Efficacy of Prednisone In the Treatment of Ocular Myasthenia
Efficacy of Prednisone In the Treatment of Ocular Myasthenia
The purpose of this study is to evaluate the efficacy and tolerability of prednisone in
 patients diagnosed with ocular myasthenia.

 Funding Source - FDA OOPD
The purpose of this study is to learn two things about prednisone in patients with ocular
 myasthenia. The first thing we aim to learn is whether or not prednisone is effective in
 improving the symptoms of double vision and drooping eyes that are experienced by patients
 with ocular myasthenia. The second thing we aim to learn is whether we can find a dose of
 prednisone that is well tolerated and safe. The overall goal is to find out whether a dose of
 prednisone that is safe and well tolerated is also effective in improving the symptoms of
 ocular myasthenia.

 After completing screening assessments to confirm eligibility, all participants will receive
 treatment with pyridostigmine. If a participant's symptoms do not resolve within the first
 month while being treated with pyridostigmine, they will be randomized to receive prednisone
 or placebo. The amount of study medication a participant receives will depend on how their
 symptoms respond to the medication and if they experience any side effects.

 After four months, participants that continue to have symptoms of ocular myasthenia and do
 not have side effects will receive open label high dose prednisone. Participants that no
 longer have symptoms will taper their dose of study drug in a double-blind fashion.
Inclusion Criteria:

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

 -  At least one of the following combinations of abnormal diagnostic testing: a) Elevated
 acetylcholine 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 any
 muscle, (d) Positive ice test and brain MRI that demonstrates no central nervous
 system pathology that mimics ocular myasthenia, or (e) Positive Tensilon test and
 brain MRI that demonstrate no central nervous system pathology that mimics ocular
 myasthenia

 -  Either no prior treatment with pyridostigmine, or participant has persistent ocular
 symptoms that are functionally limiting or troublesome despite treatment with
 pyridostigmine.

 -  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 complications
 that may arise as a consequence of steroid therapy

 -  Willing to be randomized to a trial of prednisone or placebo if symptoms respond
 inadequately 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 immune
 suppressive medication since onset of MG unless dosages of these medications and/or
 duration of therapy with these medications are clinically insignificant in the
 judgment 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 of
 osteoporotic fracture)

 -  Pregnant or lactating

 -  Renal failure, active thyroid or hepatocellular disease, chronic infection, poorly
 controlled cardiac disease, unstable psychiatric illness, untreated major depression
 or any other illness that would, in the opinion of the treating neurologist, make it
 unsafe for the patient to participate in the trial

 -  Receipt of another investigational drug within 30 days of Screening