Menin InhibitorsNCT07559695
A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance After Allo-HSCT
An observational study of Menin Inhibitors and Acute Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Menin Inhibitors, Acute Leukemia, Post Hematopoietic Stem Cell Transplantation
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Nov 2025to 31 Dec 2029
- Phase
- Observational
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with acute leukemia who underwent allogeneic hematopoietic stem cell transplantation and received post-transplant menin inhibitor maintenance therapy.
Who may be able to join
- Age ≥ 15 years.
- Patients diagnosed with acute leukemia (including AML, ALL, and MPAL) according to the World Health Organization (WHO 2022) criteria.
- Must meet one of the following characteristics: a. Harboring an NPM1 gene mutation (without concurrent FLT3-ITD or FLT3-TKD mutation); b. Harboring a KMT2A gene rearrangement or KMT2A-PTD; c. Harboring a NUP98 gene rearrangement; d. Other acute leukemia subtypes dependent on the menin-KMT2A interaction, if evidenced, may be enrolled upon discussion with and approval from the principal research team.
- Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), with ≥ 30 days elapsed since the date of graft infusion.
- Received menin inhibitor maintenance therapy after allo-HSCT and meets the following conditions: a. Received at least ≥ 2 complete cycles (7 days per cycle) of menin inhibitor therapy, or cumulative medication duration ≥ 14 days; b. Patient was in a state of CR/CRh/CRi at the initiation of maintenance therapy.
- No evidence of leukemia relapse during menin inhibitor maintenance therapy, defined as: a. Bone marrow blasts < 5%, and blasts do not exhibit morphological features of acute leukemia (e.g.Auer rods); b. No evidence of extramedullary leukemia (e.g. CNS leukemia or myeloid sarcoma).
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- The menin inhibitors used include but are not limited to: Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors.
- Capable of understanding and voluntarily signing the informed consent form.
- Complete clinical data.
Who cannot join
- Presence of any of the following at the initiation of menin inhibitor maintenance therapy (including within 28 days prior to starting treatment): a. Morphologic relapse in bone marrow (bone marrow blasts ≥ 5%); b. Presence of leukemic cells in peripheral blood.
- Active infection that is deemed uncontrolled by the investigator.
- Severe organ dysfunction, including: a. Hepatic impairment: ALT or AST ≥ 5 × ULN (Upper Limit of Normal), or total bilirubin ≥ 3 × ULN; b. Severe renal impairment: eGFR < 30 ml/min/1.73 m²; c. Cardiac dysfunction: NYHA (New York Heart Association) Class III-IV.
- Concurrent acute graft-versus-host disease (aGVHD) ≥ Grade 2 or chronic graft-versus-host disease (cGVHD) ≥ Grade 3, requiring corticosteroids ≥ 1 mg/kg and ≥ 3 types of immunosuppressive therapy (including CNI, ruxolitinib, belumosudil, etc.).
- History of other malignancies requiring ongoing treatment (except for malignancies that have undergone curative treatment or are assessed to be in complete remission and require no systemic maintenance therapy or radiotherapy).
- Any gastrointestinal disorder that may affect the intake or absorption of oral medications (e.g.dysphagia, gastroparesis, uncontrolled chronic diarrhea, intestinal GVHD, etc).
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- Patients deemed unsuitable for inclusion in this study by the investigator.
- Severely missing clinical data, precluding efficacy or safety assessment.
- Receipt of other maintenance therapies, including but not limited to hypomethylating agents, donor lymphocyte infusion (DLI), or other specific small-molecule targeted drugs.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Su-ning Chen, M.D., +8613814881746, chensuning@sina.com
About this study
Official title: A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation
The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will receive menin inhibitor maintenance therapy as part of their routine clinical practice. Acceptable agents include, but are not limited to, Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. This study imposes no additional interventions on clinical management. The specific menin inhibitor, initiation timing, dose adjustments, and treatment duration are determined at the investigator's discretion based on the patient's individual condition and clinical circumstances.
Patients will enter the follow-up phase upon initiation of menin inhibitor maintenance therapy. Efficacy and safety will be assessed at every cycle during the treatment period. Following the completion of treatment, survival follow-up visits will be conducted every three cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.
