Clinical Trial

Disease: Multiple Myeloma, MM, (NCT03399448)

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.

 

Frequency:
An estimated 36,110 people will be diagnosed with MM in 2025.
Official title:
Phase 1 Trial of Autologous T Cells Engineered to Express NY-ESO-1 TCR and CRISPR Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T Cells)
Who:

Principal Investigator: Edward Stadtmauer, MD

Sponsor:

University of Pennsylvania

Partners:

Parker Institute for Cancer Immunotherapy

Tmunity Therapeutics

Locations:

United States, Pennsylvania

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

Study start:
Sep. 5, 2018
Enrollment:
3 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock out
Gene:
T cell receptor genes, (TCRα, TCRβ) and programmed cell death protein 1 (PD-1)
Delivery method:
Lentivirus (LV) and Electroporation - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/study/NCT03399448
Safety updates:

 (28-02-2020) CRISPR-engineered T cells in patients with refractory cancer

Other links:

LinkThe first CRISPR gene therapy is safeLinkCRISPR-engineered T cells in patients with refractory cancer.LinkMultiple Myeloma Disease Information and FrequencyLinkEmerging gene therapies for cystic fibrosisLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem Qasim

Note:
Sponsor has terminated trial to pursue other targets.
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated

Description

This is a first-in-human trial proposed to test HLA-A*0201 restricted NY-ESO-1 redirected T cells with edited endogenous T cell receptor and PD-1.

Autologous T cells transduced with a lentiviral vector to express NY-ESO-1 and electroporated with CRISPR guide RNA to disrupt expression of endogenous TCRα, TCRβ and PD-1 (NYCE T Cells).

Last updated: Feb. 25, 2025
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