Disease: Relapsed/Refractory Multiple Myeloma, MM, (NCT04142619)

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 characterized by abnormalities in plasma cells, a type of white blood cell. These abnormal cells multiply out of control, 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, went off treatment and then the disease came back. 

Refractory Myeloma refers to as 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:
Phase I, Open-label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCARTCS1A (Allogenic Engineered T-cells Expressing Anti-CS1 Chimeric Antigen Receptor) in Patients With Relapsed/Refractory Multiple Myeloma
Who:

Dr. Krina Patel, Principal Investigator, Study Coordinating Investigator, Assistant Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine at MD Anderson Cancer Center 

Dr. David Siegel, Director of the Multiple Myeloma Institute at John Theuer Cancer Center at Hackensack University Medical Center in Hackensack

Partners:
Locations:

United States, New Jersey

United States, New York

United States, Texas

Study start:
Nov. 21, 2019
Enrollment:
18 participants
Gene editing method:
TALENs
Gene:
SLAMF7 SLAM family member 7 CS1
Delivery method:
In the first engineering step, genes are added to the T-cell genome with lentiviral vectors. In the second T-cell engineering step transfection is achieved with electoporation. - Ex-vivo
Note:
Chimeric Antigen Receptor T-Cell (CART-T) therapy
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting

Description

This is a Phase I, First in Human, open-label, dose escalation study evaluating Safety and Efficacy of UCART targeting CS1 in patients with Relapsed or Refractory Multiple Myeloma (MM). The purpose of this study is to evaluate the safety and clinical activity of one infusion of UCARTCS1A and to determine the Maximum Tolerated Dose (MTD).
UCART is Universal CAR-T cells. That is T Cells that have been taken from healthy donors (not from patients themselves). TALENS are used to disable the TCRαβ gene T cells use to recognize ’self’ to prevent them from attacking the host (graft vs host).

Last updated: Sep. 6, 2020
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