Trial Outcomes & Findings for Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies (NCT NCT02358538)
NCT ID: NCT02358538
Last Updated: 2023-03-21
Results Overview
Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change \& Standard Deviation)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Baseline through 52 week open label period
Results posted on
2023-03-21
Participant Flow
recruitment period May 08, 2015
Participant milestones
| Measure |
CDKL5
Cyclin-dependent kinase-like 5
|
CSWS
Continuous Spike Wave in Sleep
|
Lennox-Gastaut
Lennox-Gastaut Syndrome pediatric epilepsy
|
PCDH19
Protocadherin-19
|
|---|---|---|---|---|
|
Through Week 26
STARTED
|
7
|
2
|
10
|
11
|
|
Through Week 26
COMPLETED
|
4
|
0
|
5
|
6
|
|
Through Week 26
NOT COMPLETED
|
3
|
2
|
5
|
5
|
|
52 Week Extension Period
STARTED
|
4
|
0
|
4
|
6
|
|
52 Week Extension Period
COMPLETED
|
4
|
0
|
2
|
2
|
|
52 Week Extension Period
NOT COMPLETED
|
0
|
0
|
2
|
4
|
Reasons for withdrawal
| Measure |
CDKL5
Cyclin-dependent kinase-like 5
|
CSWS
Continuous Spike Wave in Sleep
|
Lennox-Gastaut
Lennox-Gastaut Syndrome pediatric epilepsy
|
PCDH19
Protocadherin-19
|
|---|---|---|---|---|
|
Through Week 26
Adverse Event
|
0
|
1
|
1
|
2
|
|
Through Week 26
Lack of Efficacy
|
1
|
1
|
3
|
3
|
|
Through Week 26
Withdrawal by Subject
|
1
|
0
|
0
|
0
|
|
Through Week 26
Non-Compliance
|
0
|
0
|
1
|
0
|
|
Through Week 26
Family did not see benefit from IP
|
1
|
0
|
0
|
0
|
|
52 Week Extension Period
Lack of Efficacy
|
0
|
0
|
2
|
4
|
Baseline Characteristics
Content is associated with 52-week open label period (as the label indicates)
Baseline characteristics by cohort
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=2 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=10 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=11 Participants
protocadherin-19
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=7 Participants
|
2 Participants
n=2 Participants
|
10 Participants
n=10 Participants
|
11 Participants
n=11 Participants
|
30 Participants
n=30 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=11 Participants
|
0 Participants
n=30 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=11 Participants
|
0 Participants
n=30 Participants
|
|
Age, Continuous
|
7.73 years
STANDARD_DEVIATION 3.403 • n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
11.55 years
STANDARD_DEVIATION 5.728 • n=2 Participants
|
9.25 years
STANDARD_DEVIATION 3.595 • n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
8.87 years
STANDARD_DEVIATION 4.129 • n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
8.65 years
STANDARD_DEVIATION 3.551 • n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Sex: Female, Male
Female
|
4 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
1 Participants
n=2 Participants
|
4 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
6 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
14 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Sex: Female, Male
Male
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
1 Participants
n=2 Participants
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
2 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
2 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
4 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
2 Participants
n=2 Participants
|
2 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
4 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
10 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
1 Participants
n=2 Participants
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
1 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
1 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
White
|
4 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
1 Participants
n=2 Participants
|
1 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
6 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
11 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
2 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
2 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=6 Participants • Content is associated with 52-week open label period (as the label indicates)
|
0 Participants
n=14 Participants • Content is associated with 52-week open label period (as the label indicates)
|
PRIMARY outcome
Timeframe: Baseline through 52 week open label periodPopulation: MITT Population
Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change \& Standard Deviation)
Outcome measures
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=2 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=10 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=11 Participants
protocadherin-19
|
|---|---|---|---|---|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
Percent Change from baseline (at day 91)
|
-31.23 percentage of change of frequency
Standard Deviation 41.438
|
NA percentage of change of frequency
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
122.10 percentage of change of frequency
Standard Deviation 321.124
|
52.83 percentage of change of frequency
Standard Deviation 234.084
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
Percent Change from baseline (at week 26)
|
-20.55 percentage of change of frequency
Standard Deviation 60.588
|
NA percentage of change of frequency
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
125.38 percentage of change of frequency
Standard Deviation 319.051
|
46.36 percentage of change of frequency
Standard Deviation 235.661
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
Percent change from baseline (52 week OLE through month 6)
|
-54.41 percentage of change of frequency
Standard Deviation 40.286
|
NA percentage of change of frequency
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
-38.74 percentage of change of frequency
Standard Deviation 9.292
|
-19.98 percentage of change of frequency
Standard Deviation 63.644
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
Percent change from baseline (52 week OLE period)
|
-49.20 percentage of change of frequency
Standard Deviation 50.206
|
NA percentage of change of frequency
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
-37.75 percentage of change of frequency
Standard Deviation 7.891
|
-19.95 percentage of change of frequency
Standard Deviation 63.571
|
PRIMARY outcome
Timeframe: Baseline through 52-week open- label periodPopulation: MITT Population
Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Median Percent Change)
Outcome measures
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=10 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=11 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=2 Participants
protocadherin-19
|
|---|---|---|---|---|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Percent change from baseline (at Day 91)
|
