A Prospective Single-Arm Study of VABu Conditioning Regimen in Allo-HSCT for Low-Performance or High-Comorbidity AML Patients in CR1

Summary

This study evaluates the effectiveness and safety of a new conditioning regimen called VABu before stem cell transplantation in patients with acute myeloid leukemia (AML) who are in their first complete remission (CR1), classified as non-high-risk per ELN 2022 guidelines, but have poor physical condition (ECOG performance status ≥ 2) or high comorbidity burden (HCT-CI ≥ 2). VABu combines venetoclax, azacitidine (or decitabine as an alternative), and busulfan. This is a single-arm, prospective study with 42 participants. All participants will receive the VABu regimen followed by stem cell transplantation. The primary outcome measure is 2-year overall survival (OS). Secondary outcomes include 2-year relapse-free survival (RFS), transplant-related mortality (TRM), engraftment failure rate, bloodstream infection (BSI), and 2-year all-cause mortality and non-relapse mortality (NRM). The study is conducted at the First Affiliated Hospital of Soochow University.

Conditions

• Leukemia, Myeloid, Acute(AML)
• Hematopoietic Stem Cell Transplantation (HSCT)

Interventions

DRUG

Venetoclax

Venetoclax 600 mg/m² administered orally once daily on days -12 to -7 as part of the VABu conditioning regimen for allogeneic hematopoietic stem cell transplantation. Venetoclax is a selective BCL-2 inhibitor used to enhance anti-leukemic activity.

DRUG

Azacitidine (AZA)

Azacitidine 75 mg/m² administered subcutaneously once daily on days -11 to -7 as part of the VABu conditioning regimen. This is one of the two optional hypomethylating agents; the alternative is decitabine 20 mg/m² for 5 days. Azacitidine has synergistic anti-leukemic effects when combined with venetoclax.

DRUG

Decitabine 20 mg/m²/day for 5 days

Decitabine 20 mg/m² administered intravenously once daily for 5 days on days -11 to -7 as an alternative to azacitidine in the VABu conditioning regimen. This is one of the two optional hypomethylating agents; the alternative is azacitidine 75 mg/m² once daily. Decitabine has synergistic anti-leukemic effects when combined with venetoclax.

DRUG

Cytarabine (Ara-C)

Cytarabine 2.0 g/m² administered intravenously every 12 hours on day -6 as part of the VABu conditioning regimen. Intermediate-dose cytarabine is used to further eliminate minimal residual disease prior to hematopoietic stem cell infusion.

DRUG

Busulfan (BU)

Busulfan 0.8 mg/kg administered intravenously every 6 hours on days -5 to -3 as part of the VABu conditioning regimen. Busulfan is an alkylating agent used at reduced intensity to achieve sufficient anti-leukemic effect while minimizing organ toxicity.

DRUG

Antithymocyte Globulin (ATG)

Antithymocyte globulin (ATG) 2.5 mg/kg/day administered intravenously once daily for graft-versus-host disease (GVHD) prophylaxis. Haploidentical or unrelated donors: ATG given on days -5 to -2 (4 doses). Matched sibling donors: ATG given on days -3 to -2 (2 doses).

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University

  lead OTHER

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2026-08-31

Primary Completion

2029-07-31

Completion

2029-07-31

Countries

• China

Study Locations