Clinical Trial

Disease: Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT02808442)

Disease info:

Leukaemia is cancer of the white blood cells which are responsible for fighting infection. In leukaemia, the bone marrow produces abnormal levels of white blood cells. B-cell acute lymphoblastic leukaemia (B-ALL) is an aggressive (fast-growing) type of leukaemia in which too many B-cell lymphoblasts (immature white blood cells) are found in the bone marrow and blood. It is the most common type of ALL. Also called B-cell acute lymphocytic leukaemia and precursor B-lymphoblastic leukaemia. 

Relapsed refers to when a patient has received active treatment, went off treatment and then the disease came back, whereas refractory refers to disease that is progressing despite active treatment.

ALL accounts for less than 1% of all cancers in the U.S., with around 6,540 new cases estimated in the U.S. in 2023. ALL is the most common type of cancer found in children, though it can affect adults too.
Official title:
A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia

Institut de Recherches Internationales Servier


ADIR, a Servier Group company


United States, California

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


United States, Pennsylvania

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


United States, Texas

University of Texas Southwestern Medical Center, Dallas, Texas, United States, 75390



Hôpital Robert-Debré, Paris, France, 75019



Hospital San Juan De Dios, Barcelona, Spain, 08950


United Kingdom

UCL Great Ormond Hospital, London, United Kingdom

Study start:
Jun. 3, 2016
13 participants
Gene editing method:
Type of edit:
Gene knock-out
T Cell Receptor alpha locus (TCRα), CD52 molecule
Delivery method:
Electroporation and Lentiviral (LV) - Ex-vivo
Electroporation of TALEN gene editing tools. Lentiviral transduction of CAR-T.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed


This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).

Allogeneic CD19-specific universal CAR19-expressing T lymphocytes:

A preparation of allogeneic, frozen, ‘off-the-shelf’, universal transcription activator-like effector nuclease (TALEN)-engineered, gene-edited T lymphocytes expressing a chimeric antigen receptor (CAR) targeting the tumor-associated antigen (TAA) CD19, and containing a RQR8 transgene, with potential immunostimulating and antineoplastic activities. Using TALEN technology, the T-cell receptor (TCR) alpha chain and CD52 genes are deleted from the CAR19 T cells. Upon infusion, allogeneic universal CD19-specific CAR-modified T cells (UCART19) specifically target and bind to CD19-expressing tumor cells, thereby selectively lysing CD19-expressing tumor cells. CD19 antigen is a B-cell specific cell surface antigen expressed in all B-cell lineage malignancies. Deletion of the CD52 gene makes the modified donor T cells resistant to the anti-CD52 monoclonal antibody alemtuzumab, which is used during lymphodepletion. The knockout of the TCR alpha gene eliminates TCR expression and is intended to abrogate the potential induction of graft-versus-host disease (GvHD) by the donor T cells. The gene-edited allogeneic, frozen UCART19 have reduced production times and provide off-the-shelf CAR-T cells when compared to autologous CAR-T cells, which use the patient's own cells and are produced on an individual basis. The protein expressed by the RQR8 transgene contains epitopes from CD34 and CD20, which allows tracking of the UCART19 cells with a clinically-approved anti-CD34 antibody. Additionally if the UCART19 cells cause unacceptable side effects, the CD20 portion of the protein permits selective depletion of the UCART19 cells when the anti-CD20 monoclonal antibody rituximab is administered.

Last updated: Dec. 28, 2023
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