Clinical Trial

Disease: Refractory Autoimmune Disease, (NCT06925542)

Disease info:

This trial will enrol adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).

Autoimmune diseases occur when an individual;s immune system incorrectly attacks the healthy cells of organs and tissues. There are over 80 types of different autoimmune diseases affecting various parts of the body, including the hair, liver, pancreas, and more. Symptoms vary and depend on the part of your body that's affected. Common symptoms include redness, swelling, heat and pain due to chronic inflammation. The symptoms of autoimmune diseases fluctuate between flare-ups, where symptoms are exacerbated, and remission, where symptoms alleviate, for a period of time.

The exact causes of autoimmune diseases can be complex and poorly defined. Autoimmune diseases tend to be familial, suggesting genetics may play a role in predisposition. Viruses, certain chemicals, and other environmental factors may trigger an autoimmune disease.

SLE is the most common form of lupus, a chronic autoimmune disease whereby the immune system attacks the body's own tissue and organs. SLE causes inflammation in connective tissues, such as cartilage and the lining of blood vessels.

Source: Autoimmune Diseases - MedlinePlus

 

Frequency:
Millions of Americans of all ages have autoimmune diseases. Women develop many types of autoimmune diseases much more often than men.
Official title:
A Phase 1 Dose Evaluation Study of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Subjects With Refractory Autoimmune Disease
Who:

Name: Clinical Trials

Phone Number: 1 877-214-4634

Email: medicalaffairs@crisprtx.com

Partners:
Locations:

Redwood City, California, United States, 94063 Not yet recruiting, Research Site 4

Chicago, Illinois, United States, 63110 Not yet recruiting, Research Site 2

Boston, Massachusetts, United States, 02118 Not yet recruiting, Research Site 6

Saint Louis, Missouri, United States, 63130 Recruiting, Research Site 1

Chapel Hill, North Carolina, United States, 27599 Not yet recruiting, Research Site 5

Augsburg, Germany, 86156 Not yet recruiting, Research Site 7

Hannover, Germany, 30625 Not yet recruiting, Research Site 3

Study start:
Mar. 10, 2025
Enrollment:
80
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knockout
Gene:
Multiple
Delivery method:
Adeno-associated viral (AAV) - Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).

This study may enroll up to 80 subjects in total. CTX112 is a CD19 directed chimeric antigen receptor (CAR) T cell immunotherapy comprised of allogeneic T cells prepared for the treatment of refractory autoimmune diseases. The cells are from healthy adult volunteer donors that are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA and Cas9 nuclease).

Last updated: Jun. 12, 2025
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