Clinical Trial

Disease: Multiple Myeloma, MM, (NCT04244656)

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.

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 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-BCMA Allogeneic CRISPR-Cas9-Engineered T Cells (CTX120) in Subjects With Relapsed or Refractory Multiple Myeloma

Study Director: Ewelina Morawa, MDCRISPR Therapeutics




United States, Illinois

United States, Oregon

United States, Tennessee

United States, Pennsylvania

Australia, New South Wales

Australia, Victoria

Canada, Ontario

Spain, Navarra

Spain, Salamanca

Spain, Barcelona

Study start:
Jan. 22, 2020
26 participants
Gene editing method:
Type of edit:
Gene knock-out, gene insertion
B-cell Maturation Antigen (BCMA)
Delivery method:
Undisclosed - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting


This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX120 in subjects with relapsed or refractory multiple myeloma.

CTX120 B-cell maturation antigen (BCMA)-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex -vivo using CRISPR-Cas9 gene editing components.

CTX120 is an allogeneic CRISPR-Cas9 gene-edited CAR-T cell therapy targeting BCMA for the treatment of multiple myeloma

Last updated: Jun. 5, 2023
Source: US National Institutes of Health (NIH)
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