Efficacy of Prednisone In the Treatment of Ocular Myasthenia

Ocular Myasthenia Gravis Clinical Trial

— EPITOME'  

Official title:

Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00995722
• Other study ID #: FD-R-03710-01
• Status: Terminated
• Phase: Phase 3
• First received: October 14, 2009
• Last updated: May 1, 2017
• Start date: December 2011
• Est. completion date: October 2013

Study information

• Verified date: May 2017
• Source: University of Miami
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 11
• Est. completion date: October 2013
• Est. primary completion date: October 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis
• Ocular Myasthenia Gravis

Intervention

Drug:
• Prednisone
Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.
• Placebo
Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.

Locations

Country	Name	City	State
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	Toronto General Hospital	Toronto	Ontario
United States	University at Buffalo, Buffalo General Medical Center	Buffalo	New York
United States	University of Vermont	Burlington	Vermont
United States	University of Virginia	Charlottesville	Virginia
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	Duke University Medical Center	Durham	North Carolina
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Miami	Miami	Florida
United States	Yale University	New Haven	Connecticut

Sponsors (3)

Lead Sponsor	Collaborator
Michael Benatar	University of Miami, University of Rochester

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment Failure	Failure to achive sustatined minimal manifestation status by week 16	4 months
Secondary	Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16		4 months
Secondary	Change in Quality of Life as Measured by the NEI-VFQ-25 Measures		4 months
Secondary	Change in Quality of Life as Measured by the MG-QOL-15 Score		4 Months
Secondary	Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25		4 months