-47.34 Median Percent Change in Frequency
Interval -80.9 to 36.8
|
-10.22 Median Percent Change in Frequency
Interval -68.1 to 904.3
|
-25.98 Median Percent Change in Frequency
Interval -100.0 to 723.2
|
NA Median Percent Change in Frequency
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Percent change from baseline (at week 26)
|
-37.70 Median Percent Change in Frequency
Interval -85.3 to 99.9
|
-9.19 Median Percent Change in Frequency
Interval -71.2 to 904.3
|
-24.59 Median Percent Change in Frequency
Interval -100.0 to 723.2
|
NA Median Percent Change in Frequency
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Percent change from baseline (at week 26) - with 6 duplicate diary entries removed
|
-44.4 Median Percent Change in Frequency
Interval -85.3 to 99.9
|
NA Median Percent Change in Frequency
There is no data available to support this row for this ARM. One subject in the CDKL5 cohort had duplications of data on 6 days. These data appeared to the investigator and sponsor to be erroneous. Excluding these days changes the median percent change from baseline at Week 26 to 44.4 for the CDKL5 cohort. This is reflected in the CDKL5 ARM.
|
NA Median Percent Change in Frequency
There is no data available to support this row for this ARM. One subject in the CDKL5 cohort had duplications of data on 6 days. These data appeared to the investigator and sponsor to be erroneous. Excluding these days changes the median percent change from baseline at Week 26 to 44.4 for the CDKL5 cohort. This is reflected in the CDKL5 ARM.
|
NA Median Percent Change in Frequency
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Percent change from baseline (52 week OLE through month 6)
|
-58.94 Median Percent Change in Frequency
Interval -89.4 to -10.4
|
-38.74 Median Percent Change in Frequency
Interval -45.3 to -32.2
|
-13.48 Median Percent Change in Frequency
Interval -100.0 to 59.6
|
NA Median Percent Change in Frequency
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Percent change from baseline (52 week OLE period)
|
-61.93 Median Percent Change in Frequency
Interval -89.5 to 16.6
|
-37.75 Median Percent Change in Frequency
Interval -43.3 to -32.2
|
-13.48 Median Percent Change in Frequency
Interval -99.0 to 59.6
|
NA Median Percent Change in Frequency
Both patients (2 for CSWS) discontinued so data is not available.
|
SECONDARY outcome
Timeframe: End of Week 4, End of Week 8, End of Week 17, End of Week 26, Week 44, Week 62, Week 78Population: MITT Population - n varies per visit, also CSWS is not included
Clinician Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-C scale is qualitative values and not quantitative.
Outcome measures
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=10 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=11 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=2 Participants
protocadherin-19
|
|---|---|---|---|---|
|
Summary of CGII-C
Visit 4 (End of Week 4) - Very Much Improved
|
0 Participants
|
2 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 4 (End of Week 4) - Much Improved
|
3 Participants
|
4 Participants
|
4 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 4 (End of Week 4) - Minimally Improved
|
2 Participants
|
2 Participants
|
3 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 4 (End of Week 4) - No Change
|
2 Participants
|
0 Participants
|
3 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 4 (End of Week 4) - Minimally Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 4 (End of Week 4) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 4 (End of Week 4) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - Very Much Improved
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - Much Improved
|
4 Participants
|
4 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - Minimally Improved
|
2 Participants
|
2 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - No Change
|
1 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - Minimally Worse
|
0 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 5 (End of Week 8) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - Very Much Improved
|
1 Participants
|
2 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - Much Improved
|
2 Participants
|
3 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - Minimally Improved
|
2 Participants
|
0 Participants
|
4 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - No Change
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - Minimally Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 6 (End of Week 17) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - Very Much Improved
|
0 Participants
|
1 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - Much Improved
|
3 Participants
|
1 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - Minimally Improved
|
1 Participants
|
1 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - No Change
|
2 Participants
|
2 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - Minimally Worse
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - Much Worse
|
1 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 7 (End of Week 26) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - Very Much Improved
|
0 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - Much Improved
|
3 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - Minimally Improved
|
1 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - No Change
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - Minimally Worse
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 8 (End of Week 44) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - Very Much Improved
|
0 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - Much Improved
|
4 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - Minimally Improved
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - No Change
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - Minimally Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 9 (End of Week 62) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - Very Much Improved
|
0 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - Much Improved
|
3 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - Minimally Improved
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - No Change
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - Minimally Worse
|
0 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - Much Worse
|
0 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-C
Visit 10 (End of Week 78) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
SECONDARY outcome
Timeframe: Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ]Population: MITT Population - n varies per visit, also CSWS is not included
Patient Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-P scale is qualitative values and not quantitative.
