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

NCT ID: NCT05519384

Last Updated: 2022-11-28

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-09-14
• Study Completion Date: 2025-12-31

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.

Detailed Description

The Main components of JK500 cell injection are regenerative natural killer (NK) cells derived from human embryonic stem cells and 0.9% sodium chloride solution.

Conditions

Acute Myeloid Leukemia, Childhood; Relapsed Leukemia; Refractory Leukemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

JK500 cell injection

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.

Group Type: EXPERIMENTAL

JK500 cell injection，cyclophosphamide，Fludarabine

Intervention Type: 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.

Interventions

JK500 cell injection，cyclophosphamide，Fludarabine

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Age ≤18, male or female;

2. Patients diagnosed as acute myeloid leukemia (AML) according to the revised World Health Organization (WHO) criteria in 2016;

3. Patients who failed to achieve CR after two standard-dose induction therapy or had recurrence within six months after CR;

4. 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.

Exclusion Criteria

1. Acute promyelocytic leukemia, chronic myelocytic leukemia, acute mixed-cell leukemia or known central nervous system leukemia;

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

3. 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;

4. Presence of active systemic infection; Participated in a drug trial within the past 4 weeks;

5. 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;

6. children with liver and kidney dysfunction, including:

1. Serum creatinine >2× upper limit of normal reference value;

2. Serum total bilirubin > 2× upper limit of normal reference value;

3. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) > 2× upper limit of normal reference values

7. Children who have used live attenuated vaccine 4 weeks before administration or plan to use live attenuated vaccine within 6 months after administration;

8. 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.

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bejing Institute for Stem Cell and Regenerative Medicine

UNKNOWN

Sponsor Role: collaborator

Institute for Stem cell and Regeneration, Chinese Academy of Sciences

UNKNOWN

Sponsor Role: collaborator

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Xiaofan Zhu

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC

Locations

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

Tianjin, Tianjin Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Min Ruan, MD

Role: CONTACT

ruanmin@ihcams.ac.cn

+8613602177144

Facility Contacts

Xiaofan Zhu, MD

Role: primary

xfzhu@ihcams.ac.cn

86-21-23909001

Other Identifiers

ISCRNK01001

Identifier Type: -

Identifier Source: org_study_id