Clinical Trial

Disease: Acute Lymphoblastic Leukemia, T-ALL (NCT04984356)

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:
A Phase 1/2 Dose-Escalation and Dose-Expansion Study of the Safety and Efficacy of Anti-CD7 Allogeneic CAR-T Cells (WU-CART-007) in Patients With Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)/Lymphoblastic Lymphoma (LBL)
Who:

Eileen McNulty (Wugen contact) 

Sponsor:
Partners:
Locations:

Not yet disclosed.

Study start:
Nov. 1, 2021
Enrollment:
44
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock-out
Gene:
TRAC
Delivery method:
Electroporation - Ex-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

This is a first-in-human, multi-center, Phase 1/2, single-agent study in patients with relapsed or refractory T-cell acute lymphoblastic leukaemia (ALL) who have exhausted other treatment options. The study will consist of two phases, Phase 1 and Phase 2. During the dose escalation segment (Phase 1), up to 24 patients will be treated with 1 dose of WU-CART-007, in up to 4 dose levels until maximum tolerated dose (MTD) or maximum administered dose (MAD) is determined. The dose escalation segment will enroll successive cohorts of 3 to 6 patients using a standard 3 + 3 design. Once the recommended phase 2 dose (RP2D) is defined, the Phase 2 portion (cohort expansion) will enroll expansion cohorts.

Last updated: Apr. 10, 2022
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