Outcome measures
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=10 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=11 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=2 Participants
protocadherin-19
|
|---|---|---|---|---|
|
Summary of CGII-P
Visit 6 (End of Week 17) - Minimally Worse
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - Very Much Improved
|
0 Participants
|
3 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - Much Improved
|
1 Participants
|
2 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - Minimally Improved
|
4 Participants
|
3 Participants
|
5 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - No Change
|
2 Participants
|
0 Participants
|
3 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - Minimally Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 4 (End of Week 4) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - Very Much Improved
|
0 Participants
|
2 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - Much Improved
|
1 Participants
|
2 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - Minimally Improved
|
5 Participants
|
3 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - No Change
|
0 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - Minimally Worse
|
1 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - Much Worse
|
0 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 5 (End of Week 8) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 6 (End of Week 17) - Very Much Improved
|
1 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 6 (End of Week 17) - Much Improved
|
2 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 6 (End of Week 17) - Minimally Improved
|
2 Participants
|
3 Participants
|
4 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 6 (End of Week 17) - No Change
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 6 (End of Week 17) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 6 (End of Week 17) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - Very Much Improved
|
0 Participants
|
1 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - Much Improved
|
4 Participants
|
1 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - Minimally Improved
|
0 Participants
|
3 Participants
|
3 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - No Change
|
1 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - Minimally Worse
|
1 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - Much Worse
|
1 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 7 (End of Week 26) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44) - Very Much Improved
|
0 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44) - Much Improved
|
2 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44) - Minimally Improved
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44) - No Change
|
2 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44) - Minimally Worse
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 8 (End of Week 44)- Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62) - Very Much Improved
|
2 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62) - Much Improved
|
2 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62)- Minimally Improved
|
0 Participants
|
1 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62) - No Change
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62) - Minimally Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62) - Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 9 (End of Week 62) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78) - Very Much Improved
|
1 Participants
|
0 Participants
|
2 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78) - Much Improved
|
2 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78)- Minimally Improved
|
0 Participants
|
1 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78) - No Change
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78) - Minimally Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78) - Much Worse
|
0 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Summary of CGII-P
Visit 10 (End of Week 78) - Very Much Worse
|
0 Participants
|
0 Participants
|
0 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
SECONDARY outcome
Timeframe: Month 3 and Week 26Population: MITT Population - n varies per visit, also CSWS is not included
Responder Rate in Terms of 28-day Seizure Frequency Based on the Sum of Individual Seizures and Clusters
Outcome measures
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=10 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=11 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=2 Participants
protocadherin-19
|
|---|---|---|---|---|
|
Number of Participants With Responder Rate of Seizure Frequency
Post-baseline through Month 3 (Day 91) - 25% Responder
|
4 Participants
|
4 Participants
|
6 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Number of Participants With Responder Rate of Seizure Frequency
Post-baseline through Month 3 (Day 91) - 50% Responder
|
3 Participants
|
2 Participants
|
4 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Number of Participants With Responder Rate of Seizure Frequency
Post-baseline through Month 3 (Day 91) - 75% Responder
|
1 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Number of Participants With Responder Rate of Seizure Frequency
Post-baseline 26-week open-label Period - 25% Responder
|
4 Participants
|
3 Participants
|
5 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Number of Participants With Responder Rate of Seizure Frequency
Post-baseline 26-week open-label Period - 50% Responder
|
2 Participants
|
1 Participants
|
3 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Number of Participants With Responder Rate of Seizure Frequency
Post-baseline 26-week open-label Period - 75% Responder
|
1 Participants
|
0 Participants
|
1 Participants
|
NA Participants
Both patients (2 for CSWS) discontinued so data is not available.
