Clinical Trial

Disease: Relapsed or 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 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:
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) Administered in Patients With Relapsed/Refractory Multiple Myeloma



Cellectis Central Contact

Phone: +1 347-752-4044



United States, California

UCSF Medical Center- Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States, 94115



United States, Colorado

Sarah Cannon Research Institute - Colorado Blood Cancer Institute, Denver, Colorado, United States, 80218


United States, Georgia

Winship Cancer Institute Emory University, Atlanta, Georgia, United States, 30322


United States, Minnesota

Mayo Clinical Cancer Center (MCCC), Rochester, Minnesota, United States, 55905


United States, New Jersey

Hackensack Meridian Health, Hackensack, New Jersey, United States, 07601


United States, New York

Weill Cornell Medical College, New York, New York, United States, 10065


United States, Tennessee

Sarah Cannon Research Institute - Tennessee Oncology, Nashville, Tennessee, United States, 37203-1625


United States, Texas

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

Sarah Cannon Research Institute - Methodist Healthcare, San Antonio, Texas, United States, 78229-6306  

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: Dec. 28, 2023
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