Sikkerhed og effektivitet af NK520 til behandling af pædiatrisk tilbagefald/refraktær akut myeloid leukæmi

Inclusion Criteria:

1. Participants must be between 6 and 18 years;
2. Diagnostic Criteria: Meet the 2022 World Health Organization (WHO) diagnostic criteria for acute myelocytic leukemia (AML), unsuitable for current treatments or patients with relapsed/refractory AML after ≥2 lines of therapy. The definition of relapsed/refractory acute myeloid leukemia is based on the 2017 Chinese Guidelines for Diagnosis and Treatment: a. Relapsed AML: Diagnosis is confirmed when leukemia cells reappear in the peripheral blood or bone marrow blast cells exceed 5% after complete remission (CR) (excluding reasons such as bone marrow regeneration post-consolidation chemotherapy) or there is extramedullary infiltration by leukemia cells; b. Refractory AML: Initial cases unresponsive after two cycles of standard regimen treatment; recurrence within 12 months after CR and consolidation therapy; recurrence beyond 12 months with ineffectiveness of conventional chemotherapy; those who have relapsed twice or more; or persistent extramedullary leukemia;
3. For participants under 16 years old, Lansky performance status must be ≥50%; for participants aged 16 or older, Karnofsky performance status must be ≥50%;
4. Expected survival of at least 12 weeks;
5. Normal Organ Function.

Exclusion Criteria:

1. Acute promyelocytic leukemia, chronic myeloid leukemia, acute mixed lineage leukemia, or known central nervous system leukemia;

2. AML associated with congenital syndromes, such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome, or congenital aplastic anemia;

3. Severe bleeding tendency or coagulation disorders, or currently receiving thrombolytic therapy;

4. Human immunodeficiency virus (HIV)-infected individuals, or individuals with known active syphilis infection;

5. Receipt of live attenuated vaccines within 2 weeks before the first dose or planned during the study period;

6. Participation in another clinical trial and receipt of investigational drug within 4 weeks prior to the first dose;

7. Receipt of immune-modulatory drugs (including thymosin, interferons, except for local use to manage conditions like pleural or ascites fluid) within 2 weeks before the first dose;

8. At screening, positive hepatitis B or C viral markers as follows:

   • Hepatitis B surface antigen (HBsAg) positive with serum hepatitis B virus-deoxyribonucleic acid (HBV-DNA) level ≥1×10^3 copies/mL or above normal range;
   • Positive for hepatitis C virus (HCV) antibodies;

9. Any other condition or situation in which the investigator deems the patient unsuitable for participation in this study.