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Clinical Prognosis and Progression of Myasthenia Gravis Patients

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ClinicalTrials.gov Identifier: NCT04101578
Recruitment Status : Recruiting
First Posted : September 24, 2019
Last Update Posted : August 23, 2021
Sponsor:
Information provided by (Responsible Party):
Da, Yuwei, M.D.

Brief Summary:
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.

Condition or disease Intervention/treatment
Myasthenia Gravis Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

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.

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Study Type : Observational
Estimated Enrollment : 2000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients
Actual Study Start Date : February 8, 2017
Estimated Primary Completion Date : June 1, 2023
Estimated Study Completion Date : December 31, 2024

Resource links provided by the National Library of Medicine


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 Name: 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 Name: Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate




Primary Outcome Measures :
  1. 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.

  2. 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).

  3. 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.

  4. 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).


Secondary Outcome Measures :
  1. 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.

  2. 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.


Biospecimen Retention:   Samples With DNA
Peripheral blood samples are collected annually.


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Ages Eligible for Study:   15 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with Myasthenia Gravis
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04101578


Contacts
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Contact: yuwei Da, M.D. 00-86-010-83198492 dayuwei1000@163.com

Locations
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China, Beijing
Xuan Wu Hospital, Capital Medical University Recruiting
Beijing, Beijing, China, 100053
Contact: yuwei Da, M.D.    00-86-010-83198492    dayuwei1000@163.com   
Sponsors and Collaborators
Da, Yuwei, M.D.
Investigators
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Study Chair: yuwei Da, M.D. Xuan Wu Hospital, Capital Medical University
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Responsible Party: Da, Yuwei, M.D.
ClinicalTrials.gov Identifier: NCT04101578    
Other Study ID Numbers: 2017YFC0907705
First Posted: September 24, 2019    Key Record Dates
Last Update Posted: August 23, 2021
Last Verified: August 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Pathologic Processes
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Cyclosporine
Mycophenolic Acid
Prednisone
Methylprednisolone
Cyclophosphamide
Methotrexate
Azathioprine
Bromides
Tacrolimus
Cyclosporins
Immunosuppressive Agents
Immunoglobulins
Immunoglobulins, Intravenous
Antibodies
gamma-Globulins
Rho(D) Immune Globulin
Pyridostigmine Bromide