Tongji NADs Cohort

Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-80
SponsorTongji Hospital

About this trial

Neurological Autoimmune Diseases (NADs) are disorders caused by abnormal immune system attacks on neural tissues, affecting multiple systems including the central nervous system, peripheral nervous system, and neuromuscular junctions. This study examines clinically significant NADs such as multiple sclerosis (MS), neuromyelitis optica spectrum disorders (NMOSD), myelin oligodendrocyte glycoprotein G antibody-related diseases (MOGAD), autoimmune encephalitis (AE), immune-mediated peripheral neuropathy (PN), myasthenia gravis (MG), and idiopathic inflammatory myopathy (IIM). While sharing the core pathogenesis of autoimmune response, these diseases exhibit significant heterogeneity in epidemiological patterns, clinical manifestations, therapeutic approaches, and disease progression. This heterogeneity stems from multiple factors: (1) Differences in immune targets: MS primarily involves T-cell-mediated myelin attack, NMOSD is mainly driven by astrocyte damage caused by anti-AQP4 antibodies, MOGAD results from myelin surface loss mediated by antibodies against myelin oligodendrocyte glycoprotein immunoglobulin G, while AE involves synaptic dysfunction due to antibodies against neuronal surface proteins (e.g., anti-NMDA-R antibodies); (2) Genetic-environmental interactions: MS is more prevalent in European and American populations, whereas NMOSD is more aggressive in Asian populations; (3) Variability in treatment response: Some diseases respond well to immunomodulatory therapy, but most still face challenges such as high relapse rates, progressive disability accumulation, and irreversible neurological damage.

While randomized controlled trials (RCTs) provide high-quality core evidence for drug registration, their strict inclusion/exclusion criteria, relatively homogeneous patient populations, and short-term observation designs often fail to fully capture the complex disease progression and treatment response patterns in real-world clinical settings. Additionally, long-term RCTs are frequently constrained by economic factors and sustainability challenges. Therefore, conducting comprehensive real-world observational studies (RWS) on NADs-integrating multi-disease cohorts, long-term follow-up data, and diverse clinical practices-holds significant scientific and clinical value for optimizing treatment strategies and improving long-term patient outcomes.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Participants must meet the following eligibility criteria at the time of screening to participate in this study.

≥2 clinical episodes with ≥2 objective clinical evidences of lesions.

≥2 clinical episodes with one clear historical evidence of the lesion involving specific anatomical site.

≥2 clinical episodes with objective clinical evidence of one lesion, and spatial multiplicity confirmed by clinical episodes in different CNS sites or MRI findings.

Disqualifiers

This is an observational study with no specific exclusion criteria.

The investigators identified other unexplained factors that may have disqualified participants from enrollment.

Trial population

This study aims to enroll NADs patients meeting inclusion criteria at our center from March 1, 2022 to March 1, 2032. The planned retrospective and prospective cohort will include: 300 multiple sclerosis (MS) cases, 300 AQP4 antibody-positive neuromyelitis optica spectrum disorders (NMDs), 200 MOG antibody-associated diseases, 150 autoimmune encephalitis (AE) cases, 80 immune-mediated peripheral neuropathy (IMPN) cases, 400 myasthenia gravis (MG) cases, and 120 idiopathic inflammatory myopathy (IIM) cases.

Trial design

Design model

Cohort

Time perspective

Cross-sectional

Treatments tested in this trial

  • No active interventions

    Other intervention

    The therapies are based on needs of the participants

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

1

EDSS Score

EDSS Score is the most commonly used in evaluating the neurological functions of patients with central nervous system demyelination. The range is 0-10.0.

Time frame
10 years from the date of enrollment
2

modified Rankin Score

Modified Rankin score is the most commonly used in evaluating the daily life ability of patients with neurological disorders. The range is 0-6.

Time frame
10 years from the date of enrollment
3

MMT-8 Score

MMT-8 Score is mostly used in evaluating muscle strength of patients with myopathy. The range is 0-150.

Time frame
10 years from the date of enrollment
4

QMG Score

An authoritative quantitative scale to assess the disease severity of patients with myasthenia gravis. The range is 0-39.

Time frame
10 years from the date of enrollment

Secondary outcomes

1

Quantification of pathogenic antibodies

Typical pathogenic antibodies include anti-AQP4-IgG, anti-MOG-IgG, anti-GFAP-IgG, anti-NMDAR-IgG, anti-LGI1-IgG, anti-AMPA-IgG, anti-AchR-IgG, anti-MUSK-IgG, etc, which are tested by ELISA in mmol/l.

Time frame
10 years from the date of enrollment
2

Quantification of cytokines

Common cytokines include IL2, IL2R, IL6, IL8, IL10, TNFα, etc.

Time frame
10 years from the date of enrollment
3

Quantitative and qualitative analysis of immunocyte subtypes

Common subtypes include CD3+CD19-,CD3-CD19+,CD3+CD4+,CD3+CD8+,CD3-CD16+CD56+,CD3+CD56+, etc. They are showed in cells/ul.

Time frame
10 years from the date of enrollment

Other outcomes

Sponsors and contacts

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Tongji Hospital

Lead sponsor

Shenzhen Genocury Biotech Co., Ltd.

Collaborator

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