NCT07425782

Venetoclax Combined With Azacitidine for Consolidation Therapy in AML

A Prospective, Randomized, Open-Label Study of Venetoclax Combined With Azacitidine for Consolidation Therapy in Adult Acute Myeloid Leukemia

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 216 (estimated)
• Sponsor: The First Affiliated Hospital of Soochow University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to compare the efficacy and safety of a venetoclax-based consolidation therapy versus conventional consolidation chemotherapy in newly diagnosed adult patients with high-risk acute myeloid leukemia (AML) who have achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi) after induction therapy with venetoclax and azacitidine and are planned for transplantation. The main questions it aims to answer are: Does consolidation therapy with a venetoclax-containing regimen lead to superior clinical outcomes compared to conventional chemotherapy in this specific patient population? What is the comparative safety profile of the venetoclax-containing consolidation regimen versus conventional chemotherapy in these patients? Participants will be randomly assigned to receive either the venetoclax-based consolidation therapy or the conventional consolidation chemotherapy before undergoing transplantation.

Detailed description

Background and Rationale:Current AML frontline therapy is shifting from intensive chemotherapy toward precision-based approaches. Venetoclax combined with hypomethylating agents (e.g., azacitidine) has become the standard of care for older or chemotherapy-ineligible patients and has shown comparable efficacy and improved safety in younger, fit patients compared with intensive chemotherapy.Pre-transplant consolidation remains a critical phase for reducing relapse risk; however, conventional cytarabine-based regimens are associated with high relapse rates. To date, no prospective studies have investigated venetoclax-based consolidation in AML. This trial aims to address this gap and provide evidence to guide post-remission therapy. Study Design and Interventions:Eligible patients (aged ≥18 years) with newly diagnosed high-risk AML who achieved CR/CRi after 1-2 cycles of venetoclax plus azacitidine induction will be randomized 1:1 to:Experimental arm: Venetoclax-based consolidation regimen (per protocol)；Comparator arm: Conventional intermediate-dose cytarabine consolidation (per protocol).All patients will proceed to allo-HSCT after 1-2 consolidation cycles. Endpoints:Primary: Leukemia-free survival (LFS). Key Secondary: MRD-negative rate before transplantation, overall survival (OS), cumulative incidence of relapse (CIR), non-relapse mortality (NRM), and safety profile. Significance:This study will provide high-level evidence to guide consolidation strategies for high-risk AML in the venetoclax era, with the goal of improving transplant outcomes and long-term survival.

Conditions

• Acute Myeloid Leukemia
• Consolidation Therapy
• Venentoclax
• High Risk
• Hematopoietic Stem Cell Transplant (HSCT)

Interventions

Type	Name	Description
DRUG	Ara-C Group	Ara-C 1-2 g/m², every 12 hours, intravenous infusion, days 1-3; may be combined with anthracyclines/anthraquinones, followed by allo-HSCT after consolidation within 2 cycles.
DRUG	VEN/AZA condsolidation	Venetoclax 400 mg, orally, days 1-28 (the dose of Venetoclax should be adjusted according to the drug dosage instructions/guidelines when combined with CYP3A4 inhibitors); AZA: Azacitidine 75 mg/m² per day, subcutaneous injection, days 1-7. Patients will proceed to allo-HSCT after consolidation within 2 cycles.

Timeline

• Start date: 2026-02-01
• Primary completion: 2028-11-26
• Completion: 2028-11-26
• First posted: 2026-02-23
• Last updated: 2026-02-23