Different immunosuppressants and treatments for new-onset ocular myasthenia gravis

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

This project tries different immunosuppressants and symptomatic treatments to see which works best and is safest for adults with new-onset ocular myasthenia gravis.

Quick facts

• Study type: Observational
• Enrollment: 200 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Tang-Du Hospital (Academic / other)
• Locations: 1 site (Xi'an, Shaanxi)
• Trial ID: NCT04182984 on ClinicalTrials.gov

What this trial studies

This multicenter observational cohort collects real-world clinical data from adults with new-onset ocular myasthenia gravis at neurology departments across several hospitals in China. Investigators record clinical manifestations, laboratory results (including antibody testing), chest imaging, and thymectomy history, and collect peripheral blood samples annually. Treatment choices—including pyridostigmine, steroids, or immunosuppressants such as azathioprine, tacrolimus, or mycophenolate mofetil—are made by physicians together with patient preference, and patients are followed prospectively to monitor outcomes and adverse events. The study aims to enroll about 200 patients to compare treatment outcomes and identify risk factors for conversion to generalized myasthenia gravis.

Who should consider this trial

Good fit: Adults aged 18–74 with new-onset ocular myasthenia gravis who meet diagnostic criteria, have not received prior treatment for OMG, and agree to testing and regular follow-up are ideal candidates.

Not a fit: Patients who already have generalized myasthenia gravis at presentation, who have received prior immunosuppressive therapy, who are pregnant, or who cannot attend in-person follow-up visits are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, the study could identify which treatments reduce ocular symptoms, lower side effects, and decrease the risk of progression to generalized myasthenia gravis.

How similar studies have performed: Previous trials and observational studies show steroids and several immunosuppressants can improve symptoms in myasthenia gravis and may reduce conversion risk, but direct comparative real-world data focused on ocular-only onset remain limited.

Eligibility criteria

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.

Where this trial is running

Xi'an, Shaanxi

• Tangdu Hospotal — Xi'an, Shaanxi, China (Recruiting)

Study contacts

• Study coordinator: Jun Guo, M.D.

Conditions Ocular Myasthenia Gravis; Ocular myasthenia gravis