Azacitidine + Venetoclax VS Azacitidineas Maintenance Therapy in AML

A Study of Azacitidine With or Without Venetoclax as Maintenance Therapy in Acute Myeloid Leukemia

This is a prospective, multicenter, randomized, open-label, phase III trial evaluating the efficacy and safety of azacitidine plus venetoclax versus azacitidine alone as maintenance therapy in patients with acute myeloid leukemia (AML) who have achieved first complete remission (CR) or CR with incomplete count recovery (CRi) after induction and consolidation. Eligible patients aged 14 to 74 years are randomized 1:1 to receive either azacitidine 50 mg/m²/day on days 1-5 every 6 weeks for up to 12 cycles, or the same azacitidine regimen combined with venetoclax 400 mg on days 1-7 per cycle. The primary endpoint is disease-free survival (DFS). Secondary endpoints include overall survival (OS), relapse-free survival (RFS), cumulative incidence of relapse (CIR), minimal residual disease (MRD) conversion rate, and safety. A total of 788 patients are planned with stratification by prior venetoclax exposure and MRD status.

Study Overview

• Status: Not yet recruiting
• Conditions: AML
• Intervention / Treatment: Drug: Venetoclax; Drug: Azacitidine

• Study Type: Interventional
• Enrollment (Estimated): 788
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Hui Wei, MD; Phone Number: 13132507161; Email: weihui@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Diagnosis of AML per WHO 2022 or ICC criteria, or MDS/AML (10-20% blasts) per ICC
• Age ≥14 and <75 years
• ECOG performance status 0-2
• First complete remission (CR) or CR with incomplete count recovery (CRi) after induction and consolidation
• Received at least 2 cycles of intermediate- or high-dose cytarabine (cumulative dose ≥6 g/m² per cycle)
• Time from first CR/CRi to enrollment ≤10 months, and time from last treatment to enrollment ≤3 months
• MRD-negative or low-level MRD detectable; MRD-positive patients are excluded
• Signed informed consent

Exclusion Criteria:

• Acute promyelocytic leukemia with PML-RARA
• AML with BCR-ABL
• Active central nervous system involvement
• Prior allogeneic hematopoietic stem cell transplantation
• Planned allogeneic HSCT within 6 months
• Deemed unsuitable by investigator

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: VA; Azacitidine 50 mg/m²/day on days 1-5 + Venetoclax 400 mg on days 1-7, every 6 weeks for 12 cycles	Drug: Venetoclax; 400 mg on days 1-7, every 6 weeks for 12 cycles; Drug: Azacitidine; 50 mg/m²/day on days 1-5, every 6 weeks for 12 cycles
Active Comparator: AZA; Azacitidine 50 mg/m²/day on days 1-5, every 6 weeks for 12 cycles	Drug: Azacitidine; 50 mg/m²/day on days 1-5, every 6 weeks for 12 cycles

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Disease-Free Survival	up to 2 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Cumulative Incidence of Relapse (CIR)	Up to 5 years
Overall Survival (OS)	up to 2 years
Relapse-Free Survival (RFS)	up to 2 years
Complete Remission Duration (CRd)	up to 1 year
DFS-MRD	Up to 2 years
RFS-MRD	Up to 2 years
CIR-MRD	up to 5 years
MRD Negativity Conversion Rate	up to 5 years

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Estimated): April 29, 2026
• Primary Completion (Estimated): April 1, 2030
• Study Completion (Estimated): April 1, 2031

Study Registration Dates

• First Submitted: April 13, 2026
• First Submitted That Met QC Criteria: April 13, 2026
• First Posted (Actual): April 20, 2026

Study Record Updates

• Last Update Posted (Actual): April 20, 2026
• Last Update Submitted That Met QC Criteria: April 13, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleic Acids, Nucleotides, and Nucleosides; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Aza Compounds; Nucleosides; Ribonucleosides; Azacitidine; venetoclax
• Other Study ID Numbers: IIT2026028

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