Trial Outcomes & Findings for Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (NCT NCT01399047)
NCT ID: NCT01399047
Last Updated: 2019-05-21
Results Overview
The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Baseline to 8 weeks
Results posted on
2019-05-21
Participant Flow
Participant milestones
| Measure |
Group A: Mycophenolate to Placebo Crossover
Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.
|
Group B: Placebo to Mycophenolate Crossover
Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
9
|
|
Overall Study
COMPLETED
|
10
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Group A: Mycophenolate to Placebo Crossover
Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.
|
Group B: Placebo to Mycophenolate Crossover
Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Baseline characteristics by cohort
| Measure |
Group A: Mycophenolate to Placebo Crossover
n=10 Participants
Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.
|
Group B: Placebo to Mycophenolate Crossover
n=9 Participants
Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
10.50 years
STANDARD_DEVIATION 5.99 • n=99 Participants
|
14.78 years
STANDARD_DEVIATION 2.44 • n=107 Participants
|
12.53 years
STANDARD_DEVIATION 5.04 • n=206 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Race · White/Caucasian
|
9 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Race · More than one Race
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=99 Participants
|
9 participants
n=107 Participants
|
19 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline to 8 weeksPopulation: All subjects.
The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.
Outcome measures
| Measure |
Mycophenolate
n=19 Participants
Includes data from all subjects while on mycophenolate, regardless of treatment order.
|
Placebo
n=19 Participants
Includes data from all subjects while on placebo, regardless of treatment order.
|
|---|---|---|
|
Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose
|
17 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: Baseline to 8 weeksPopulation: All subjects who completed 8 weeks of the respective treatment arm assignment.
The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Motor impairment was measured by the physical subscale of the UBDRS with a range of 0-112 with zero indicating better outcome. The overall treatment effect was determined - mean difference in physical subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Outcome measures
| Measure |
Mycophenolate
n=17 Participants
Includes data from all subjects while on mycophenolate, regardless of treatment order.
|
Placebo
n=18 Participants
Includes data from all subjects while on placebo, regardless of treatment order.
|
|---|---|---|
|
Unified Batten Disease Rating Scale Physical Subscale Change
|
-1.18 units on a scale
Interval -4.39 to 2.04
|
2.17 units on a scale
Interval -1.02 to 5.35
|
SECONDARY outcome
Timeframe: Baseline to 8 weeksPopulation: All subjects who completed 8 weeks of the respective treatment arm assignment.
The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Seizure severity was measured by the seizure subscale of the UBDRS with a range of 0-54 with zero indicating better outcome. The overall treatment effect was determined - mean difference in seizure subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Outcome measures
| Measure |
Mycophenolate
n=17 Participants
Includes data from all subjects while on mycophenolate, regardless of treatment order.
|
Placebo
n=18 Participants
Includes data from all subjects while on placebo, regardless of treatment order.
|
|---|---|---|
|
Unified Batten Disease Rating Scale Seizure Subscale Change
|
-0.24 units on a scale
Interval -1.15 to 0.68
|
-1.44 units on a scale
Interval -3.03 to 0.14
|
SECONDARY outcome
Timeframe: Baseline to 8 weeksPopulation: All subjects who completed 8 weeks of the respective treatment arm assignment.
The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Mood and behavior severity was measured by the behavior subscale of the UBDRS with a range of 0-55 with zero indicating better outcome. The overall treatment effect was determined - mean difference in behavior subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Outcome measures
| Measure |
Mycophenolate
n=17 Participants
Includes data from all subjects while on mycophenolate, regardless of treatment order.
|
Placebo
n=18 Participants
Includes data from all subjects while on placebo, regardless of treatment order.
|
|---|---|---|
|
Unified Batten Disease Rating Scale Behavior Subscale Change
|
-1.28 units on a scale
Interval -4.12 to 1.56
|
-0.37 units on a scale
Interval -2.94 to 2.2
|
SECONDARY outcome
Timeframe: Baseline to 8 weeksPopulation: All subjects who completed 8 weeks of the respective treatment arm assignment.
The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Capability severity was measured by the capability subscale of the UBDRS with a range of 0-14 with higher scores indicating better outcome. The overall treatment effect was determined - mean difference in capability subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.
Outcome measures
| Measure |
Mycophenolate
n=17 Participants
Includes data from all subjects while on mycophenolate, regardless of treatment order.
|
Placebo
n=18 Participants
Includes data from all subjects while on placebo, regardless of treatment order.
|
|---|---|---|
|
Unified Batten Disease Rating Scale Capability Subscale Change
|
0.33 units on a scale
Interval -0.18 to 0.85
|
0.47 units on a scale
Interval -0.02 to 0.97
|
Adverse Events
Mycophenolate
Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mycophenolate
n=19 participants at risk
Includes data from all subjects while on mycophenolate, regardless of order.
|
Placebo
n=19 participants at risk
Includes data from all subjects while on placebo, regardless of order.
|
|---|---|---|
|
Metabolism and nutrition disorders
Hyperkalemia
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
Other adverse events
| Measure |
Mycophenolate
n=19 participants at risk
Includes data from all subjects while on mycophenolate, regardless of order.
|
Placebo
n=19 participants at risk
Includes data from all subjects while on placebo, regardless of order.
|
|---|---|---|
|
Cardiac disorders
ECG Abnormality
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Ear and labyrinth disorders
Haemotympanum
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Ear and labyrinth disorders
Ear pain
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Endocrine disorders
Weight decreased
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Eye disorders
Photophobia
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Vomiting
|
31.6%
6/19 • Number of events 8
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 3
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Diarrhea
|
15.8%
3/19 • Number of events 3
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Retching
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Abdominal Discomfort
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
General disorders
Oedema peripheral
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
General disorders
Chest pain
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
General disorders
Fatigue
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Otitis media
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Strep pharyngitis
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Sinusitis
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Upper Respiratory Infection
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Gingivitis
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Ear Infection
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Infections and infestations
Otitis externa
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Blood prolactin increased
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Weight increased
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Blood calcium decreased
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Cardiac murmur
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Electrocardiogram abnormality
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Electrocardiogram T wave inversion
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Investigations
Protein urine present
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Musculoskeletal and connective tissue disorders
Muscle Stiffness
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Musculoskeletal and connective tissue disorders
Muscle twitching
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Headache
|
15.8%
3/19 • Number of events 3
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
15.8%
3/19 • Number of events 3
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Tremor
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Dyskinesia
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Insomnia
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Aphasia
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Convulsion
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Disorientation
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Dysarthria
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Motor dysfunction
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Movement disorder
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Seizure
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Speech disorder
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Dizziness
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Nervous system disorders
Ataxia
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Abnormal behavior
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Mental status changes
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Mood altered
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Aggression
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Anger
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Irritability
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Nightmare
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Psychiatric disorders
Perseveration
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Renal and urinary disorders
Haematuria
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.8%
3/19 • Number of events 3
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
10.5%
2/19 • Number of events 2
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
0.00%
0/19
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
5.3%
1/19 • Number of events 1
Reporting of adverse events were sought at each encounter and were considered in the evaluation of each safety assessment (laboratory studies, electrophysiology).
|
Additional Information
Erika Augustine, MD, MS
University of Rochester Medical Center
Phone: 585-275-2808
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place