Read the detailed description
AML with KMT2A rearrangements are grouped as a high risk subtype. Allogenic hematopoietic stem cell transplantation is the only curative strategy for this subtype. However, patients with KMT2A rearrangements have a high incidence of relapse rates. Menin inhibitors have shown promising remission rates in patients with relpased or refractory patients haboring specific fusions including KMT2A rearrangements. The role of menin inhibitors in post-HSCT maintenance therapy remains unknown.
The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will receive menin inhibitor maintenance therapy as part of their routine clinical practice. Acceptable agents include, but are not limited to, Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. This study imposes no additional interventions on clinical management. The specific menin inhibitor, initiation timing, dose adjustments, and treatment duration are determined at the investigator's discretion based on the patient's individual condition and clinical circumstances.
Patients will enter the follow-up phase upon initiation of menin inhibitor maintenance therapy. Efficacy and safety will be assessed at every cycle during the treatment period. Following the completion of treatment, survival follow-up visits will be conducted every three cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Post-alloHSCT menin maintenance | Patients with acute leukemia who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) and received menin inhibitor maintenance therapy after transplantation. Menin inhibitors include but are not limited to Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-year rate of RFS
It is measured as the proportions of numbers of patients with hematologic relapse to the numbers of the overall enrolled patients at 2 years from the first day of taking menin inhibitor post allo-HSCT.
Measured: 2 years
Secondary outcomes (5)
OS
Overall survival (OS): It is measured from the date of the first day of taking menin inhibitor post allo-HSCT to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive.
Measured: 2 years
EFS
Event-free survival(EFS): It is measured from he date from the first day of taking menin inhibitor post allo-HSCT to the first documented hematologic relapse, initiation of other maintenance therapy stragies such as hypomethylation agents, interferon, donor lymphocyte infusion, Bcl-2 inhibitor or death from any cause. Patients who die without evidence of relapse are typically censored at the last disease-free assessment.
Measured: 2 years
CIR
Cumulative incidence of relapse (CIR): It is measured as the estimated rate of hematologic relapse or extramedullary relapse since the first day of taking menin inhibitor post allo-HSCT.
Measured: 2 years
NRM
non-relapse mortality (NRM): It is measured the probability of death attributable to treatment-related complications or comorbidities, excluding disease relapse/progression since the first day of taking menin inhibitor post allo-HSCT.
Measured: 2 years
Incidence and severity of GVHD
Incidence and severity of acute and chronic GVHD since the first day or taking menin inhibitor post allo-HSCT.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2026
- Study start1 Nov 2025
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Menin InhibitorsStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Suning, The First Affiliated Hospital of Soochow University
- Investigators
- Su-ning Chen, M.D., Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07559695
- Sponsor's protocol ID
- menin inhibitors maintenance
- Status last verified
- Apr 2026
- First submitted
- 24 Apr 2026
- Last updated
- 30 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07559695
- Acronym / sponsor ID
- menin inhibitors maintenance
- Conditions
- Menin Inhibitors, Acute Leukemia, Post Hematopoietic Stem Cell Transplantation, Maintenance Therapy, KMT2A Rearrangement, NPM1 Mutation
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Nov 2025 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 30 Apr 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.
The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will receive menin inhibitor maintenance therapy as part of their routine clinical practice. Acceptable agents include, but are not limited to, Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. This study imposes no additional interventions on clinical management. The specific menin inhibitor, initiation timing, dose adjustments, and treatment duration are determined at the investigator's discretion based on the patient's individual condition and clinical circumstances.
Patients will enter the follow-up phase upon initiation of menin inhibitor maintenance therapy. Efficacy and safety will be assessed at every cycle during the treatment period. Following the completion of treatment, survival follow-up visits will be conducted every three cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.
Detailed description
AML with KMT2A rearrangements are grouped as a high risk subtype. Allogenic hematopoietic stem cell transplantation is the only curative strategy for this subtype. However, patients with KMT2A rearrangements have a high incidence of relapse rates. Menin inhibitors have shown promising remission rates in patients with relpased or refractory patients haboring specific fusions including KMT2A rearrangements. The role of menin inhibitors in post-HSCT maintenance therapy remains unknown.
