Precision Diagnosis and Prospective Cohort Study for Myasthenia Gravis: Multicenter Analysis in China

The present study is a prospective cohort study aiming to improve the clinical capacity in the diagnosis and natural history of Chinese patients with myasthenia gravis (MG). 300 MG patients are planned to recruit, document and prospectively follow up. Management of screening test and cohort manifestation are studied.

Study Overview

• Status: Recruiting
• Conditions: Myasthenia Gravis
• Intervention / Treatment: Other: Biomarkers and omics data

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

• Study Contact: Name: Chongbo Zhao, MD; Phone Number: 86-021-52889999; Email: zhao_chongbo@fudan.edu.cn

Study Locations

• China
  • Shanghai, China, 200040
    • Recruiting
    • Huashan Hospital
    • Contact: Chongbo Zhao, MD; Phone Number: +86 21 52889999; Email: zhao_chongbo@fudan.edu.cn
  • Shanghai
    • Shanghai, Shanghai, China, 200000
      • Recruiting
      • Shanghai Chest Hospital
      • Contact: Changlu Wang, MD; Phone Number: 86-021-22200000
    • Shanghai, Shanghai, China, 201102
      • Recruiting
      • Children's Hospital of Fudan University
      • Contact: Wenhui Li, MD; Phone Number: 86-021-64931923
    • Shanghai, Shanghai, China, 200011
      • Recruiting
      • Obsterics and Gynecology Hospital of Fudan University
      • Contact: Weirong Gu, MD; Phone Number: 86-021-33189900

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 86 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients from the outpatient clinics and inpatient wards are planned to be enrolled, as long as meeting the inclusion criteria.

Description

Inclusion Criteria:

• fluctuating muscle weakness and fatigability, along with one of the below:
• more than 10% amplitude decrement in low frequency repetitive nerve stimulation,less than 10% amplitude increment in high frequency repetitive nerve stimulation;
• anti-AChR or MuSK antibody positivity;
• positive to the neostigmine test;
• understanding and assigning the informed consent form, and having a good compliance with the follow up.

Exclusion Criteria:

• excluding the possible diagnosis of Lambert-Eaton syndrome, congenital myasthenia syndrome,botulism injection, chronic progressive extraocular ophthalmoplegia, etc;
• poor compliance to the follow up.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
myasthenia gravis; 300 MG patients are anticipated for precision diagnosis and disease monitoring.	Other: Biomarkers and omics data; To detect disease-related biomarkers and omics data, in this prospective MG cohort.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Myasthenia gravis foundation of America post intervention status	Myasthenia gravis foundation of America post intervention status (PIS) is assessed by the investigators during follow up. According to history inquiry and physical examination, participants are rated as clinical remission (no complain of myasthenia, no weakness at physical examination, and no therapies concerning MG for one year), pharmocological remission (no complain of myasthenia, no weakness at physical examination, but undertaking MG therapies at the last year), minimal manifestation (no complain of weakness, but showing weakness at physical examination), improvement (symptoms and signs), unchanged (symptoms and signs) , worsening(symptoms and signs), exacerbation(symptoms and signs), and death (medical record).	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at the follow up	A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity.	Baseline, 1 year, 2 year, and 3 year
Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at the follow up	The MG-QOL15 is helpful in informing the clinician about the patient's perception of the extent of and dissatisfaction with MG-related dysfunction. Total MG-QOL15 score range from 0 (none) to 60 (extreme disease severity), higher score indicated more disease severity.	Baseline, 1 year, 2 year, and 3 year
Change From Baseline in Activities of Daily Living (ADL) Scores at the follow up	The MG-ADL scale assesses the impact of MG on patients' daily functions. Physicians use this tool to score a patient's MG symptoms based on the patient's recall of symptoms during the prior week. A person's score can range from zero (normal) to 24 (most severe).	Baseline, 1 year, 2 year, and 3 year

Collaborators and Investigators

• Sponsor: Huashan Hospital
• Investigators: Study Chair: Chongbo Zhao, MD, Huashan Hospital, Shanghai,China

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2024
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): May 1, 2028

Study Registration Dates

• First Submitted: August 23, 2020
• First Submitted That Met QC Criteria: August 27, 2020
• First Posted (Actual): September 2, 2020

Study Record Updates

• Last Update Posted (Actual): May 28, 2025
• Last Update Submitted That Met QC Criteria: May 27, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: myasthenia gravis; precision diagnosis; antibody detection
• Additional Relevant MeSH Terms: Neurologic Manifestations; Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Manifestations; Pathologic Processes; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: 2020-999

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No