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

This study has been terminated.
(Slow recruitment rate)
Sponsor:
Collaborators:
University of Miami
University of Rochester
Information provided by (Responsible Party):
Michael Benatar, University of Miami
ClinicalTrials.gov Identifier:
NCT00995722
First received: October 14, 2009
Last updated: May 5, 2014
Last verified: May 2014
  Purpose

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 Intervention Phase
Ocular Myasthenia Gravis
Drug: Prednisone
Drug: Placebo
Phase 3

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 ]

Enrollment: 11
Study Start Date: December 2011
Study Completion Date: October 2013
Primary Completion Date: October 2013 (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 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00995722

Locations
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
Investigators
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
  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: May 5, 2014
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 July 22, 2014