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.


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

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
48 participants
Gene editing method:
Type of edit:
Gene knock-out, gene knock-in
T-cell Receptor Alpha Constant (TRAC), Tumor Necrosis Factor Receptor Superfamily Member (TNFRSF17 or BCMA)
Delivery method:
- Ex-vivo
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.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed


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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