Acute Myeloid Leukemia (AML)Phase 3NCT07686965
Lisaftoclax Plus Azacitidine Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia Patients at High Risk of Relapse
A Phase 3 study testing Lisaftoclax Plus Azacitidine in people with Acute Myeloid Leukemia (AML) and Maintenance Treatment, run by Institute of Hematology & Blood Diseases Hospital, China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Maintenance Treatment, Hematopoetic Stem Cell Transplantation
- What is being tested?
- Lisaftoclax Plus Azacitidine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 191 participants
- Where
- No sites listed yet
- Study dates
- 10 Aug 2026to 10 Aug 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- 1) 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.
- 3. Undergoing first allogeneic hematopoietic stem cell transplantation (allo-HSCT).
- 4. 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.
- 5. 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.
- 8. Written informed consent may be provided by the participant or an authorized immediate family member in accordance with local regulations.
Who cannot join
- 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.
About this study
Official 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
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:
Read more
- 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
- Being tested
- Lisaftoclax Plus Azacitidine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lisaftoclax Plus AzacitidineDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Lisaftoclax plus Azacitidine Maintenance Therapy | Experimental | Not described Receives: Drug: Lisaftoclax Plus Azacitidine |
| Observation | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Secondary outcomes (5)
Cumulative Incidence of Relapse
The cumulative incidence of relapse will be assessed during the follow-up period.
Measured: 2 years
Overall Survival
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.
Measured: 2 years.
Cumulative Incidence of Pre-emptive Therapy.
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.
Measured: 2 years
Cumulative Incidence of Non-Relapse Mortality
Non-relapse mortality, defined as death without prior disease relapse or progression, assessed as a cumulative incidence.
Measured: 2-years.
Treatment-Related Adverse Events
Incidence, severity, and type of adverse events will be evaluated and graded according to CTCAE v5.0 during maintenance therapy and follow-up.
Measured: From initiation of maintenance therapy to 30 days after last dose of study drug.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2026
- Study start10 Aug 2026 (estimated)
- Primary completion10 Aug 2030 (estimated)
- Study completion10 Aug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 191 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07686965
- Sponsor's protocol ID
- IIT2026015
- Status last verified
- Jun 2026
- First submitted
- 30 Jun 2026
- Last updated
- 7 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07686965
- Acronym / sponsor ID
- IIT2026015
- Conditions
- Acute Myeloid Leukemia (AML), Maintenance Treatment, Hematopoetic Stem Cell Transplantation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 191 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Aug 2026 (estimated)
- Primary completion
- 10 Aug 2030 (estimated)
- Study completion
- 10 Aug 2030 (estimated)
- First posted
- 7 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lisaftoclax plus Azacitidine Maintenance Therapy | Experimental | Drug: Lisaftoclax Plus Azacitidine |
| Observation | No intervention | – |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
- 1) 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.
- 3. Undergoing first allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lisaftoclax Plus AzacitidineParticipants 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. | Drug | Lisaftoclax plus Azacitidine Maintenance Therapy |
Primary outcome
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.
5 secondary outcomes
Cumulative Incidence of Relapse
Time frame: 2 years
Overall Survival
Time frame: 2 years.
Cumulative Incidence of Pre-emptive Therapy.
Time frame: 2 years
Cumulative Incidence of Non-Relapse Mortality
Time frame: 2-years.
Treatment-Related Adverse Events
Time frame: From initiation of maintenance therapy to 30 days after last dose of study drug.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lisaftoclax Plus Azacitidine in people with Acute Myeloid Leukemia (AML) and Maintenance Treatment, run by Institute of Hematology & Blood Diseases Hospital, China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease-Free Survival (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026.