Skip to main content
OpenTrials
Not Yet Recruiting

NCT Number: NCT07686965

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

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.

Not Yet Recruiting

Trial opening soon.

Get Notified

Key information

Conditions

Acute Myeloid Leukemia (AML) Hematologic Diseases Hematopoetic Stem Cell Transplantation Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Maintenance Treatment Neoplasms Neoplasms by Histologic Type

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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.

Exclusion criteria

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.

Treatment and study plan

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.

Primary outcomes

  1. Disease-Free Survival

    Time frame: 2 years

    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.

Secondary outcomes

  1. Cumulative Incidence of Relapse

    Time frame: 2 years

    The cumulative incidence of relapse will be assessed during the follow-up period.

  2. Overall Survival

    Time frame: 2 years.

    Overall survival (OS) is defined as the time from randomization to death from any cause. Prespecified subgroup analyses will be conducted according to pre-transplant measurable residual disease (MRD) status to assess the consistency of treatment effects between MRD-positive and MRD-negative patients.

  3. Cumulative Incidence of Pre-emptive Therapy.

    Time frame: 2 years

    Pre-emptive therapy rate is defined as the proportion of patients who receive additional anti-leukemic treatment triggered by measurable residual disease positivity or other molecular evidence of impending relapse after allogeneic hematopoietic stem cell transplantation.

  4. Cumulative Incidence of Non-Relapse Mortality

    Time frame: 2-years.

    Non-relapse mortality, defined as death without prior disease relapse or progression, assessed as a cumulative incidence.

  5. Treatment-Related Adverse Events

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

    Incidence, severity, and type of adverse events will be evaluated and graded according to CTCAE v5.0 during maintenance therapy and follow-up.

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Official study title

Maintenance Therapy With Lisaftoclax Plus Azacitidine After Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia Patients at High Risk of Relapse: A Multicenter, Open-Label, Randomized Controlled Trial

Important dates

Study start
2026
Primary completion
2030
Study completion
2030
First posted
Jul 7, 2026
Registry last updated
Jul 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.