Clinical Trial

Disease: Acute Myeloid Leukemia, AML, (NCT05066165)

Disease info:

Leukaemia is cancer of the white blood cells. White blood cells help your body to fight infection. Blood cells form in the bone marrow. In leukaemia, the bone marrow produces abnormal white blood cells, which outnumber healthy blood cells, thus compromising the function of blood. 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 gets advances quickly if it is not treated. 

The estimated number of new cases of acute myeloid leukaemia is 4.3 per 100,000 men and women per year. The estimated number of deaths is 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 1/2a, Single Dose Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

United States, Florida

United States, Massachusetts

United States, Oregon

United States, Texas

United States, Wisconsin

United Kingdom

United States, California

Study start:
Oct. 1, 2021
6 participants
Gene editing method:
Type of edit:
Gene knock-out and gene knock-in
Wilms tumour 1- WT1 specific TCR (knock-in), T Cell Receptor Alpha Constant-TRAC (knock-out), T Cell Receptor Beta Constant-TRBC (knock-out)
Delivery method:
Undisclosed - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated


This 2-part first in human (FIH) study is comprised of two open-label arms. It is a multi-center, Phase 1/2a study evaluating the safety and activity of NTLA-5001 in subjects with persistent or recurrent acute myeloid leukaemia after first line of therapy or later therapy. NTLA-5001 comprises autologous WT1-directed TCR-T cells engineered ex vivo using CRISPR-Cas9, and the treatment is adminstrered as an intravenous infusion.

Last updated: Jan. 2, 2023
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