A Study in Patients With Myasthenia Gravis in China (MG Cohort)

A Multicenter, Prospective Cohort Study in Patients With Myasthenia Gravis in China

This study is a multi-center, prospective cohort study designed to characterize current clinical practice, clinical and patient-reported outcomes (PROs), disease prognosis, treatment patterns and healthcare resource utilization for Chinese patients with myasthenia gravis (MG). This study will enroll patients with MG as diagnosed by physician. Approximately 1,200 MG patients are intended to be recruited from approximately 40 sites across majority of regions in China. The clinical and PROs included MGFA class, MGFA PIS, MG-ADL (Activities of Daily Living), QMG (Quantitative MG score), MG QOL-15R, EQ-5D etc. All MG patients enrolled will be followed up every 6 months until end of 2027.

Study Overview

• Status: Active, not recruiting
• Conditions: Myasthenia Gravis

• Study Type: Observational
• Enrollment (Actual): 1199

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Research Site
  • Chengdu, China
    • Research Site
  • Fuzhou, China
    • Research Site
  • Guangzhou, China
    • Research Site
  • Guiyang, China
    • Research Site
  • Harbin, China
    • Research Site
  • Hebei, China
    • Research Site
  • Henan, China
    • Research Site
  • Inner Mongolia, China
    • Research Site
  • Jiangsu, China
    • Research Site
  • Lanzhou, China
    • Research Site
  • Nanning, China
    • Research Site
  • Qingdao, China
    • Research Site
  • Taiyuan, China
    • Research Site
  • Tianjin, China
    • Research Site
  • Wenzhou, China
    • Research Site
  • Wuhan, China
    • Research Site
  • Xi'an, China
    • Research Site
  • Yinchuan, China
    • Research Site
  • Ürümqi, China
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This study will recruit patients diagnosed with MG by a physician. The diagnosis of MG patients newly diagnosed prior to enrollment should be based on the clinical characteristics of MG and at least one of the following criteria: (a) Positive serology tests for acetylcholine receptor antibody (AChR Ab), muscle-specific kinase antibody (MuSK Ab), or lipoprotein-related protein 4 (LRP4 Ab); (b) Results of electrophysiological studies (repetitive stimulation, single-fiber electromyography, or both) related to synaptic transmission disorder at the neuromuscular junction; (c) Positive pharmacological test results (such as neostigmine methylsulfate test). Previously diagnosed patients must have a documented MG diagnosis accepted by clinical physician.

Description

Inclusion Criteria:

This study will enroll patients of all ages who are diagnosed with MG of all MGFA classifications by physicians.

1. Patients must have the following data to be enrolled in this study:

   • MGFA classification
   • MG-ADL score
2. MG patients or their legal guardians/representatives are able to sign the Informed Consent Form (ICF).

Exclusion Criteria:

1. Patients aged ≥18 years with ocular myasthenia gravis (MGFA class I) lasting for more than 2 years;
2. Patients who are currently participating in an interventional clinical trial cannot be enrolled in this study.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical outcome	MGFA classification (e.g I, II, III, IV, V)	Every 6 months up to 3 years
Treatment method	Targeted treatment methods (list of methods, including medication, immunoglobulin, plasma exchange, surgery etc. )	Every 6 months up to 3 years
Patient-reported outcome	MG-Activities of Daily Living (MG-ADL, aggregated value in a scale, ranged 0- best to 24- worst)	Every 6 months up to 3 years
Clinical outcome	MGFA Post-intervention Status (e.g. CSR, PR, MM)	Every 6 months up to 3 years
Clinical outcome	Quantitative MG score (QMG, aggregated value in a scale, ranged 0- best to 39-worst)	Every 6 months up to 3 years
Clinical outcome	Physician-reported MG crisis	Every 6 months up to 3 years
Clinical outcome	Death	Every 6 months up to 3 years
Patient-reported outcome	MG-Quality of Life 15-Revised (MG-QoL15-r, aggregated value in a scale for quality of life, ranged 0- best to 60- worst)	Every 6 months up to 3 years
Patient-reported outcome	EuroQol-5D (separate value for five dimensions in a scale for quality of life)	Every 6 months up to 3 years
Treatment duration	Therapies start and end date	Every 6 months up to 3 years
Treatment dose	Dose (e.g. mg)	Every 6 months up to 3 years
Treatment frequency	Frequency (e.g. times/day)	Every 6 months up to 3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Demographic factors	Age (years); Gender (male, female); Residence place (rural, urban); Education; Household income	Baseline
Healthcare resource utilization	MG-related hospitalization (days)	Every 6 months up to 3 years
Cormobidity	Comorbidities (list of related diseases, e.g. autoimmune diseases, cardiovascular disease, hypertension, diabetes etc.)	Every 6 months up to 3 years
Healthcare resource utilization	MG-related healthcare costs	Every 6 months up to 3 years
MG diagnosis history	MG first time diagnosis date	Every 6 months up to 3 years
Vital sign - blood pressure	Blood pressure (mmHg)	Every 6 months up to 3 years
Laboratory test - blood cell test	Blood cell counts by cell type tests (e.g. counts/L)	Every 6 months up to 3 years
Laboratory tests etc.) - MG-related antibodies	MG-related antibodies including AChR, MuSK, LRP4 etc. (positive or negative).	Every 6 months up to 3 years
Vital sign - pulse	Pulse (count/minute)	Every 6 months up to 3 years

Collaborators and Investigators

• Sponsor: AstraZeneca
• Investigators: Principal Investigator: Fudong Shi, MD, PhD, Beijing Tiantan Hospital

Study record dates

Study Major Dates

• Study Start (Actual): September 26, 2024
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2027

Study Registration Dates

• First Submitted: September 30, 2024
• First Submitted That Met QC Criteria: November 19, 2024
• First Posted (Actual): November 22, 2024

Study Record Updates

• Last Update Posted (Actual): March 16, 2026
• Last Update Submitted That Met QC Criteria: March 12, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Chinese; Myasthenia Gravis; Observational Study; Cohort Study; Patient Registry; Prospective Study; Generalized Myasthenia Gravis; Refractory Myasthenia Gravis
• Additional Relevant MeSH Terms: Nervous System Diseases; 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; Myasthenia Gravis
• Other Study ID Numbers: D9030R00001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All requests will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

IPD Sharing Access Criteria

When a request has been approved AstraZeneca will provide access to the de-identified individual patient-level data in an approved sponsored tool. Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

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