Clinical Research Directory

The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders

This is a single-center, randomized, open-label, placebo-controlled, dose-escalation trial. The objective of this research is to evaluate the safety, tolerability, and efficacy of intrathecal administration of human-induced neural stem cell-derived extracellular vesicles (NouvSoma001) for the treatment of neuromyelitis optica spectrum disorders.

A Clinical Study of B001 Injection in the Treatment of Neuromyelitis Optica Spectrum Disorders(NMOSD)

To evaluate the efficacy and safety of B001 injection in aquaporin-4 antibody positive patients with neuromyelitis optica spectrum disorder.

Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Study to Assess Disease Activity in Apparently Stable Patients

The present research is an observational clinical study. The project aims to investigate astrocytic damage, assessed through biological findings such as an increase in GFAP level and/or MRI water index, in patients with NMOSD and its potential role in predicting demyelinating relapses or affecting disability outcomes. It will also explore predictors of astrocytic relapses, focusing on demographic, clinical, and immunological biomarkers like T cell responses, B cell repopulation, and cytokine levels. The goal is to identify unrecognized disease activity, providing insights for future research and clinical trials. The study will involve 8 sites in Italy: 6 NMOSD clinical centers for patient enrolment and 2 centers for bioengineering and biological analysis. Centralized analysis of the MRI images of all patients enrolled in the clinical centers will be performed by the Neuroimaging Research Unit Fase 1 of San Raffaele Hospital. A total of 50 patients will be included and they will be followed for 12 months. Comprehensive evaluation of patients, including clinical assessment, bioengineering evaluation, MRI, and blood samples, will be conducted at baseline, month 6, and month 12. To assess silent astrocytic relapses, a specialized evaluation will take place at months 3 and 9, including clinical analysis, blood samples to assess biomarkers like GFAP, a reduced MRI protocol to assess MRI water index, and bioengineering evaluation. In the case of a classical relapse, a dedicated visit will occur within 5 days of symptom onset, using the same evaluation protocol as at months 3 and 9 (MRI and biomarkers will be evaluated if not done in the month before).

A Phase III Study of TACI-antibody Fusion Protein Injection (RC18) in Subjects With Neuromyelitis Optica Spectrum Disorders

The purpose of this study is to initially observe the safety and effectivity of RC18 in Participants with Neuromyelitis Optica Spectrum Disorders.

Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/ BCMA（QT-019C） in Patients With r/ r Neurological Autoimmune Diseases

This is an open label, single-site, dose-escalation study in up to 15 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells（QT-019C）.

Normal Chinese Lifespan Brain Charts Initiative (NCLBCI)

Major brain diseases (including neuroimmune diseases, neurodegenerative diseases, and cerebrovascular diseases) are leading causes of death and disability in humans. This research will gather brain imaging data from more than 10,000 participants across multiple age groups from multiple centers nationwide. Using standardized modeling methods, the investigators aim to establish normal brain imaging reference values for the Chinese population. (1) The study will reveal population-level patterns of brain structure changes and determine normal reference values for brain structure at different ages. (2) Non-invasive imaging methods will be applied to evaluate blood-brain barrier changes without the need for invasive procedures, establishing age-related permeability patterns and normal values in healthy populations. Additionally, functional magnetic resonance imaging (fMRI) will be combined to provide brain function indicators at various scales, creating comprehensive multi-dimensional function charts. (3) These brain structure and function charts will be applied to the individualized diagnosis and treatment of major brain diseases, including neuroimmune diseases (Multiple Sclerosis \[MS\], Neuromyelitis Optica Spectrum Disorders \[NMOSD\]), neurodegenerative diseases (Alzheimer's disease \[AD\], Parkinson's disease \[PD\]), and cerebrovascular diseases (Cerebral Small Vessel Diseases \[cSVD\]). This approach will facilitate early disease diagnosis and provide precise, individualized assessments of treatment efficacy and prognosis prediction. The established brain structure and function charts will be accessible via interactive websites or software, enabling real-time online data updates and individualized percentile outputs for brain structure and function. By inputting the target data, users can obtain individualized evaluation metrics for brain structure and function. The project's implementation and application hold great potential for broad use, promotion, and high translational potential.

Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD

The primary objective of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in Chinese Adults with Neuromyelitis Optica Spectrum Disorders (NMOSD).

Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation Antigen （CD19/BCMA） in Patients With r/r Neurological Autoimmune Diseases

This is an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.

Integrating Metabolism, Connectivity, and Mesoscale Imaging at Ultra-high Field to Decipher Mechanisms of Resilience and Neurodegeneration in Neurological Diseases and Healthy Aging

