Phase 2 Trial of BN104 as Post-HSCT Maintenance in Acute Leukemia

NCT07101497 | Recruiting Phase 2

• 1 other identifier: BN104 maintenance post-HSCT
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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). 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.

Conditions

Menin Inhibitors; Post Hematopoietic Stem Cell Transplantation; Maintenance Therapy; Acute Leukemia; KMT2A Rearrangement; NPM1 Mutation; NUP98 Gene Rearrangement

Outcome Measures

Primary Outcomes (1)

• 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.

  2 years

Secondary Outcomes (5)

• OS

  2 years

• EFS

  2 years

• CIR

  2 years

• NRM

  2 years

• Incidence and severity of GVHD

  2 years

Other Outcomes (2)

• Pre-/post-transplant MRD dynamics

  2 years

• Impact of BN104 on immune recovery

  2 years

Study Arms (1)

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.

Drug: BN104 monotherapy

Interventions

BN104 monotherapy DRUG

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%.

BN104 maitenenace

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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).
• 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.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

RECRUITING

Study Officials

• Su-ning Chen, M.D.

  (0086)13814881746

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Su-ning Chen, M.D.

CONTACT

(0086)13814881746; chensuning@sina.com

Hai-ping Dai, M.D.

CONTACT

(0086)13914086271

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof.

Study Record Dates

• First Submitted: July 28, 2025
• First Posted: August 3, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 30, 2029
• Last Updated: August 3, 2025

Record last verified: 2025-07

Locations

CN (1)