Epilepsy
Conditions
Keywords
PCDH19, ganaxolone, neurosteroid, CDKL5, LGS, CSWS
Brief summary
To evaluate the efficacy of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation and other rare genetic epilepsies in an open-label proof-of-concept study.
Detailed description
The purpose of this proof-of-concept study is to evaluate ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutations and other rare genetic epilepsies. After establishing baseline seizure frequency, qualifying subjects will enter the study and be treated with open-label ganaxolone for up to six months.
Interventions
DRUGGanaxolone
oral suspension or capsules
Sponsors
Marinus Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Have parent or legal guardian available and willing to give written informed consent. 2. Male and female outpatients between 2 and 18 years of age years of age at time of consent. 3. Have any of the following epilepsy syndromes: PCDH19; CDKL5; Dravet Syndrome; Lennox Gastaut Syndrome (LGS); Continuous Spikes and Waves during Sleep (CSWS) 4. Have uncontrolled cluster seizures and/or non-clustered seizures. 5. Subjects should be on a stable regimen of anti-epileptic medication, and generally in good health. 6. Parent or guardian is able and willing to maintain an accurate and complete daily written seizure calendar. 7. Able and willing to take study medication with food, two or three times daily. Key
Exclusion criteria
1. Have had previous exposure to ganaxolone. 2. Known sensitivity or allergy to any component in the study drug, progesterone, or other related steroid compounds. 3. Exposure to any investigational drug or device \< 90 days prior to screening, or plans to participate in another drug or device trial at any time during the study. 4. Concurrent use of vigabatrin, tiagabine, or ezogabine is not permitted. 5. Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome, including but not limited to: clinically significant cardiac, renal, pulmonary, gastrointestinal, hematologic or hepatic conditions; or a condition that affects the absorption, distribution, metabolism or excretion of drugs. 6. Have active suicidal plan/intent, or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 3 years. 7. Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels \> 3 times upper limits of normal (ULN), or total bilirubin \>1.5 time ULN at the screening and baseline visits.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation) | Baseline through 52 week open label period | Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change & Standard Deviation) |
| Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change) | Baseline through 52-week open- label period | Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Median Percent Change) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Summary of CGII-C | End of Week 4, End of Week 8, End of Week 17, End of Week 26, Week 44, Week 62, Week 78 | Clinician Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-C scale is qualitative values and not quantitative. |
| Summary of CGII-P | Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ] | Patient Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-P scale is qualitative values and not quantitative. |
| Number of Participants With Responder Rate of Seizure Frequency | Month 3 and Week 26 | Responder Rate in Terms of 28-day Seizure Frequency Based on the Sum of Individual Seizures and Clusters |
| Mean Percentage Change of Individual Seizure-free Days | Baseline, Day 91, Week 26, 52-week OLE through month 6, 52-week OLE Period | Mean Percentage Change of Individual Seizure-free days per 28-day period (through 52-week OLE) period relative to baseline |
Countries
Italy, United States
Participant flow
Recruitment details
recruitment period May 08, 2015
Participants by arm
| Arm | Count |
|---|---|
| CDKL5 Cyclin-dependent kinase-like 5 | 7 |
| CSWS Continuous Spike and Waves during Sleep | 2 |
| Lennox- Gastaut Lennox- Gastaut syndrome | 10 |
| PCDH19 protocadherin-19 | 11 |
| Total | 30 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| 52 Week Extension Period | Lack of Efficacy | 0 | 0 | 2 | 4 |
| Through Week 26 | Adverse Event | 0 | 1 | 1 | 2 |
| Through Week 26 | Family did not see benefit from IP | 1 | 0 | 0 | 0 |
| Through Week 26 | Lack of Efficacy | 1 | 1 | 3 | 3 |
| Through Week 26 | Non-Compliance | 0 | 0 | 1 | 0 |
| Through Week 26 | Withdrawal by Subject | 1 | 0 | 0 | 0 |
Baseline characteristics
| Characteristic | CDKL5 | CSWS | Lennox- Gastaut | PCDH19 | Total |
|---|---|---|---|---|---|
