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Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
Phase 2
Waitlist Available
Research Sponsored by Marinus Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up end of the double-blind 17 week treatment period
Awards & highlights
All Individual Drugs Already Approved
Summary
This trial is testing ganaxolone, a medication that may help reduce seizures, in female children and young adults with a specific genetic mutation (PCDH19). The study will evaluate if ganaxolone is effective and safe. Ganaxolone works by balancing brain signals to make seizures less likely. Ganaxolone, a synthetic neuroactive steroid, is approved by the US Food and Drug Administration (FDA) to treat seizures in patients with CDKL5 deficiency disorder.
Eligible Conditions
- Epilepsy
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ end of the double-blind 17 week treatment period
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~end of the double-blind 17 week treatment period
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)
Secondary study objectives
50% Primary Seizure Reduction
Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)
Side effects data
From 2014 Phase 2 trial • 112 Patients • NCT0133968919%
Somnolence
14%
Headache
8%
Fatigue
8%
Sedation
7%
Dizziness
7%
Nightmare
7%
Vision blurred
7%
Myalgia
5%
Confusional state
5%
Dysarthria
3%
Vertigo
3%
Gastrointestinal disorder
3%
Pain in extremity
3%
Tremor
3%
Neck pain
3%
Nasopharyngitis
3%
Increased appetite
3%
Ataxia
3%
Balance disorder
3%
Cough
3%
Insomnia
3%
Diarrhoea
3%
Lethargy
2%
Confusion
2%
Splenomegaly
2%
Road traffic accident
2%
Protein urine present
2%
Dry mouth
2%
Urinary tract infection
2%
Tandem galt test abnormal
2%
Arthralgia
2%
Balance Issues
2%
Upper respiratory tract infection
2%
Pulmonary congestion
2%
Rhinorrhoea
2%
Vomiting
2%
Chest pain
2%
Decreased appetite
2%
Abnormal dreams
2%
Nausea
2%
Chest discomfort
2%
Feeling drunk
2%
Irritability
2%
Pharyngitis streptococcal
2%
Blood urine present
2%
Weight increased
2%
Back pain
2%
Muscle spasms
2%
Depressed level of consciousness
2%
Disturbance in attention
2%
Motor dysfunction
2%
Anger
2%
Depressed
2%
Euphoric mood
2%
Initial insomnia
2%
Psychomotor retardation
2%
Sleep disorder
2%
Upper airway resistance syndrome
2%
Anorgasmia
2%
Suicidal ideation
2%
Haematuria
2%
Suicide of companion
2%
Product used for unknown indication
2%
Dry Eye
2%
Abdominal discomfort
2%
Alanine aminotransferase increased
2%
Liver function test abnormal
2%
Tic
2%
Migraine
2%
Suicidal Ideation
2%
Fever
2%
Sinus congestion
2%
Rash
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ganaxolone
Placebo
Awards & Highlights
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: GanaxoloneExperimental Treatment1 Intervention
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Group II: PlaceboPlacebo Group1 Intervention
placebo suspension 3x's /day for 17 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ganaxolone
FDA approved
Find a Location
Who is running the clinical trial?
Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,744 Total Patients Enrolled
6 Trials studying Epilepsy
577 Patients Enrolled for Epilepsy
Maciej Gasior, M.D., Ph.DStudy DirectorMarinus Pharmaceuticals, Inc.
Paula Bokesk, M.D., FAAPStudy DirectorMarinus Pharmaceuticals, Inc.