Clinical Trial

Disease: Relapsed or Refractory Multiple Myeloma, MM, (NCT04171843)

Disease info:

Multiple myeloma is a cancer that develops in the bone marrow, the spongy tissue found in the centre of most bones. Multiple myeloma is characterised by abnormalities in plasma cells, a type of white blood cell. In myeloma, these abnormal cells multiply uncontrollably, increasing from about one percent of cells in the bone marrow to the majority of bone marrow cells. The abnormal cells form tumours within the bone, causing bone pain and an increased risk of fractures.

Relapsed myeloma refers to when a patient had active treatment that their disease responded to, went off treatment and then the disease came back. 

Refractory myeloma is a disease that is progressing despite active treatment.

 

Frequency:
Multiple myeloma occurs in approximately 4 per 100,000 people per year; there are currently about 100,000 affected individuals in the United States.
Official title:
A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion Study to Evaluate the Safety and Clinical Activity of PBCAR269A, With or Without Nirogacestat, in Study Participants With Relapsed/Refractory Multiple Myeloma
Who:

Study Chair: Monika Vainorius, MD   Precision BioSciences, Inc.

Principal Investigator: Maung Myo Htut, MD City of Hope

Principal Investigator: Nina Shah, MD University of California San Francisco

Principal Investigator: Omar Nadeem, MD  Dana Farber Cancer Institute

Principal Investigator: Ran Reshef, MD Columbia University Irving Medical Center

Principal Investigator: Krina Patel, MD MD Anderson Cancer Center 

 

Sponsor:

Precision BioSciences, Inc.

Partners:
Locations:

United States, California

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

University of California San Francisco, San Francisco, California, United States, 94115

 

United States, Massachusetts

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

 

United States, New York

Columbia University Irving Medical Center, New York, New York, United States, 10032

 

United States, Texas

MD Anderson, Houston, Texas, United States, 77030

Study start:
Apr. 30, 2020
Enrollment:
48 participants
Gene editing method:
Meganucleases
Type of edit:
Gene knock-out, gene knock-in
Gene:
T-cell Receptor Alpha Constant (TRAC), Tumor Necrosis Factor Receptor Superfamily Member (TNFRSF17 or BCMA)
Delivery method:
- Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT04171843
Safety updates:

(08-06-2022) Precision BioSciences Provides Update on Allogeneic CAR T Programs and Path Forward with Its Lead PBCAR0191 Candidate for CAR T Relapsed Patient Population

(11-12-2021) Precision BioSciences Reports Clinical Program Updates for Its Allogeneic CAR T Pipeline

(09-09-2020): Precision BioSciences Receives Fast Track Designation from U.S. Food and Drug Administration for PBCAR269A, an Investigational Allogeneic CAR T Therapy for Relapsed/Refractory Multiple Myeloma

Other links:

LinkMultiple Myeloma Disease Information and 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 CancerLinkPrecision BioSciences and SpringWorks Therapeutics Dose First Patient in Expanded Phase 1/2a Clinical Trial Evaluating PBCAR269A with Nirogacestat in Patients with Relapsed/Refractory Multiple Myeloma

Note:
PBCAR269A 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 clinical activity of PBCAR269A in adults with r/r Multiple Myeloma

Before initiating the study treatment PBCAR269A, all study participants will be administered lymphodepletion chemotherapy. The initial lymphodepletion chemotherapy regimen will be composed of fludarabine and cyclophosphamide during the Screening Period. On Day 0 of the Treatment Period, study participants will receive a single intravenous (IV) infusion of PBCAR269A. All subjects are monitored during the treatment period through Day 28. All subjects who receive a dose of PBCAR269A will be followed in a separate long-term follow-up (LTFU) study for 15 years after exiting this study.

Last updated: Dec. 28, 2023
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