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

NCT04182984 | Recruiting FDA Drug

• 1 other identifier: 2019TDSNOMG
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Ocular Myasthenia Gravis

Keywords

Ocular myasthenia gravis

Outcome Measures

Primary Outcomes (5)

• Conversion rate from ocular to generalized MG at the last follow-up visit.

  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.

  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.

  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.

  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.

  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 Outcomes (3)

• Changes in titers of MG antibodies.

  Baseline, 48 weeks, 96 weeks, 144 weeks

• Improvement of repeated frequency electrical stimulation tests (RNS) and single fiber electromyography (SFEMG).

  12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks

• Relapse rate during follow-up period.

  Baseline, 144 weeks

Study Arms (1)

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)

Interventions

Symptomatic Treatment, Steroids, Immunosuppressive Agents( azathioprine, tacrolimus, Mycophenolate Mofetil) DRUG

Treatment regimens are determined according to the physician's judgment and preferences of the patients.

Also known as: Pyridostigmine Bromide, Prednisone,Azathioprine, Tacrolimus, Mycophenolate Mofetil,

Patients with autoimmune ocular MG

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with newly-onset ocular myasthenia gravis

You may qualify if:

• Age \>18 years and\<75 years;
• 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;
• Willingness to sample collection, imaging study and other disease-related examinations and assessments;
• 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;
• Patients with informed consent;
• Predicted survival time is longer than 3 years.

You may not qualify if:

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

Sponsors & Collaborators

• Tang-Du Hospital: lead

Study Sites (1)

Tangdu Hospotal

Xi'an, Shaanxi, China

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Steroids; Tacrolimus; Mycophenolic Acid; Pyridostigmine Bromide; Prednisone; Azathioprine

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Fused-Ring Compounds; Polycyclic Compounds; Macrolides; Lactones; Organic Chemicals; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Pyridinium Compounds; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pregnadienediols; Pregnadienes; Pregnanes; Thionucleosides; Sulfur Compounds; Mercaptopurine; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides

Study Officials

• Jun Guo

  Tangdu Hospital, Air Force Military Medical University

  STUDY CHAIR

Central Study Contacts

Jun Guo, M.D.

CONTACT

86-29-8477 8844; guojun_81@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: November 26, 2019
• First Posted: December 2, 2019
• Study Start: November 4, 2019
• Primary Completion (Estimated): April 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: January 30, 2026

Record last verified: 2026-01

Locations

CN (1)