Menin InhibitorsPhase 2NCT07101497
Phase 2 Trial of BN104 as Post-HSCT Maintenance in Acute Leukemia
A Phase 2 study testing BN104 monotherapy in people with Menin Inhibitors and Post Hematopoietic Stem Cell Transplantation, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Menin Inhibitors, Post Hematopoietic Stem Cell Transplantation, Maintenance Therapy
- What is being tested?
- BN104 monotherapy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2025to 30 Dec 2029
- Phase
- Phase 2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patients.
- Adult and adolescent patients aged ≥12 years who must weight ≥35 kg.
- Diagnosed with acute myeloid leuekmia, acute lymphoblastic leukemia or ambiguous acute leukemia according to the World Health Organization classification of hematologic neoplams (WHO 2022).
- Intermediate or high risk accroding to the ELN risk stratification.
- Harboring one of the following genetic aberrations: a. somatic NPM1 mutation (without FLT3-ITD/TKD co-mutations); b. KMT2A rearrangement/KMT2A-PTD; c. NUP98 rearrangement; d. other genetic alterations dependent on menin-KMT2A.
- Received allogenic hematopoietic stem cell transplantation within 30-180 days at the initiation of BN104 maintenance therapy.
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- Achieved full donor chimerism and hematologic recovery, with acute neuthrophil count (ANC) ≥1.0×10⁹/L, platelets ≥75×10⁹/L (no red blood cells /platelets transfusion within 7 days, no G-CSF or GM-CSF within 72 hours).
- Complete hematological remission (CHR) after first allo-SCT. CHR must be confirmed by bone marrow analysis within 14 days before entering the study (CHR criteria are: "< 5% marrow blasts, no peripheral blasts, blood platelet count > 75×10⁹/L, WBC count > 3.5 G/L, ANC ≥ 1.0×10⁹/L).
- No extramedullary leukemia.
- Eastern Cooperative Oncology Group (ECOG) performance status score 0-2.
- Adequate organ function
- Provided informed consent by all patients and the guardians ( aged 12-17 years).
- Written informed consent.
- ECOG ≥ 2.
Who cannot join
- Complicated with active and uncontrolled infections.
- Activation of virus, (e.g., CMV viremia with CMV DNA copies > 400 copies/ml, EBV viremia with EBV DNA copies > 400 copies/ml, and proof of activation of adenovirus and Human Parvovirus B19 ).
- Activation of hepatitis B, hepatitis C, or human immunodeficiency virus.
- Cardiac disease as followings: a. inherited long QT syndrome. b. Congestive heart failure with NYHA ≥ grade 2.
- ≥ grade 2 acute GVHD or ≥ grade 3 chronic GVHD which requiring systemic therapy.
- Have received other maintenance therapies (e.g., hypomethylating agents, targetd drugs such as Bcl-2 inhibitors, FLT3 inhibitors, IDH1/2 inhibitors, interferon, interleukin-2, donor lymphocyte infusion and chemotherapy).
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- History of other malignancies which needed systemic treatment (excluding those in stable remission without maintenance therapy).
- Any gastrointestinal condition that may interfere with oral drug intake or absorption (e.g., dysphagia, gastroparesis, uncontrolled chronic diarrhea, intestinal graft versus host disease.
- Pregnancy, breastfeeding
- Hypersensitivity to BN104.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Hai-ping Dai, M.D. contact
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: Phase 2 Trial of Menin Inhibitor BN104 as Post Hematopoietic Stem Cell Transplantation Maintenance in Patients With Acute Leukemia
This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 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. BN104 is a menin inhibitor. It has shown promising remission rates in patients with relpased or refractory patients haboring specific fusions including KMT2A rearrangements. The role of BN104 in post-HSCT maintenance therapy remains unknown.
This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 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, incidence of acute and chronic graft versus host diseas, and safety.
