Efficacy of Prednisone In the Treatment of Ocular Myasthenia

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00995722
• Date of registration: 14/10/2009
• Prospective Registration: Yes
• Primary sponsor: Michael Benatar
• Public title: Efficacy of Prednisone In the Treatment of Ocular Myasthenia EPITOME'
• Scientific title: Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study
• Date of first enrolment: December 2011
• Target sample size: 11
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00995722
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Canada, United States

Contacts

• Name: Gil Wolfe, MD
• Affiliation: State University of New York at Buffalo

• Name: Donald Sanders, MD
• Affiliation: Duke University

• Name: Michael Benatar, MBChB, DPhil
• Affiliation: University of Miami

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Ocular Myasthenia Gravis

Intervention(s)

Drug: Prednisone

Drug: Placebo

Primary Outcome(s)

Treatment Failure [Time Frame: 4 months]

Secondary Outcome(s)

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 NEI-VFQ-25 Measures [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]

Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16 [Time Frame: 4 months]

Secondary ID(s)

FD-R-03710-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

University of Rochester

University of Miami

Results

• Results available: Yes
• Date Posted: 02/11/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00995722