Disease: B- cell Acute Lymphoblastic Leukaemia, ALL, (NCT03666000)

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 1/2a, Open-label, Dose-escalation/Expansion, Parallel Assignment Study to Evaluate Safety and Clinical Activity of PBCAR0191 in Subjects With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma and r/r B-cell Acute Lymphoblastic Leukemia
Who:

No information 

Partners:
Locations:

United States, California

United States, Florida

United States, Georgia

United States, Indiana

United States, Massachusetts

United States, New York

United States, Texas

Study start:
Mar. 11, 2019
Enrollment:
92 participants
Gene editing method:
Meganucleases
Type of edit:
gene knock-out and gene knock-in
Gene:
T-cell Receptor Alpha Constant (TRAC), CD19 molecule
Delivery method:
unknown - 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 Phase 1/2a, nonrandomized, open-label, parallel assignment, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of PBCAR0191 in adults with r/r B ALL (Cohort A) and in adults with r/r B-cell NHL (Cohort N).

PBCAR0191Off-The-Shelf CAR T Platform
PBCAR0191 is an allogeneic 'off-the-shelf' CAR T therapy. Using T cells derived from healthy donors as starting material, then utilizes the ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a cell product that is designed to prevent graft-versus-host disease.

Before initiating PBCAR0191, subjects will be administered lymphodepletion chemotherapy composed of fludarabine and cyclophosphamide. At Day 0 of the Treatment Period, subjects will receive an intravenous (IV) infusion of PBCAR0191. Subjects who receive a split dose will also receive an IV infusion of PBCAR0191 on Day 10 and/or Day 14. Subjects may be considered for retreatment. All subjects are monitored during the treatment period through Day 28. All subjects who receive a dose of PBCAR0191 will be followed in a separate long-term follow-up (LTFU) study for 15 years after exiting this study.

Intervention:

  • Genetic: PBCAR0191

    Single dose of Allogeneic Anti-CD19 CAR T cells will be infused

    Other Name: Allogeneic Anti-CD19 CAR T cells

  • Drug: Fludarabine

    Fludarabine is used for lymphodepletion.

  • Drug: Cyclophosphamide

    Cyclophosphamide is used for lymphodepletion.

Last updated: Nov. 20, 2020
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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