Clinical Trial

Disease: T-cell Acute Lymphoblastic Leukaemia, T-ALL, (NCT05397184)

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 particular 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.

The most common signs and symptoms of T-ALL are caused by the bone marrow being unable to produce enough normal blood cells. T-ALL often causes swollen 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 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 therap: Usually only given in conjunction with 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 1 Study of Base Edited CAR7 T Cells to Treat T Cell Malignancies (TvT CAR7)


Name: Robert Chiesa, Dr.  

Phone: 020 7405 9200 ext 8434



Name: Agnieszka B Kubat, MSc.

Phone: 07502269573   



Great Ormond Street Hospital for Children NHS Foundation Trust


UCL Great Ormond Street Institute of Child Health

Medical Research Council


United Kingdom

Ilyas Ali, London, United Kingdom

Study start:
Apr. 19, 2022
Gene editing method:
Base editing
Type of edit:
Gene knock-out
Delivery method:
Undisclosed - Ex-vivo
Safety updates:
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


Single-dose intravenous infusion (weight-based dosing) of CAR7+ T cells/kg BECAR7.

Total duration of treatment: 28 days.

Follow-up: 12 months.

Patients will undergo careful screening to confirm that this treatment is adequate for them.

Chemotherapy will be given prior to BE CAR-7 infusion. Patients will then receive a single infusion of the BE CAR-7 cells and will be closely monitored in hospital via blood and bone marrow tests for safety and to check the levels of BE CAR-7 and leukaemia cells. Patients are expected to be in hospital for 4-6 weeks for the BE CAR-7 therapy and the transplant will be scheduled 2-4 weeks after the end of BE CAR7 if leukaemia cells are no longer detectable. Patients will be monitored on the study for 1 year every month for the first 3 months and then every 6 months and then long term in routine clinics.

Last updated: Dec. 28, 2023
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