Clinical Trial

Disease: Acute Myeloid Leukaemia, AML, (2018-001018-14)

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.

 

Frequency:
An estimated 22,010 people will be diagnosed with AML in the United States in 2025.
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
Who:

Contact

Phone: +33181691713

E-mail: clinicaltrialinfo@cellectis.com

Sponsor:

Cellectis S.A.

Partners:
Locations:

France

8 rue de la Croix Jarry, Paris, 75013, France

 

Study start:
Apr. 2, 2019
Enrollment:
18 participants in the EEA
Gene editing method:
TALENs
Type of edit:
Gene knock-out
Gene:
CD123 or IL3RA interleukin 3 receptor subunit alpha, T Cell Receptor (TCR)
Delivery method:
Electroporation and Lentiviral - Ex-vivo
Trial link:
https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-001018-14/GB
Safety updates:

(28-05-2018) Approval of UCART123 Amendment in AML to Accelerate Clinical Development

Other links:

LinkAcute Myeloid Leukemia Disease InformationLinkAcute Myeloid Leukemia FrequencyLinkWhat is UCART123?LinkStraightforward Generation of Ultrapure Off-the-Shelf Allogeneic CAR-T CellsLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem Qasim

Note:
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.
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated

Description

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: May. 20, 2025
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