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

ConditionsLeukemia, Myeloid, Acute(AML)Hematopoietic Stem Cell Transplantation (HSCT)
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-70
SponsorThe First Affiliated Hospital of Soochow University

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • 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.

  • 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.

  • 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.

  • 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.

  • 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.

  • 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).

Treatment groups

42 Participants
are divided into 1 treatment group
Group A: VABu Conditioning RegimenExperimental treatment 6 interventions

Trial outcomes

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.

Time frame
2 years

Secondary outcomes

1

2-Year Relapse-Free Survival (RFS)

Relapse-free survival is defined as the time from enrollment to the first occurrence of relapse or death from any cause, whichever occurs first. Participants alive and relapse-free at the end of follow-up will be censored at their last known disease-free date. Relapse is defined as disease recurrence confirmed by bone marrow morphology, cytogenetics, or molecular testing per ELN 2022 criteria.

Time frame
2 years
2

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

All-cause mortality is defined as death from any cause within 2 years of enrollment. Transplant-related mortality (TRM) is defined as death from any cause other than disease relapse or progression, with a focus on transplant-related complications (e.g., infection, GVHD, organ toxicity). Non-relapse mortality (NRM) is defined as death from any cause other than disease relapse or progression, and will be reported as a competing risk for relapse outcomes. The cumulative incidences of TRM and NRM will be estimated separately, and all-cause mortality will be reported as the overall proportion of deaths.

Time frame
2 years
3

Engraftment Failure Rate

Engraftment failure is defined as the failure to achieve neutrophil engraftment by day 28 post-transplantation. Neutrophil engraftment is defined as an absolute neutrophil count ≥ 0.5 × 10⁹/L for three consecutive days. The rate of engraftment failure will be reported as the proportion of participants who fail to achieve engraftment by day 28.

Time frame
Day 28 post-transplantation
4

Bloodstream Infection (BSI) Rate

Bloodstream infection is defined as the occurrence of at least one positive blood culture for a bacterial or fungal pathogen during the peri-transplantation period. The BSI rate will be reported as the proportion of participants with at least one episode of bloodstream infection from transplantation to day 28 post-transplantation.

Time frame
Day 28 post-transplantation

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

The First Affiliated Hospital of Soochow University

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: