guangdong Second Provincial General Hospital — Guangzhou

Identifiers

NCT: NCT07537257 · GD2H-2026-481

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