Clinical trial NCT05943314: Clinical Study on Safety and Efficacy of Anti-CLL1 /+CD33 CAR T Cells in the Treatment of Acute Myeloid Leukemia
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Acute Myeloid Leukemia
Keywords: AML
CLL1
CD33
Lead sponsor: Guangzhou Bio-gene Technology Co., Ltd
Sponsor class: INDUSTRY
First posted: 2023-07-13
Enrollment: 0
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Changes in cytokine level after CLL1/+CD33 CAR-T infusion
timeFrame: CAR T cell infusion before and 12 months after infusion
measure: The change characteristics of chimeric antigen receptor(CAR)-T cell number in patients after infusion.
timeFrame: CAR T cell infusion before and 12 months after infusion
measure: The change characteristics of chimeric antigen receptor(CAR)-T cell copy number in patients after infusion.
timeFrame: CAR T cell infusion before and 12 months after infusion
Secondary outcomes:
measure: Event-free survival
timeFrame: Up to 12 months after CLL1/+CD33 CAR-T infusion
measure: Overall survival
timeFrame: Up to 12 months after CLL1/+CD33 CAR-T infusion
measure: Duration of Overall Response
timeFrame: Up to 12 months after CLL1/+CD33 CAR-T infusion
measure: MRD negative rate
timeFrame: Up to 12 months after CLL1/+CD33 CAR-T infusion
Other outcomes:
Locations:
city: Fuzhou
country: China
facility: Fujian Provincial Children's Hospital
state: Fujian
Study officials:
affiliation: Children's Hospital of Fujian Province
name: Hui Zhang, doctor
role: PRINCIPAL_INVESTIGATOR
References: