Safety and Preliminary Efficacy of JK500 Cell Injection in Relapsed/Refractory Pediatric Acute Myeloid Leukemia

NCT05519384 | Unknown Early Phase 1

• 1 other identifier: ISCRNK01001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a prospective, single-center, single-arm exploratory clinical study, aiming to complete the preliminary clinical observation of 12 children with relapsed/refractory acute myeloid leukemia treated with JK500 cell injection to evaluate the safety of clinical infusion and the initial efficacy of JK500 cell injection in the treatment of children with relapsed/refractory acute myeloid leukemia.

Conditions

Acute Myeloid Leukemia, Childhood; Relapsed Leukemia; Refractory Leukemia

Outcome Measures

Primary Outcomes (1)

• Dose-limiting toxicity (DLT)

  DLT evaluation is defined as adverse events or laboratory abnormalities that occur within 4 weeks after investigational drug administration, are unrelated to external causes such as progressive disease, concomitant disease, and concomitant medications, including hematologic and non-hematologic adverse events (grade according to NCI CTCAE 5.0).

  28 days

Secondary Outcomes (2)

• Objective Response Rate (ORR)

  24 months

• Minimal Residual Disease (MRD)

  24 months

Study Arms (1)

JK500 cell injection

EXPERIMENTAL

They were divided into three dose groups: 10\^6, 5x10\^6 and 3x10\^7 JK500 cells injection /kg/ time, three times a week, a total of 6 times. According to the principle of dose escalation, 3 patients were assigned to each dose group. After completing the treatment of 3 patients in each dose group, the Safety Review Committee (SRC) discussed whether to enter the next dose group.

Drug: JK500 cell injection，cyclophosphamide，Fludarabine

Interventions

JK500 cell injection，cyclophosphamide，Fludarabine DRUG

After enrollment, patients received JK500 cell injection infusion after reinduction therapy. Pretreatment regimen before cell infusion: intravenous infusion of cyclophosphamide 60mg/kg day -7, intravenous infusion of fludarabine 25mg/㎡/day day -6 to -2 days. In order to activate and expand circulating NK cells, on the day of each infusion of JK500 cells, the patients were first subcutaneously injected with 100WU/ m2 of interleukin (IL)-2 0.5h-1h in advance, for a total of 6 doses.

JK500 cell injection

Eligibility Criteria

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

You may qualify if:

• Age ≤18, male or female;
• Patients diagnosed as acute myeloid leukemia (AML) according to the revised World Health Organization (WHO) criteria in 2016;
• Patients who failed to achieve CR after two standard-dose induction therapy or had recurrence within six months after CR;
• The subject or the guardian of the subject must fully understand the purpose, nature, method and possible adverse reactions of the test, agree the subject as the subject, and sign the informed consent.

You may not qualify if:

• Acute promyelocytic leukemia, chronic myelocytic leukemia, acute mixed-cell leukemia or known central nervous system leukemia;
• AML associated with congenital syndromes such as Down syndrome, Fanconi's anemia, Bloom's syndrome, Koch's syndrome, or congenital aplastic anemia;
• The subjects have active virus infection, and during the screening period, the serum virology test is performed, and the human immunodeficiency virus (HIV) antibody is positive, hepatitis B surface antigen or E antigen is positive, hepatitis C antibody is positive, or treponema pallidum antibody is positive;
• Presence of active systemic infection; Participated in a drug trial within the past 4 weeks;
• Patients who suffered from a clinically significant disease within 28 days before receiving the study product or underwent a major surgical operation within 28 days before receiving the study product, or are expected to need major surgery during the trial;
• children with liver and kidney dysfunction, including:
• Serum creatinine \>2× upper limit of normal reference value;
• Serum total bilirubin \> 2× upper limit of normal reference value;
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \> 2× upper limit of normal reference values
• Children who have used live attenuated vaccine 4 weeks before administration or plan to use live attenuated vaccine within 6 months after administration;
• Have any other conditions that may cause the subject to be unable to complete the study or present a significant risk to the subject in the opinion of the Investigator.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Bejing Institute for Stem Cell and Regenerative Medicine: collaborator
• Institute for Stem cell and Regeneration, Chinese Academy of Sciences: collaborator

Study Sites (1)

Department of Pediatrics, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Leukemia

Condition Hierarchy (Ancestors)

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

Study Officials

• Xiaofan Zhu

  Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Min Ruan, MD

CONTACT

+8613602177144; ruanmin@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: They were divided into three dose groups: 10\^6, 5x10\^6 and 3x10\^7 JK500 cells injection /kg/ time, three times a week, a total of 6 times. According to the principle of dose escalation, 3 patients were assigned to each dose group. After completing the treatment of 3 patients in each dose group, the Safety Review Committee (SRC) discussed whether to enter the next dose group.

Study Record Dates

• First Submitted: August 25, 2022
• First Posted: August 29, 2022
• Study Start: September 14, 2022
• Primary Completion: November 1, 2023
• Study Completion: December 1, 2025
• Last Updated: November 28, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)