Clinical Trial

Disease: Acute Myeloid Leukaemia, AML, (ISRCTN14430213)

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 I study of base-edited CAR T cells against acute myeloid leukaemia (AML): deep conditioning ahead of allogeneic stem cell transplantation


Name; Mr Ahmed Batoul
Phone: +44 (0)2031085573


Great Ormond Street Hospital for Children NHS Foundation Trust


Wellcome Trust




United Kingdom

Study start:
Jan. 11, 2023
10 participants
Gene editing method:
Type of edit:
Gene enhancement
CD33 receptor
Delivery method:
Lentivirus (LV) - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This phase I study will treat children aged 6 months up to 16 years old with acute myeloid leukaemia (AML), which has come back (relapsed). The new product is made from white blood cells (T cells) collected from a healthy donor and changed so they can kill leukaemia cells. These ‘ready-made’ CAR T cells have been made using a new technique called Base Editing and have been given the code name ‘BE CAR-33’. They look for and attack cells showing a flag called ‘CD33’. This technique allows them to work after chemotherapy and also disarms them to prevent effects against normal cells. The main purpose of this study is to assess the safety of the ‘BE CAR-33’ therapy and to see if ready-made CAR T cells can get rid of leukaemia ahead of a planned bone marrow transplant that will hopefully prevent the leukaemia from returning.

Last updated: Feb. 5, 2024
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