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Slow recruitment rate
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| Name | Class |
|---|---|
| University of Miami | OTHER |
| University of Rochester | OTHER |
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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
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Prednisone + Pyridostigmine | Experimental | Corticosteroid |
|
| Placebo + Pyridostigmine | Placebo Comparator | Matched, inactive substance |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Prednisone | Drug | Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Failure | Failure to achive sustatined minimal manifestation status by week 16 | 4 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16 | 4 months | |
| Change in Quality of Life as Measured by the NEI-VFQ-25 Measures | 4 months | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michael Benatar, MBChB, DPhil | University of Miami | Principal Investigator |
| Gil Wolfe, MD | State University of New York at Buffalo | Study Director |
| Donald Sanders, MD | Duke University | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University | New Haven | Connecticut | 06510 | United States | ||
| University of Miami |
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| ID | Title | Description |
|---|---|---|
| FG000 | Prednisone | Corticosteroid Prednisone: Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone. Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant. |
| FG001 | Placebo | Matched, inactive substance Placebo: Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo. Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Double Blinded: 16 Weeks |
| |||||||||||||
| Overall Study: 36 Weeks |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Prednisone | Corticosteroid Prednisone: Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone. Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Treatment Failure | Failure to achive sustatined minimal manifestation status by week 16 | Posted | Number | 95% Confidence Interval | percentage of participants | 4 months |
|
Adverse Events After Randomization
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Prednisone | Corticosteroid Prednisone: Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone. Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| SINUS TACHYCARDIA | Cardiac disorders | CTCAE 3.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| NAUSEA | Gastrointestinal disorders | OTHER | Systematic Assessment |
Early termination leading to small numbers of subjects analyzed
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Benatar | University Of Miami | 305-243-6480 | MBenatar@med.miami.edu |
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| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D011241 | Prednisone |
| D013256 | Steroids |
| D000073893 | Sugars |
| ID | Term |
|---|---|
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D000072473 | Fused-Ring Compounds |
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|
| Placebo | Drug | Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo. |
|
|
| Change in Quality of Life as Measured by the MG-QOL-15 Score |
| 4 Months |
| Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25 | 4 months |
| Miami |
| Florida |
| 33136 |
| United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| University at Buffalo, Buffalo General Medical Center | Buffalo | New York | 14203 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| University of Vermont | Burlington | Vermont | 05401 | United States |
| University of Virginia | Charlottesville | Virginia | 22908 | United States |
| University of Alberta Hospital | Edmonton | Alberta | T6G 2P4 | Canada |
| Toronto General Hospital | Toronto | Ontario | M5G 2C4 | Canada |
| NOT COMPLETED |
|
| BG001 | Placebo | Matched, inactive substance Placebo: Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo. Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Secondary | Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16 | Posted | Mean | 95% Confidence Interval | units on a scale | 4 months |
|
|
|
|
| Secondary | Change in Quality of Life as Measured by the NEI-VFQ-25 Measures | Posted | Mean | 95% Confidence Interval | units on a scale | 4 months |
|
|
|
|
| Secondary | Change in Quality of Life as Measured by the MG-QOL-15 Score | Posted | Mean | 95% Confidence Interval | units on a scale | 4 Months |
|
|
|
|
| Secondary | Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25 | Posted | Mean | 95% Confidence Interval | units on a scale | 4 months |
|
|
|
|
| 1 |
| 6 |
| 6 |
| 6 |
| EG001 | Placebo | Matched, inactive substance Placebo: Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo. Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant. | 2 | 5 | 4 | 5 |
| DIVERTICULAR ABCESS | Infections and infestations | CTCAE 3.0 | Systematic Assessment |
|
| PALPITATIONS | Cardiac disorders | CTCAE 3.0 | Systematic Assessment |
|
| GI BLEED | Gastrointestinal disorders | CTCAE 3.0 | Systematic Assessment |
|
| DIARRHEA | Gastrointestinal disorders | OTHER | Systematic Assessment |
|
| HEADACHE | Nervous system disorders | OTHER | Systematic Assessment |
|
| INSOMNIA | Nervous system disorders | OTHER | Systematic Assessment |
|
| AGITATION | Nervous system disorders | OTHER | Systematic Assessment |
|
| ANXIETY | Nervous system disorders | OTHER | Systematic Assessment |
|
| ARRHYTMIA | Cardiac disorders | OTHER | Systematic Assessment |
|
| BLEPHAROSPASM | Nervous system disorders | OTHER | Systematic Assessment |
|
| BRONCHITIS | Respiratory, thoracic and mediastinal disorders | OTHER | Systematic Assessment |
|
| CONSTIPATION | Gastrointestinal disorders | OTHER | Systematic Assessment |
|
| CONDUCTION DISORDER | Cardiac disorders | other | Systematic Assessment |
|
| DIZZINESS | Nervous system disorders | other | Systematic Assessment |
|
| EDEMA: HEAD AND NECK | Musculoskeletal and connective tissue disorders | other | Systematic Assessment |
|
| FACE FASCICULATION (RIGHT SIDE) | Nervous system disorders | other | Systematic Assessment |
|
| HEMATOMA | Vascular disorders | other | Systematic Assessment |
|
| IMPAIRED GLUCOSE TOLERANCE | Endocrine disorders | other | Systematic Assessment |
|
| EYE PAIN(POCKET IN EYE) | Eye disorders | other | Systematic Assessment |
|
| DYSPHAGIA | Gastrointestinal disorders | other | Systematic Assessment |
|
| EYE FASCICULLATION | Nervous system disorders | other | Systematic Assessment |
|
| WEIGHT GAIN | Endocrine disorders | other | Systematic Assessment |
|
| FLATULENCE | Gastrointestinal disorders | OTHER | Systematic Assessment |
|
| MYALGIA | Musculoskeletal and connective tissue disorders | OTHER | Systematic Assessment |
|
| BLURRED VISION | Nervous system disorders | CTCAE 3.0 | Systematic Assessment |
|
| PALPITATIONS | Cardiac disorders | CTCAE 3.0 | Systematic Assessment |
|
| SINUS TACHYCARDIA | Cardiac disorders | CTCAE 3.0 | Systematic Assessment |
|
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| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D011083 |
| Polycyclic Compounds |
| D002241 | Carbohydrates |
N=80, (40 per group), 80% power to detect a group difference of 30%-35% and 5% significance level.