Disease: T-Cell Chronic Lymphoblastic Leukaemia, CLL, (NCT04264078)

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. 
Frequency:
The American Cancer Society’s estimates for acute lymphocytic leukemia (ALL) in the United States for 2021 are: About 5,690 new cases of ALL (3,000 in males and 2,690 in females) About 1,580 deaths from ALL (900 in males and 680 in females).
Official title:
Anti-CD7 Universal CAR-T Cells for CD7+ T/NK Cell Hematologic Malignancies: a Multi-center, Uncontrolled Trial
Who:

Principal Investigator:Xi Zhang, MD    Xinqiao Hospital of Chongqing

Partners:

Gracell Biotechnologies (Shanghai) Co., Ltd

920th Hospital of Joint Logistics Support Force

The Second Affiliated Hospital of Chongqing Medical University

The Affiliated Hospital Of Guizhou Medical University

Central South University

The First Affiliated Hospital of Kunming Medical College

The General Hospital of Western Theater Command

Second Affiliated Hospital of Xi'an Jiaotong University

Nanfang Hospital of Southern Medical University

Fujian Medical University Union Hospital

The First Affiliated Hospital of Anhui Medical University

Tang-Du Hospital

Locations:

China, Chongqing

Study start:
Mar. 1, 2020
Enrollment:
30 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock-out
Gene:
T Cell Receptor Alpha Constant (TRAC), CD7 molecule
Delivery method:
- Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

The primary outcome of the trial is to test the anti-tumour efficiency of anti-CD7 UCAR-T cells and the secondary outcome of the trial is the long-term efficiency of anti-CD7 UCAR-T cells. GC027 is a first-in-human, universal CAR-T therapy manufactured from T cells of human leukocyte antigen (HLA) unmatched healthy donors using TruUCAR™ technology.

To reduce the possibility of graft-versus-host disease GvHD from allogeneic T cells, CRISPR-Cas9 is used to disrupt the T cell receptor alpha constant (TRAC) locus to eliminate surface expression of the TCR complex of TruUCAR product candidates. Furthermore, to eliminate potential fratricide (self-killing of CAR T cells during the production process), CRISPR-Cas9 is employd to disrupt CD7, a pan T and NK marker on the CAR T cells.

All patients received a single infusion of TruUCAR™ GC027.

Last updated: Jul. 11, 2021
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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