Clinical Trial

Disease: Relapsed or Refractory Acute Myeloid Leukemia, AML, (NCT05984199)

Disease info:

Leukaemia is cancer of the white blood cells which are responsible for fighting infection. In leukaemia, the bone marrow produces abnormal levels of white blood cells. Acute myeloid leukaemia (AML) starts in the bone marrow but often quickly spreads into the bloodstream. AML sometimes spreads to other parts of the body such as the lymph nodes, liver, central nervous system and testicles. 

Commonly, AML develops from cells other than lymphocytes that would normally develop into white blood cells. The World Health Organization (WHO) divides AML into several subtypes based off genetic association. AML is also known as acute myelocytic leukaemia, acute myelogenous leukaemia, acute granulocytic leukaemia, and acute non-lymphocytic leukaemia.

Relapsed refers to when a patient has received active treatment, went off treatment and then the disease came back, whereas refractory refers to disease that is progressing despite active treatment.

The American Cancer Society estimates approximately 20,380 cases of AML will be diagnosed in the U.S. in 2023, and approximately 11,310 deaths. AML is predominantly found in adults and is one of the most common types of leukaemia in adults.
Official title:
Phase 1/2 Study of Donor-Derived Anti-CD33 Chimeric Antigen Receptor Expressing T Cells (VCAR33) in Patients With Relapsed or Refractory Acute Myeloid Leukemia After Allogeneic Hematopoietic Cell Transplantation


Name: Jennifer Whangbo, MD, PhD

Phone Number: 617-655-6580




United States, California

University of California San Diego Moores Cancer Center, La Jolla, California, United States, 92093

Stanford Cancer Institute, Stanford, California, United States, 94305

United States, Florida
Miami Cancer Institute, Miami, Florida, United States, 33176

United States, Kansas
The University of Kansas Cancer Center, Fairway, Kansas, United States, 66205

United States, Michigan
University of Michigan Health, Ann Arbor, Michigan, United States, 48109

Karmanos Cancer Institute, Detroit, Michigan, United States, 48201

United States, Missouri
Washington University School of Medicine Siteman Cancer Center, Saint Louis, Missouri, United States, 63110

United States, New Jersey
John Theurer Cancer Center at Hackensack University Medical Center, Hackensack, New Jersey, United States, 07601

United States, Ohio
University Hospitals Seidman Cancer Center, Cleveland, Ohio, United States, 44106

United States, Utah
University of Utah Huntsman Cancer Institute, Salt Lake City, Utah, United States, 84112

Study start:
Dec. 1, 2023
24 participants
Gene editing method:
Type of edit:
Gene KO
Delivery method:
Undisclosed - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


CD33 is a preferential target for AML CAR T cell therapy due to its surface expression on the majority (>80%) of AML blasts and due to the extensive prior clinical experience demonstrating safety and efficacy of targeting CD33 with Mylotarg (gemtuzumab ozogamicin). VCAR33 is being developed as a potential new treatment for patients with relapsed/refractory (R/R) AML after alloHCT. In this Phase 1/2 trial, the safety and efficacy of lentiviral-transduced CD33-directed CAR T cells (VCAR33) generated from the patient's prior allogeneic stem cell donor will be tested. It is hypothesized that CAR T cell production from healthy donors will not only eliminate delays in production due to lymphopenia but also reduce concerns for suboptimal T cell function from exposure to systemic immunosuppression or chemotherapeutic agents. Approximately 24 eligible patients with R/R AML after alloHCT will be enrolled in one of two separate arms based on disease burden (morphologic disease versus measurable residual disease (MRD ) positive). The maximum tolerated dose of VCAR33 will be determined using a 3+3 trial design within each arm. Dose escalation can only occur after a minimum of 3 patients have completed the dose-limiting toxicity (DLT) observation period.

Last updated: Apr. 20, 2024
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