VEN+IDAC vs IDAC in AML

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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: AML (Acute Myelogenous Leukemia)
• Intervention / Treatment: Drug: Ventoclax; Drug: Cytarabine

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.

• Study Type: Interventional
• Enrollment (Estimated): 232
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age between 18 and 65 years, any gender;
2. 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;
3. Baseline MRD testing (using multiparameter flow cytometry, MFC) must be completed at enrollment.
4. Eastern Cooperative Oncology Group (ECOG) performance status score ≤2;
5. 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%;
6. 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:

1. 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);
2. Central nervous system leukemia;
3. Existence of uncontrolled severe infection (such as sepsis, fungal pneumonia, active tuberculosis, etc.);
4. 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.;
5. 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;
6. Known allergy to venetoclax, cytarabine, or other components of the combination regimen in the study;
7. Having mental illness (such as schizophrenia, major depressive disorder, etc.) or cognitive dysfunction that prevents cooperation with study follow-up and treatment;
8. Active hepatitis B virus infection (HBV DNA positive), active hepatitis C virus infection (HCV RNA positive), or human immunodeficiency virus (HIV) infection;
9. Undergoing major surgery, radiation therapy, other chemotherapy drugs, or investigational drug treatments within 3 weeks prior to study drug treatment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VEN+IDAC; Venetoclax 400mg per day from day 1 to day 7; cytarabine 1.5g/m^2 q12h from day 1 to day 3.	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.
Active Comparator: IDAC; Cytarabine 1.5g/m^2 q12h from day 1 to day 3	Drug: Cytarabine; Cytarabine 1.5g/m^2 q12h from day 1 to day 3.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Measurable residual disease (MRD) negative	MRD is monitored using flow cytometric analysis with a positive MRD threshold of 0.1%.	At the end of cycle 2 (28 days for a cycle)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative Incidence of Recurrence (CIR)	Assess the risk of leukemia recurrence after the consolidation	2 years
Overall survival (OS)	OS is calculated from enrollment to death or the last follow-up.	2 years
Disease Free Survival (DFS)	It refers to the time from the start of the consolidation to disease recurrence or death from any cause or the last follow-up.	2 years
Aderse events	Side effects of the consolidations including haematological and non-haematological toxicity.	At the end of cycle 2 (28 days for a cycle)
The rate of being bridged to allogeneic hematopoietic stem cell transplantation	To assess how many patients are needed to accept allogeneic hematopoietic stem cell transplantation after the consolidation.	2 years

Collaborators and Investigators

• Sponsor: Guangdong Second Provincial General Hospital
• Collaborators: Guangzhou First People's Hospital; Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University; Shenzhen Second People's Hospital; Dongguan People's Hospital; Zhongshan People's Hospital, Guangdong, China; First Affiliated Hospital of Guangxi Medical University; Nanfang Hospital, Southern Medical University; Shunde Hospital, Sothen Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): April 1, 2029

Study Registration Dates

• First Submitted: April 3, 2026
• First Submitted That Met QC Criteria: April 12, 2026
• First Posted (Actual): April 17, 2026

Study Record Updates

• Last Update Posted (Actual): April 17, 2026
• Last Update Submitted That Met QC Criteria: April 12, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Cytarabine; Venetoclax; Acute myeloid leukemia; Consolidation; Measurable residual disease
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Neoplastic Processes; Leukemia, Myeloid; Leukemia; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Neoplasm, Residual; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleic Acids, Nucleotides, and Nucleosides; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Nucleosides; Arabinonucleosides; Cytarabine
• Other Study ID Numbers: GD2H-2026-481

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No