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Efficacy of Prednisone In the Treatment of Ocular Myasthenia (EPITOME')

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00995722
Recruitment Status : Terminated (Slow recruitment rate)
First Posted : October 15, 2009
Results First Posted : November 1, 2015
Last Update Posted : June 2, 2017
University of Miami
University of Rochester
Information provided by (Responsible Party):
Michael Benatar, University of Miami

Brief 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

Condition or disease Intervention/treatment Phase
Ocular Myasthenia Gravis Drug: Prednisone Drug: Placebo Phase 3

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study
Study Start Date : December 2011
Actual Primary Completion Date : October 2013
Actual Study Completion Date : October 2013

Resource links provided by the National Library of Medicine

Drug Information available for: Prednisone

Arm Intervention/treatment
Experimental: Prednisone + Pyridostigmine
Drug: Prednisone
Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.
Other Name: Steroid

Placebo Comparator: Placebo + Pyridostigmine
Matched, inactive substance
Drug: Placebo
Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.
Other Name: Sugar pill

Primary Outcome Measures :
  1. Treatment Failure [ Time Frame: 4 months ]
    Failure to achive sustatined minimal manifestation status by week 16

Secondary Outcome Measures :
  1. Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16 [ Time Frame: 4 months ]
  2. Change in Quality of Life as Measured by the NEI-VFQ-25 Measures [ Time Frame: 4 months ]
  3. Change in Quality of Life as Measured by the MG-QOL-15 Score [ Time Frame: 4 Months ]
  4. Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25 [ Time Frame: 4 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00995722

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United States, Connecticut
Yale University
New Haven, Connecticut, United States, 06510
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, New York
University at Buffalo, Buffalo General Medical Center
Buffalo, New York, United States, 14203
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
United States, Vermont
University of Vermont
Burlington, Vermont, United States, 05401
United States, Virginia
University of Virginia
Charlottesville, Virginia, United States, 22908
Canada, Alberta
University of Alberta Hospital
Edmonton, Alberta, Canada, T6G 2P4
Canada, Ontario
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
Sponsors and Collaborators
Michael Benatar
University of Miami
University of Rochester
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Principal Investigator: Michael Benatar, MBChB, DPhil University of Miami
Study Director: Gil Wolfe, MD State University of New York at Buffalo
Study Director: Donald Sanders, MD Duke University
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Responsible Party: Michael Benatar, MBChB, DPhil, University of Miami
ClinicalTrials.gov Identifier: NCT00995722    
Other Study ID Numbers: FD-R-03710-01
First Posted: October 15, 2009    Key Record Dates
Results First Posted: November 1, 2015
Last Update Posted: June 2, 2017
Last Verified: May 2017
Keywords provided by Michael Benatar, University of Miami:
Ocular myasthenia gravis
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents