Efficacy of Prednisone In the Treatment of Ocular Myasthenia (EPITOME')
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| ClinicalTrials.gov Identifier: NCT00995722 |
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Recruitment Status :
Terminated
(Slow recruitment rate)
First Posted : October 15, 2009
Results First Posted : November 1, 2015
Last Update Posted : June 2, 2017
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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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Ocular Myasthenia Gravis | Drug: Prednisone Drug: Placebo | Phase 3 |
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 : | Interventional (Clinical Trial) |
| Actual Enrollment : | 11 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study |
| Study Start Date : | December 2011 |
| Actual Primary Completion Date : | October 2013 |
| Actual Study Completion Date : | October 2013 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Prednisone + Pyridostigmine
Corticosteroid
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Drug: Prednisone
Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.
Other Name: Steroid |
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Placebo Comparator: Placebo + Pyridostigmine
Matched, inactive substance
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Drug: Placebo
Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.
Other Name: Sugar pill |
- Treatment Failure [ Time Frame: 4 months ]Failure to achive sustatined minimal manifestation status by week 16
- 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 ]
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00995722
| 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 | |
| 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 |
| Responsible Party: | Michael Benatar, MBChB, DPhil, University of Miami |
| ClinicalTrials.gov Identifier: | NCT00995722 |
| Other Study ID Numbers: |
FD-R-03710-01 |
| First Posted: | October 15, 2009 Key Record Dates |
| Results First Posted: | November 1, 2015 |
| Last Update Posted: | June 2, 2017 |
| Last Verified: | May 2017 |
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Ocular myasthenia gravis Prednisone Steroids |
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Myasthenia Gravis Muscle Weakness Muscular Diseases Musculoskeletal Diseases Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathologic Processes Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Neoplasms Paraneoplastic Syndromes Autoimmune Diseases of the Nervous System |
Neurodegenerative Diseases Neuromuscular Junction Diseases Neuromuscular Diseases Autoimmune Diseases Immune System Diseases Prednisone Anti-Inflammatory Agents Glucocorticoids Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs Antineoplastic Agents, Hormonal Antineoplastic Agents |