Trial Outcomes & Findings for Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study) (NCT NCT03865732)
NCT ID: NCT03865732
Last Updated: 2023-06-26
Results Overview
Summary of 28-Day Seizure Frequency for Seizure Types through 17 week Post-Baseline Phase (Median Percent Change)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
29 participants
Primary outcome timeframe
End of the double-blind 17 week treatment period
Results posted on
2023-06-26
Participant Flow
Participant milestones
| Measure |
Placebo
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Ganaxolone: active drug
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
10
|
|
Overall Study
COMPLETED
|
11
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Placebo
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Ganaxolone: active drug
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
Baseline Characteristics
Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
Baseline characteristics by cohort
| Measure |
Placebo
n=11 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: non-active drug
|
Ganaxolone
n=10 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Ganaxolone: active drug
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
7.5 years
STANDARD_DEVIATION 3.93 • n=5 Participants
|
6.7 years
STANDARD_DEVIATION 3.40 • n=7 Participants
|
7.1 years
STANDARD_DEVIATION 3.62 • n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Hungary
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: ITT Population
Summary of 28-Day Seizure Frequency for Seizure Types through 17 week Post-Baseline Phase (Median Percent Change)
Outcome measures
| Measure |
Placebo
n=11 Participants
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
n=10 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
|
|---|---|---|
|
Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)
|
-23.97 Median % Change in Number of Seizures
Interval -88.24 to 4.89
|
-61.52 Median % Change in Number of Seizures
Interval -95.85 to -33.4
|
SECONDARY outcome
Timeframe: [Time Frame: End of the double-blind 17 week treatment period]Population: ITT Population specific to subjects in the Biomarker-positive Stratum
Summary of 28-day Seizure Frequency for Seizure Types for Subjects in the Biomarker-positive Stratum through 17 weeks (Median Percent Change)
Outcome measures
| Measure |
Placebo
n=7 Participants
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
n=6 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
|
|---|---|---|
|
Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)
|
-18.71 Median % Change in Number of Seizures
Interval -85.74 to 70.73
|
-35.90 Median % Change in Number of Seizures
Interval -86.48 to -24.32
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: ITT Population
Percent of subjects experiencing a greater than or equal to 50% reduction in 28-day primary seizure frequency relative to the 12-week baseline
Outcome measures
| Measure |
Placebo
n=11 Participants
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
n=10 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
|
|---|---|---|
|
50% Primary Seizure Reduction
|
4 Participants
|
5 Participants
|
Adverse Events
Placebo
Serious events: 5 serious events
Other events: 11 other events
Deaths: 0 deaths
Ganaxolone
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=11 participants at risk
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
n=10 participants at risk
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Ganaxolone: active drug
|
|---|---|---|
|
Psychiatric disorders
Psychogenic Seizure
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Nervous system disorders
Febrile Convulsion
|
9.1%
1/11 • Number of events 3 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Seizure
|
27.3%
3/11 • Number of events 4 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Seizure Cluster
|
18.2%
2/11 • Number of events 2 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
Other adverse events
| Measure |
Placebo
n=11 participants at risk
Placebo suspension 3x's/day for 17 weeks
|
Ganaxolone
n=10 participants at risk
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Ganaxolone: active drug
|
|---|---|---|
|
Psychiatric disorders
Aggression
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
20.0%
2/10 • Number of events 2 • Screening through Week 17
|
|
Psychiatric disorders
Agitation
|
0.00%
0/11 • Screening through Week 17
|
20.0%
2/10 • Number of events 2 • Screening through Week 17
|
|
Psychiatric disorders
Behavior Disorder
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Psychiatric disorders
Irritability
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Psychiatric disorders
Psychogenic Seizure
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Psychiatric disorders
Restlessness
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Psychiatric disorders
Disinhibition
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Somnolence
|
27.3%
3/11 • Number of events 3 • Screening through Week 17
|
40.0%
4/10 • Number of events 4 • Screening through Week 17
|
|
Nervous system disorders
Ataxia
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Nervous system disorders
Lethargy
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 2 • Screening through Week 17
|
|
Nervous system disorders
Dizziness
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Febrile Convulsion
|
9.1%
1/11 • Number of events 3 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Seizure
|
36.4%
4/11 • Number of events 5 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Nervous system disorders
Seizure Cluster
|
18.2%
2/11 • Number of events 2 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Gastrointestinal disorders
Constipation
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Gastrointestinal disorders
Dental Caries
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Gastrointestinal disorders
Diarrhoea
|
18.2%
2/11 • Number of events 2 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Gastrointestinal disorders
Eructation
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Gastrointestinal disorders
Abdominal Pain
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Gastrointestinal disorders
Vomiting
|
18.2%
2/11 • Number of events 2 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
General disorders
Fatigue
|
18.2%
2/11 • Number of events 2 • Screening through Week 17
|
20.0%
2/10 • Number of events 2 • Screening through Week 17
|
|
General disorders
Gait Disturbance
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Investigations
Alanine Aminotransferase Increased
|
0.00%
0/11 • Screening through Week 17
|
20.0%
2/10 • Number of events 2 • Screening through Week 17
|
|
Investigations
Protein Urine Present
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
0.00%
0/11 • Screening through Week 17
|
20.0%
2/10 • Number of events 2 • Screening through Week 17
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Infections and infestations
Impetigo
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Infections and infestations
Pharyngitis Streptococcal
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Infections and infestations
Vulvovaginal Candidiasis
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Skin and subcutaneous tissue disorders
Papule
|
0.00%
0/11 • Screening through Week 17
|
10.0%
1/10 • Number of events 1 • Screening through Week 17
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Skin and subcutaneous tissue disorders
Rash
|
18.2%
2/11 • Number of events 2 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Injury, poisoning and procedural complications
Eye Injury
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Injury, poisoning and procedural complications
Skin Laceration
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
|
Renal and urinary disorders
Pollakiuria
|
9.1%
1/11 • Number of events 1 • Screening through Week 17
|
0.00%
0/10 • Screening through Week 17
|
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