Akut Lösemide Nakil Sonrası İdame Olarak BN104'ün Faz 2 Çalışması

Sade dilde özet

Bu çalışma, allojenik kök hücre nakli (HSCT) sonrası akut lösemi (acute leukemia) hastalarında idame tedavisi olarak BN104 ilacının tek başına kullanımını değerlendirmektedir. Çalışmaya, belirli genetik değişiklikler (NPM1 mutasyonu, KMT2A ya da NUP98 yeniden düzenlenmesi gibi) taşıyan, 12 yaş ve üzeri, nakil sonrası tam remisyona ulaşmış hastalar katılabilmektedir. Çalışma Faz 2 aşamasında yürütülmekte ve halen katılımcı almaktadır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 2 (Phase 2)

Durum

Katılımcı alıyor

Sponsor

The First Affiliated Hospital of Soochow University

Müdahale

BN104 monotherapy

Başlangıç

01.08.2025

Tahmini bitiş

30.12.2029

Ülkeler

China

İlgili genler

Merkezler

• Suzhou, China

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

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.