TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study (TREAT-OMG)

August 16, 2022 updated by: Jun Guo, MD, Tang-Du Hospital
This study collects the clinical data of new-onset ocular myasthenia gravis (OMG) patients, assesses outcomes and adverse effects of different treatment options, and evaluate risk factors of conversion to generalized MG(GMG).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a multicenter, observational cohort trial in the real-world clinical setting recruiting new-onset OMG patients from Neurology Departments of 7 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Treatment option 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. The investigators plan to recruit a final sample of 200 patients for analysis.

Study Type

Observational

Enrollment (Anticipated)

200

Contacts and Locations

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

Study Contact

Study Locations

  • China
    • Shaanxi
      • Xi'an, Shaanxi, China
        • Recruiting
        • Tangdu Hospotal
        • Contact:

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

16 years to 73 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with newly-onset ocular myasthenia gravis

Description

Inclusion Criteria:

  1. Age >18 years and<75 years;

  2. Clinical Diagnosis of MG with supporting evidence:

    Patients with ocular muscle type MG who have never received treatment meet one of the diagnoses (1) and (2)-(5): (1) The patient's symptoms involve the extraocular muscles, except for drooping eyelids and diplopia, no other clinical symptoms; (2) unequivocal clinical response to pyridostigmine; (3) positive acetylcholine receptor antibody or musk antibody; (4) decrement of more than 10% in repetitive nerve stimulations study (RNS); (5) the "trembling" of the single fiber electromyography (SFEMG) is broadened with or without blockade;

  3. Willingness to sample collection, imaging study and other disease-related examinations and assessments;
  4. The results of pregnancy tests for female subjects with fertility during the screening period should be negative and effective contraception was used by the patient and her spouse during the study period;
  5. Patients with informed consent;
  6. Predicted survival time is longer than 3 years.

Exclusion Criteria:

  1. History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue;
  2. Patients who may have other diseases that may lead to eyelid drooping, peripheral muscle weakness or diplopia;
  3. Age ≤18 years or ≥75 years;
  4. Patients who have been taking glucocorticoids or associated immunosuppressants due to other immune system diseases;
  5. Patients who cannot use immunosuppressants due to other chronic diseases;
  6. Patients who are unable to cooperate with follow-up and self-assessment due to severe mental illness or cognitive impairment;
  7. Pregnant women, lactating women and patients with fertility plans during the trial;
  8. Patients who have suffered from severe infections or malignant tumors in the last 1 month and are unable to receive immunosuppressants treatments;
  9. Patients who are not willing to cooperate with repeated frequency electrical stimulation tests and chest CT examinations;
  10. Patients who are not willing to participate in this study;
  11. Patients who are unable to sign informed consent;
  12. Predicted survival time is shorter than 3 years;
  13. Patients who are not suitable to participated in the trial after researchers' assessment.

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

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with autoimmune ocular MG
Newly-onset OMG patients who agreed to join the follow-up cohort
Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents( azathioprine, tacrolimus, Mycophenolate Mofetil)
Treatment regimens are determined according to the physician's judgment and preferences of the patients.
Other Names:
  • Pyridostigmine Bromide, Prednisone,Azathioprine, Tacrolimus, Mycophenolate Mofetil,

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Conversion rate from ocular to generalized MG at the last follow-up visit.
Time Frame: 144 weeks
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.
144 weeks
Changes in the ratio of patients who achieve the level 2 of MG Status and Treatment Intensity (MGSTI) scale or better.
Time Frame: Baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
Ocular MG patients are followed up to determine the ratio of patients that achieve the status of MGSTI 2 or better at each period piont of follow-up. The clinical records will be retrospectively analyzed to search for risk factors prolonged the regimens reduction.
Baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
Changes in proportion of patients with treatment-related adverse events.
Time Frame: Baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
Treatment-Related Adverse Events (AEs) are evaluated in patients receiving different treatment protocol
Baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
Changes in scores of Quantitative Myasthenia Gravis (QMG) scale and of Absolute and Relative Score of MG(ARS-MG) scales from Baseline.
Time Frame: 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
The improvement of clinic status of OMG patients was assessed by investigators according to QMG and ARS-MG score. 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.The ARS-MG is a 8-item scale which has a more detailed measure to assess ocular function . The total score ranges from 0 (no myasthenic findings) to 60 (maximal myasthenic deficits) obtained by summing the responses to each individual item.
12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
Changes in scores of MG-specific Activities of Daily Living (MG-ADL) scale from Baseline.
Time Frame: 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
The improvement of clinic status of OMG patients was assessed by patients themselves according to MG-ADL score. The 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.
12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in titers of MG antibodies.
Time Frame: Baseline, 48 weeks, 96 weeks, 144 weeks
MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.
Baseline, 48 weeks, 96 weeks, 144 weeks
Improvement of repeated frequency electrical stimulation tests (RNS) and single fiber electromyography (SFEMG).
Time Frame: 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
RNS and SFEMG will be monitored on time to assess the clinic status.
12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks
Relapse rate during follow-up period.
Time Frame: Baseline, 144 weeks
Ocular MG patients are followed up to determine the relapse rate at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of recurrence.
Baseline, 144 weeks

Collaborators and Investigators

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

Sponsor

Tang-Du Hospital

Investigators

  • Study Chair: Jun Guo, Tangdu hospital, Air force Military 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)

November 4, 2019

Primary Completion (Anticipated)

November 4, 2024

Study Completion (Anticipated)

November 4, 2024

Study Registration Dates

First Submitted

November 26, 2019

First Submitted That Met QC Criteria

November 29, 2019

First Posted (Actual)

December 2, 2019

Study Record Updates

Last Update Posted (Actual)

August 18, 2022

Last Update Submitted That Met QC Criteria

August 16, 2022

Last Verified

August 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2019TDSNOMG

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

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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