|
SECONDARY outcome
Timeframe: Baseline, Day 91, Week 26, 52-week OLE through month 6, 52-week OLE PeriodPopulation: MITT Population - n varies per visit, also CSWS is not included
Mean Percentage Change of Individual Seizure-free days per 28-day period (through 52-week OLE) period relative to baseline
Outcome measures
| Measure |
CDKL5
n=7 Participants
Cyclin-dependent kinase-like 5
|
CSWS
n=10 Participants
Continuous Spike and Waves during Sleep
|
Lennox- Gastaut
n=11 Participants
Lennox- Gastaut syndrome
|
PCDH19
n=2 Participants
protocadherin-19
|
|---|---|---|---|---|
|
Mean Percentage Change of Individual Seizure-free Days
Mean Percentage Change of individual seizure-free days from baseline to Week 26
|
11.80 Percentage Change
Standard Deviation 20.968
|
-2.13 Percentage Change
Standard Deviation 22.042
|
7.94 Percentage Change
Standard Deviation 18.016
|
NA Percentage Change
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Mean Percentage Change of Individual Seizure-free Days
Mean Percentage Change of individual seizure-free days from baseline to Day 91
|
11.84 Percentage Change
Standard Deviation 18.472
|
-1.12 Percentage Change
Standard Deviation 24.928
|
7.87 Percentage Change
Standard Deviation 17.843
|
NA Percentage Change
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Mean Percentage Change of Individual Seizure-free Days
Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE (181 days)
|
21 Percentage Change
Standard Deviation 30.85
|
16.35 Percentage Change
Standard Deviation 2.916
|
17.12 Percentage Change
Standard Deviation 27.040
|
NA Percentage Change
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
|
Mean Percentage Change of Individual Seizure-free Days
Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE Period
|
20.44 Percentage Change
Standard Deviation 28.788
|
14.95 Percentage Change
Standard Deviation 4.897
|
17.02 Percentage Change
Standard Deviation 26.904
|
NA Percentage Change
Standard Deviation NA
Both patients (2 for CSWS) discontinued so data is not available.
|
Adverse Events
CDKL5
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
CSWS
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Lennox-Gastaut
Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths
PCDH19
Serious events: 3 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CDKL5
n=7 participants at risk
Cyclin-dependent kinase-like 5
|
CSWS
n=2 participants at risk
Continuous Spike Wave in Sleep
|
Lennox-Gastaut
n=10 participants at risk
Lennox-Gastaut Syndrome pediatric epilepsy
|
PCDH19
n=11 participants at risk
Protocadherin-19
|
|---|---|---|---|---|
|
Nervous system disorders
Seizure
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 4 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Hepatobiliary disorders
Hepatic failure
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
50.0%
1/2 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Pneumonia
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Somnolence
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
50.0%
1/2 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Pneumonia Viral
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
General disorders
Unintentional Medical Device Removal
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
Other adverse events
| Measure |
CDKL5
n=7 participants at risk
Cyclin-dependent kinase-like 5
|
CSWS
n=2 participants at risk
Continuous Spike Wave in Sleep
|
Lennox-Gastaut
n=10 participants at risk
Lennox-Gastaut Syndrome pediatric epilepsy
|
PCDH19
n=11 participants at risk
Protocadherin-19
|
|---|---|---|---|---|
|
Nervous system disorders
Somnolence
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
50.0%
1/2 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
20.0%
2/10 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
54.5%
6/11 • Number of events 6 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Seizure
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
20.0%
2/10 • Number of events 4 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
36.4%
4/11 • Number of events 6 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Headache
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
36.4%
4/11 • Number of events 4 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Dizziness
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
50.0%
1/2 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Hypersomnia
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
20.0%
2/10 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Sedation
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
General disorders
Pyrexia
|
71.4%
5/7 • Number of events 22 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
63.6%
7/11 • Number of events 16 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
General disorders
Fatigue
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
36.4%
4/11 • Number of events 4 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Nasopharyngitis
|
28.6%
2/7 • Number of events 8 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Influenza
|
28.6%
2/7 • Number of events 5 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Otitis media
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Rhinitis
|
14.3%
1/7 • Number of events 3 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Upper respiratory tract infection
|
42.9%
3/7 • Number of events 3 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Gastrointestinal disorders
Vomiting
|
42.9%
3/7 • Number of events 9 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
50.0%
1/2 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 3 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
27.3%
3/11 • Number of events 3 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Psychiatric disorders
Abnormal behavior
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Psychiatric disorders
Emotional disorder
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Psychiatric disorders
Restlessness
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Investigations
Weight decreased
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
27.3%
3/11 • Number of events 4 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Skin and subcutaneous tissue disorders
Rash
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Injury, poisoning and procedural complications
Contusion
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
27.3%
3/11 • Number of events 3 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Ataxia
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Nervous system disorders
Balance Disorder
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Fungal Infection
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
20.0%
2/10 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Pneumonia
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Infections and infestations
Sinusitis
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
50.0%
1/2 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
9.1%
1/11 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
14.3%
1/7 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
10.0%
1/10 • Number of events 1 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Renal and urinary disorders
Enuresis
|
0.00%
0/7 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
18.2%
2/11 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
|
Surgical and medical procedures
Gastrostomy
|
28.6%
2/7 • Number of events 2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/2 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/10 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
0.00%
0/11 • Combined 26-week open label period and 52-week open label extension period
Overall Summary of Adverse Events (Safety Population)
|
Additional Information
Marinus Clinical Trials Submission Manager
Marinus Pharmaceuticals, Inc.
Phone: 484-801-4670
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place