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

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ClinicalTrials.gov Identifier: NCT00995722
Recruitment Status : Terminated (Slow recruitment rate)
First Posted : October 15, 2009
Results First Posted : November 2, 2015
Last Update Posted : June 2, 2017
Sponsor:
Collaborators:
University of Miami
University of Rochester
Information provided by (Responsible Party):
Michael Benatar, University of Miami

Tracking Information
First Submitted Date  ICMJE October 14, 2009
First Posted Date  ICMJE October 15, 2009
Results First Submitted Date  ICMJE September 28, 2015
Results First Posted Date  ICMJE November 2, 2015
Last Update Posted Date June 2, 2017
Study Start Date  ICMJE December 2011
Actual Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 1, 2015)
Treatment Failure [ Time Frame: 4 months ]
Failure to achive sustatined minimal manifestation status by week 16
Original Primary Outcome Measures  ICMJE
 (submitted: October 14, 2009)
The proportion of subjects who 'fail treatment' during the four months of the double blind phase of the study. [ Time Frame: 4 months ]
Change History Complete list of historical versions of study NCT00995722 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 1, 2015)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: October 14, 2009)
  • Time to sustained minimal manifestation status [ Time Frame: 4 months ]
  • Time to an ocular quantitative myasthenia gravis (QMG) score of zero [ Time Frame: 4 months ]
  • Change in quality of life as measured by the NEI-VFQ-25, the 10-item neuro-ophthalmological supplement to the VFQ-25 and the INQoL. [ Time Frame: 4 months ]
  • Occurrence of adverse events [ Time Frame: 4 Months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy of Prednisone In the Treatment of Ocular Myasthenia
Official Title  ICMJE Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study
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

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Ocular Myasthenia Gravis
Intervention  ICMJE
  • Drug: Prednisone
    Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.
    Other Name: Steroid
  • Drug: Placebo
    Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.
    Other Name: Sugar pill
Study Arms  ICMJE
  • Experimental: Prednisone + Pyridostigmine
    Corticosteroid
    Intervention: Drug: Prednisone
  • Placebo Comparator: Placebo + Pyridostigmine
    Matched, inactive substance
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: November 7, 2013)
11
Original Estimated Enrollment  ICMJE
 (submitted: October 14, 2009)
88
Actual Study Completion Date  ICMJE October 2013
Actual Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00995722
Other Study ID Numbers  ICMJE FD-R-03710-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Michael Benatar, University of Miami
Study Sponsor  ICMJE Michael Benatar
Collaborators  ICMJE
  • University of Miami
  • University of Rochester
Investigators  ICMJE
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
PRS Account University of Miami
Verification Date May 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP