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:

Contact

Name: Eileen McNulty

Phone: 636-385-5306

Email: emcnulty@wugen.com

Sponsor:
Partners:
Locations:

United States, California

City of Hope, Duarte, California, United States, 91010

Children's Hospital Los Angeles, Los Angeles, California, United States, 90027

 

United States, Florida

Moffit Cancer Center, Tampa, Florida, United States, 33612

 

United States, Missouri

Washington University, Saint Louis, Missouri, United States, 63110

 

United States, Pennsylvania

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

 

United States, Tennessee

Vanderbilt University, Nashville, Tennessee, United States, 37212

 

United States, Wisconsin

University of Wisconsin, Madison, Wisconsin, United States, 53792

 

Australia, Victoria

Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia

Study start:
Jan. 14, 2022
Enrollment:
44
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock-out
Gene:
CD7 & 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: Jun. 5, 2023
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