Trial Outcomes & Findings for Efficacy of Prednisone In the Treatment of Ocular Myasthenia (NCT NCT00995722)
NCT ID: NCT00995722
Last Updated: 2017-06-02
Results Overview
Failure to achive sustatined minimal manifestation status by week 16
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
11 participants
Primary outcome timeframe
4 months
Results posted on
2017-06-02
Participant Flow
Participant milestones
| Measure |
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.
|
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.
|
|---|---|---|
|
Double Blinded: 16 Weeks
STARTED
|
6
|
5
|
|
Double Blinded: 16 Weeks
COMPLETED
|
5
|
4
|
|
Double Blinded: 16 Weeks
NOT COMPLETED
|
1
|
1
|
|
Overall Study: 36 Weeks
STARTED
|
5
|
4
|
|
Overall Study: 36 Weeks
COMPLETED
|
3
|
2
|
|
Overall Study: 36 Weeks
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy of Prednisone In the Treatment of Ocular Myasthenia
Baseline characteristics by cohort
| Measure |
Prednisone
n=6 Participants
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.
|
Placebo
n=5 Participants
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.
|
Total
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
64 years
STANDARD_DEVIATION 18 • n=5 Participants
|
62 years
STANDARD_DEVIATION 9 • n=7 Participants
|
63 years
STANDARD_DEVIATION 13.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 monthsFailure to achive sustatined minimal manifestation status by week 16
Outcome measures
| Measure |
Prednisone
n=6 Participants
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.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Treatment Failure
|
17 percentage of participants
Interval 0.0 to 64.0
|
100 percentage of participants
Interval 48.0 to 100.0
|
SECONDARY outcome
Timeframe: 4 monthsOutcome measures
| Measure |
Prednisone
n=6 Participants
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.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16
|
-2.25 units on a scale
Interval -5.56 to 1.05
|
-0.05 units on a scale
Interval -3.71 to 3.6
|
SECONDARY outcome
Timeframe: 4 monthsOutcome measures
| Measure |
Prednisone
n=6 Participants
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.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Change in Quality of Life as Measured by the NEI-VFQ-25 Measures
|
10.7 units on a scale
Interval 4.75 to 16.64
|
4.14 units on a scale
Interval -2.54 to 10.82
|
SECONDARY outcome
Timeframe: 4 MonthsOutcome measures
| Measure |
Prednisone
n=6 Participants
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.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Change in Quality of Life as Measured by the MG-QOL-15 Score
|
-6.3 units on a scale
Interval -10.11 to -2.5
|
-2.5 units on a scale
Interval -6.3 to 1.31
|
SECONDARY outcome
Timeframe: 4 monthsOutcome measures
| Measure |
Prednisone
n=6 Participants
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.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25
|
15.28 units on a scale
Interval -0.62 to 31.17
|
-1.7 units on a scale
Interval -19.83 to 16.43
|
Adverse Events
Prednisone
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Prednisone
n=6 participants at risk
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.
|
Placebo
n=5 participants at risk
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.
|
|---|---|---|
|
Cardiac disorders
SINUS TACHYCARDIA
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Infections and infestations
DIVERTICULAR ABCESS
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Cardiac disorders
PALPITATIONS
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Gastrointestinal disorders
GI BLEED
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
Other adverse events
| Measure |
Prednisone
n=6 participants at risk
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.
|
Placebo
n=5 participants at risk
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.
|
|---|---|---|
|
Gastrointestinal disorders
NAUSEA
|
33.3%
2/6 • Number of events 2 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Gastrointestinal disorders
DIARRHEA
|
33.3%
2/6 • Number of events 2 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Nervous system disorders
HEADACHE
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Nervous system disorders
INSOMNIA
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Nervous system disorders
AGITATION
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Nervous system disorders
ANXIETY
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Cardiac disorders
ARRHYTMIA
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Nervous system disorders
BLEPHAROSPASM
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Respiratory, thoracic and mediastinal disorders
BRONCHITIS
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Gastrointestinal disorders
CONSTIPATION
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Cardiac disorders
CONDUCTION DISORDER
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Nervous system disorders
DIZZINESS
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Musculoskeletal and connective tissue disorders
EDEMA: HEAD AND NECK
|
16.7%
1/6 • Number of events 2 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Nervous system disorders
FACE FASCICULATION (RIGHT SIDE)
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Vascular disorders
HEMATOMA
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Endocrine disorders
IMPAIRED GLUCOSE TOLERANCE
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Eye disorders
EYE PAIN(POCKET IN EYE)
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Gastrointestinal disorders
DYSPHAGIA
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Nervous system disorders
EYE FASCICULLATION
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Endocrine disorders
WEIGHT GAIN
|
33.3%
2/6 • Number of events 2 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Gastrointestinal disorders
FLATULENCE
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Musculoskeletal and connective tissue disorders
MYALGIA
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Nervous system disorders
BLURRED VISION
|
16.7%
1/6 • Number of events 1 • Adverse Events After Randomization
|
0.00%
0/5 • Adverse Events After Randomization
|
|
Cardiac disorders
PALPITATIONS
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
|
Cardiac disorders
SINUS TACHYCARDIA
|
0.00%
0/6 • Adverse Events After Randomization
|
20.0%
1/5 • Number of events 1 • Adverse Events After Randomization
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place