Clinical Study on Safety and Efficacy of Anti-CLL1 /+CD33 CAR T Cells in the Treatment of Acute Myeloid Leukemia

Sponsor

Guangzhou Bio-gene Technology Co., Ltd (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05943314

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a single-center, single-arm, open, intravenous drug administration of the safety and efficacy of clinical study.

Condition or Disease	Intervention/Treatment	Phase
Acute Myeloid Leukemia	Biological: CLL1/+CD33 CAR-T	N/A

Detailed Description

The primary objective of the clinical trial was to evaluate the safety and efficacy of single dose infusion of anti-CLL1 /+CD33 CAR T cells in patients with refractory/recurrent acute myeloid leukemia. A total of about 5 patients with refractory/recurrent acute myeloid leukemia were enrolled in this study, and the target dose range was 1.00~2.50x10^6/kgCAR-positive T cells.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

5 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Safety and Efficacy of Anti-CLL1 /+CD33 CAR T Cells in Refractory/Recurrent Acute Myeloid Leukemia: a Single-arm, Non-blind Clinical Study

Anticipated Study Start Date :

Jul 1, 2023

Anticipated Primary Completion Date :

Jun 1, 2025

Anticipated Study Completion Date :

Jun 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: CLL1/+CD33 CAR-T The target dose range for subjects was set to be 1.00~2.50x10^6/kg CAR-positive T cells.	Biological: CLL1/+CD33 CAR-T CLL1/+CD33 CAR T is a type of CAR T cell therapy for patients with treating/relapsed acute myeloid leukemia.

Arm

Intervention/Treatment

Experimental: CLL1/+CD33 CAR-T

The target dose range for subjects was set to be 1.00~2.50x10^6/kg CAR-positive T cells.

Biological: CLL1/+CD33 CAR-T

CLL1/+CD33 CAR T is a type of CAR T cell therapy for patients with treating/relapsed acute myeloid leukemia.

Outcome Measures

Primary Outcome Measures

Changes in cytokine level after CLL1/+CD33 CAR-T infusion [CAR T cell infusion before and 12 months after infusion]
Calculate the change of cytokine level in peripheral blood by flow cytometry after CAR-T infusion.
The change characteristics of chimeric antigen receptor(CAR)-T cell number in patients after infusion. [CAR T cell infusion before and 12 months after infusion]
Track CAR-T cells expansion in patients after infusion by flow cytometry
The change characteristics of chimeric antigen receptor(CAR)-T cell copy number in patients after infusion. [CAR T cell infusion before and 12 months after infusion]
Track CAR-T cells expansion in patients after infusion by Real-time Quantitative Polymerase Chain Reaction（qPCR）

Secondary Outcome Measures

Event-free survival [Up to 12 months after CLL1/+CD33 CAR-T infusion]
Counting from the beginning of cell transfusion until treatment failure, recurrence, or death (various causes). Subjects without any of these events were counted up to the last follow-up examination date. For patients without CR or CRi, EFS is calculated from the beginning of cell transfusion until disease progression or death. Based on the initial event.
Overall survival [Up to 12 months after CLL1/+CD33 CAR-T infusion]
Death from any cause from the beginning of cell transfusion
Duration of Overall Response [Up to 12 months after CLL1/+CD33 CAR-T infusion]
The time from the start of cell infusion when CR or PR is first achieved to disease progression.
MRD negative rate [Up to 12 months after CLL1/+CD33 CAR-T infusion]
The rate of MRD negative subjects was determined by flow cytometry.

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

The patient or his/her legal guardian volunteers for the trial and signs an informed consent form;
Age range 1-18 years;
Acute myeloid leukemia (AML) with CLL1 and CD33 markers (including secondary patients) was diagnosed by pathology, histology and flow cytometry, or complete hematologic remission could not be achieved after 1 course of chemotherapy for hematologic relapse after drug withdrawal ;
The main organ functions of the patients were good: (1) liver function: ALT/AST < 3 times the upper limit of normal (ULN) and bilirubin ≤34.2 μmol/l; (2) renal function: creatinine < 220 μmol/l; (3) lung function: oxygen saturation ≥95% ; (4) cardiac function: left ventricular ejection fraction (LVEF)≥40% ;
The blood flow of peripheral superficial vein was unobstructed, which could meet the demands of intravenous drip and mononuclear cell collection;
ECOG score was 0-2.

Exclusion Criteria:

The patients had uncontrollable infectious diseases within 4 weeks before the enrollment;
Active hepatitis B/C virus;
HIV infection, treponema syphilis positive patients;
Pathological diagnosis of primary tumors other than acute myeloid leukemia;
Suffering from serious autoimmune diseases or immunodeficiency diseases;
The patient is allergic to antibodies or cytokines and other macromolecular biological drugs;
Pregnant or lactating women;
Patients who were considered ineligible for study for other reasons.