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

Plain-language summary

This study evaluates the drug BN104 used on its own as maintenance therapy in patients with acute leukemia after allogeneic stem cell transplantation (HSCT). It is open to patients aged 12 and older who carry certain genetic changes (such as an NPM1 mutation or KMT2A or NUP98 rearrangement) and have reached complete remission after transplant. The study is being conducted in Phase 2 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Recruiting

Sponsor

The First Affiliated Hospital of Soochow University

Intervention

BN104 monotherapy

Start date

8/1/2025

Estimated completion

12/30/2029

Countries

China

Related genes

Locations

• Suzhou, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.