VA Induction and Bridging Transplantation for Hypoplastic/Secondary AML

NCT07635485 | Not Yet Recruiting DMC

• 2 other identifiers: 202653109021420250003
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to explore the efficacy and safety of venetoclax combined with azacitidine（VA) induction followed by bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of hypoplastic and secondary acute myeloid leukemia (AML).

Conditions

Secondary AML (Adult); Hypoplastic Acute Myeloid Leukemia

Keywords

Venetoclax; Azacitidine; Allo-HSCT

Outcome Measures

Primary Outcomes (1)

• Overall Survival (OS)

  Time from the date of diagnosis to death due to any cause.

  2 year

Secondary Outcomes (6)

• Complete Remission Rate（CRR）

  Day 28 of induction therapy and 56 days post-transplantation

• Composite Complete Remission Rate (CCRR)

  Day 28 of induction therapy and 56 days post-transplantation

• Disease-free Survival (DFS)

  2 year

• GVHD-free and Relapse-free Survival (GRFS)

  2 year

• Cumulative Incidence of Relapse (CIR)

  2 year

Study Arms (1)

A

EXPERIMENTAL

VA induction+Allo-HSCT consolidation

Drug: Venetoclax (VEN); Drug: Azacitidine (AZA); Procedure: Allo-HSCT

Interventions

Venetoclax (VEN) DRUG

Venetoclax was administered at 100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-28 of cycle 1.

A

Azacitidine (AZA) DRUG

Azacitidine was administered at 75 mg/m² on days 1-7 of cycle 1.

A

Allo-HSCT PROCEDURE

All patients proceeded directly to allogeneic HSCT after cycle 1, regardless of remission. Myeloablative or intensified conditioning was preferred; reduced-intensity conditioning was allowed for intolerant patients. Donor source and transplant type were not restricted.

A

Eligibility Criteria

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

You may qualify if:

• Age ≥18 years.
• Previously untreated acute myeloid leukemia (AML) diagnosed according to the 2022 ELN guidelines. Patients with isolated extramedullary disease (i.e., no evidence of AML in bone marrow or peripheral blood) are not eligible.
• Bone marrow biopsy demonstrating cellularity \<20% or concurrent myelofibrosis; Or a prior history of an antecedent hematologic disorder, radiotherapy/chemotherapy-related history, or presence of myelodysplasia-related changes (AML-MRC according to WHO-HEAM5).
• The patient is deemed suitable for allogeneic hematopoietic stem cell transplantation as assessed by the treating physician.
• Adequate organ function, defined as follows: a. Good liver function: serum total bilirubin ≤2.0 × upper limit of normal (ULN); if considered due to Gilbert's disease or leukemia, serum total bilirubin \<3.0 × ULN. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemia. b. Good renal function: serum creatinine ≤2.0 × ULN or creatinine clearance \>30 mL/min calculated using the Cockcroft-Gault formula. c. No history of chronic lung disease and no dyspnea. Otherwise, documented diffusing capacity of the lung for carbon monoxide ≤40% (adjusted for hemoglobin if available) and forced expiratory volume in 1 second/forced vital capacity ≥50%.
• ECOG performance status score 0-2.
• Ability to understand and voluntarily sign informed consent.
• Women of childbearing potential must have a negative serum pregnancy test before initiation of study treatment.

You may not qualify if:

• Prior treatment for AML, except non-cytotoxic therapy given to stabilize disease.
• White blood cell count ≥10×10⁹/L, or presence of proliferation-associated gene mutations such as FLT3.
• Favorable risk group according to the 2022 ELN prognostic stratification, e.g., t(8;21), inv(16)/t(16;16), NPM1 mutation, or CEBPA bZIP in-frame mutation.
• No suitable stem cell donor available.
• Acute promyelocytic leukemia (APL).
• Clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia.
• Life-threatening immediate complications of leukemia, such as uncontrolled bleeding, hypoxic pneumonia, sepsis, and/or disseminated intravascular coagulation (DIC). Expected survival \<12 weeks.
• Prior allogeneic hematopoietic stem cell transplantation for a hematologic disorder.
• Current use of strong CYP3A4 inducers or narrow-therapeutic-window CYP3A4 substrates; enrollment is allowed only if these drugs can be switched to alternatives ≥5 half-lives before the first dose of study treatment.
• Active, uncontrolled systemic fungal, bacterial, or viral infection despite appropriate antibiotic, antiviral, or other therapy.
• Known infection with human immunodeficiency virus (HIV) or active hepatitis B virus (HBV) or hepatitis C virus (HCV) that cannot be controlled by therapy.
• Another active malignancy, unless the patient has been disease-free for ≥5 years before initiation of study treatment. However, patients with the following history/concurrent conditions or similar indolent cancers are eligible: Basal cell or squamous cell carcinoma of the skin Carcinoma in situ of the cervix Carcinoma in situ of the breast Prostate cancer found incidentally on histology.
• Significant active cardiac disease within 6 months before initiation of study treatment, including New York Heart Association (NYHA) Class III or IV congestive heart failure, myocardial infarction, unstable angina, and/or stroke.
• Uncontrolled hypertension (systolic blood pressure \>180 mmHg or diastolic blood pressure \>100 mmHg).
• Dysphagia, short-bowel syndrome, gastroparesis, or other conditions that limit oral intake or gastrointestinal absorption.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead
• Nanfang Hospital, Southern Medical University: collaborator
• Ruijin Hospital North Shanghai Jiao Tong University School of Medicine: collaborator

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

Location

MeSH Terms

Interventions

venetoclax; Azacitidine

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Suning Chen, Doctor

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zhen Shen, Doctor

CONTACT

0512-67976802; zhenshen96@sina.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: June 1, 2026
• First Posted: June 9, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): May 31, 2029
• Study Completion (Estimated): August 31, 2029
• Last Updated: June 9, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)