Efficacy of Prednisone In the Treatment of Ocular Myasthenia

Inclusion Criteria:

• Weakness confined to the extra-ocular muscles, eyelid levator and eye closure with anocular-QMG1 score ≥ 1

• At least one of the following combinations of abnormal diagnostic testing: a) Elevatedacetylcholine receptor antibody titers, (b) Abnormal repetitive nerve stimulation (> 10% decrement following slow repetitive nerve stimulation) of any nerve-muscle pair, (c) Abnormal jitter on single fiber or concentric needle electromyography in anymuscle, (d) Positive ice test and brain MRI that demonstrates no central nervoussystem pathology that mimics ocular myasthenia, or (e) Positive Tensilon test andbrain MRI that demonstrate no central nervous system pathology that mimics ocularmyasthenia

• Either no prior treatment with pyridostigmine, or participant has persistent ocularsymptoms that are functionally limiting or troublesome despite treatment withpyridostigmine.

• Age 18 years or older, male or female

• Capable of providing informed consent and complying with study procedures

• Identifiable primary care physician to assist with management of medical complicationsthat may arise as a consequence of steroid therapy

• Willing to be randomized to a trial of prednisone or placebo if symptoms respondinadequately to pyridostigmine.

Exclusion Criteria:

• Disease duration (time since symptom onset) > 5 years

• Treatment with prednisone or other corticosteroids within 90 days of randomization

• Treatment with azathioprine, cyclosporine, mycophenolate mofetil or other immunesuppressive medication since onset of MG unless dosages of these medications and/orduration of therapy with these medications are clinically insignificant in thejudgment of the PI

• Intravenous immunoglobulin or plasma exchange within 90 days of randomization

• Prior thymectomy or history of thymoma

• Contraindication to steroids (poorly controlled diabetes, glaucoma or hypertension,history of prior steroid intolerance, obesity [BMI > 39.9kg/m2] or a history ofosteoporotic fracture)

• Pregnant or lactating

• Renal failure, active thyroid or hepatocellular disease, chronic infection, poorlycontrolled cardiac disease, unstable psychiatric illness, untreated major depressionor any other illness that would, in the opinion of the treating neurologist, make itunsafe for the patient to participate in the trial

• Receipt of another investigational drug within 30 days of Screening