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

United States, Michigan

United States, Minnesota

United States, Rhode Island

Study start:
Mar. 11, 2019
Enrollment:
92 participants
Gene editing method:
Meganucleases
Type of edit:
Gene knock-out, gene knock-in
Gene:
T-cell Receptor Alpha Constant (TRAC), CD19 molecule
Delivery method:
- Ex-vivo
Note:
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.
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).

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: Apr. 18, 2021
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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