Clinical Trial

Disease: T-Cell Acute Lymphoblastic Leukaemia, T-ALL, (ISRCTN15323014)

Disease info:

T-cell acute lymphoblastic leukaemia (T-ALL) is a type of acute leukaemia meaning that it is aggressive and progresses quickly. It affects the lymphoid-cell-producing stem cells, in paticular a type of white blood cell called T lymphocytes as opposed to acute lymphoblastic leukaemia (ALL) which commonly affects B lymphocytes. A lymphoid stem cell becomes a lymphoblast cell and then one of three types of lymphocytes (white blood cells):

  • B lymphocytes that make antibodies to help fight infection.
  • T lymphocytes that help B lymphocytes make the antibodies that help fight infection.
  • Natural killer cells that attack cancer cells and viruses.

There are no specific signs or symptoms which would allow a diagnosis of T-ALL to be made. The most common signs and symptoms are caused by the bone marrow being unable to produce enough normal blood cells. T-ALL often causes swolen lymph nodes in the middle part of the chest (mediastinum) which may affect breathing or the circulation. The results of a simple blood count will usually indicate leukaemia although, rarely, a blood count may be normal. Virtually all patients with T-ALL will have bone marrow samples taken to confirm the diagnosis and to help to determine exactly what type of leukaemia a patient has. 

The main ways in which leukaemia is treated are:

  • Chemotherapy – Cell-killing drugs. Steroids are normally used along with chemotherapy for T-ALL
  • Radiation therapy – Usually only given as part of a stem cell transplant in T-ALL
  • Stem cell transplant – Younger/fitter patients may be given a stem cell transplant (bone marrow transplant). This is done using healthy stem cells from a donor. This is also done for T-ALL if chemotherapy does not cure the disease. 


The American Cancer Society’s estimates approximately 6,540 new cases of Acute Lymphoblastic Leukaemia (ALL) in 2023, accounting for less than 1% of all cancers in the United States.
Official title:
Phase I study of base edited CAR7 T cells to treat T cell malignancies (TvT CAR7)


Principal Investigator: Dr Waseem Qasim
Phone: +44 (0)207 405 9200


Great Ormond Street Hospital


United Kingdom, England

Great Ormond Street Hospital

Study start:
Jan. 4, 2022
10 participants
Gene editing method:
Type of edit:
Gene enhancement
TRBC1 , TRBC2, CD7, and CD52
Delivery method:
Electroporation and lentivirus
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This phase I clinical trial will treat children aged 6 months up to 16 years with T cell leukaemia which has come back (relapsed) after chemotherapy or is not responding to chemotherapy (refractory). The cell therapy is made from white blood cells (T cells) collected from a healthy donor and changed so they can kill other T cells, including leukaemia cells. These ‘ready-made’ CAR T cells have been made using a new technique called CRISPR base editing to modify their DNA code and have been given the name BE CAR-7. This technique allows them to work after chemotherapy and also disarms them to prevent effects against normal cells. The main aim of this study is to assess the safety of the BE CAR-7 treatment and to see if ready-made CAR T cells can eradicate T cell leukaemia ahead of a planned bone marrow transplant.

Last updated: Apr. 20, 2024
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