Clinical Trial

Disease: Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT04150497)

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.

Frequency:
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:
Open Label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCART22 (Allogeneic Engineered T-cells Expressing Anti-CD22 Chimeric Antigen Receptor) in Patients With Relapsed or refractory CD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL)
Who:

Contact:

Cellectis Central

Phone: +1 (347) 752-4044

Email: clinicaltrials@cellectis.com

Sponsor:

Cellectis S.A.

Partners:
Locations:

United States, Texas

MD Anderson Cancer Center, Houston, Texas, United States, 77030

 

United States, New York

Memorial Sloan Kettering Cancer Center (MSKCC) David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center, New York, New York, United States, 10021

Weill Medical College of Cornell University, New York, New York, United States, 10065

 

United States, Illinois

University of Chicago, Chicago, Illinois, United States, 60647

 

United States, California

University of California, Los Angeles (UCLA) - Medical Center, Los Angeles, California, United States, 90095

 

United States, Massachusetts

Dana Farber Cancer Institute, Boston, Massachusetts, United States, 02215

 

France, Paris 

CHU de Nantes - Hôtel-Dieu, Nantes, France, 44093

Hôpital Saint Louis, Unité d'Hématologie Adolescents et Jeunes Adultes Département d'HématologieRecruitingParis, France, 75010

Hôpital Robert Debré - Service d'hémato-immunologieRecruitingParis, France, 75019

Hôpital Lyon Sud, Pierre-Benite, France, 69310

CHU Rennes - Hopital Pontchaillou, Rennes, France, 35033

 

United States, Pennsylvania

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

Study start:
Oct. 14, 2019
Enrollment:
40 participants
Gene editing method:
TALENs
Type of edit:
Gene Knock-out
Gene:
T-cell receptor alpha constant (TRAC) and CD52 genes
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/study/NCT04150497
Safety updates:

(12-04-2023) Cellectis doses first France patient in BALLI-01 trial of UCART22

(11-12-2021) Cellectis Reports Encouraging Clinical Data from BALLI-01 Study in Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia

(05-11-2020) Preliminary Results of Balli-01: A Phase I Study of UCART22 (allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor) in Adult Patients with Relapsed or Refractory (R/R) CD22+ B-Cell Acute Lymphoblastic Leukemia (B-ALL)

(04-11-2020) American Society of Hematology (ASH) Abstract Shows Initial Anti-Leukemic Activity of UCART22 in BALLI-01 Phase 1 Study in R/R Adult B-ALL

Other links:

LinkB-cell Acute Lymphoblastic Leukemia Disease InformationLinkAcute Lymphoblastic Leukemia FrequencyLinkWhat is UCART22?LinkStraightforward Generation of Ultrapure Off-the-Shelf Allogeneic CAR-T CellsLinkPre-Clinical Activity of Allogeneic Anti-CD22 CAR-T Cells for the Treatment of B-Cell Acute Lymphoblastic LeukemiaLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem Qasim

Note:
UCART22 consists of allogeneic engineered T-cells Expressing Anti-CD22 Chimeric Antigen Receptor. In the first engineering step, genes are added to the T-cell genome with lentiviral vectors. In the second T-cell engineering step transfection is achieved with electroporation.
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

This is a Phase I, first-in-human, open-label, dose escalation and expansion study of UCART22 administered intravenously to patients with relapsed or refractory B-cell acute Lymphoblastic Leukemia (B-ALL). The purpose of this study is to evaluate the safety, tolerance, clinical activity of UCART22 and to determine the Maximum Tolerated Dose (MTD).

UCART is Universal CAR-T cells. That is T Cells that have been taken from healthy donors (not from patients themselves). TALENS are used to disable the TCRαβ gene T cells use to recognize ’self’ to prevent them from attacking the host (graft vs host).

Allogeneic Engineered T-cells Expressing Anti-CD22 Chimeric Antigen Receptor.

Experimental: Dose Escalation

Part 1: Dose Escalation One administration of UCART22 in the dose escalation phase will explore 4 doses of UCART22 and continue until the Maximum Tolerated Dose (MTD) is identified.

Part 2: Dose Expansion One administration of UCART22 at the MTD.

Last updated: Apr. 20, 2024
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