Clinical Trial

Disease: Relapsed or Refractory Lymphoma and Leukaemia, (NCT04030195)

Disease info:

B-cell malignancies are cancers that arise from abnormalities in B cells, and include a type of non-Hodgkin lymphoma (NHL) called B-cell lymphoma, and B-cell acute lymphoblastic leukaemia (B-ALL). 

B-cell lymphoma refers to types of non-Hodgkin lymphoma that are characterised by abnormalities of the "B cells" (a type of white blood cell that makes antibodies to help fight infection). The condition may grow and spread slowly with few symptoms (also known as indolent lymphoma) or may be very aggressive with severe symptoms. Non-Hodgkin lymphoma (also known as non-Hodgkin’s lymphoma, NHL, or sometimes just lymphoma) is a cancer that starts in white blood cells called lymphocytes, which are part of the body’s immune system. NHL is a term that's used for many different types of lymphoma that all share some of the same characteristics. NHL usually starts in lymph nodes or other lymph tissue, but it can sometimes affect the skin. Non-Hodgkin lymphoma is one of the most common cancers in the United States, accounting for about 4% of all cancers. 

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. Chronic Lymphocytic Leukemia (CLL)  and Small Lymphocytic Leukemia are essentially the same disease with the differenc being where the cancer is located. CLL occurs when the disease is located in the bloodstream and bone marrow, while SLL occurs when the cancer is located mostly in the lymph nodes.

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:
NHL accounts for about 4% of all cancers in the U.S. The American Cancer Society estimates 80,550 people will be diagnosed with NHL in 2023. ALL accounts for less than 1% of cancers in the U.S., with around 6,540 new cases estimated in the U.S. in 2023.
Official title:
A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate the Safety and Clinical Activity of PBCAR20A in Subjects With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) or r/r Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)
Who:

Study Chair: Alan List, MD, Precision BioSciences, Inc.

Sponsor:

Precision BioSciences, Inc.

Partners:
Locations:

United States, California

City of Hope, Duarte, California, United States, 91010

Stanford University, Stanford, California, United States, 94305

 

United States, New York

Columbia University, New York, New York, United States, 10032

 

United States, Ohio

Cleveland Clinic, Cleveland, Ohio, United States, 44195

 

United States, Texas

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

Study start:
Mar. 24, 2020
Enrollment:
18 participants
Gene editing method:
Meganucleases
Type of edit:
Gene knock out, gene knock-in
Gene:
T-cell Receptor Alpha Constant (TRAC), CD20 molecule
Delivery method:
Adeno–associated virus (AAV) vectors/lipid nanoparticles (LNPs) - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT04030195
Safety updates:

(16- 09-2019) Precision BioSciences Announces FDA Clearance of the IND for PBCAR20A, a CD20 Targeting Genome Edited Allogeneic CAR T Therapy

Other links:

LinkNon Hodgkin's lymphoma- B cell malignancies informationLinkNon-Hodgkin’s lymphoma frequencyLinkPrecision Biosciences Allogeneic CAR TLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem QasimLinkLatest Gene Editing Clinical Trials for Cancer

Note:
PBCAR20A 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: Completed

Description

This is a multicenter, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study to evaluate the safety and tolerability, find an appropriate dose to optimize safety and efficacy, and evaluate clinical activity of PBCAR20A in subjects with relapsed/refractory (r/r) Non-Hodgkin Lymphoma (NHL) or r/r Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL). Before initiating PBCAR20A, subjects will be administered lymphodepletion chemotherapy composed of fludarabine and cyclophosphamide. At Day 0 of the Treatment Period, subjects will receive a single intravenous (IV) infusion of PBCAR20A. All subjects are monitored during the treatment period through Day 28. All subjects who receive a dose of PBCAR20A will be followed in a separate long-term follow-up (LTFU) study for 15 years after exiting this study.

Intervention:

  • Genetic: PBCAR20A

    Single dose of Allogeneic Anti-CD20 CAR T cells will be infused, and a classic "3+3" dose escalation will be applied.

    Other Name: Allogeneic Anti-CD20 CAR T cells

  • Drug: Fludarabine

    Fludarabine is used for lymphodepletion.

  • Drug: Cyclophosphamide

    Cyclophosphamide is used for lymphodepletion.

Last updated: Dec. 28, 2023
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