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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
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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
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Secondary ID [1]
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2018-001180-23
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Secondary ID [2]
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1042-CDD-3001
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Universal Trial Number (UTN)
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Trial acronym
Marigold
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
CDKL5 Deficiency Disorder
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Summary of 28-day Seizure Frequency for Major Motor Seizure Types
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Assessment method [1]
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Summary of 28-day seizure frequency for Major Motor Seizure Types during the double-blind treatment period relative to the 6-week prospective baseline period
Note: Summaries are based on the sum of the individual seizures, the countable seizures, and the clusters with uncountable seizures (each cluster with uncountable seizures counts as 1 seizure). Within the baseline and post baseline intervals, 28-day seizure frequency was calculated as the total number of seizures in the interval divided by the number of days with available seizure data in the interval, multiplied by 28.
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Timepoint [1]
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End of the double-blind 17 week treatment period
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Secondary outcome [1]
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Caregiver Global Impression of Change in Attention
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Assessment method [1]
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Caregiver global impression of change in attention during the double-blind treatment period of ganaxolone compared to placebo. Investigators and caregivers reported improvements in attention, mood, behavior and sleep via investigator narratives
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Timepoint [1]
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End of the double-blind 17 week treatment period
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Secondary outcome [2]
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Caregiver Global Impression of Change in Target Behavior
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Assessment method [2]
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Caregiver global impression of change in target behavior during the double-blind treatment period of ganaxolone compared to placebo. Investigators and caregivers reported improvements in attention, mood, behavior and sleep via investigator narratives.
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Timepoint [2]
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End of the double-blind 17 week treatment period
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Secondary outcome [3]
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Clinical Global Impression of Improvement - Parent/Caregiver
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Assessment method [3]
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Clinical global impression of improvement during the double-blind treatment period of ganaxolone compared to placebo. The CGI is rated on a 7-point scale, with the severity of illness scale using a range of responses.
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Timepoint [3]
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End of the double-blind 17 week treatment period
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Secondary outcome [4]
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Clinical Global Impression of Improvement - Clinician
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Assessment method [4]
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Clinical global impression of improvement during the double-blind treatment period of ganaxolone compared to placebo
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Timepoint [4]
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[Time Frame: End of the double-blind 17 week treatment period]
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Secondary outcome [5]
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Percentage of Seizure-free Days for Major Motor Seizure Types
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Assessment method [5]
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Percentage of Seizure-free Days for Major Motor Seizure types during the double-blind treatment period of ganaxolone compared to placebo. The major motor seizure types include bilateral tonic (sustained motor activity = 3 seconds), generalized tonic-clonic, atonic/drop, bilateral clonic, and focal to bilateral tonic-clonic.
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Timepoint [5]
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End of the double-blind 17 week treatment period
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Secondary outcome [6]
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Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure Types
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Assessment method [6]
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Arithmetic change in longest seizure free interval, based on primary seizure types during the double-blind treatment period of ganaxolone compared to placebo
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Timepoint [6]
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End of the double-blind 17 week treatment period
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Secondary outcome [7]
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Caregiver Global Impression of Change in Seizure Intensity and Duration
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Assessment method [7]
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Caregiver global impression of change in seizure intensity and duration during the double-blind treatment period of ganaxolone compared to placebo. CGI-C is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention.
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Timepoint [7]
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End of the double-blind 17 week treatment period
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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
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Minimum age
2
Years
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Maximum age
21
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/06/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
28/05/2021
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Sample size
Target
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Accrual to date
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Final
101
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Recruitment in Australia
Recruitment state(s)
QLD,VIC
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Recruitment hospital [1]
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Marinus Research Site - Brisbane
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Recruitment hospital [2]
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Marinus Research Site - Heidelberg
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Recruitment hospital [3]
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Marinus Research Site - Melbourne
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Recruitment postcode(s) [1]
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4101 - Brisbane
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Recruitment postcode(s) [2]
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3084 - Heidelberg
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Recruitment postcode(s) [3]
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3168 - Melbourne
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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Florida
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Illinois
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France
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Paris
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Israel
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Ramat Gan
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Italy
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Firenze
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Italy
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Milano
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Italy
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Pavia
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Italy
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Italy
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Verona
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Bydgoszcz
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Kraków
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Russian Federation
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Moscow
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Russian Federation
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Nizhniy Novgorod
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Russian Federation
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Novosibirsk
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Birmingham
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Bristol
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Glasgow
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Marinus Pharmaceuticals
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Ethics approval
Ethics application status
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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 children and young adults with genetically confirmed CDKL5 gene mutation.
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Trial website
https://clinicaltrials.gov/study/NCT03572933
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Trial related presentations / publications
Knight EMP, Amin S, Bahi-Buisson N, Benke TA, Cross JH, Demarest ST, Olson HE, Specchio N, Fleming TR, Aimetti AA, Gasior M, Devinsky O; Marigold Trial Group. Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2022 May;21(5):417-427. doi: 10.1016/S1474-4422(22)00077-1. Erratum In: Lancet Neurol. 2022 Jul;21(7):e7. doi: 10.1016/S1474-4422(22)00230-7.
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Public notes
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Contacts
Principal investigator
Name
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Joseph Hulihan, MD
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Address
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Marinus Pharmaceuticals, Inc.
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/33/NCT03572933/Prot_002.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/33/NCT03572933/SAP_003.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03572933
Download to PDF