Using NK520 to treat children with relapsed or refractory acute myeloid leukemia

An Open, Single Center Exploratory Study to Evaluate Safety and Efficacy of NK520 for Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia

This study is testing if a new treatment using special immune cells can help children with relapsed or hard-to-treat acute myeloid leukemia feel better.

Quick facts

• Phase: EARLY_PHASE1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 6 Years to 18 Years
• Sex: All
• Sponsor: Base Therapeutics (Shanghai) Co., Ltd. (industry)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06541405 on ClinicalTrials.gov

What this trial studies

This open-label, single-arm study evaluates the safety and efficacy of allogenic NK520 cells administered via intravenous injection in pediatric patients with relapsed or refractory acute myeloid leukemia. Participants aged 6 to 18 years will receive weekly infusions of NK520, and the study will monitor for dose-limiting toxicity and determine the maximum tolerable dose. The findings will inform future clinical trials regarding dosage and treatment plans for this patient population.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients aged 6 to 18 years with relapsed or refractory acute myeloid leukemia who have failed at least two lines of therapy.

Not a fit: Patients who are not diagnosed with acute myeloid leukemia or those who are not relapsed or refractory after multiple treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children with difficult-to-treat acute myeloid leukemia.

How similar studies have performed: Other studies using allogenic NK cell therapies have shown promise, suggesting potential for success in this novel application.

Eligibility criteria

Inclusion Criteria:

1. Participants must be between 6 and 18 years;
2. Diagnostic Criteria: Meet the 2022 World Health Organization (WHO) diagnostic criteria for 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. 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:

   * HBsAg positive with serum HBV-DNA level ≥1×10\^3 copies/mL or above normal range;
   * Positive for HCV antibodies;
9. Any other condition or situation in which the investigator deems the patient unsuitable for participation in this study.

Where this trial is running

Shanghai, Shanghai Municipality

• Shanghai Children's Medical Center — Shanghai, Shanghai Municipality, China (RECRUITING)

Study contacts

• Principal investigator: wenting Hu — Shanghai children's medical center
• Study coordinator: Jun Yan

Conditions: Relapsed/Refractory Acute Myeloid Leukemia