Disease: Relapsed/Refractory Multiple Myeloma, (NCT04142619)

Disease info:

Multiple myeloma is a cancer that develops in the bone marrow, the spongy tissue found in the center 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 tumors 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 occus in aproximately 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

Cellectis S.A.


United States, New Jersey

United States, New York

United States, Texas

Study start:
Nov. 21, 2019
18 participants
Gene editing method:
No information
Chimeric Antigen Receptor T-Cell (CART-T) therapy
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: Apr. 25, 2020