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NCT Number: NCT07583888

VABu Conditioning in Elderly AML HSCT

This is an open-label, multi-center, single-arm clinical study evaluating the efficacy and safety of the VABu conditioning regimen in elderly patients (≥60 years) with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (HSCT).

The VABu regimen consists of Venetoclax, Azacitidine, Semustine, Cytarabine, and Busulfan. All enrolled participants will receive the VABu regimen as conditioning therapy prior to HSCT.

The study aims to enroll 20 participants from multiple centers in China. The primary objectives are to evaluate the overall response rate, cumulative relapse rate, overall survival, graft-versus-host disease (GVHD)-free relapse-free survival (GRFS), non-relapse mortality (NRM), incidence of acute and chronic GVHD, and reactivation rates of cytomegalovirus (CMV) and Epstein-Barr virus (EBV). Safety outcomes include treatment-related toxicities, such as bone marrow suppression, infection, and organ dysfunction.

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Key information

Conditions

Allogeneic Hematopoietic Stem Cell Transplantation Recipient Acute Myeloid Leukemia (AML) Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type

Age range

60 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

Location status: Recruiting

Location contact

Xin Kong

CONTACT

[email protected]

67781856

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 60 years.
  • Confirmed diagnosis of acute myeloid leukemia (AML) according to WHO classification, with intermediate or high-risk prognosis.
  • Previous response to Venetoclax-based therapy.
  • Planned to undergo allogeneic hematopoietic stem cell transplantation (HSCT).
  • Donor availability: Related donor matched at least 5/10 at HLA-A, -B, -C, -DQB1, and -DRB1; OR unrelated donor matched at least 8/10 at the same loci.
  • Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-CI) score ≤ 4.
  • ECOG performance status 0-2.
  • Adequate organ function as defined by: Creatinine ≤ 1.5 × ULN or creatinine clearance ≥ 45 mL/min (Cockcroft-Gault formula or 24-hour urine collection)； AST ≤ 3.0 × ULN and ALT ≤ 3.0 × ULN； Total bilirubin ≤ 1.5 × ULN； Left ventricular ejection fraction (LVEF) > 50%； Baseline oxygen saturation > 92%； DLCO ≥ 40% and FEV1 ≥ 50%；
  • Ability to understand and provide written informed consent.

Exclusion criteria

  • Age < 60 years.
  • Poor response to prior Venetoclax-based therapy.
  • Unstable systemic disease (unstable angina, myocardial infarction, cerebrovascular accident within 3 months; NYHA Class III-IV heart failure; severe arrhythmia; pulmonary hypertension).
  • Active uncontrolled infection or active bleeding in vital organs.
  • CNS symptoms grade ≥ 2 requiring treatment.
  • Major organ surgery within 6 weeks.
  • History of malignant disease other than AML within 5 years.
  • History of thrombosis, embolism, or cerebral hemorrhage within 1 year.
  • ECOG performance status > 2.
  • HCT-CI score > 4.
  • Organ failure meeting specified criteria.
  • Known HIV, active HBV, or active HCV infection.
  • History of autoimmune disease requiring systemic immunosuppressive therapy.
  • Pregnancy, breastfeeding, or unwillingness to use effective contraception in patients of childbearing potential.
  • Drug abuse or chronic alcoholism.
  • Psychiatric disorder or other condition compromising informed consent or compliance.
  • Any other condition making the patient unsuitable for study participation in the investigator's judgment.

Treatment and study plan

VaBU

Drug

The VABu conditioning regimen is administered prior to allogeneic hematopoietic stem cell transplantation (HSCT). The regimen consists of the following drugs administered sequentially:

Venetoclax: 600 mg/m² orally once daily on days -10 to -5. Azacitidine: 75 mg/m² subcutaneously once daily on days -10 to -6. Semustine: 250 mg/m² orally once on day -10. Cytarabine: 2 g/m² intravenously once daily on day -1. Busulfan: 0.8 mg/kg intravenously every 6 hours on days -4 to -2 (total of 12 doses).

The conditioning regimen is completed before HSCT. Dose adjustments may be made based on toxicity, concomitant medications (especially CYP3A4 inhibitors), and individual patient tolerance.

Primary outcomes

  1. Progression-Free Survival (PFS)

    Time frame: 1 year and 2 years post-transplantation

    Progression-free survival is defined as the time from the date of transplantation to the date of disease progression, relapse, or death from any cause, whichever occurs first. Participants who are alive without progression or relapse at the last follow-up are censored.

Secondary outcomes

  1. Overall Survival (OS)

    Time frame: 1 year and 2 years post-transplantation

    Overall survival is defined as the time from the date of transplantation to the date of death from any cause. Participants who are alive at the last follow-up are censored.

  2. Cumulative Incidence of Relapse (CIR)

    Time frame: 1 year, 2 years

  3. Non-relapse Mortality (NRM)

    Time frame: Day 100, 1 year, 2 years

  4. GVHD-free Relapse-free Survival (GRFS)

    Time frame: 1 year, 2 years

  5. Incidence of Acute GVHD

    Time frame: Day 100, Day 180

  6. Incidence of Chronic GVHD

    Time frame: 1 year, 2 years

  7. CMV Reactivation Rate

    Time frame: 1 year

  8. EBV Reactivation Rate

    Time frame: 1 year

    include EBV-related post-transplant lymphoproliferative disorder (PTLD)

  9. Engraftment Rate

    Time frame: Day 30

  10. Safety and Tolerability (TEAEs)

    Time frame: Treatment period to 30 days post-treatment

Study contacts

Contact information is provided by the study sponsor or research team.

Huiying Qiu, M.D.

CONTACT

[email protected]

+8618151578096 ext. +8613912792913

Sponsors and collaborators

Lead sponsor

The First Affiliated Hospital of Soochow University

Other

Collaborators

  • Henan Cancer Hospital
  • Huai'an Second People's Hospital and the Affiliated Huai'an Hospital of Xuzhou Medical University
  • Shenzhen People's Hospital
  • Suzhou Hongci Hematology Hospital, Suzhou, China

Registry information

Official study title

Efficacy and Safety of the VABu Conditioning Regimen in Elderly Patients With Acute Myeloid Leukemia Undergoing Hematopoietic Stem Cell Transplantation: An Open-Label, Multicenter, Single-Arm Clinical Study

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
May 13, 2026
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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