Venetoclax Combined With Azacitidine for Consolidation Therapy in AML

February 14, 2026 updated by: CHEN Jia, The First Affiliated Hospital of Soochow University

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

216

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of AML confirmed by bone marrow morphology, flow cytometry, and molecular genetics, meeting WHO 2022 classification criteria;
  • Age ≥ 18 years;
  • Classified as high-risk according to the European LeukemiaNet (ELN) prognostic risk stratification for AML, including AML with myelodysplasia-related changes (AML-MRC) and therapy-related acute myeloid leukemia (t-AML);
  • Achieved CR or CRi after ≤ 2 cycles of VA induction chemotherapy;
  • Availability of a suitable donor, with plans to undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT);
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2;
  • Creatinine clearance ≥ 50 mL/min (calculated using the Cockcroft-Gault formula); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 times the upper limit of normal (ULN), total bilirubin ≤ 2 times ULN; left ventricular ejection fraction (LVEF) ≥ 50% as shown by echocardiography (ECHO); expected survival > 8 weeks;
  • Voluntarily signed the informed consent form and can understand and comply with study requirements.

Exclusion Criteria:

  • Presence of clinically active cardiovascular disease, such as uncontrolled ventricular arrhythmia, uncontrolled hypertension, congestive heart failure, cardiac disease classified as Class 3 or 4 according to the New York Heart Association (NYHA) Functional Classification, or a history of myocardial infarction within 3 months prior to screening;
  • Active central nervous system leukemia (CNSL) or extramedullary infiltration of leukemia;
  • Other serious diseases that may limit the patient's participation in this trial (e.g., severe infection, renal failure);
  • Known human immunodeficiency virus (HIV) infection or uncontrolled severe viral hepatitis;
  • Pregnant or breastfeeding women;
  • Inability to understand, comply with the study protocol, or sign the informed consent form;
  • Any other conditions deemed by the investigator as unsuitable for participation in this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Ara-C Consolidation Arm
AraC (Cytarabine) 1-2 g/m², administered intravenously every 12 hours on days 1-3, may be combined with anthracycline/anthraquinone agents. After completing 1-2 cycles of consolidation therapy, the patient proceeds to allogeneic hematopoietic stem cell transplantation (allo-HSCT).
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.
Experimental: VA Consolidation Chemotherapy Arm
VEN: 400 mg, orally, days 1-28 (Venetoclax dosage must be adjusted per prescribing information/guidelines based on concomitant use of CYP3A4 inhibitors); AZA: 75 mg/m²/day, subcutaneously, days 1-7; Proceed to allogeneic hematopoietic stem cell transplantation (allo-HSCT) after completing 1-2 cycles of consolidation therapy.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Leukemia free survival
Time Frame: From date of complete remission until the date of first documented relapse or date of death from any cause, whichever came first, assessed up to 2 years.
Time from date of achieving complete remission until date of first documented hematologic relapse, extramedullary relapse, or death from any cause, whichever occurs first.
From date of complete remission until the date of first documented relapse or date of death from any cause, whichever came first, assessed up to 2 years.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pretransplantation MRD negative rate
Time Frame: From the end of the last consolidation therapy to the initiation of conditioning regimen for allogeneic hematopoietic stem cell transplantation, within approximately 1 month.
Proportion of patients who achieve minimal residual disease (MRD) negativity prior to hematopoietic stem cell transplantation, as assessed by multicolor flow cytometry, measured from bone marrow samples.
From the end of the last consolidation therapy to the initiation of conditioning regimen for allogeneic hematopoietic stem cell transplantation, within approximately 1 month.
Overall survival
Time Frame: From the first day of randomization to the date of death, assesed up to 2 years.
From the first day of randomization to the date of death from any cause
From the first day of randomization to the date of death, assesed up to 2 years.
Cumulative relapse rate
Time Frame: From date of achieving remission to the date of death from any cause, assessed up to 2 years.
From the date of achieving remission to the date of hematologic relapse.
From date of achieving remission to the date of death from any cause, assessed up to 2 years.
Non-relapse mortality
Time Frame: From date of randomization until date of death without prior relapse or disease progression, assessed up to 2 years.
The proportion of patients who died from causes unrelated to disease relapse/progression after achieving remission or receiving an intervention
From date of randomization until date of death without prior relapse or disease progression, assessed up to 2 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The First Affiliated Hospital of Soochow University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 1, 2026

Primary Completion (Estimated)

November 26, 2028

Study Completion (Estimated)

November 26, 2028

Study Registration Dates

First Submitted

February 3, 2026

First Submitted That Met QC Criteria

February 14, 2026

First Posted (Actual)

February 23, 2026

Study Record Updates

Last Update Posted (Actual)

February 23, 2026

Last Update Submitted That Met QC Criteria

February 14, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SCH-AML-VenAza-C

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Acute Myeloid Leukemia

Clinical Trials on Ara-C Group

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Poland

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe