Clinical Trial

Disease: Acute Myeloid Leukaemia, AML, (NCT05066165)

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.


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 1/2a, Single Dose Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

United States, California

Research Site 2, Los Angeles, California, United States, 90095


United States, Florida

Research Site 5, Tampa, Florida, United States, 33612


United States, Massachusetts

Research Site 1, Boston, Massachusetts, United States, 02114


United States, Oregon

Research Site 6, Portland, Oregon, United States, 97239


United States, Texas

Research Site 3, Houston, Texas, United States, 77030


United States, Wisconsin

Research Site 4, Milwaukee, Wisconsin, United States, 53226


United Kingdom

Research Site 10, Leeds, United Kingdom

Research Site 8, London, United Kingdom

Research Site 9, London, United Kingdom

Research Site 7, Manchester, United Kingdom

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. 6, 2024
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