Venetoclax and Azacitidine Combined With Homoharringtonine, Followed by Allo-HSCT for Intermediate and High-risk AML.

February 28, 2026 updated by: Xianmin Song, MD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Efficacy and Safety of Venetoclax and Azacitidine Combined With Low-Intensity Homoharringtonine Chemotherapy Followed by Allogeneic Hematopoietic Stem Cell Transplantation in Intermediate/High-Risk Newly Diagnosed Acute Myeloid Leukemia

This study is a single-center, single-arm, prospective phase II clinical trial evaluating the efficacy and safety of the VAH (Venetoclax and Azacitidine combined with Homoharringtonine) regimen, followed by allo-HSCT for intermediate and high-risk AML. Eligible patients receive two cycles of the VAH chemotherapy regimen. If minimal residual disease (MRD) is negative after these two cycles, patients proceed to the transplantation process. If MRD remains positive, patients receive an additional two cycles of the VAH regimen. Upon achieving MRD negativity, they then proceed to the transplantation process.

The conditioning regimen includes fludarabine at 30 mg/m²/day from day -7 to day -3 (5 days), cytarabine at 1-1.5 g/m²/day from day -7 to day -3 (5 days), and busulfan at 3.2 mg/kg/day from day -5 to day -3 (3 days). Conditioning begins on day -6, and donor hematopoietic stem cell infusion is performed on day 0.

All patients will undergo bone marrow examination on day 14 and day 28 post-transplant, followed by bone marrow examinations every 30 days within the first year after transplantation, and every 60 days within the second year. If disease relapse is suspected during the follow-up period, bone marrow or extramedullary relapse site examinations will be conducted at any time.

The primary endpoint is the 1-year and 2-year overall survival (OS). Secondary endpoints include the complete response (CR) rate after 1 and 2 cycles of chemotherapy; 1-year and 2-year disease-free survival (DFS) following the achievement of CR through induction therapy; cumulative relapse rate; non-relapse mortality (NRM); incidence of acute graft-versus-host disease (GVHD) within 180 days post-transplant; and the cumulative incidence of chronic GVHD within 1 year and 2 years post-transplant.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

56

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

Study Locations

  • China
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China
        • Recruiting
        • Shanghai General hospital,Shanghai Jiao Tong University School of Medicine
        • 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

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age between 18 and 55 years, inclusive, regardless of gender.
  2. Diagnosed with AML (excluding APL) according to the 2022 International Consensus Classification (ICC) for AML [1].
  3. Evaluated as intermediate/high-risk AML based on the 2022 ELN AML guidelines (see Appendix 1).
  4. No prior treatment for acute leukemia, including hypomethylating agents used for leukemia or myelodysplastic syndromes (MDS), except for hydroxyurea.
  5. Patients must have a suitable hematopoietic stem cell donor.
  6. Hematopoietic cell transplantation comorbidity index (HCT-CI) score ≤ 2.
  7. ECOG (Eastern Cooperative Oncology Group) performance status: 0-2.

  8. Adequate liver, kidney, and cardiopulmonary function, meeting the following requirements:

    1. Serum creatinine ≤ 1.5x ULN (the upper limit of normal);
    2. Cardiac function: Ejection fraction ≥ 50%;
    3. Baseline oxygen saturation > 92%;
    4. Total bilirubin ≤ 1.5 x ULN; ALT and AST ≤ 2.0 x ULN;

    5. Pulmonary function: DLCO (corrected for hemoglobin) ≥ 40% and FEV1 (Forced Expiratory Volume in 1 second) ≥ 50%.

      9) Patients must have the ability to understand and be willing to participate in this study and sign an informed consent form.

Exclusion Criteria:

  1. Acute promyelocytic leukemia.
  2. Presence of extramedullary disease manifestations at diagnosis or during treatment, including central nervous system involvement.
  3. History of malignancies other than myeloid neoplasms within the past 5 years prior to screening, except adequately treated cervical carcinoma in situ, basal cell carcinoma, squamous cell carcinoma of the skin, and localized prostate cancer after radical surgery, and ductal carcinoma in situ after radical surgery.

4) ECOG > 2. 5) HCT-CI score ≥ 3. 6) Any unstable systemic diseases, including but not limited to unstable angina, recent cerebrovascular accidents or transient ischemic attacks within the 3 months prior to screening, myocardial infarction within the 3 months prior to screening, congestive heart failure (New York Heart Association [NYHA] class ≥ III), severe arrhythmias requiring drug treatment after pacemaker implantation, significant liver, kidney, or metabolic diseases, and pulmonary arterial hypertension.

7) Active, uncontrolled infections, including those associated with hemodynamic instability, new or worsening infection symptoms or signs, new infectious lesions on imaging, or persistent unexplained fever without signs or symptoms of infection.

8) Conditions requiring treatment such as grade 2 or higher seizures, paralysis, aphasia, recent severe cerebral infarction, severe traumatic brain injury, dementia, Parkinson's disease, or schizophrenia.

9) HIV-infected individuals. 10) Active hepatitis B (HBV) or active hepatitis C (HCV) requiring antiviral therapy.

Patients at risk of HBV reactivation, are defined as those who are positive for hepatitis B surface antigen or core antibody without receiving antiviral therapy.

11) History of autoimmune diseases. 12) Pregnant or breastfeeding women. 13)Fertile males and females unwilling to use contraception during the treatment period and for 12 months after treatment.

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VAH regimen combined with allo-HSCT
Eligible patients receive two cycles of the VAH chemotherapy regimen. If minimal residual disease (MRD) is negative after these two cycles, patients proceed to the transplantation process. If MRD remains positive, patients receive an additional two cycles of the VAH regimen. Upon achieving MRD negativity, they then proceed to the transplantation process.
Drug: Venetoclax, Azacitidine and Homoharringtonine
Patients will receive the VAH regimen for the treatment of AML
Other Names:
  • VAH

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
1y and 2y-OS
Time Frame: up to 2 years
1-year and 2-year overall survival (OS) after enrollment
up to 2 years

Collaborators and Investigators

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

Sponsor

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Investigators

  • Principal Investigator: Xianmin Song, MD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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 (Actual)

July 2, 2024

Primary Completion (Estimated)

July 1, 2026

Study Completion (Estimated)

July 1, 2028

Study Registration Dates

First Submitted

June 26, 2024

First Submitted That Met QC Criteria

July 1, 2024

First Posted (Actual)

July 3, 2024

Study Record Updates

Last Update Posted (Actual)

March 3, 2026

Last Update Submitted That Met QC Criteria

February 28, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SHSYXY-202403-VAH

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.

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