Clinical Trial

Disease: B-cell Acute Lymphoblastic Leukemia, ALL, (NCT04150497)

Disease info:

B-cell Acute Lymphoblastic Leukemia is an aggressive (fast-growing) type of leukemia (blood cancer) 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 acute lymphoblastic leukemia (ALL). Also called B-cell acute lymphocytic leukemia and precursor B-lymphoblastic leukemia.

Frequency:
The American Cancer Society’s estimates for acute lymphocytic leukemia (ALL) in the United States for 2020 are: About 6,150 new cases of ALL (3,470 in males and 2,680 in females) About 1,520 deaths from ALL (860 in males and 660 in females)
Official title:
Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)
Who:

Principal Investigator: Nitin Jain, MD  M.D. Anderson Cancer Center

Sponsor:

Cellectis S.A.

Partners:
Locations:

United States, Texas

United States, New York

United States, Illinois

United States, California

United States, Massachusetts

France, Paris 

United States, Pennsylvania

Study start:
Oct. 19, 2019
Enrollment:
30 participants
Gene editing method:
TALENs
Type of edit:
Gene Knock-out
Gene:
T Cell Receptor alpha and beta locus (TCRαβ)
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/study/NCT04150497
Safety updates:

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

(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: Jan. 2, 2023
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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