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Efficacy of Prednisone In the Treatment of Ocular Myasthenia (EPITOME')
This study is currently recruiting participants.
Verified February 2012 by University of Miami

First Received on October 14, 2009.   Last Updated on February 3, 2012   History of Changes
Sponsor: Michael Benatar
Collaborator: Food and Drug Administration (FDA)
Information provided by (Responsible Party): Michael Benatar, University of Miami
ClinicalTrials.gov Identifier: NCT00995722
  Purpose

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


Condition Intervention Phase
Ocular Myasthenia Gravis
Drug: Prednisone
Drug: Placebo
Phase III

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study

Resource links provided by NLM:


Further study details as provided by University of Miami:

Primary Outcome Measures:
  • The proportion of subjects who 'fail treatment' during the four months of the double blind phase of the study. [ Time Frame: 4 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Time to sustained minimal manifestation status [ Time Frame: 4 months ] [ Designated as safety issue: No ]
  • Time to an ocular quantitative myasthenia gravis (QMG) score of zero [ Time Frame: 4 months ] [ Designated as safety issue: No ]
  • Change in quality of life as measured by the NEI-VFQ-25, the 10-item neuro-ophthalmological supplement to the VFQ-25 and the MG-QoL-15. [ Time Frame: 4 months ] [ Designated as safety issue: No ]
  • Occurrence of adverse events [ Time Frame: 4 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 88
Study Start Date: December 2011
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prednisone
Corticosteroid
Drug: Prednisone
Prednisone and placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone or matching placebo.
Other Name: Steroid
Placebo Comparator: Placebo
Matched, inactive substance
Drug: Placebo
Prednisone and placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone or matching placebo.
Other Name: Sugar pill

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 or an inadequate trial of pyridostigmine (where inadequate is defined as a trial in which the dosage was titrated to neither efficacy [MMS] nor adverse events that did not resolve with the addition of a selective muscarinic antagonist)
  • 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) > 2 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 > 30kg/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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00995722

Contacts
Contact: Michael Benatar, MBChB, DPhil mbenatar@med.miami.edu
Contact: Farheen Hussain, MS Farheen_Hussain@URMC.Rochester.edu

Locations
United States, Florida
Michael Benatar Recruiting
Miami, Florida, United States, 33136
Contact: Michael Benatar, MBChB, DPhil     305-243-6480     mbenatar@med.miami.edu    
Contact: Alexandra Waltz, MA     305-243-3799     awaltz@med.miami.edu    
Principal Investigator: Michael Benatar, MBChB, DPhil            
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Richard Barohn, MD     913-588-6094     rbarohn@kumc.edu    
Contact: Laura Herbelin     913-588-5095     lherbelin@kumc.edu    
Principal Investigator: Richard Barohn, MD            
United States, North Carolina
Duke University Medical Center Not yet recruiting
Durham, North Carolina, United States, 27710
Contact: Vern Juel, MD            
Principal Investigator: Vern Juel, MD            
United States, Texas
University of Texas Southwestern Medical Center Not yet recruiting
Dallas, Texas, United States, 75390
Contact: Srikanth Muppidi, MD            
Principal Investigator: Srikanth Muppidi, MD            
United States, Virginia
University of Virginia Not yet recruiting
Charlottesville, Virginia, United States, 22908
Contact: Ted Burns, MD            
Principal Investigator: Ted Burns, MD            
Sponsors and Collaborators
Michael Benatar
Food and Drug Administration (FDA)
Investigators
Principal Investigator: Michael Benatar, MBChB, DPhil University of Miami
Study Director: Gil Wolfe, MD University of Texas Southwestern Medical Center
Study Director: Donald Sanders, MD Duke University
  More Information

No publications provided

Responsible Party: Michael Benatar, MBChB, DPhil, University of Miami
ClinicalTrials.gov Identifier: NCT00995722     History of Changes
Other Study ID Numbers: FD-R-03710-01
Study First Received: October 14, 2009
Last Updated: February 3, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Miami:
Ocular myasthenia gravis
Prednisone
Steroids

Additional relevant MeSH terms:
Myasthenia Gravis
Muscle Weakness
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Pathologic Processes
Signs and Symptoms
Prednisone
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Therapeutic Uses
Anti-Inflammatory Agents

ClinicalTrials.gov processed this record on February 09, 2012