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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03865732
Ethics application status
Date submitted
5/03/2019
Date registered
7/03/2019

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
Trial website
Trial related presentations / publications
Public notes

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

Data sharing statement


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol
Statistical analysis plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03865732