A Study of VABu Conditioning Regimen in Allo-HSCT for AML Patients

NCT07762508 | Not Yet Recruiting

• 1 other identifier: 2026863
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief 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)

Keywords

Acute Myeloid Leukemia; Allogeneic Hematopoietic Stem Cell Transplantation; Conditioning Regimen; Venetoclax; Azacitidine; VABu; Low Performance Status; High Comorbidity Index; Single-Arm Study; Prospective Study; AML; Allo-HSCT

Outcome Measures

Primary Outcomes (1)

• 2-Year Overall Survival (OS)

  Overall survival is defined as the time from enrollment to death from any cause. Participants alive at the end of follow-up will be censored at their last known alive date.

  2 years

Secondary Outcomes (4)

• 2-Year Relapse-Free Survival (RFS)

  2 years

• 2-Year All-Cause Mortality, Transplant-Related Mortality (TRM), and Non-Relapse Mortality (NRM)

  2 years

• Engraftment Failure Rate

  Day 28 post-transplantation

• Bloodstream Infection (BSI) Rate

  Day 28 post-transplantation

Study Arms (1)

VABu Conditioning Regimen

EXPERIMENTAL

Participants receive the VABu conditioning regimen followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The VABu regimen consists of venetoclax 600 mg/m² once daily on days -12 to -7, azacitidine 75 mg/m² once daily on days -11 to -7 (or decitabine 20 mg/m² for 5 days), cytarabine 2.0 g/m² every 12 hours on day -6, and busulfan 0.8 mg/kg every 6 hours on days -5 to -3. Antithymocyte globulin (ATG) is administered at 2.5 mg/kg/day according to donor type: haploidentical or unrelated donors receive ATG on days -5 to -2 (4 doses), while matched sibling donors receive ATG on days -3 to -2 (2 doses). Hematopoietic stem cell infusion occurs on day 0, followed by standard graft-versus-host disease (GVHD) prophylaxis.

Drug: Venetoclax; Drug: Azacitidine (AZA); Drug: Decitabine 20 mg/m²/day for 5 days; Drug: Cytarabine (Ara-C); Drug: Busulfan (BU); Drug: Antithymocyte Globulin (ATG)

Interventions

Venetoclax DRUG

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.

VABu Conditioning Regimen

Azacitidine (AZA) DRUG

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.

VABu Conditioning Regimen

Decitabine 20 mg/m²/day for 5 days DRUG

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.

VABu Conditioning Regimen

Cytarabine (Ara-C) DRUG

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.

VABu Conditioning Regimen

Busulfan (BU) DRUG

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.

VABu Conditioning Regimen

Antithymocyte Globulin (ATG) DRUG

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).

VABu Conditioning Regimen

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 to 70 years.
• Diagnosis of acute myeloid leukemia (AML) other than acute promyelocytic leukemia (APL), with first complete remission (CR1) achieved after induction chemotherapy.
• Classified as non-high-risk per ELN 2022 risk stratification guidelines.
• ECOG performance status ≥ 2 (not due to leukemia) OR Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-CI) ≥ 2.
• Willing to undergo allogeneic hematopoietic stem cell transplantation and has a suitable donor.
• Voluntarily signs informed consent form after full understanding of the study.

You may not qualify if:

• Allergy to any component of the study drugs.
• Psychiatric or psychological disorders that prevent cooperation with treatment.
• Uncontrolled systemic or local severe infection.
• Severe dysfunction of major organs (heart, lung, liver, kidney) that, in the investigator's opinion, makes the patient unsuitable for enrollment.
• Currently participating in or planning to participate in any other clinical study.
• Any other conditions that, in the investigator's opinion, make the patient unsuitable for enrollment.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

venetoclax; Azacitidine; Decitabine; Cytarabine; Busulfan; Antilymphocyte Serum

Condition Hierarchy (Ancestors)

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

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; Butylene Glycols; Glycols; Alcohols; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Central Study Contacts

Depei Wu, MD, PhD

CONTACT

512-67781856; wudepei@suda.edu.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: August 8, 2026
• First Posted: August 13, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: August 19, 2026

Record last verified: 2026-08

Locations

CN (1)