VABu Conditioning in Elderly AML HSCT

NCT07583888 | Recruiting Phase 2

• 1 other identifier: ZSQ003
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Allogeneic Hematopoietic Stem Cell Transplantation Recipient; Acute Myeloid Leukemia (AML)

Keywords

Acute Myeloid Leukemia; Elderly; Hematopoietic Stem Cell Transplantation; Venetoclax; Busulfan; Preconditioning

Outcome Measures

Primary Outcomes (1)

• Progression-Free Survival (PFS)

  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.

  1 year and 2 years post-transplantation

Secondary Outcomes (10)

• Overall Survival (OS)

  1 year and 2 years post-transplantation

• Cumulative Incidence of Relapse (CIR)

  1 year, 2 years

• Non-relapse Mortality (NRM)

  Day 100, 1 year, 2 years

• GVHD-free Relapse-free Survival (GRFS)

  1 year, 2 years

• Incidence of Acute GVHD

  Day 100, Day 180

Study Arms (1)

VaBU

EXPERIMENTAL

Drug: VaBU

Interventions

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.

VaBU

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead
• Suzhou Hongci Hematology Hospital, Suzhou, China: collaborator
• Shenzhen People's Hospital: collaborator
• Henan Cancer Hospital: collaborator
• Huai'an Second People's Hospital and the Affiliated Huai'an Hospital of Xuzhou Medical University: collaborator

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Huiying Qiu, M.D.

CONTACT

+8618151578096; mumukongxin@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician；Professor

Model Details: All enrolled participants receive the same VABu conditioning regimen (Venetoclax, Azacitidine, Semustine, Cytarabine and Busulfan) prior to allogeneic hematopoietic stem cell transplantation. No comparator or control group is included.

Study Record Dates

• First Submitted: May 7, 2026
• First Posted: May 13, 2026
• Study Start: May 7, 2026
• Primary Completion (Estimated): August 15, 2027
• Study Completion (Estimated): August 15, 2027
• Last Updated: May 14, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)