Clinical Research Directory

Modified Zipper Therapy for AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder

Study Title: A National, Multicenter, Randomized Controlled Trial of the Modified Zipper Therapy in AQP4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder (ELITE Study) Brief Summary: The goal of this clinical trial is to evaluate the efficacy and safety of a novel sequential immunomodulation strategy, termed "Modified Zipper Therapy," in patients with acute attacks of Aquaporin-4 antibody-positive Neuromyelitis Optica Spectrum Disorder (AQP4-IgG+ NMOSD). The therapy aims to enhance neurological recovery by combining plasma exchange (PE) with immediate complement inhibition using eculizumab, following high-dose corticosteroid pulse therapy. The main questions this trial aims to answer are: Efficacy: Does the Modified Zipper Therapy (high-dose corticosteroids + plasma exchange + eculizumab) lead to a higher rate of neurological improvement at Week 12 compared to standard therapy (high-dose corticosteroids + plasma exchange alone)? For patients with NMOSD-related optic neuritis (NMOSD-ON), improvement is defined as a gain of ≥10 letters on the ETDRS chart or a decrease of ≥0.2 LogMAR in best-corrected visual acuity (BCVA). For patients with NMOSD-related longitudinally extensive transverse myelitis (NMOSD-LETM), improvement is defined as a reduction of ≥2 points on the Expanded Disability Status Scale (EDSS). Safety: What is the nature and frequency of adverse events experienced by participants receiving the Modified Zipper Therapy compared to those receiving standard therapy? Researchers will compare the Modified Zipper Therapy group to the Standard Therapy group to see if the novel combination is more effective in improving visual and functional outcomes in acute AQP4-IgG+ NMOSD. Participants will: Be randomly assigned (like a coin toss) to receive either the Modified Zipper Therapy or the Standard Therapy. Undergo a treatment period involving intravenous corticosteroids and a series of plasma exchange sessions. The Modified Zipper Therapy group will also receive intravenous eculizumab infusions timed around the plasma exchange procedures. Be followed for 24 weeks after treatment completion. Attend scheduled clinic visits for comprehensive assessments including: Visual acuity testing (using ETDRS, Snellen, and low-contrast charts). Neurological function evaluations (EDSS and OSIS scores). Optical coherence tomography (OCT) and visual evoked potential (VEP) tests. Magnetic resonance imaging (MRI) scans of the optic nerves. Safety monitoring (physical exams, lab tests, ECGs).

A Study of C-CAR168 in the Treatment of Central Nervous System Autoimmune Diseases Refractory to Standard Therapy

This is an investigator-initiated, single-center, open-label study of C-CAR168, an autologous bi-specific CAR-T therapy targeting CD20 and BCMA, for the treatment of adult patients with central nervous system autoimmune diseases refractory to standard therapy

Slow vs. Rapid Glucocorticoids Tapering With Inebilizumab in NMOSD

Neuromyelitis optica spectrum disorder (NMOSD) is a central nervous system autoimmune condition mainly involving the spinal cord, optic nerves, and area postrema. The anti-aquaporin-4 (AQP4)-Immunoglobulin G (IgG) is a specific biomarker for NMOSD. Glucocorticoids(GCs) are used as first-line treatment for NMOSD. Oral glucocorticoids tapering is always suggested following the pused therapy in the maintenance phase. Inebilizumab, a humanized monoclonal antibody targeting CD19, has been proven effective in preventing NMOSD relapses. This study aims to evaluate and compare the efficacy and differences between glucocorticoids slow-tapering and rapid-tapering strategies combined with inebilizumab in preventing relapses in AQP4-IgG-seropositive NMOSD patients following an acute attack, with the goal of determining the optimal approach to steroid tapering and discontinuation after initiation of inebilizumab.

A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy

This is an investigator-initiated, multicenter, open-label study of C-CAR168, an autologous bi-specific CAR-T therapy targeting CD20 and BCMA, for the treatment of adult patients with autoimmune diseases refractory to standard therapy

HANDLE-a Real World Study on Satralizumab in NMOSD

This study is a single-center, retrospective-prospective, non-interventional cohort study to assess the clinical outcomes of Chinese NMOSD patients treated with satralizumab in a real-world patient management model by collecting follow-up data in clinical practice.