- Conditions
- Menin Inhibitors, Post Hematopoietic Stem Cell Transplantation, Maintenance Therapy, Acute Leukemia, KMT2A Rearrangement, NPM1 Mutation
- Being tested
- BN104 monotherapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BN104 monotherapyDrug
BN104 will be started at 200 mg or 300mg, to a target dose of 400mg, twice daily, orally, 28 days a cycle, for a total of 24 to 36 cycles . When used in combination with posaconazoles and voriconazole, the dose of BN104 should be reduced by 50%.
| Study group | Role | What they receive |
|---|---|---|
| BN104 maitenenace | Experimental | Eligible patients will take BN104 start from 200 or 300mg, to a targent dose of 400mg, twice daily orally, 28 days for a cycle. When used in combination with posaconazoles and voriconazole, the dose of BN104 should be reduced by 50%. A total of 24 to 36 cycles of BN104 was planned. Receives: Drug: BN104 monotherapy |
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 BN104 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 BN104 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-freesurvival: It is measured from he date from the first day of taking BN104 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 BN104 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 BN104 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 BN104 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 posted3 Aug 2025
- Study start1 Aug 2025 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion30 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 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, (0086)13814881746
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07101497
- Sponsor's protocol ID
- BN104 maintenance post-HSCT
- Status last verified
- Jul 2025
- First submitted
- 28 Jul 2025
- Last updated
- 3 Aug 2025
- 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 3 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Trial of Menin Inhibitor BN104 as Post Hematopoietic Stem Cell Transplantation Maintenance in Patients With Acute Leukemia
- ClinicalTrials.gov ID
- NCT07101497
- Acronym / sponsor ID
- BN104 maintenance post-HSCT
- Conditions
- Menin Inhibitors, Post Hematopoietic Stem Cell Transplantation, Maintenance Therapy, Acute Leukemia, KMT2A Rearrangement, NPM1 Mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Aug 2025 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 30 Dec 2029 (estimated)
- First posted
- 3 Aug 2025 (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 is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 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. BN104 is a menin inhibitor. It has shown promising remission rates in patients with relpased or refractory patients haboring specific fusions including KMT2A rearrangements. The role of BN104 in post-HSCT maintenance therapy remains unknown.
This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Eligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 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, incidence of acute and chronic graft versus host diseas, and safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BN104 maitenenace | Experimental | Drug: BN104 monotherapyEligible patients will take BN104 start from 200 or 300mg, to a targent dose of 400mg, twice daily orally, 28 days for a cycle. When used in combination with posaconazoles and voriconazole, the dose of BN104 should be reduced by 50%. A total of 24 to 36 cycles of BN104 was planned. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- Male or female patients.
- Adult and adolescent patients aged ≥12 years who must weight ≥35 kg.
- Diagnosed with acute myeloid leuekmia, acute lymphoblastic leukemia or ambiguous acute leukemia according to the World Health Organization classification of hematologic neoplams (WHO 2022).
- Intermediate or high risk accroding to the ELN risk stratification.
- Harboring one of the following genetic aberrations: a. somatic NPM1 mutation (without FLT3-ITD/TKD co-mutations); b. KMT2A rearrangement/KMT2A-PTD; c. NUP98 rearrangement; d. other genetic alterations dependent on menin-KMT2A.
Key exclusion criteria (10 listed)
- Complicated with active and uncontrolled infections.
- Activation of virus, (e.g., CMV viremia with CMV DNA copies > 400 copies/ml, EBV viremia with EBV DNA copies > 400 copies/ml, and proof of activation of adenovirus and Human Parvovirus B19 ).
- Activation of hepatitis B, hepatitis C, or human immunodeficiency virus.
- Cardiac disease as followings: a. inherited long QT syndrome. b. Congestive heart failure with NYHA ≥ grade 2.
- ≥ grade 2 acute GVHD or ≥ grade 3 chronic GVHD which requiring systemic therapy.
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 |
|---|---|---|
| BN104 monotherapyBN104 will be started at 200 mg or 300mg, to a target dose of 400mg, twice daily, orally, 28 days a cycle, for a total of 24 to 36 cycles . When used in combination with posaconazoles and voriconazole, the dose of BN104 should be reduced by 50%. | Drug | BN104 maitenenace |
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 BN104 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 3 Aug 2025.
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A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance After Allo-HSCT
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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.
Hai-ping Dai, M.D. contact
Su-ning Chen, M.D. principal investigator
(0086)13814881746
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BN104 monotherapy in people with Menin Inhibitors and Post Hematopoietic Stem Cell Transplantation, 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 14 inclusion and 10 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 3 Aug 2025. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
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 3 Aug 2025; AzTrials last refreshed its copy on 6 Nov 2026.