Myasthenia Gravis Registry in China

Myasthenia Gravis Clinical Trial

Official title:

Myasthenia Gravis Registry in China

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06241521
• Other study ID #: 2023M-022
• Secondary ID: 2022YFC350130382
• Status: Recruiting
• Start date: February 1, 2024
• Est. completion date: January 30, 2035

Study information

• Verified date: January 2024
• Source: Huashan Hospital
• Contact: Sushan Luo, MD, PhD
• Phone: 86 2152889999
• Email: luosushan[@]fudan.edu.cn
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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

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.).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1500
• Est. completion date: January 30, 2035
• Est. primary completion date: February 1, 2034
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis

Locations

Country	Name	City	State
China	Beijing Hospital	Beijing	Beijing
China	Peking Union Medical College Hospital	Beijing	Beijing
China	Xuanwu hospital, Capital medical university	Beijing	Beijing
China	The First Hospital, Jilin University	Changchun	Jilin
China	Xiangya hospital, Central south university	Changsha	Hunan
China	West China Hospital, Sichuan University	Chengdu	Sichuan
China	The First Affiliated Hospital of Chongqing Medical University	Chongqing	Chongqing
China	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong
China	The Third Hospital, Sun Yat-sen University	Guangzhou	Guangdong
China	The First Affiliated Hospital of Nanchang University	Nanchang	Jiangxi
China	Huashan Hospital, Fudan University	Shanghai	Shanghai
China	The First Hospital, Soochow University	Suzhou	Jiangsu
China	Tianjin Medical University General Hospital	Tianjin	Tianjin
China	Tangdu Hospital, The Air Force Medical University	Xi'an	Shanxi
China	Affiliated Hospital of Xuzhou Medical University	Xuzhou	Jiangsu
China	First Affiliated Hospital of Zhengzhou University	Zhengzhou	Henan

Sponsors (4)

Lead Sponsor	Collaborator
Huashan Hospital	China Alliance for Rare Diseases, China myasthenia gravis collaborating group (CMGCG), National Center for Neurological Disorders (China)

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	MSE proportion	The proportion of patients achieving minimal symptom expression (MSE, defined as reaching an ADL score of 0 or 1) after treatment.	Ten years
Secondary	Safety events	Safety events including upper respiratory tract infection, lower respiratory tract infection, urinary tract infection, and other infections;	Ten years
Secondary	Relapse of MG symptoms	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.	Ten years