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NCT07538713

PHASE1

Functionally Optimized CD33 CAR-T Cell Therapy Targeting Recurrent/Refractory Acute Myeloid Leukemia

Sponsor: Qi deng

View on ClinicalTrials.gov

Summary

Relapsed/refractory acute myeloid leukemia (AML) currently lacks effective CAR-T therapy products due to the absence of specific target antigens. Most AML antigens are frequently expressed in normal hematopoietic stem/progenitor cells (HSPCs) or healthy organ tissues, thereby increasing the risks of target toxicity and non-neoplastic toxicity. CD33 is expressed in leukemia cells from over 80% of AML patients. Compared to other targets such as CLL-1 and CD123, CD33 typically exhibits higher expression levels across various AML subtypes, reducing the risk of treatment failure and relapse caused by tumor antigen escape, making it an ideal therapeutic target for AML. However, conventional CD33 CAR-T therapies have demonstrated suboptimal efficacy in clinical trials and face challenges such as toxicity and insufficient expansion. To further investigate the safety and efficacy of CAR-T therapy for AML, our center has initiated a clinical study on functionally optimized CD33 CAR-T (FO33 CAR-T) cell therapy for refractory/refractory AML.

Official title: Clinical Study on the Efficacy and Safety of Functionally Optimized CD33 CAR-T Cells (FO33 CAR-T) Therapy Targeting CD33-Positive Recurrent/Refractory Acute Myeloid Leukemia

Key Details

Gender

All

Age Range

14 Years - 75 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-06-01

Completion Date

2028-05-31

Last Updated

2026-04-20

Healthy Volunteers

Conditions

CAR T Cell Therapy CD33 Positive Acute Myelogenous Leukemia

Interventions

BIOLOGICAL

Functionally optimized CD33 CAR-T

Functionally optimized CD33 CAR-T intravenous infusion

Inclusion Criteria: * 1\. Subjects diagnosed with refractory/recurrent acute myeloid leukemia (excluding M3) who meet any of the following criteria: 1. Relapse: Recurrence of leukemia cells in peripheral blood or ≥5% blast cells in bone marrow after complete remission (CR) of AML (excluding other causes such as bone marrow regeneration following consolidation chemotherapy), or extramedullary leukemia infiltration. 2. Refractory: First-time cases unresponsive to two cycles of standard therapy; relapse within 12 months after consolidation therapy following CR; relapse after 12 months without response to conventional chemotherapy; two or more relapses; persistent extramedullary leukemia. 2\. During enrollment screening, bone marrow flow cytometry must demonstrate a CD33+ expression rate of ≥80% in leukemia cells and/or pathological immunohistochemical confirmation of CD33+ extramedullary lesions; 3. Estimated survival duration exceeding 3 months as of the date of informed consent signing; 4. Participants with Eastern Cooperative Oncology Group (ECOG) physical status scores ranging from 0 to 2; 5. Age range of 14 years ≤ ≤ 75 years, inclusive, with no gender restriction; 6. Hemoglobin (HGB) level ≥70 g/L with transfusion capability; 7. Liver/kidney function and cardiopulmonary function meeting the following criteria:  1. Creatinine ≤1.5×ULN; 2. Left ventricular ejection fraction ≥50%; 3. Blood oxygen saturation\>90%; 4. Total bilirubin ≤1.5×ULN; ALT and AST ≤2.5×ULN; 8. Acceptance of autologous CART cells with peripheral blood tumor burden ≤ 30%; 9. The subject or guardian understands and signs the informed consent form. Exclusion Criteria: * 1\. Presence of one of the following cardiac criteria: atrial fibrillation; myocardial infarction (MI) within the past 12 months; prolonged QT syndrome or secondary QT prolongation as determined by the investigator. Echocardiographic left ventricular systolic fraction (LVSF) \<30% or left ventricular ejection fraction (LVEF) \<50%; clinically significant pericardial effusion; New York Heart Association (NYHA) class III or IV heart failure (confirmed by echocardiography within 12 months after treatment). 2\. Active graft-versus-host disease (GVHD); 3. History of severe pulmonary dysfunction; 4. Concurrent other progressive malignancies; 5. Concurrent severe or persistent infections that cannot be effectively controlled; 6. Concurrent severe autoimmune diseases or congenital immunodeficiency; 7. Active hepatitis (HBV-DNA ≥ 500 IU/ml with abnormal liver function or HCV antibody \[HCV-Ab\] positivity, HCV-RNA exceeding the detection limit of analytical methods with abnormal liver function); 8. Human immunodeficiency virus (HIV) infection or syphilis infection; 9. History of severe allergic reactions to biological products (including antibiotics); 10. Presence of central nervous system disorders such as uncontrolled epilepsy, cerebrovascular ischemia/hemorrhage, dementia, or cerebellar diseases; 11. Female patients in pregnancy or lactation, or planning pregnancy within 12 months; 12. Situations where investigators consider may increase subject risk or interfere with trial outcomes.