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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03572933




Registration number
NCT03572933
Ethics application status
Date submitted
19/06/2018
Date registered
28/06/2018
Date last updated
14/04/2023

Titles & IDs
Public title
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
Scientific title
A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment
Secondary ID [1] 0 0
2018-001180-23
Secondary ID [2] 0 0
1042-CDD-3001
Universal Trial Number (UTN)
Trial acronym
Marigold
Linked study record

Health condition
Health condition(s) or problem(s) studied:
CDKL5 Deficiency Disorder 0 0
Condition category
Condition code

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

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

Placebo comparator: Placebo - placebo suspension 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 for Major Motor Seizure Types
Timepoint [1] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [1] 0 0
Caregiver Global Impression of Change in Attention
Timepoint [1] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [2] 0 0
Caregiver Global Impression of Change in Target Behavior
Timepoint [2] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [3] 0 0
Clinical Global Impression of Improvement - Parent/Caregiver
Timepoint [3] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [4] 0 0
Clinical Global Impression of Improvement - Clinician
Timepoint [4] 0 0
[Time Frame: End of the double-blind 17 week treatment period]
Secondary outcome [5] 0 0
Percentage of Seizure-free Days for Major Motor Seizure Types
Timepoint [5] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [6] 0 0
Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure Types
Timepoint [6] 0 0
End of the double-blind 17 week treatment period
Secondary outcome [7] 0 0
Caregiver Global Impression of Change in Seizure Intensity and Duration
Timepoint [7] 0 0
End of the double-blind 17 week treatment period

Eligibility
Key inclusion criteria
* Genetically confirmed CDKL5 gene mutation, seizure onset by 1 year of age and lack of independent ambulation by 2 years of age
* Failure to control seizures despite 2 or more anti-seizure medications
* At least 16 seizures per 28 days of primary seizure types
* On a stable regimen of 0-4 anti-seizure medications (Vagus nerve stimulator, ketogenic diet, and modified Atkins diet do not count towards this limit)
* Additional Inclusion Criteria apply and can be discussed with study team
Minimum age
2 Years
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Previous exposure to ganaxolone
* West Syndrome with hypsarrhythmia pattern on EEG or seizures predominantly of Infantile Spasms type
* Use of adrenocorticotropic hormone (ACTH), prednisone or other glucocorticoid or use of moderate or strong inducers or inhibitors of CYP3A4/5/7 are prohibited
* Use of tetrahydrocannabinol (THC) or cannabidiol (CBD) is prohibited during the double-blind phase, unless patient has a prescription of Epidiolex®
* Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening
* Plasma allopregnanolone-sulfate (Allo-S) levels greater than or equal to 6.0 ng/ml at screening visit
* Additional Exclusion Criteria apply and can be discussed with study team

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 3
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
30/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/05/2021
Sample size
Target
Accrual to date
Final
101
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Marinus Research Site - Brisbane
Recruitment hospital [2] 0 0
Marinus Research Site - Heidelberg
Recruitment hospital [3] 0 0
Marinus Research Site - Melbourne
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment postcode(s) [2] 0 0
3084 - Heidelberg
Recruitment postcode(s) [3] 0 0
3168 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New Jersey
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
Israel
State/province [16] 0 0
Ramat Gan
Country [17] 0 0
Italy
State/province [17] 0 0
Firenze
Country [18] 0 0
Italy
State/province [18] 0 0
Milano
Country [19] 0 0
Italy
State/province [19] 0 0
Pavia
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
Country [21] 0 0
Italy
State/province [21] 0 0
Verona
Country [22] 0 0
Poland
State/province [22] 0 0
Bydgoszcz
Country [23] 0 0
Poland
State/province [23] 0 0
Kraków
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Moscow
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Nizhniy Novgorod
Country [26] 0 0
Russian Federation
State/province [26] 0 0
Novosibirsk
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Birmingham
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Bristol
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Glasgow
Country [30] 0 0
United Kingdom
State/province [30] 0 0
London

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
Joseph Hulihan, MD
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT03572933