Azacitidine + Venetoclax VS Azacitidineas Maintenance Therapy in AML

NCT07539818 | Not Yet Recruiting Phase 3

• 1 other identifier: IIT2026028
• Study Type: interventional
• Target: 788
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

AML

Outcome Measures

Primary Outcomes (1)

• Disease-Free Survival

  up to 2 years

Secondary Outcomes (8)

• 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

Study Arms (2)

VA

EXPERIMENTAL

Azacitidine 50 mg/m²/day on days 1-5 + Venetoclax 400 mg on days 1-7, every 6 weeks for 12 cycles

Drug: Venetoclax; Drug: Azacitidine

AZA

ACTIVE COMPARATOR

Azacitidine 50 mg/m²/day on days 1-5, every 6 weeks for 12 cycles

Drug: Azacitidine

Interventions

Venetoclax DRUG

400 mg on days 1-7, every 6 weeks for 12 cycles

VA

Azacitidine DRUG

50 mg/m²/day on days 1-5, every 6 weeks for 12 cycles

AZA; VA

Eligibility Criteria

• Age: 14 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

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

MeSH Terms

Interventions

venetoclax; Azacitidine

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Central Study Contacts

Hui Wei, MD

CONTACT

13132507161; weihui@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 13, 2026
• First Posted: April 20, 2026
• Study Start: April 29, 2026
• Primary Completion (Estimated): April 1, 2030
• Study Completion (Estimated): April 1, 2031
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share