Clinical Trial

Disease: Acute Myeloid Leukaemia, AML, (NCT04106076)

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 Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of Multiple Infusions of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor) in Patients With Adverse Genetic Risk Acute Myeloid Leukaemia
Study start:
Jul. 11, 2019
0 participants
Gene editing method:
Type of edit:
Gene knock-out
T Cell Receptor alpha and beta locus TCRαβ, CD52 molecule
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
UCART123 consists of 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: Terminated


This is a Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). The purpose of this study is to evaluate the safety and clinical activity of multiple infusions of UCART123 and to 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: Dose escalation:

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

Last updated: Jun. 26, 2023
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