Ocular Myasthenia GravisPhase 3EPITOME'NCT00995722
Efficacy of Prednisone In the Treatment of Ocular Myasthenia
A Phase 3 study testing Prednisone in people with Ocular Myasthenia Gravis, run by Michael Benatar at 10 sites in 2 countries.
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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ocular Myasthenia Gravis
- What is being tested?
- Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 11 participants
- Where
- 10 sitesin 2 countries
- Study dates
- Dec 2011to Oct 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
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- 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
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States8 sites
University at Buffalo, Buffalo General Medical Center
Buffalo, New York, 14203
University of Vermont
Burlington, Vermont, 05401
University of Virginia
Charlottesville, Virginia, 22908
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Miami
Miami, Florida, 33136
Yale University
New Haven, Connecticut, 06510
Canada2 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2P4
Toronto General Hospital
Toronto, Ontario, M5G 2C4
About this study
Official title: Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study
The purpose of this study is to evaluate the efficacy and tolerability of prednisone in patients diagnosed with ocular myasthenia.
Funding Source - FDA OOPD
Read the 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.
- Conditions
- Ocular Myasthenia Gravis
- Being tested
- Prednisone, Placebo
- Keywords
- Ocular myasthenia gravis, Prednisone, Steroids
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
Also called Steroid
Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.
PlaceboDrug
Also called Sugar pill
Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.
| Study group | Role | What they receive |
|---|---|---|
| Prednisone + Pyridostigmine | Experimental | Corticosteroid Receives: Drug: Prednisone |
| Placebo + Pyridostigmine | Placebo comparator | Matched, inactive substance Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Failure
Failure to achive sustatined minimal manifestation status by week 16
Measured: 4 months
Secondary outcomes (4)
Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16
Measured: 4 months
Change in Quality of Life as Measured by the NEI-VFQ-25 Measures
Measured: 4 months
Change in Quality of Life as Measured by the MG-QOL-15 Score
Measured: 4 Months
Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Oct 2009 (estimated)
- Study startDec 2011
- Primary completionOct 2013
- Study completionOct 2013
Why it stopped: Slow recruitment rate
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Ocular Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Michael Benatar
- Sponsor type
- University, hospital or other
- Collaborators
- University of Miami, University of Rochester
- Responsible party
- Sponsor-investigator, Michael Benatar, University of Miami
- Investigators
- Michael Benatar, MBChB, DPhil, Principal investigator, University of MiamiGil Wolfe, MD, Study director, State University of New York at BuffaloDonald Sanders, MD, Study director, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00995722
- Sponsor's protocol ID
- FD-R-03710-01
- Acronym
- EPITOME'
- Status last verified
- May 2017
- First submitted
- 14 Oct 2009
- Last updated
- 2 Jun 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study
- ClinicalTrials.gov ID
- NCT00995722
- Acronym / sponsor ID
- EPITOME' / FD-R-03710-01
- Conditions
- Ocular Myasthenia Gravis
- Keywords
- Ocular myasthenia gravis, Prednisone, Steroids
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow recruitment rate
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Michael BenatarUniversity, hospital or other
- Collaborators
- University of Miami, University of Rochester
- Study start
- Dec 2011
- Primary completion
- Oct 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 15 Oct 2009 (estimated)
- Results
- Posted 2 Nov 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prednisone + Pyridostigmine | Experimental | Drug: PrednisoneCorticosteroid |
| Placebo + Pyridostigmine | Placebo comparator | Drug: PlaceboMatched, inactive substance |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PrednisoneAlso: SteroidPrednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone. | Drug | Prednisone + Pyridostigmine |
| PlaceboAlso: Sugar pillPlacebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo. | Drug | Placebo + Pyridostigmine |
Primary outcome
Treatment Failure
Time frame: 4 months
Failure to achive sustatined minimal manifestation status by week 16
4 secondary outcomes
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Benatar, MBChB, DPhil principal investigator
University of Miami
Gil Wolfe, MD study director
State University of New York at Buffalo
Donald Sanders, MD study director
Duke University
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone in people with Ocular Myasthenia Gravis, run by Michael Benatar at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Nov 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment Failure (time frame: 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2017.