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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
11
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.
Yale University
New Haven, Connecticut, United States
University of Miami
Miami, Florida, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Treatment Failure
Failure to achive sustatined minimal manifestation status by week 16
Time frame: 4 months
Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16
Time frame: 4 months
Change in Quality of Life as Measured by the NEI-VFQ-25 Measures
Time frame: 4 months
Change in Quality of Life as Measured by the MG-QOL-15 Score
Time frame: 4 Months
Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25
Time frame: 4 months
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University at Buffalo, Buffalo General Medical Center
Buffalo, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Vermont
Burlington, Vermont, United States
University of Virginia
Charlottesville, Virginia, United States
University of Alberta Hospital
Edmonton, Alberta, Canada
Toronto General Hospital
Toronto, Ontario, Canada