Safety and Efficacy of NK520 to Treat Pediatric Relapsed/Refractory Acute Myeloid Leukemia

NCT06541405 | Recruiting Early Phase 1

• 1 other identifier: NK520-01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection. The safety and efficacy of this treatment will be evaluated.

Conditions

Relapsed/Refractory Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (2)

• Dose-Limiting Toxicity

  To evaluate the DLT during N520 treatment

  From the first infusion of NK520 to 4 weeks after last infusion of NK520

• Complete Response Rate (CRR)

  Effectiveness Metrics

  from the date of first infusion of NK510 up to 104 weeks

Secondary Outcomes (4)

• Overall response rate (ORR)

  from the date of first infusion of NK510 up to 104 weeks

• Duration of Response (DOR)

  From the date of enrollment up to 104 weeks

• Event-Free Survival (EFS)

  From date of enrollment up to 104 weeks, or progression, or date of death, whichever came first.

• Overall Survival (OS)

  From date of enrollment up to 104 weeks, or date of death, whichever came first.

Study Arms (3)

Group A(low-dose group)

EXPERIMENTAL

NK520: 5x10\^7 NK/kg

Drug: NK520

Group B(medium-dose group)

EXPERIMENTAL

NK520: 1.5×10\^8NK/kg

Drug: NK520

Group C(high-dose group)

EXPERIMENTAL

NK520: 3×10\^8NK/kg

Drug: NK520

Interventions

NK520 DRUG

The number of NK520 cell infused for each dosing should be calculated base on the body weight of subject. NK520 will be administered through intravenous infusion.

Also known as: genetic modified NK cell

Group A(low-dose group); Group B(medium-dose group); Group C(high-dose group)

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participants must be between 6 and 18 years;
• 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;
• For participants under 16 years old, Lansky performance status must be ≥50%; for participants aged 16 or older, Karnofsky performance status must be ≥50%;
• Expected survival of at least 12 weeks;
• Normal Organ Function.

You may not qualify if:

• Acute promyelocytic leukemia, chronic myeloid leukemia, acute mixed lineage leukemia, or known central nervous system leukemia;
• AML associated with congenital syndromes, such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome, or congenital aplastic anemia;
• Severe bleeding tendency or coagulation disorders, or currently receiving thrombolytic therapy;
• HIV-infected individuals, or individuals with known active syphilis infection;
• Receipt of live attenuated vaccines within 2 weeks before the first dose or planned during the study period;
• Participation in another clinical trial and receipt of investigational drug within 4 weeks prior to the first dose;
• 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;
• 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;
• Any other condition or situation in which the investigator deems the patient unsuitable for participation in this study.

Sponsors & Collaborators

• Base Therapeutics (Shanghai) Co., Ltd.: lead
• Shanghai Children's Medical Center: collaborator

Study Sites (1)

Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, 200127, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• wenting Hu

  Shanghai children's medical center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jun Yan

CONTACT

13817651474; yanjun@basetherapeutics.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Dose escalation study

Study Record Dates

• First Submitted: June 7, 2024
• First Posted: August 7, 2024
• Study Start: July 1, 2024
• Primary Completion: June 1, 2025
• Study Completion: June 1, 2025
• Last Updated: August 7, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)