Leukaemia is cancer of the white blood cells, which are the infection-fighting cells of the immune system. Blood cells form in the bone marrow. In leukaemia, 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 leukaemia (AML), there are too many of a specific type of white blood cell called a myeloblast.
AML is the most common type of acute leukaemia in adults. This type of cancer usually progresses 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.
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
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
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 leukaemia (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, i.e. originating from T cells that have been taken from healthy donors. TALENS are used to disable the TCRαβ gene T cells use to recognise ’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: Apr. 8, 2022
Source: United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) www.gov.uk