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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03865732




Registration number
NCT03865732
Ethics application status
Date submitted
5/03/2019
Date registered
7/03/2019
Date last updated
26/06/2023

Titles & IDs
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.
Secondary ID [1] 0 0
2018-004496-12
Secondary ID [2] 0 0
1042-PCDH19-3002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
PCDH19-Related Epilepsy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ganaxolone
Treatment: Drugs - Placebo

Placebo Comparator: Placebo - placebo suspension 3x's /day for 17 weeks

Experimental: Ganaxolone - ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks


Treatment: Drugs: Ganaxolone
active drug

Treatment: Drugs: Placebo
inactive
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)
Timepoint [1] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [1] 0 0
Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)
Timepoint [1] 0 0
[Time Frame: End of the double-blind 17 week treatment period]
Secondary outcome [2] 0 0
50% Primary Seizure Reduction
Timepoint [2] 0 0
End of the double-blind 17 week treatment period

Eligibility
Key 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)
Minimum age
1 Year
Maximum age
17 Years
Sex
Females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/05/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/06/2022
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Utah
Country [6] 0 0
Hungary
State/province [6] 0 0
Budapest
Country [7] 0 0
Italy
State/province [7] 0 0
Firenze
Country [8] 0 0
Italy
State/province [8] 0 0
Rome
Country [9] 0 0
Netherlands
State/province [9] 0 0
Heeze
Country [10] 0 0
Netherlands
State/province [10] 0 0
Zwolle
Country [11] 0 0
Poland
State/province [11] 0 0
Krakow

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Marinus Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone
therapy compared to placebo for the treatment of seizures in female children and young adults
with genetically confirmed PCDH19 gene mutation.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03865732
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Maciej Gasior, M.D., Ph.D
Address 0 0
Marinus Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03865732