Functionally Optimized CD33 CAR-T for AML, Phase 1 Trial NCT07538713
ClinicalTrials.gov Studies
Summary
NIH registers Phase 1 trial NCT07538713 evaluating functionally optimized CD33 CAR-T (FO33 CAR-T) cell therapy in patients with recurrent/refractory acute myeloid leukemia (AML). The single-arm study will assess safety, tolerability, and preliminary efficacy of the CD33-targeted therapy. CD33 is expressed in leukemia cells from over 80% of AML patients and was selected as the target antigen due to higher expression levels across AML subtypes compared to alternative targets such as CLL-1 and CD123.
What changed
NIH has registered a new Phase 1 clinical trial (NCT07538713) for a functionally optimized CD33 CAR-T (FO33 CAR-T) cell therapy in patients with recurrent/refractory acute myeloid leukemia. The trial will evaluate safety, tolerability, and preliminary efficacy of the CD33-targeted therapy, which was developed to address limitations of conventional CD33 CAR-T including suboptimal efficacy, toxicity, and insufficient expansion observed in prior clinical trials.\n\nHealthcare providers and pharmaceutical companies involved in cell therapy development should note that this trial addresses a significant gap in AML treatment, as relapsed/refractory AML currently lacks approved CAR-T products. The CD33 target was chosen because it is expressed in over 80% of AML patients and exhibits higher expression levels across AML subtypes, potentially reducing relapse risk from tumor antigen escape.
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Apr 21, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Functionally Optimized CD33 CAR-T Cell Therapy Targeting Recurrent/Refractory Acute Myeloid Leukemia
Phase 1 NCT07538713 Kind: PHASE1 Apr 20, 2026
Abstract
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.
Conditions: CAR T Cell Therapy, CD33 Positive Acute Myelogenous Leukemia
Interventions: Functionally optimized CD33 CAR-T
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