| Age, Categorical <=18 years | 7 Participants | 2 Participants | 10 Participants | 11 Participants | 30 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 7.73 years STANDARD_DEVIATION 3.403 | 11.55 years STANDARD_DEVIATION 5.728 | 9.25 years STANDARD_DEVIATION 3.595 | 8.87 years STANDARD_DEVIATION 4.129 | 8.65 years STANDARD_DEVIATION 3.551 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 4 Participants | 3 Participants | 7 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 7 Participants | 2 Participants | 6 Participants | 8 Participants | 10 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 3 Participants |
| Race (NIH/OMB) White | 4 Participants | 1 Participants | 4 Participants | 6 Participants | 11 Participants |
| Sex: Female, Male Female | 6 Participants | 1 Participants | 4 Participants | 6 Participants | 14 Participants |
| Sex: Female, Male Male | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 2 | 0 / 10 | 0 / 11 |
| other Total, other adverse events | 7 / 7 | 1 / 2 | 7 / 10 | 11 / 11 |
| serious Total, serious adverse events | 1 / 7 | 1 / 2 | 2 / 10 | 3 / 11 |
Outcome results
Primary
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change & Standard Deviation)
Time frame: Baseline through 52 week open label period
Population: MITT Population
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CSWS | 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) | NA percentage of change of frequency | — |
| CSWS | 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) | NA percentage of change of frequency | — |
| CSWS | 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) | NA percentage of change of frequency | — |
| CSWS | 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) | NA percentage of change of frequency | — |
| Lennox- Gastaut | 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) | -38.74 percentage of change of frequency | Standard Deviation 9.292 |
| Lennox- Gastaut | 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) | 125.38 percentage of change of frequency | Standard Deviation 319.051 |
| Lennox- Gastaut | 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) | -37.75 percentage of change of frequency | Standard Deviation 7.891 |
| Lennox- Gastaut | 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) | 122.10 percentage of change of frequency | Standard Deviation 321.124 |
| PCDH19 | 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) | -19.95 percentage of change of frequency | Standard Deviation 63.571 |
| PCDH19 | 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) | 46.36 percentage of change of frequency | Standard Deviation 235.661 |
| PCDH19 | 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) | 52.83 percentage of change of frequency | Standard Deviation 234.084 |
| PCDH19 | 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) | -19.98 percentage of change of frequency | Standard Deviation 63.644 |
Primary
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Median Percent Change)
Time frame: Baseline through 52-week open- label period
Population: MITT Population
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CSWS | 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 | NA Median Percent Change in Frequency |
| CSWS | 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) | -38.74 Median Percent Change in Frequency |
| CSWS | 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) | -37.75 Median Percent Change in Frequency |
| CSWS | 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) | -9.19 Median Percent Change in Frequency |
| CSWS | 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) | -10.22 Median Percent Change in Frequency |
| Lennox- Gastaut | 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 | NA Median Percent Change in Frequency |
| Lennox- Gastaut | 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) | -25.98 Median Percent Change in Frequency |
| Lennox- Gastaut | 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) | -24.59 Median Percent Change in Frequency |
| Lennox- Gastaut | 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) | -13.48 Median Percent Change in Frequency |
| Lennox- Gastaut | 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) | -13.48 Median Percent Change in Frequency |
| PCDH19 | 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) | NA Median Percent Change in Frequency |
| PCDH19 | 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) | NA Median Percent Change in Frequency |
| PCDH19 | 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) | NA Median Percent Change in Frequency |
| PCDH19 | 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 | NA Median Percent Change in Frequency |
| PCDH19 | 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) | NA Median Percent Change in Frequency |
Secondary
Mean Percentage Change of Individual Seizure-free Days
Mean Percentage Change of Individual Seizure-free days per 28-day period (through 52-week OLE) period relative to baseline
