Clinical Trial

Disease: Relapsed or Refractory Multiple Myeloma, MM, (NCT04244656)

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:
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

University of Chicago, Chicago, Illinois, United States, 60637


United States, Oregon

Oregon Health and Science University, Portland, Oregon, United States, 97239


United States, Pennsylvania

University of Pennsylvania, Philadelphia, Pennsylvania, United States, 19104


United States, Tennessee

Sarah Cannon Research Institute, Nashville, Tennessee, United States, 37203


Australia, New South Wales

Royal Prince Alfred Hospital, Sydney, New South Wales, Australia, 2050


Australia, Victoria

Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia, 3000


Canada, Ontario

University Health Network, Princess Margaret Cancer Centre, Toronto, Ontario, Canada, M5G 1X6



Institut Catala d'Oncologia Hospital Germans Trias i Pujol, Badalona, Barcelona, Spain, 08916

Universidad de Navarra, Pamplona, Navarra, Spain, 31008

Hospital Universitario de Salamanca, Salamanca, Spain, 37007

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:
- 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: Dec. 28, 2023
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