Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML

NCT07490288 | Not Yet Recruiting

• 1 other identifier: IIT2026021
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a Phase I/II, single-arm, open-label clinical trial evaluating the safety and preliminary efficacy of a novel induction regimen combining Venetoclax, Azacitidine, and Liposomal Mitoxantrone (VAM) in adult patients with newly diagnosed Acute Myeloid Leukemia (AML) who are eligible for intensive chemotherapy. The study plans to enroll 30 participants. Patients will receive VAM induction therapy, followed by three cycles of intermediate-dose cytarabine consolidation. Allogeneic hematopoietic stem cell transplantation is recommended for high-risk or MRD-positive patients in remission.

Conditions

AML

Outcome Measures

Primary Outcomes (1)

• Composite complete remission rate after induction

  up to 42 days

Secondary Outcomes (6)

• Event-free survival

  Up to 2 years

• Overall survival

  Up to 5 years

• Relapse-free survival

  Up to 5 years

• MRD negativity rate by flow cytometry and molecular methods (PCR/NGS) after 1 and 2 induction cycles

  At the end of the first and second induction cycles

• 30-day mortality

  up to 30 days

Study Arms (1)

VAM Induction Regimen

EXPERIMENTAL

Single-arm, phase I/II study evaluating venetoclax, azacitidine, and liposomal mitoxantrone (VAM) as induction in newly diagnosed AML patients fit for intensive chemotherapy. Patients receive 1-2 cycles of VAM induction, followed by 3 cycles of intermediate-dose cytarabine consolidation. Induction is dose-exploratory: first 10 patients receive venetoclax days 3-9; next 10 receive venetoclax days 3-14; subsequent 20 receive the selected regimen. High-risk or MRD+ patients may proceed to allogeneic HSCT.

Drug: Venetoclax; Drug: Azacitidine; Drug: Liposomal Mitoxantrone; Drug: Cytarabine; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Interventions

Venetoclax DRUG

BCL-2 inhibitor. Oral. Induction: 100 mg day 1, 200 mg day 2, then 400 mg days 3-9 or 3-14.

VAM Induction Regimen

Azacitidine DRUG

Hypomethylating agent. 75 mg/m²/day IV/SC on days 1-7 of induction.

VAM Induction Regimen

Liposomal Mitoxantrone DRUG

Liposomal topoisomerase II inhibitor. 24 mg/m² IV on day 1 of each induction cycle.

VAM Induction Regimen

Cytarabine DRUG

Antimetabolite. Consolidation: 2 g/m² (age \<60) or 1 g/m² (age ≥60) q12h IV on days 1-3 for 3 cycles.

VAM Induction Regimen

Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) PROCEDURE

Recommended for high-risk or MRD+ patients after response.

VAM Induction Regimen

Eligibility Criteria

• Age: 14 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients diagnosed with AML according to the WHO (2022) or ICC criteria, or with MDS/AML as defined by ICC (with 10%-20% blasts in the bone marrow)
• Age ≥ 14 years, male or female.
• Eastern Cooperative Oncology Group Performance Status (ECOG-PS) score of 0-2.
• Meet the following laboratory requirements (tests must be performed within 7 days prior to treatment):
• i. Total bilirubin ≤ 1.5 times the upper limit of normal (ULN) for the corresponding age group.
• ii. AST and ALT ≤ 2.5 times ULN for the corresponding age group. iii. Serum creatinine \< 1.5 times ULN for the corresponding age group. iv. Cardiac enzymes \< 2 times ULN for the corresponding age group. v. Left ventricular ejection fraction (LVEF) within the normal range as measured by echocardiography (ECHO).

You may not qualify if:

• Acute promyelocytic leukemia with PML::RARA fusion gene.
• Acute myeloid leukemia with RUNX1::RUNX1T1 fusion gene.
• Acute myeloid leukemia with BCR::ABL1 fusion gene.
• Previously treated patients (defined as having received prior induction chemotherapy for AML/MDS; prior use of cytoreductive agents like hydroxyurea is allowed).
• Concurrent active malignancy of other organs (requiring treatment).
• Active cardiac disease, defined as one or more of the following:
• i. History of uncontrolled or symptomatic angina. ii. Myocardial infarction within 6 months prior to study enrollment. iii. History of clinically significant arrhythmia requiring medication or causing severe symptoms.
• iv. Uncontrolled or symptomatic congestive heart failure (\> New York Heart Association \[NYHA\] Class 2).
• Active, uncontrolled infectious diseases (e.g., untreated tuberculosis, pulmonary aspergillosis).
• Any other condition that, in the opinion of the investigator, makes the patient unsuitable for study participation.

Sponsors & Collaborators

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

MeSH Terms

Interventions

venetoclax; Azacitidine; Cytarabine

Intervention Hierarchy (Ancestors)

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

Central Study Contacts

Hui Wei, MD

CONTACT

13132507161; weihui@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2026
• First Posted: March 24, 2026
• Study Start: April 30, 2026
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): March 1, 2029
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share