Clinical trial NCT06241521: Myasthenia Gravis Registry in China
Official title: Myasthenia Gravis Registry in China
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myasthenia Gravis
Keywords: Myasthenia Gravis
Prospective study
Observational study
Patient registry
Lead sponsor: Huashan Hospital
Sponsor class: OTHER
Collaborators: name: China Alliance for Rare Diseases
name: National Center for Neurological Disorders (China)
name: China myasthenia gravis collaborating group (CMGCG)
First posted: 2024-02-05
Enrollment: 1500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: MG patients mostly of Chinese Han ethnicity (ocular MG type no more than 30%).

Brief summary: 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

Detailed description: 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.).

Eligibility criteria: 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.

Primary outcomes:
measure: MSE proportion
timeFrame: Ten years
Secondary outcomes:
measure: Safety events
timeFrame: Ten years
measure: Relapse of MG symptoms
timeFrame: Ten years
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Hospital
state: Beijing
city: Beijing
country: China
facility: Xuanwu hospital, Capital medical university
state: Beijing
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Chongqing
country: China
facility: The First Affiliated Hospital of Chongqing Medical University
state: Chongqing
city: Guangzhou
country: China
facility: The First Affiliated Hospital, Sun Yat-sen University
state: Guangdong
city: Guangzhou
country: China
facility: The Third Hospital, Sun Yat-sen University
state: Guangdong
city: Zhengzhou
country: China
facility: First Affiliated Hospital of Zhengzhou University
state: Henan
city: Changsha
country: China
facility: Xiangya hospital, Central south university
state: Hunan
city: Suzhou
country: China
facility: The First Hospital, Soochow University
state: Jiangsu
city: Xuzhou
country: China
facility: Affiliated Hospital of Xuzhou Medical University
state: Jiangsu
city: Nanchang
country: China
facility: The First Affiliated Hospital of Nanchang University
state: Jiangxi
city: Changchun
country: China
facility: The First Hospital, Jilin University
state: Jilin
city: Shanghai
country: China
facility: Huashan Hospital, Fudan University
state: Shanghai
city: Xi'an
country: China
facility: Tangdu Hospital, The Air Force Medical University
state: Shanxi
city: Chengdu
country: China
facility: West China Hospital, Sichuan University
state: Sichuan
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
state: Tianjin
Study officials:
References: