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NCT Number: NCT06241521

Myasthenia Gravis Registry in China

Myasthenia gravis (MG) is an autoimmune disease caused by dysfunction at the neuromuscular junction, characterized by partial or generalized skeletal muscle weakness and fatigability. The estimated annual incidence rate of MG in China is 0.68/100,000, with a high rate of relapse and poor treatment compliance. This study is a prospective, observational, multi-center patient registry across China. To support standardized management and follow-up of MG patients in China, a Cloud-based MG Patient Management Platform (CN MG Base) was established in September 2023 with the support of the Chinese Rare Diseases Alliance. The platform aims to collect longitudinal clinical data including demographic information, age of onset, medical history, comorbidities, medication usage, treatment responses, and others. It is intended to collect newly registered MG cases each year and follow up with these patients at one-year intervals for ten years

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Key information

Sex eligibility

All sexes

Study type

Observational

Primary location

Beijing Hospital, Beijing, Beijing Municipality, China

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About this study

This study is a observational nationwide multicenter MG registry research. Starting from the baseline, annual follow-ups will be conducted to prospectively collect information on treatment medications, prognosis, and safety events. The following classification definitions can be applied: ① MG clinical subtypes (ocular, early-onset generalized, thymoma-related, late-onset generalized, MuSK-related, and seronegative); ② Different treatment methods (classic immunotherapy group, B-cell-targeted therapy, complement inhibition therapy, rapid-acting therapy, and IL-6 pathway inhibitors, etc.).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Fluctuating weakness in skeletal muscles, such as extraocular, bulbar, limb muscles, etc.. Demonstrating fluctuation and fatigability: Weakness is usually worse in the morning and improves with rest; worsens with sustained activity.
  • Patients themselves or their guardians can understand and sign the informed consent form.

To meet the eligibility criteria, at least one of the following additional criteria must be met:

  • a. Positive Tensilon test;
  • b. Decrease in compound muscle action potential by more than 10% with low-frequency repetitive nerve stimulation or widening of the "jitter" (the variability in time of the second action potential relative to the first) on single-fiber electromyography, with or without blocking;
  • c. Positive antibodies: Serum testing positive for AChR antibodies, MuSK antibodies, or LRP4 antibodies.

Exclusion criteria

  • Patients with mental illness who cannot cooperate.
  • Patients with multiple organ dysfunction or in extremely critical condition, unable to complete relevant medical history collection or evaluation.

Treatment and study plan

Primary outcomes

  1. MSE proportion

    Time frame: Ten years

    The proportion of patients achieving minimal symptom expression (MSE, defined as reaching an ADL score of 0 or 1) after treatment.

Secondary outcomes

  1. Safety events

    Time frame: Ten years

    Safety events including upper respiratory tract infection, lower respiratory tract infection, urinary tract infection, and other infections;

  2. Relapse of MG symptoms

    Time frame: Ten years

    Having reached MSE for over a year but experiencing a symptom relapse, manifested as an increase in ADL score greater than or equal to 2 points.

Study contacts

Contact information is provided by the study sponsor or research team.

Sushan Luo, MD, PhD

CONTACT

[email protected]

86 2152889999

Sponsors and collaborators

Lead sponsor

Huashan Hospital

Other

Collaborators

  • China Alliance for Rare Diseases
  • China myasthenia gravis collaborating group (CMGCG)
  • National Center for Neurological Disorders (China)

Registry information

Important dates

Study start
2024
Primary completion
2034
Study completion
2035
First posted
Feb 5, 2024
Registry last updated
Feb 5, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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