Clinical Trial

Disease: Acute Myeloid Leukaemia, AML, (NCT04106076)

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:
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 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:

Principal Investigator:Ghulam Mufti,Pr Kings college London NHS Foundation Trust

Sponsor:

Cellectis S.A.

Partners:
Locations:
Study start:
Jul. 11, 2019
Enrollment:
0 participants
Gene editing method:
TALENs
Type of edit:
Gene knock-out
Gene:
T Cell Receptor alpha and beta locus TCRαβ, CD52 molecule
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT04106076
Safety updates:

(13-12-2022) Cellectis Announces Positive Preliminary Clinical Data for UCART22 in ALL and UCART123 in AML

(15-01-2020) First Patient Dosed with Cellectis’ New Allogeneic UCART123 Product Candidate for Relapsed/Refractory Acute Myeloid Leukemia

Other links:

LinkAcute Myeloid Leukaemia Disease InformationLinkAcute Myeloid Leukaemia FrequencyLinkWhat is UCART123?LinkStraightforward Generation of Ultrapure Off-the-Shelf Allogeneic CAR-T CellsLinkPre-clinical Studies of Allogeneic Anti-CD123 CAR-T Cells for the Treatment of Blastic plasmacytoid dendritic cell neoplasm (BPDCN)LinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem Qasim

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

Status: Terminated

Description

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: Dec. 28, 2023
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