Clinical Trial

Disease: Relapsed or Refractory Acute Myeloid Leukaemia, AML, (NCT03190278)

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 AML refers to when a  patient had active treatment, went off treatment and then the disease came back. 

Refractory AML refers to as 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 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: Dec. 28, 2023
Search CRISPR Medicine