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

Not Applicable

Withdrawn

• Conditions: Acute Myeloid Leukemia
• Interventions: Biological: CLL1/+CD33 CAR-T

• Registration Number: NCT05943314
• Lead Sponsor: Guangzhou Bio-gene Technology Co., Ltd

Brief Summary

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

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 Timeline

• Study First Submitted: 2023-06-09
• Study First Submitted QC: 2023-07-10
• Study Start: 2023-07-12
• Study First Posted: 2023-07-13
• Primary Completion: 2023-08-28
• Study Completion: 2023-08-28
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-31
• Last Update Posted: 2023-11-02

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. The patient or his/her legal guardian volunteers for the trial and signs an informed consent form;
2. Age range 1-18 years;
3. 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 ;
4. 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% ;
5. The blood flow of peripheral superficial vein was unobstructed, which could meet the demands of intravenous drip and mononuclear cell collection;
6. ECOG score was 0-2.

Exclusion Criteria

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CLL1/+CD33 CAR-T	CLL1/+CD33 CAR-T	The target dose range for subjects was set to be 1.00\~2.50x10\^6/kg CAR-positive T cells.

Primary Outcome Measures

Name	Time	Method
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
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 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

Name	Time	Method
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.
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.
MRD negative rate	Up to 12 months after CLL1/+CD33 CAR-T infusion	The rate of MRD negative subjects was determined by flow cytometry.

Trial Locations

Locations (1)

Fujian Provincial Children's Hospital; 🇨🇳 Fuzhou, Fujian, China