Clinical Trial

Disease: Neuromyelitis Optica Spectrum Disease, NMOSD, (NCT06633042)

Disease info:

Neuromyelitis optica spectrum disorder (NMOSD) or Devic disease is a chronic condition affecting the brain and spinal cord. The condition is characterised by inflammation of the optic nerve (optic neuritis) and spinal cord (myelitis). While once thought to be a single, non-recurring episode of inflammation, it is now understood that most NMOSD patients experience repeated attacks separated by remission periods, which can vary from weeks to years. In its early stages, NMOSD can be mistaken for multiple sclerosis (MS) due to overlapping symptoms.

Source: https://rarediseases.org/rare-diseases/neuromyelitis-optica/

Frequency:
The prevalence of NMOSD is estimated at 1-10 per 100,000 individuals worldwide.
Official title:
The Safety and Efficacy of Universal CAR-T Cells Targeting BCMA in the Treatment of Refractory AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disease
Who:

Contact

Name: Xiaohui Wang, PhD

Phone: 18701533866

Email: xiaoxiao512006@163.com

Partners:

Wenzhou Medical University Affiliated Ophthalmology Hospital

Locations:
Study start:
Nov. 12, 2024
Enrollment:
18 participants
Gene editing method:
CRISPR
Gene:
B cell maturation antigen (BCMA) CD19
Delivery method:
- Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

This is an open label, Multi-center,dose-escalation study in up to 18 participants with refractory NMOSD. This study aims to evaluate the safety and efficacy of universal CAR-T Cells targeting BCMA in the Treatment of refractory NMOSD.

This is a multi-center, single-arm, open-label clinical study, and the sample size is set to 12-18 subjects. Based on the "3 + 3" dose escalation design principle, subjects will be divided into 3 groups from low dose to high dose in sequence (Group A; Group B; Group C).

Last updated: Nov. 2, 2024
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