|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
3 July 2023 |
|
Main ID: |
NCT03865732 |
|
Date of registration:
|
05/03/2019 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
|
|
Scientific 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. |
|
Date of first enrolment:
|
May 17, 2019 |
|
Target sample size:
|
29 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT03865732 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
Australia
|
France
|
Hungary
|
Italy
|
Netherlands
|
Poland
|
United States
| |
|
Contacts
|
|
Name:
|
Maciej Gasior, M.D., Ph.D |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Marinus Pharmaceuticals, Inc. |
|
|
Name:
|
Paula Bokesk, M.D., FAAP |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Marinus Pharmaceuticals, Inc. |
| |
|
Key inclusion & exclusion criteria
|
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
Age minimum:
1 Year
Age maximum:
17 Years
Gender:
Female
|
|
Health Condition(s) or Problem(s) studied
|
|
PCDH19-Related Epilepsy
|
|
Intervention(s)
|
|
Drug: Placebo
|
|
Drug: Ganaxolone
|
|
Primary Outcome(s)
|
|
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]
|
|
Secondary Outcome(s)
|
|
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]]
|
|
50% Primary Seizure Reduction
[Time Frame: End of the double-blind 17 week treatment period]
|
|
Secondary ID(s)
|
|
2018-004496-12
|
|
1042-PCDH19-3002
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|