Efficacy of Prednisone In the Treatment of Ocular Myasthenia
Condition: Ocular Myasthenia Gravis
MeSH terms: Myasthenia Gravis; Muscle Weakness
Intervention: Prednisone; Placebo
Intervention description: Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.; Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.
Intervention MeSH terms: Prednisone
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Enrollment: 11.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Ocular myasthenia gravis; Prednisone; Steroids

Summary:

      The purpose of this study is to evaluate the efficacy and tolerability of prednisone in
      patients diagnosed with ocular myasthenia.

      Funding Source - FDA OOPD
    

Eligibility criteria:

        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