Time frame: Baseline, Day 91, Week 26, 52-week OLE through month 6, 52-week OLE Period
Population: MITT Population - n varies per visit, also CSWS is not included
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CDKL5 | 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 |
| CSWS | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to Week 26 | -2.13 Percentage Change | Standard Deviation 22.042 |
| CSWS | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE (181 days) | 16.35 Percentage Change | Standard Deviation 2.916 |
| CSWS | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE Period | 14.95 Percentage Change | Standard Deviation 4.897 |
| CSWS | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to Day 91 | -1.12 Percentage Change | Standard Deviation 24.928 |
| Lennox- Gastaut | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE (181 days) | 17.12 Percentage Change | Standard Deviation 27.04 |
| Lennox- Gastaut | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to Week 26 | 7.94 Percentage Change | Standard Deviation 18.016 |
| Lennox- Gastaut | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE Period | 17.02 Percentage Change | Standard Deviation 26.904 |
| Lennox- Gastaut | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to Day 91 | 7.87 Percentage Change | Standard Deviation 17.843 |
| PCDH19 | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE Period | NA Percentage Change | — |
| PCDH19 | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to Week 26 | NA Percentage Change | — |
| PCDH19 | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to Day 91 | NA Percentage Change | — |
| PCDH19 | Mean Percentage Change of Individual Seizure-free Days | Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE (181 days) | NA Percentage Change | — |
Secondary
Number of Participants With Responder Rate of Seizure Frequency
Responder Rate in Terms of 28-day Seizure Frequency Based on the Sum of Individual Seizures and Clusters
Time frame: Month 3 and Week 26
Population: MITT Population - n varies per visit, also CSWS is not included
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| CDKL5 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 25% Responder | 4 Participants |
| CDKL5 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 50% Responder | 3 Participants |
| CDKL5 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 75% Responder | 1 Participants |
| CDKL5 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 25% Responder | 4 Participants |
| CDKL5 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 50% Responder | 2 Participants |
| CDKL5 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 75% Responder | 1 Participants |
| CSWS | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 75% Responder | 0 Participants |
| CSWS | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 25% Responder | 3 Participants |
| CSWS | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 25% Responder | 4 Participants |
| CSWS | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 75% Responder | 0 Participants |
| CSWS | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 50% Responder | 2 Participants |
| CSWS | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 50% Responder | 1 Participants |
| Lennox- Gastaut | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 50% Responder | 4 Participants |
| Lennox- Gastaut | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 75% Responder | 1 Participants |
| Lennox- Gastaut | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 25% Responder | 5 Participants |
| Lennox- Gastaut | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 75% Responder | 1 Participants |
| Lennox- Gastaut | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 50% Responder | 3 Participants |
| Lennox- Gastaut | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 25% Responder | 6 Participants |
| PCDH19 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 50% Responder | NA Participants |
| PCDH19 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 75% Responder | NA Participants |
| PCDH19 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 50% Responder | NA Participants |
| PCDH19 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline 26-week open-label Period - 25% Responder | NA Participants |
| PCDH19 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 25% Responder | NA Participants |
| PCDH19 | Number of Participants With Responder Rate of Seizure Frequency | Post-baseline through Month 3 (Day 91) - 75% Responder | NA Participants |
Secondary
Summary of CGII-C
Clinician Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-C scale is qualitative values and not quantitative.
