Clinical Trial

Disease: Acute Myeloid Leukemia, AML, (NCT03190278)

Disease info:

Leukemia is cancer of the white blood cells. White blood cells help your body fight infection. White blood cells help your body fight infection. Your blood cells form in your bone marrow. In leukemia, however, the bone marrow produces abnormal white blood cells. These cells crowd out the healthy blood cells, making it hard for blood to do its work. In acute myeloid leukemia (AML), there are too many of a specific type of white blood cell called a myeloblast.

AML is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. 

The number of new cases of acute myeloid leukemia was 4.3 per 100,000 men and women per year. The number of deaths was 2.8 per 100,000 men and women per year. These rates are age-adjusted and based on 2012-2016 cases and deaths.
Official title:
Phase I, Open Label Dose Escalation and Dose-Expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Relapsed/Refractory Acute Myeloid Leukemia



Cellectis Central

Phone: 1-347-752-4044



United States, California

University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States, 94143


United States, Florida

Sylvester Comprehensive Cancer CenterR, Miami, Florida, United States, 33136

H. Lee Moffitt Cancer Center & Research Institute,Tampa, Florida, United States, 33612


United States, Illinois

Northwestern University, Chicago, Illinois, United States, 60201


United States, Massachusetts

Dana-Farber Cancer Institute, Boston, Massachusetts, United States, 02215


United States, New York

Roswell Park Cancer Institute, Buffalo, New York, United States, 14263

Weill Medical College of Cornell University, New York, New York, United States, 10021


United States, Pennsylvania

University of Pennsylvania - Abramson Cancer Center, Philadelphia, Pennsylvania, United States, 19104


United States, Texas

MD Anderson Cancer Center, Houston, Texas, United States, 77030

Study start:
Jun. 19, 2017
65 participants
Gene editing method:
Type of edit:
Gene knock-out
T-cell receptor alpha constant (TRAC) and CD52
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
UCART123 is Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor. In the first engineering step, genes are added to the T-cell genome with lentiviral vectors. In the second T-cell engineering step transfection is achieved with electroporation.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


Phase I, first-in-human, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of UCART targeting CD123 in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of one infusion of UCART123 and determine the Maximum Tolerated Dose (MTD).

UCART is Universal CAR-T cells. That is T Cells that have been taken from healthy donors (not from patients themselves). TALENS are used to disable the TCRαβ gene T cells use to recognize ’self’ to prevent them from attacking the host (graft vs host).

Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor.

Experimental: Part 1: Dose Escalation

Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified

Dose Expansion: UCART123 administered at the selected dose determined from the dose escalation phase

Last updated: Jun. 26, 2023
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