Clinical trial NCT05519384: 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
Overall status: RECRUITING
Phase: EARLY_PHASE1
Conditions: Acute Myeloid Leukemia, Childhood
Relapsed Leukemia
Refractory Leukemia
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
Collaborators: name: Bejing Institute for Stem Cell and Regenerative Medicine
name: Institute for Stem cell and Regeneration, Chinese Academy of Sciences
First posted: 2022-08-29
Enrollment: 12
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model 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.
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Dose-limiting toxicity (DLT)
timeFrame: 28 days
Secondary outcomes:
measure: Objective Response Rate (ORR)
timeFrame: 24 months
measure: Minimal Residual Disease (MRD)
timeFrame: 24 months
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Department of Pediatrics, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences
state: Tianjin
Study officials:
affiliation: Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC
name: Xiaofan Zhu
role: PRINCIPAL_INVESTIGATOR
References: