Trial Outcomes & Findings for Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (NCT NCT02849457)
NCT ID: NCT02849457
Last Updated: 2024-08-19
Results Overview
The primary outcome measure will be the standardized Cognitive scale scores on the Bayley Scales of Infant and Toddler Development- Third Edition at 24 months. The Cognitive scale Composite score is a standard score derived from the observed and elicited performance of the child on cognitive assessment tasks, with a mean of 100 and standard deviation of 10. The range for the Cognitive scale Composite score is 55 to 145. The score is calculated using standard procedures available in the manual for this measure. A higher score is considered better performance. The Bayley Scales of Infant and Toddler Development at 24 months will be used for the data analysis and to compare the developmental impact of early versus delayed treatment with vigabatrin.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
84 participants
Primary outcome timeframe
24 months
Results posted on
2024-08-19
Participant Flow
The PREVeNT clinical trial was conducted at 12 TSC Clinics in the U.S. with the first participant enrolled in December 2016 and last participant enrolled in March 2020. The study enrolled 84 TSC infants who were 6 months of age or younger and met the diagnostic criteria for TSC, with no history of seizures nor evidence of subclinical electrographic seizures on EEG. Participants were excluded if they were born prematurely, received any anti-seizure medication (ASM), or an mTOR inhibitor.
Participant milestones
| Measure |
Delayed Vigabatrin (Placebo)
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Enrolled, Pre-randomized Period
STARTED
|
0
|
0
|
84
|
0
|
|
Enrolled, Pre-randomized Period
COMPLETED
|
0
|
0
|
72
|
0
|
|
Enrolled, Pre-randomized Period
NOT COMPLETED
|
0
|
0
|
12
|
0
|
|
Randomized Period
STARTED
|
27
|
29
|
12
|
4
|
|
Randomized Period
COMPLETED
|
22
|
29
|
11
|
4
|
|
Randomized Period
NOT COMPLETED
|
5
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Delayed Vigabatrin (Placebo)
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Enrolled, Pre-randomized Period
Excluded prior to randomization due to discovery of exclusion criteria, e.g. prenatal mTOR inhibitor
|
0
|
0
|
12
|
0
|
Baseline Characteristics
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Baseline characteristics by cohort
| Measure |
Delayed Vigabatrin (Placebo)
n=27 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
n=12 Participants
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Total
n=68 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
1.9 Months
STANDARD_DEVIATION 1.3 • n=5 Participants
|
2.3 Months
STANDARD_DEVIATION 1.4 • n=7 Participants
|
4.4 Months
STANDARD_DEVIATION 2.1 • n=5 Participants
|
2.5 Months
STANDARD_DEVIATION 1.7 • n=4 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
34 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
34 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
23 Participants
n=5 Participants
|
28 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
61 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 24 monthsPopulation: The analysis population was the intent to treat population. Three participants withdrew prior to 24 months of age, and one participant did not complete the Bayley assessment at 24 months.
The primary outcome measure will be the standardized Cognitive scale scores on the Bayley Scales of Infant and Toddler Development- Third Edition at 24 months. The Cognitive scale Composite score is a standard score derived from the observed and elicited performance of the child on cognitive assessment tasks, with a mean of 100 and standard deviation of 10. The range for the Cognitive scale Composite score is 55 to 145. The score is calculated using standard procedures available in the manual for this measure. A higher score is considered better performance. The Bayley Scales of Infant and Toddler Development at 24 months will be used for the data analysis and to compare the developmental impact of early versus delayed treatment with vigabatrin.
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=23 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
n=11 Participants
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
n=4 Participants
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Cognitive Assessment Scores and Developmental Impact
|
83.9 score on a scale
Standard Deviation 17.3
|
80.9 score on a scale
Standard Deviation 15.6
|
97.3 score on a scale
Standard Deviation 16.9
|
85.0 score on a scale
Standard Deviation 21.6
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: One participant in the Delayed Vigabatrin group withdrew prior to 24 months and prior to the development of any seizures. They were therefore excluded from count endpoints and from cognitive assessments at 24 months, although they were included in time to event analyses (censored at drop-out).
Evaluate the number of subjects that develop seizures when treated with vigabatrin or placebo as a seizure prevention.
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=26 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Number of Subjects That Develop Seizures When Treated With Study Drug During the Randomized Phase of the Study.
|
19 Participants
|
20 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsTime to the subject's first clinical seizure will be measured for both subjects on placebo and vigabatrin.
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=27 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Time to the Subject's First Clinical Seizure From Randomization
|
2.77 Months from randomization
Interval 1.1 to 11.07
|
9.47 Months from randomization
Interval 4.8 to 22.27
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Three participants in the Delayed Vigabatrin group withdrew prior to age 24 months and could therefore not be classified as has having drug resistant epilepsy or not at age 24 months.
The count of participants with drug resistant epilepsy. Drug resistant epilepsy classified according to International League Against Epilepsy (ILAE) definition, specifically defined as any participant on 2 or more anti-seizure medications experiencing persistent seizures (seizures occurring within 3 months of the 24 month participant visit).
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=24 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Count of Participants With Drug Resistant Epilepsy at 24 Months of Age.
|
14 Participants
|
14 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 months, 24 months and 36 monthsPopulation: The analysis population was the intent to treat population. Three participants withdrew prior to 24 months of age, and 2 withdrew prior to 12 months of age.
The range for the Vineland-II Adaptive Behavior Composite is 20 to 160 The Vineland-II ABC standard score has a mean of 100 and standard deviation of 15, with higher scores indicating better overall adaptive functioning. The ABC standard score is a composite derived from obtained scores on the Communication, Daily Living Skills, Socialization, and Motor Skills domains on the Vineland-II and is calculated according to standardized procedures described in the Vineland-II manual.
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=25 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
n=12 Participants
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
n=4 Participants
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Evaluate Vineland II ABC Scores and Impact of Early Versus Late Treatment
12 months
|
86.9 score on a scale
Standard Deviation 13.1
|
86.0 score on a scale
Standard Deviation 9.8
|
97.8 score on a scale
Standard Deviation 14.2
|
86.3 score on a scale
Standard Deviation 7.0
|
|
Evaluate Vineland II ABC Scores and Impact of Early Versus Late Treatment
24 months
|
90.6 score on a scale
Standard Deviation 14.3
|
84.9 score on a scale
Standard Deviation 11.9
|
96.3 score on a scale
Standard Deviation 5.8
|
85.3 score on a scale
Standard Deviation 10.7
|
|
Evaluate Vineland II ABC Scores and Impact of Early Versus Late Treatment
36 months
|
86.2 score on a scale
Standard Deviation 17.5
|
78.6 score on a scale
Standard Deviation 14.0
|
93.6 score on a scale
Standard Deviation 8.8
|
76.8 score on a scale
Standard Deviation 14.4
|
SECONDARY outcome
Timeframe: 24 months and 36 monthsPopulation: This outcome was not assessed because it required an in-person evaluation that could not be performed while wearing a face mask, which would have invalidated the assessment, and was thus eliminated as an outcome measure at the start of the COVID-19 pandemic.
Evaluate ADOS2 scores and the impact of early versus late treatment at 24 and 36 months.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsNumber of subjects with vigabatrin related adverse events, severe adverse events as assessed by CTCAE v4.0 and risk evaluation and mitigation strategy (REMS) measures as required by the FDA.
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=27 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
Number of Subjects With Vigabatrin Related Adverse Events and Severe Adverse Events
|
6 Participants
|
2 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsFeasibility of the routine 1 hour video EEG in determining the EEG biomarker for developing epilepsy. Outcome was determined as the number of participants developing seizures amongst those developing the biomarker (epileptiform activity).
Outcome measures
| Measure |
Delayed Vigabatrin (Placebo)
n=26 Participants
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Vigabatrin
n=29 Participants
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
n=11 Participants
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin)
n=4 Participants
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin.
|
|---|---|---|---|---|
|
EEG Biomarker for Developing Epilepsy
|
19 Participants
|
20 Participants
|
0 Participants
|
4 Participants
|
Adverse Events
Delayed Vigabatrin (Placebo), Pre-randomization
Serious events: 3 serious events
Other events: 4 other events
Deaths: 0 deaths
Early Vigabatrin, Pre-randomization
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Delayed Vigabatrin (Placebo), Post-randomization
Serious events: 14 serious events
Other events: 15 other events
Deaths: 0 deaths
Early Post-randomization
Serious events: 15 serious events
Other events: 17 other events
Deaths: 0 deaths
Watchful Waiting (Control Group)
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Watchful Waiting (Open Label Vigabatrin), Before Use of Vigabatrin
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Watchful Waiting (Open Label Vigabatrin), After Starting Vigabatrin
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Delayed Vigabatrin (Placebo), Pre-randomization
n=27 participants at risk
Patients who were eventually randomized to the Delayed Vigabatrin (Placebo) arm. The adverse events reflected in this arm occurred prior to randomization and initiation of study drug.
|
Early Vigabatrin, Pre-randomization
n=29 participants at risk
Patients who were eventually randomized to the Early Vigabatrin arm. The adverse events reflected in this arm occurred prior to randomization and initiation of study drug.
|
Delayed Vigabatrin (Placebo), Post-randomization
n=27 participants at risk
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Post-randomization
n=29 participants at risk
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
n=12 participants at risk
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin), Before Use of Vigabatrin
n=4 participants at risk
Participants in this group eventually had a seizure and were placed on open label vigabatrin; however, the adverse events in this arm occurred prior to beginning open label vigabatrin.
|
Watchful Waiting (Open Label Vigabatrin), After Starting Vigabatrin
n=4 participants at risk
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin. The adverse events in this arm occurred after beginning open label vigabatrin.
|
|---|---|---|---|---|---|---|---|
|
Infections and infestations
Infections and infestations
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
7.4%
2/27 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Investigations
Investigations
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Nervous system disorders
Nervous system disorders
|
7.4%
2/27 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
40.7%
11/27 • Number of events 30 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
34.5%
10/29 • Number of events 16 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
50.0%
2/4 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
50.0%
2/4 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Cardiac disorders
Cardiac disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
11.1%
3/27 • Number of events 3 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
13.8%
4/29 • Number of events 6 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Surgical and medical procedures
Surgical and medical procedures
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Congenital, familial and genetic disorders
Congenital, familial and genetic disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Eye disorders
Eye disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
8.3%
1/12 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
Other adverse events
| Measure |
Delayed Vigabatrin (Placebo), Pre-randomization
n=27 participants at risk
Patients who were eventually randomized to the Delayed Vigabatrin (Placebo) arm. The adverse events reflected in this arm occurred prior to randomization and initiation of study drug.
|
Early Vigabatrin, Pre-randomization
n=29 participants at risk
Patients who were eventually randomized to the Early Vigabatrin arm. The adverse events reflected in this arm occurred prior to randomization and initiation of study drug.
|
Delayed Vigabatrin (Placebo), Post-randomization
n=27 participants at risk
Study drug (in this case, placebo) is given for administration, the entire content of one sachet (500 mg placebo) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Early Post-randomization
n=29 participants at risk
Study drug (in this case, vigabatrin) is given for administration, the entire content of one sachet (500 mg vigabatrin) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID).
Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
|
Watchful Waiting (Control Group)
n=12 participants at risk
Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.
|
Watchful Waiting (Open Label Vigabatrin), Before Use of Vigabatrin
n=4 participants at risk
Participants in this group eventually had a seizure and were placed on open label vigabatrin; however, the adverse events in this arm occurred prior to beginning open label vigabatrin.
|
Watchful Waiting (Open Label Vigabatrin), After Starting Vigabatrin
n=4 participants at risk
Participants in this group experienced seizures prior to detection of EEG epileptiform activity and where immediately placed on open label Vigabatrin. The adverse events in this arm occurred after beginning open label vigabatrin.
|
|---|---|---|---|---|---|---|---|
|
Immune system disorders
Immune system disorders
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
11.1%
3/27 • Number of events 4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
10.3%
3/29 • Number of events 3 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
8.3%
1/12 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Infections and infestations
Infections and infestations
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
6.9%
2/29 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.9%
7/27 • Number of events 30 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
27.6%
8/29 • Number of events 24 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
41.7%
5/12 • Number of events 9 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
6.9%
2/29 • Number of events 3 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
8.3%
1/12 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Investigations
Investigations
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Nervous system disorders
Nervous system disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
13.8%
4/29 • Number of events 4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Psychiatric disorders
Psychiatric disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Cardiac disorders
Cardiac disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
3.4%
1/29 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
14.8%
4/27 • Number of events 5 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
24.1%
7/29 • Number of events 9 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
33.3%
4/12 • Number of events 7 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
7.4%
2/27 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
6.9%
2/29 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
14.8%
4/27 • Number of events 6 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
13.8%
4/29 • Number of events 4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
16.7%
2/12 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
50.0%
2/4 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Surgical and medical procedures
Surgical and medical procedures
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
8.3%
1/12 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders
|
3.7%
1/27 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
11.1%
3/27 • Number of events 4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
10.3%
3/29 • Number of events 7 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
3/12 • Number of events 3 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Eye disorders
Eye disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
11.1%
3/27 • Number of events 3 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
10.3%
3/29 • Number of events 3 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
8.3%
1/12 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
3.7%
1/27 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
11.1%
3/27 • Number of events 5 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
6.9%
2/29 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
16.7%
2/12 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
General disorders
General disorders
|
7.4%
2/27 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
11.1%
3/27 • Number of events 8 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
13.8%
4/29 • Number of events 11 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 2 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
|
Renal and urinary disorders
Renal and urinary disorders
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/27 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/29 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/12 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
25.0%
1/4 • Number of events 1 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
0.00%
0/4 • Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.
|
Additional Information
E. Martina Bebin M.D., M.P.A.
University of Alabama at Birmingham
Phone: 205-975-2890
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place