The MESO7 study is a prospective observational research project designed to investigate the mechanisms of resilience and neurodegeneration in neurological diseases and healthy aging. It leverages advanced multiparametric brain and spinal cord imaging at high (3T) and ultra-high magnetic fields (7T) to assess structural, functional, metabolic, and mesoscale changes in the central nervous system (CNS). Particular emphasis is placed on sodium (23Na-MRI) and phosphorus (31P-MRI) imaging, along with layer-dependent brain connectivity analysis. The primary objective is to evaluate the impact of neuronal energy failure, measured via sodium concentration, on functional and structural reorganization in both healthy individuals and patients with various neurological conditions. Directed brain network models will be constructed from MRI data to quantify the connectivity strength (in- and out-degree) of cortical nodes. These connectivity metrics will be correlated with sodium concentrations to assess energy failure and its role in network reorganization. Longitudinal follow-up over two years is planned for subgroups with clinically progressive diseases. Secondary objectives include decoding metabolic, microstructural, and functional signatures of successful aging at the laminar level; characterizing disease-specific patterns of cortical and spinal microstructure associated with physical and cognitive dysfunction; describing longitudinal mesoscale and metabolic changes; and generating representative normative imaging datasets for the neuroscience community. The study plans to enroll a total of 540 patients across 9 neurological conditions:Multiple Sclerosis (MS), Neuromyelitis Optica Spectrum Disorders (NMOSD), MOG Antibody Disease (MOGAD), Alzheimer's disease, Parkinson's disease, Amyotrophic Lateral Sclerosis (ALS), temporal and non-temporal epilepsy, and mild traumatic brain injury (mTBI),in addition to 160 age- and sex-matched healthy controls, totaling 700 participants. Imaging and clinical assessments will be performed at the CEMEREM center at Timone University Hospital, AP-HM, Marseille, France. Each participant will undergo multiparametric brain and spinal cord MRI, including DTI, BOLD, MP2RAGE, SWI, quantitative sodium and phosphorus imaging, and functional assessments including neuropsychological testing, visual and motor function tests. Disease-specific assessments such as OCT, evoked potentials, and disability scores (e.g., EDSS for MS) will also be included when appropriate. The study is expected to improve understanding of CNS adaptation mechanisms and support the development of more accurate diagnostic and prognostic tools for neurodegenerative diseases

Protein A immuNoaDsorption for the Treatment of Acute Episodes of Neuromyelitis Optica Spectrum Disorder

To clarify the efficacy and safety of protein A immunoadsorption therapy for acute exacerbations of neuromyelitis optica spectrum disorders(NMOSD), we designed a multicenter, open-label, superiority randomized controlled clinical trial, planning to enroll 144 patients with NMOSD. We plan to treat patients with acute NMOSD using protein A immunoadsorption combined with high-dose intravenous methylprednisolone, and compare this with treatment using high-dose intravenous methylprednisolone alone. The aim is to observe the impact and safety of protein A immunoadsorption on the treatment efficacy for these patients experiencing acute exacerbations of NMOSD, ultimately providing more comprehensive clinical evidence to support treatment protocols for the acute phase of NMOSD.

Frequency of FCGR3A Gene Polymorphisms in Patients With Neuromyelitis Optica Spectrum Disorders, Anti-oligodendrocyte Myelin Protein Antibody Disease, and Multiple Sclerosis.

The goal of this study is to assess the frequency of genetic polymorphisms of the FCG3A in a cohort of Italian patients affected by neuromyelitis optica spectrum disorder (NMOSD) and mog antibody associated disease (MOGAD) and in a a comparison group of patients affected with Multiple Sclerosis (MS). The study will involve adult patients diagnosed with MS, NMOSD, or MOGAD, followed at various clinical centers in the Lazio region. Patients from the participating clinical centers will be selected, and their medical records will be analyzed to collect clinical and neuroimaging data. The data will include demographic information such as age, sex and body mass index and clinical information such as age at disease onset, disease duration, antibody status (AQP4+/- and MOG+/-), disease-modifying therapies, as well as MRI data and the Expanded Disability Status Scale (EDSS) score. Each patient included in the study will undergo a single blood draw of approximately 5 ml of peripheral venous blood during routine blood tests, which will be used for DNA extraction and polimorphysm analysis. Demographic and clinical differences between patients with NMOSD and MOGAD, with and without the polymorphism, will be assessed and compared with the group of patients with MS.

Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.

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.

A Study to Evaluate the Efficacy and Safety of JYP0061 in Patients With Acute Neuromyelitis Spectrum Disease (NMOSD)

The goal of this clinical trial is to evaluate the efficacy and safety, of JYP0061 in acute NMOSD patients. The main questions it aims to answer are: efficacy and safety of JYP0061 in acute NMOSD patients. Participants will be treated with low-dose JYP0061 in combination with reduced dose glucocorticoids or standard dose glucocorticoids or high-dose JYP0061. Efficacy and safety evaluations will be conducted according to the protocol.

Efficacy and Safety of Divozilimab in Patients With Neuromyelitis Optica Spectrum Disorders (AQUARELLE)

The goal of this clinical trial is to study the efficacy and safety of BCD-132 (divozilimab) in subjects with neuromyelitis optica spectrum disorders (NMOSD).

Sun Yat-Sen Cohort of CNS Idiopathic Inflammatory Demyelinating Diseases

The goal of this observational study is to learn about pathogenesis and clinical prognosis of CNS IIDD in the Chinese population and to provide evidence-based clues for clinical treatment decisions. The main questions it aims to answer are: Question 1: Clarify the clinical characteristics and prognostic factors of various diseases (MS, NMOSD, MOGAD, etc.) within IIDD in the Chinese population. Question 2: Analyze the relationship between biomarkers and the occurrence, progression, and prognosis of CNS IIDD cases in our hospital. Participants will 1. Receive the recommended diagnosis and treatment plans from current international and national guidelines or expert consensus, without additional special interventions. 2. Receive clinical evaluation, follow-up, and management from dedicated neuroimmunology specialists.

MS-ResearchBiomarkerS

This study is being conducted to investigate risk factors for disability progression in Multiple Sclerosis and related disorders (MSRD). The primary goal is to assess whether combining information from visual assessment, blood markers, as well as historical and ongoing longitudinal MRIs of the brain, orbit (the part of the skull where eyes are located), and/or spinal cord can predict changes in quantitative disability measures related to MSRD and neurological disease.