Clinical Trial

Disease: Refractory Autoimmune Diseases, (NCT06485232)

Disease info:

This trial is for refractory autoimmune diseases of the nervous system including Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Multiple Sclerosis, and Chronic Inflammatory Demyelinating Polyradiculoneuropathy.

Autoimmune diseases occur when an individuals 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 nervous system, 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.

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:
An Exploratory Study on the Safety and Efficacy of Universal CAR-T Cells Targeting BCMA and CD19 in the Treatment of Refractory Autoimmune Diseases of the Nervous System
Who:

Contact:

Name: Junwei Hao, MD;PhD

Phone: 01083198277

Email: haojunwei@vip.163.com

Sponsor:

Xuanwu Hospital, Beijing

Locations:

China, Beijing

Xuanwu Hospital, Capital Medical University, Beijing, Beijing, China, 100053

Study start:
Aug. 1, 2024
Enrollment:
25 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene insertion (CAR) and gene knockout
Gene:
CD19-targeting CAR, genes involved in mediating graft versus host disease (GHVD), genes involved in mediating Immune rejection
Delivery method:
Electroporation - Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting

Description

This is an open label, single-site, dose-escalation study in up to 25 participants with refractory autoimmune diseases of nervous system. This study aims to evaluate the safety and efficacy of the treatment with universal BCMA and CD19 CART.

Last updated: Sep. 1, 2024
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