Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
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ClinicalTrials.gov Identifier: NCT03865732 |
Recruitment Status :
Completed
First Posted : March 7, 2019
Results First Posted : September 8, 2022
Last Update Posted : September 8, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
PCDH19-Related Epilepsy | Drug: Ganaxolone Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 29 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | The double-blind phase will randomize subjects to adjunctive ganaxolone or placebo at a 1:1 ratio to standard of care. |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment. |
Actual Study Start Date : | May 17, 2019 |
Actual Primary Completion Date : | January 19, 2021 |
Actual Study Completion Date : | June 20, 2022 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Placebo
placebo suspension 3x's /day for 17 weeks
|
Drug: Placebo
inactive
Other Name: Placebo (for ganaxolone) |
Experimental: Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
|
Drug: Ganaxolone
active drug |
- Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change) [ Time Frame: End of the double-blind 17 week treatment period ]Summary of 28-Day Seizure Frequency for Seizure Types through 17 week Post-Baseline Phase (Median Percent Change)
- Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change) [ Time Frame: [Time Frame: End of the double-blind 17 week treatment period] ]Summary of 28-day Seizure Frequency for Seizure Types for Subjects in the Biomarker-positive Stratum through 17 weeks (Median Percent Change)
- 50% Primary Seizure Reduction [ Time Frame: End of the double-blind 17 week treatment period ]Percent of subjects experiencing a greater than or equal to 50% reduction in 28-day primary seizure frequency relative to the 12-week baseline

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Ages Eligible for Study: | 1 Year to 17 Years (Child) |
Sexes Eligible for Study: | Female |
Gender Based Eligibility: | Yes |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant
- Failure to control seizures despite 2 or more anti-seizure medications
- 12 seizures over a 12-week period of primary seizure types prior to screening
- On a stable regimen of concomitant AEDs, Ketogenic diets, and modified Atkins diet should be unchanged for 3 months prior to screening)
Exclusion Criteria:
- Previous exposure to ganaxolone
- > 8 consecutive weeks of seizure freedom during the 12 weeks prior to screening
- Concurrent use of strong inducers or inhibitors of CYP3A4/5/7 is not permitted
- Use of tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) is prohibited during the double-blind phase
- Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03865732
United States, Arkansas | |
Marinus Research Site | |
Little Rock, Arkansas, United States, 72202 | |
United States, California | |
Marinus Research Site | |
Los Angeles, California, United States, 90095 | |
Marinus Research Site | |
San Francisco, California, United States, 94158 | |
United States, North Carolina | |
Marinus Research Site | |
Durham, North Carolina, United States, 27710 | |
United States, Pennsylvania | |
Marinus Research Site | |
York, Pennsylvania, United States, 17403 | |
United States, Utah | |
Marinus Research Site | |
Salt Lake City, Utah, United States, 84113 | |
Hungary | |
Marinus Research Site | |
Budapest, Hungary, 1083 | |
Italy | |
Marinus Research Site | |
Firenze, Italy, 50139 | |
Marinus Research Site | |
Rome, Italy, 00165 | |
Netherlands | |
Marinus Research Site | |
Heeze, Netherlands, 5591 | |
Marinus Research Site | |
Zwolle, Netherlands, 8025 | |
Poland | |
Marinus Research Site | |
Krakow, Poland, 30-363 |
Study Director: | Maciej Gasior, M.D., Ph.D | Marinus Pharmaceuticals, Inc. | |
Study Director: | Paula Bokesk, M.D., FAAP | Marinus Pharmaceuticals, Inc. |
Documents provided by Marinus Pharmaceuticals:
Responsible Party: | Marinus Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03865732 |
Other Study ID Numbers: |
1042-PCDH19-3002 2018-004496-12 ( EudraCT Number ) |
First Posted: | March 7, 2019 Key Record Dates |
Results First Posted: | September 8, 2022 |
Last Update Posted: | September 8, 2022 |
Last Verified: | May 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
refractory seizures epilepsy in children seizure disorder |
Epilepsy Brain Diseases Central Nervous System Diseases Nervous System Diseases Pregnanolone Ganaxolone Neurosteroids |
Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs GABA Modulators GABA Agents Anesthetics Central Nervous System Depressants |