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

Primary Purpose

Acute Myeloid Leukemia, Childhood, Relapsed Leukemia, Refractory Leukemia

Status

Recruiting

Phase

Early Phase 1

Locations

China

Study Type

Interventional

Intervention

JK500 cell injection，cyclophosphamide，Fludarabine

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia, Childhood

Eligibility Criteria

undefined - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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:
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
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.

Sites / Locations

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

JK500 cell injection

Arm Description

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.

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

Objective Response Rate (ORR)

ORR was defined as complete response (CR)+ incomplete bone marrow recovery (CRi) + partial response (PR). Treatment response will be assessed periodically until the end of treatment and up to 24 months after the first injection.

Minimal Residual Disease (MRD)

To observe the MRD status in bone marrow.

Full Information

NCT ID

NCT05519384

First Posted

August 25, 2022

Last Updated

November 23, 2022

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Collaborators

Bejing Institute for Stem Cell and Regenerative Medicine, Institute for Stem cell and Regeneration, Chinese Academy of Sciences

1. Study Identification

Unique Protocol Identification Number

NCT05519384

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2022

Overall Recruitment Status

Recruiting

Study Start Date

September 14, 2022 (Actual)

Primary Completion Date

November 2023 (Anticipated)

Study Completion Date

December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Collaborators

Bejing Institute for Stem Cell and Regenerative Medicine, Institute for Stem cell and Regeneration, Chinese Academy of Sciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acute Myeloid Leukemia, Childhood, Relapsed Leukemia, Refractory Leukemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Single Group Assignment

Model Description

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

JK500 cell injection

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

JK500 cell injection，cyclophosphamide，Fludarabine

Intervention Description

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.

Primary Outcome Measure Information:

Title

Dose-limiting toxicity (DLT)

Description

Time Frame

28 days

Secondary Outcome Measure Information:

Title

Objective Response Rate (ORR)

Description

Time Frame

24 months

Title

Minimal Residual Disease (MRD)

Description

To observe the MRD status in bone marrow.

Time Frame

24 months

10. Eligibility

Sex

All

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Min Ruan, MD

Phone

+8613602177144

ruanmin@ihcams.ac.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Xiaofan Zhu

Organizational Affiliation

Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC

Official's Role

Principal Investigator

Facility Information:

Facility Name

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

City

Tianjin

State/Province

Tianjin

ZIP/Postal Code

300020

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Xiaofan Zhu, MD

Phone

86-21-23909001

xfzhu@ihcams.ac.cn

First Name & Middle Initial & Last Name & Degree

Min Ruan, MD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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