Time frame: End of Week 4, End of Week 8, End of Week 17, End of Week 26, Week 44, Week 62, Week 78
Population: MITT Population - n varies per visit, also CSWS is not included
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - Much Improved | 3 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Improved | 1 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - Much Improved | 2 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Improved | 2 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - Much Improved | 4 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - Much Improved | 4 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Improved | 2 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - Much Improved | 3 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - No Change | 1 Participants |
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Improved | 2 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Improved | 1 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - Much Improved | 3 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - Much Worse | 1 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - No Change | 2 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Improved | 1 Participants |
| CDKL5 | Summary of CGII-C | Visit 10 (End of Week 78) - Much Improved | 3 Participants |
| CDKL5 | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Improved | 0 Participants |
| CDKL5 | Summary of CGII-C | Visit 7 (End of Week 26) - No Change | 2 Participants |
| CDKL5 | Summary of CGII-C | Visit 5 (End of Week 8) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - No Change | 2 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - Much Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Worse | 1 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - Much Worse | 1 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - No Change | 0 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Improved | 1 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - Much Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - No Change | 1 Participants |
| CSWS | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Worse | 1 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - Much Improved | 4 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Improved | 2 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Improved | 1 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Improved | 2 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - No Change | 1 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - Much Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - No Change | 0 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Improved | 2 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - Much Improved | 3 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Improved | 0 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - No Change | 1 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - Much Improved | 4 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - No Change | 0 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Improved | 1 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Improved | 2 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - Much Improved | 1 Participants |
| CSWS | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Improved | 1 Participants |
| CSWS | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Improved | 1 Participants |
| CSWS | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - Much Improved | 4 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Improved | 3 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - No Change | 3 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - No Change | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Worse | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - Much Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Improved | 4 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - No Change | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - Much Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - Much Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Worse | 0 Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - No Change | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - No Change | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 6 (End of Week 17) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - No Change | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - No Change | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - No Change | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 5 (End of Week 8) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 8 (End of Week 44) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 4 (End of Week 4) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 9 (End of Week 62) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 7 (End of Week 26) - No Change | NA Participants |
| PCDH19 | Summary of CGII-C | Visit 10 (End of Week 78) - No Change | NA Participants |
Secondary
Summary of CGII-P
Patient Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-P scale is qualitative values and not quantitative.
Time frame: 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
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - Much Improved | 4 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - No Change | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Improved | 4 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Improved | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - Much Improved | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - Much Improved | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Improved | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - Much Improved | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Improved | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Improved | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44) - No Change | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78) - Much Improved | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62)- Minimally Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44) - Much Improved | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78)- Minimally Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44)- Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Worse | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62) - No Change | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44) - Very Much Improved | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Improved | 5 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78) - Minimally Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - Much Worse | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 9 (End of Week 62) - Much Improved | 2 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Worse | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 8 (End of Week 44) - Much Worse | 0 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - No Change | 1 Participants |
| CDKL5 | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62)- Minimally Improved | 1 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Improved | 3 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62) - No Change | 0 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Improved | 3 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - No Change | 1 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78) - Much Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78)- Minimally Improved | 1 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78) - No Change | 0 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - No Change | 0 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Improved | 1 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - Much Improved | 1 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Improved | 3 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - No Change | 0 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Worse | 1 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Improved | 1 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - Much Improved | 1 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Improved | 3 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - Much Improved | 2 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - No Change | 1 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - Much Worse | 1 Participants |
| CSWS | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44) - Very Much Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44) - Much Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Improved | 2 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44) - No Change | 1 Participants |
| CSWS | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Improved | 3 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Worse | 1 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44) - Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 5 (End of Week 8) - Much Improved | 2 Participants |
| CSWS | Summary of CGII-P | Visit 8 (End of Week 44)- Very Much Worse | 0 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Improved | 0 Participants |
| CSWS | Summary of CGII-P | Visit 9 (End of Week 62) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78) - Much Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - Much Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78)- Minimally Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Improved | 5 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62)- Minimally Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44)- Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Improved | 4 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - Much Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - No Change | 3 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - No Change | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Improved | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44) - No Change | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - Much Worse | 1 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 4 (End of Week 4) - Much Worse | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Improved | 2 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Improved | 3 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 9 (End of Week 62) - Much Improved | 0 Participants |
| Lennox- Gastaut | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Worse | 0 Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 4 (End of Week 4) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 5 (End of Week 8) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 6 (End of Week 17) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 7 (End of Week 26) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 8 (End of Week 44)- Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62)- Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62) - Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 9 (End of Week 62) - Very Much Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78) - Very Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78) - Much Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78)- Minimally Improved | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78) - No Change | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78) - Minimally Worse | NA Participants |
| PCDH19 | Summary of CGII-P | Visit 10 (End of Week 78) - Much Worse | NA Participants |