Clinical Trial

Disease: T-Cell Acute Lymphoblastic Leukaemia, T-ALL, (ChiCTR1900025311)

Disease info:

T-cell acute lymphoblastic leukaemia (T-ALL) is a type of acute leukaemia that 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.

There are no specific signs or symptoms that would allow a T-ALL diagnosis 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 swollen lymph nodes in the middle part of the chest (mediastinum) which may affect breathing and circulation. The results of a simple blood count will usually indicate leukaemia, although in rare cases a blood count may be normal even though leukaemia is present. 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 is present. 

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 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
Frequency:
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:
Clinical research for anti-CD7 U-CAR-T bridging HST for CD7+ T/NK hematologic malignancies
Who:

Principal Investigator:Xi Zhang, MD    

Sponsor:

Xinqiao Hospital of Chongqing, Second Affiliated Hospital of the Army Medical University, Hematology Department

Partners:

 Foundation of the Innovative Scientific Research of Third Military Medical University 

Locations:

China, Chongqing

China, Guizhou 

China, Sichuan 

China, Yunnan 

Study start:
Sep. 2, 2019
Enrollment:
30 participants
Gene editing method:
CRISPR-Cas9
Gene:
T Cell Receptor Alpha Constant TRAC, CD7 molecule
Delivery method:
Lentivirus packaging - Ex-vivo
Trial link:
https://www.researchsquare.com/article/rs-2431426/v1
Safety updates:

(31-01-2023) CD7 targeted “off-the-shelf” CAR-T demonstrates robust in vivo expansion and high efficacy in the treatment of patients with relapsed and refractory T-cell malignancies. Note that this is a non peer-reviewed article preprint. 

(10-04-2021) Gracell Biotechnologies Reports Long-term Follow-up Data on TruUCAR-enabled GC027 in Relapsed/Refractory T-ALL at the AACR 2021 Annual Meeting

 

Other links:

LinkT-cell Acute Lymphoblastic Leukaemia Disease InformationLinkAcute Lymphocytic Leukemia (ALL) FrequencyLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem QasimLinkTrial Update: CRISPR-Edited Therapies for Sickle Cell Disease and Blood Cancer

IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

The primary outcome of this trial is to test the anti-tumour efficiency of anti-CD7 UCAR T-cells and the secondary outcome of the trial is to assess 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 receive a single infusion of TruUCAR™ GC027.

Last updated: Dec. 28, 2023
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