Lisaftoclax Plus Azacitidine Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia Patients at High Risk of Relapse

NCT07686965 | Not Yet Recruiting Phase 3

• 1 other identifier: IIT2026015
• Study Type: interventional
• Target: 191
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study evaluates the efficacy and safety of maintenance therapy with lisaftoclax plus azacitidine after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adults with acute myeloid leukemia (AML) at high risk of relapse. The main questions this study aims to answer are:

• Does maintenance therapy with lisaftoclax plus azacitidine improve disease-free survival compared with observation alone after allo-HSCT?
• Does maintenance therapy reduce relapse and improve overall survival?
• What adverse events and safety outcomes are associated with this treatment strategy? Researchers will compare maintenance therapy with lisaftoclax plus azacitidine with observation or best supportive care in patients with AML at high risk of relapse following allo-HSCT. Participants will:
• Be randomly assigned in a 2:1 ratio to receive either maintenance therapy with lisaftoclax plus azacitidine or observation.
• Receive study treatment for up to 12 cycles or undergo observation according to the study assignment.
• Undergo regular follow-up assessments, disease monitoring, and safety evaluations after transplantation.

Conditions

Acute Myeloid Leukemia (AML); Maintenance Treatment; Hematopoetic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• Disease-Free Survival

  Disease status (including relapse) and survival outcomes will be evaluated for the assessment of disease-free survival (DFS). DFS is defined as the time from randomization to the first occurrence of relapse or death from any cause. Prespecified subgroup analyses will be conducted according to pre-transplant measurable residual disease (MRD) status (MRD-positive vs. MRD-negative) to assess the consistency of treatment effects across MRD subgroups.

  2 years

Secondary Outcomes (5)

• Cumulative Incidence of Relapse

  2 years

• Overall Survival

  2 years.

• Cumulative Incidence of Pre-emptive Therapy.

  2 years

• Cumulative Incidence of Non-Relapse Mortality

  2-years.

• Treatment-Related Adverse Events

  From initiation of maintenance therapy to 30 days after last dose of study drug.

Study Arms (2)

Lisaftoclax plus Azacitidine Maintenance Therapy

EXPERIMENTAL

Drug: Lisaftoclax Plus Azacitidine

Observation

NO INTERVENTION

Interventions

Lisaftoclax Plus Azacitidine DRUG

Participants will receive maintenance therapy with lisaftoclax plus azacitidine after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Lisaftoclax will be administered orally at a dose of 400 mg once daily on Days 1-7 of each treatment cycle. Azacitidine will be administered at a dose of 32 mg/m² by subcutaneous injection or intravenous infusion on Days 1-5 of each treatment cycle. Each treatment cycle is 28 days in length. Maintenance therapy will be administered for up to 12 cycles or until 15 months after allo-HSCT, whichever occurs first. The dose of lisaftoclax may be modified according to concomitant medications and treatment-related hematologic toxicities. The interval between treatment cycles may be extended based on individual tolerability and hematologic recovery.

Lisaftoclax plus Azacitidine Maintenance Therapy

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must meet all of the following criteria to be eligible for enrollment in this study:
• Diagnosed with acute myeloid leukemia (AML), excluding acute promyelocytic leukemia, according to the WHO 2022 classification, and without FLT3-ITD or FLT3-TKD mutations.
• Presence of at least one of the following high-risk features:
• \) Adverse-risk AML according to the ELN 2022 risk classification; 2) Refractory AML; 3) Relapsed AML; 4) Persistent measurable residual disease positivity prior to transplantation; 5) Secondary AML transformed from myelodysplastic syndrome or myeloproliferative neoplasm.
• \. Undergoing first allogeneic hematopoietic stem cell transplantation (allo-HSCT).
• \. Stable hematologic recovery, defined as: Absolute neutrophil count ≥ 1.0 × 10⁹/L without G-CSF support; and Platelet count ≥ 50 × 10⁹/L without platelet transfusion within 7 days prior to randomization.
• \. Age ≥18 years and ≤75 years. 6. Eastern Cooperative Oncology Group performance status of 0-2. 7. Ability to provide written informed consent before initiation of any study procedures.
• \. Written informed consent may be provided by the participant or an authorized immediate family member in accordance with local regulations.

You may not qualify if:

• Participants meeting any of the following criteria will be excluded from the study:
• Evidence of disease relapse or impending relapse prior to randomization after transplantation, as determined by morphologic assessment or flow cytometry.
• Active or uncontrolled acute graft-versus-host disease (aGVHD) requiring systemic immunosuppressive therapy.
• Uncontrolled active infection requiring systemic antibacterial, antifungal, or antiviral therapy within 7 days prior to enrollment.
• Moderate or severe hepatic impairment, as defined by the Child-Pugh classification.
• Severe renal impairment, defined as creatinine clearance \<30 mL/min (calculated using the Cockcroft-Gault formula) or requirement for dialysis.
• Pregnancy, or unwillingness/inability to use adequate contraception during the study treatment period.
• Psychiatric disorders or other medical conditions that, in the investigator's judgment, would interfere with compliance with study treatment, monitoring, or study procedures.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Lisaftoclax; Azacitidine

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2026
• First Posted: July 7, 2026
• Study Start (Estimated): August 10, 2026
• Primary Completion (Estimated): August 10, 2030
• Study Completion (Estimated): August 10, 2030
• Last Updated: July 7, 2026

Record last verified: 2026-06