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

Launched by THE FIRST AFFILIATED HOSPITAL OF SOOCHOW UNIVERSITY · Jul 28, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medicine called BN104 to help prevent acute leukemia from coming back after patients have had a stem cell transplant from a donor. This treatment is for people whose leukemia has certain specific genetic changes and who are in remission (meaning their disease is under control) after their transplant. The goal is to see if taking BN104 by mouth twice daily can keep the leukemia from returning and to check if the medicine is safe to use over a longer period.

To join this study, patients need to be at least 12 years old, weigh at least 35 kg (about 77 pounds), and have had a stem cell transplant within the past 1 to 6 months. They must have certain types of acute leukemia with specific genetic markers and be doing well enough after transplant, with healthy blood counts and no active infections or serious complications. Participants will take BN104 in cycles lasting about a month each, for up to 2 to 3 years, and will be closely monitored to see how well the treatment works and if there are any side effects. This study is currently recruiting patients, and those interested should talk with their doctor to learn more about whether this trial might be a good fit for them.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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.
• Exclusion Criteria:
• • 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.