Clinical Trial

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.

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:
Study Evaluating Safety and Efficacy of UCART Targeting CS1 in Patients With Relapsed/Refractory Multiple Myeloma (MELANI-01)

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


United States, New Jersey

United States, New York

United States, Texas

United States, California 

United States, Minnesota


United States, Colorado


United States, Tennessee

Study start:
Nov. 21, 2019
18 participants
Gene editing method:
Type of edit:
Gene knock-out
SLAM family member 7 CS1 (SLAMF7)
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
Chimeric Antigen Receptor T-Cell (CAR-T) therapy. 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 electroporation.
Upon transfusion of allogeneic CS1-specific universal CAR-expressing T lymphocytes UCARTCS1A, these cells target and bind to cancer cells expressing CS1. This induces selective toxicity in and causes lysis of CS1-expressing tumor cells. SLAMF7 is a member of the signaling lymphocytic activation molecule (SLAM) family of transmembrane receptors that modulate the function of immune cells through immunoreceptor tyrosine-based switch motifs (ITSMs) and intracellular adaptor proteins.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


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: Jan. 1, 2023
Source: US National Institutes of Health (NIH)
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