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

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

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

Study Overview

• Status: Recruiting
• Conditions: Relapsed/Refractory Acute Myeloid Leukemia
• Intervention / Treatment: Drug: NK520

Detailed Description

This open label, single-arm study aims to evaluate the efficacy and safety of allogenic NK cells in subjects with relapsed/refractory acute myeloid leukemia. Allogenic NK cells will be infused once a week. After infusion, the investigators will observe the characteristics of dose limited toxicity (DLT), and determine the maximum tolerable dose(MTD). To provide basis for the dosage and treatment plan of cell products in follow-up clinical trials.

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Jun Yan; Phone Number: 13817651474; Email: yanjun@basetherapeutics.com

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 201202
      • Recruiting
      • Shanghai Pudong Hospital
      • Contact: Minghua Yu; Phone Number: 18017821601; Email: minghuamd@fudan.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Participants must be between 18 and 75 years;

2. Diagnostic Criteria:

   Meet the 2016 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:

   1. 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;
   2. 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. Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2;
4. Expected survival of at least 12 weeks;
5. Normal Organ Function.

Exclusion Criteria:

1. Acute promyelocytic leukemia;
2. Severe bleeding tendency or coagulation disorders, or currently receiving thrombolytic therapy;
3. Active tuberculosis (TB), currently undergoing anti-TB treatment, or treated for TB within 1 year prior to the study;
4. HIV-infected individuals, or known active syphilis infection;
5. Use of immunosuppressive drugs within 1 week before the first dose, excluding topical, inhaled, or other locally administered glucocorticoids, or physiologic doses of systemic glucocorticoids (not exceeding 10 mg/day prednisone equivalent) for allergic reactions or for managing respiratory distress from asthma, COPD, etc;
6. Receipt of live attenuated vaccines within 2 weeks before the first dose or planned during the study;
7. Participation in another clinical trial and receipt of investigational drug within 4 weeks prior to the first dose;
8. Receipt of immune-modulating drugs (including thymosin, interferons, except for local use to control pleural or ascitic fluid) within 2 weeks prior to the first dose;

9. At screening, hepatitis B or C viral tests positive according to either:

   • HBsAg positive with serum HBV-DNA titer ≥1×10^3 copies/mL or above normal limits;
   • HCV antibody positive;
10. Any other condition or situation in which the investigator deems the patient unsuitable for participation in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Group A(low-dose group); NK520: 3×10^7NK/kg	Drug: NK520; The number of NK520 cell infused for each dosing will be calculated based on body weight of subject. NK520 should be administered through intravenous infusion once a week, for a total of four times.; Other Names: genetic modified NK cell
Experimental: Group B(medium-dose group); NK520: 6×10^7NK/kg	Drug: NK520; The number of NK520 cell infused for each dosing will be calculated based on body weight of subject. NK520 should be administered through intravenous infusion once a week, for a total of four times.; Other Names: genetic modified NK cell
Experimental: Group C(high-dose group); NK520: 9×10^7NK/kg	Drug: NK520; The number of NK520 cell infused for each dosing will be calculated based on body weight of subject. NK520 should be administered through intravenous infusion once a week, for a total of four times.; Other Names: genetic modified NK cell

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose-Limiting Toxicity	To evaluate the DLT during NK520 treatment	From the first dose of NK520 to 4 weeks after last infusion of NK520
Complete Response Rate (CRR)	Effectiveness Metrics	From the date of first infusion of NK520 up to 104 weeks.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate (ORR)	Effectiveness Metrics	From the date of first infusion of NK520 up to 104 weeks.
Event-Free Survival (EFS)	Effectiveness Metrics	From date of enrollment up to 104 weeks, or date of progression, or date of death, whichever came first.
Overall Survival (OS)	Effectiveness Metrics	From date of enrollment up to 104 weeks or date of death, whichever came first.
Duration of Response (DOR)	Effectiveness Metrics	From the date of enrollment up to 104 weeks

Collaborators and Investigators

• Sponsor: Base Therapeutics (Shanghai) Co., Ltd.
• Collaborators: Shanghai Pudong Hospital
• Investigators: Principal Investigator: zhiguo Long, Shanghai Pudong Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: June 7, 2024
• First Submitted That Met QC Criteria: August 5, 2024
• First Posted (Actual): August 7, 2024

Study Record Updates

• Last Update Posted (Actual): August 7, 2024
• Last Update Submitted That Met QC Criteria: August 5, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: NK520-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No