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

NCT04535843 — Recruiting, 500 participants, Huashan Hospital

ClinicalTrials Daily  ·  Areas  ·  Conditions  ·  Drugs  ·  Sponsors  ·  
RegistryNCT04535843
StatusRecruiting
Study typeObservational
Enrollment500 (Estimated)
SponsorHuashan Hospital (Other)
ConditionsMyasthenia Gravis
InterventionsBiomarkers and omics data (Other)
Datesstart 2024-02-01, primary completion 2028-02-01 (estimated), completion 2028-05-01 (estimated), first posted 2020-09-02, last updated 2025-05-28
Locations4 sites in China

Related Papers Related Patents Related Grants Related Experts
All trials for Myasthenia Gravis All trials of Biomarkers and omics data

-Summary

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.

Expand all sections  ·  Collapse all

+Arms and interventions

ArmTypeInterventionsDescription
myasthenia gravisOther: Biomarkers and omics data300 MG patients are anticipated for precision diagnosis and disease monitoring.
  • Biomarkers and omics data (Other): To detect disease-related biomarkers and omics data, in this prospective MG cohort.

+Outcome measures

Primary outcomes

  • Myasthenia gravis foundation of America post intervention status [3 years]: 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).

Secondary outcomes

  • Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at the follow up [Baseline, 1 year, 2 year, and 3 year]: 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.
  • Change From Baseline in Activities of Daily Living (ADL) Scores at the follow up [Baseline, 1 year, 2 year, and 3 year]: 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).
  • Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at the follow up [Baseline, 1 year, 2 year, and 3 year]: 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.

+Eligibility

Sex: All, ages: 0 Years to 90 Years

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.

+Locations (4)

FacilityCityCountryStatus
Shanghai Chest HospitalShanghai, Shanghai MunicipalityChinaRecruiting
Obsterics and Gynecology Hospital of Fudan UniversityShanghai, Shanghai MunicipalityChinaRecruiting
Children's Hospital of Fudan UniversityShanghai, Shanghai MunicipalityChinaRecruiting
Huashan HospitalShanghaiChinaRecruiting

+Publications

  • Results publication: Jin L, Zou Z, Wang Q, Zeng W, Jiang Q, Chen J, Shi J, Yu Y, Hong D, Zeng Q, Tan S, Yue Y, Zhang Z, Zhang Y, Guo X, Du L, Zhao Z, Huang S, Chen Y, Wu Z, Yan C, Xi J, Song J, Luo S, Zhao C. Patterns and predictors of therapeutic response to efgartigimod in acetylcholine receptor-antibody generalized myasthenia gravis subtypes. Ther Adv Neurol Disord. 2025 Feb 18;18:17562864251319656. doi: 10.1177/17562864251319656. eCollection 2025. PMID 39974170
  • Results publication: Jin L, He D, Zeng Q, Tan S, Shi J, Liu Y, Zou Z, Song J, Yan C, Huan X, Wang Y, Yang L, Xi J, Wu Z, Liu Z, Zheng J, Zhao C, Chu X, Luo S. Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study. Ther Adv Neurol Disord. 2024 Dec 25;17:17562864241309431. doi: 10.1177/17562864241309431. eCollection 2024. PMID 39735403

+Investigators