Clinical Prognosis and Progression of Myasthenia Gravis Patients

August 19, 2021 updated by: Da, Yuwei, M.D.

Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients

This study collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a multicenter, observational cohort trial in the real-world clinical setting recruiting MG patients from Neurology Departments of 6 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Patients will be classified by clinical manifestation as well as antibody status, and treatment regimens are determined according to the physician's judgment and preferences of the patients. Patients are followed up prospectively on regular to assess the outcomes of treatments and monitor any side effects. Peripheral blood samples are collected annually. Patients' clinical records are uploaded to an online database. The investigators plan to recruit a final sample of 2000 patients for analysis.

Study Type

Observational

Enrollment (Anticipated)

2000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100053
        • Recruiting
        • Xuan Wu Hospital, Capital Medical University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

15 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with Myasthenia Gravis

Description

Inclusion Criteria:

  1. Age >14.

  2. Clinical Diagnosis of MG with supporting evidence:

    1. unequivocal clinical response to pyridostigmine
    2. positive antibody testing
    3. decrement >10% in repetitive nerve stimulations study (RNS) .
  3. Willingness to sample collection, imaging study and other disease-related examinations and assessments.
  4. Patients with informed consent.

Exclusion Criteria:

  1. History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
  2. Age ≤14 years.
  3. Severe anxiety, depression or schizophrenia.
  4. Cognitive impairment or mini-mental state examination (MMSE) score ≤24.
  5. Severe systemic illness with life-expectancy less than 4 years.
  6. Unwillingness to consent for collection of biological samples.
  7. Inability to provide informed consent.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Ocular MG
Patients with autoimmune MG whose symptoms restricted to extraocular muscles
Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)
Treatment regimens are determined according to the physician's judgment and preferences of the patients.
Other Names:
  • Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate
Generalized MG
Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure
Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)
Treatment regimens are determined according to the physician's judgment and preferences of the patients.
Other Names:
  • Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Conversion rates from ocular to generalized MG at the last visit and risk factors.
Time Frame: Baseline, 48 months
Ocular MG patients are followed up to determine the ratio of conversion to generalized disease at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of progressing.
Baseline, 48 months
Change in Quantitative Myasthenia Gravis (QMG) Scores from Baseline to 48 months.
Time Frame: Baseline, 12 months, 24 months, 36 months, 48 months
The QMG is a 13-item scale which measures ocular, bulbar, limb function and respiratory function. The total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) obtained by summing the responses to each individual item (None=0, Mild=1, Moderate=2, Severe=3).
Baseline, 12 months, 24 months, 36 months, 48 months
Change in MG-specific Activities of Daily Living scale (MG-ADL).
Time Frame: Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months
The MG-ADL is an 8-item scale to assess symptoms of myasthenia gravis patients obtained by summing the responses to each individual item (Grades: 0,1,2,3). The score ranges from 0 to 24.
Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months
The proportion of patients reaching minimal manifestations (MM) or better.
Time Frame: 48 months
Clinical statuses of patients are assessed and categorized according to Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS). MM or better includes Minimal Manifestation (MM), Pharmacologic Remission (PR) or Complete Remission (CR).
48 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of Patients with Treatment-related Adverse Experiences.
Time Frame: 3 months, 6 months, 12 months, 24 months, 36 months, 48 months
Treatment-Related Adverse Events (AEs) are evaluated in patients of different regimens.
3 months, 6 months, 12 months, 24 months, 36 months, 48 months
Changes in titers of MG antibodies.
Time Frame: Baseline, 12 months, 24 months, 36 months, 48 months
MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.
Baseline, 12 months, 24 months, 36 months, 48 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Da, Yuwei, M.D.

Investigators

  • Study Chair: yuwei Da, M.D., Xuan Wu Hospital, Capital Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

February 8, 2017

Primary Completion (ANTICIPATED)

June 1, 2023

Study Completion (ANTICIPATED)

December 31, 2024

Study Registration Dates

First Submitted

September 16, 2019

First Submitted That Met QC Criteria

September 23, 2019

First Posted (ACTUAL)

September 24, 2019

Study Record Updates

Last Update Posted (ACTUAL)

August 23, 2021

Last Update Submitted That Met QC Criteria

August 19, 2021

Last Verified

August 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2017YFC0907705

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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