The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will receive menin inhibitor maintenance therapy as part of their routine clinical practice. Acceptable agents include, but are not limited to, Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. This study imposes no additional interventions on clinical management. The specific menin inhibitor, initiation timing, dose adjustments, and treatment duration are determined at the investigator's discretion based on the patient's individual condition and clinical circumstances.
Patients will enter the follow-up phase upon initiation of menin inhibitor maintenance therapy. Efficacy and safety will be assessed at every cycle during the treatment period. Following the completion of treatment, survival follow-up visits will be conducted every three cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Post-alloHSCT menin maintenance | – | –Patients with acute leukemia who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) and received menin inhibitor maintenance therapy after transplantation. Menin inhibitors include but are not limited to Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with acute leukemia who underwent allogeneic hematopoietic stem cell transplantation and received post-transplant menin inhibitor maintenance therapy.
Key inclusion criteria (9 listed)
- Age ≥ 15 years.
- Patients diagnosed with acute leukemia (including AML, ALL, and MPAL) according to the World Health Organization (WHO 2022) criteria.
- Must meet one of the following characteristics: a. Harboring an NPM1 gene mutation (without concurrent FLT3-ITD or FLT3-TKD mutation); b. Harboring a KMT2A gene rearrangement or KMT2A-PTD; c. Harboring a NUP98 gene rearrangement; d. Other acute leukemia subtypes dependent on the menin-KMT2A interaction, if evidenced, may be enrolled upon discussion with and approval from the principal research team.
- Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), with ≥ 30 days elapsed since the date of graft infusion.
- Received menin inhibitor maintenance therapy after allo-HSCT and meets the following conditions: a. Received at least ≥ 2 complete cycles (7 days per cycle) of menin inhibitor therapy, or cumulative medication duration ≥ 14 days; b. Patient was in a state of CR/CRh/CRi at the initiation of maintenance therapy.
Key exclusion criteria (9 listed)
- Presence of any of the following at the initiation of menin inhibitor maintenance therapy (including within 28 days prior to starting treatment): a. Morphologic relapse in bone marrow (bone marrow blasts ≥ 5%); b. Presence of leukemic cells in peripheral blood.
- Active infection that is deemed uncontrolled by the investigator.
- Severe organ dysfunction, including: a. Hepatic impairment: ALT or AST ≥ 5 × ULN (Upper Limit of Normal), or total bilirubin ≥ 3 × ULN; b. Severe renal impairment: eGFR < 30 ml/min/1.73 m²; c. Cardiac dysfunction: NYHA (New York Heart Association) Class III-IV.
- Concurrent acute graft-versus-host disease (aGVHD) ≥ Grade 2 or chronic graft-versus-host disease (cGVHD) ≥ Grade 3, requiring corticosteroids ≥ 1 mg/kg and ≥ 3 types of immunosuppressive therapy (including CNI, ruxolitinib, belumosudil, etc.).
- History of other malignancies requiring ongoing treatment (except for malignancies that have undergone curative treatment or are assessed to be in complete remission and require no systemic maintenance therapy or radiotherapy).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
2-year rate of RFS
Time frame: 2 years
It is measured as the proportions of numbers of patients with hematologic relapse to the numbers of the overall enrolled patients at 2 years from the first day of taking menin inhibitor post allo-HSCT.
5 secondary outcomes
OS
Time frame: 2 years
EFS
Time frame: 2 years
CIR
Time frame: 2 years
NRM
Time frame: 2 years
Incidence and severity of GVHD
Time frame: 2 years
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 30 Apr 2026.
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Phase 2 Trial of BN104 as Post-HSCT Maintenance in Acute Leukemia
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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.
Su-ning Chen, M.D. principal investigator
The First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Menin Inhibitors and Acute Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in 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 9 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
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?
Contact Su-ning Chen, M.D. ((0086)13814881746, chensuning@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-year rate of RFS (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 30 Apr 2026.