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    Clinical Trial Results:
    A controlled double-blind crossover trial of ganaxolone in children with fragile X syndrome

    Summary
    EudraCT number
    		 2014-000251-89
    Trial protocol
    		 BE  
    Global end of trial date
    15 Nov 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Sep 2021
    First version publication date
    04 Sep 2021
    Other versions
    Summary report(s)
    		 Trial results publication

    Trial information

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    Trial identification
    Sponsor protocol code
    1042-0800
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01725152
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Marinus Pharmaceuticals
    Sponsor organisation address
    100 Matsonford Rd, Radnor, United States,
    Public contact
    Cognitive Genetics Group, Antwerp University Hospital, +32 32759756, medische.genetica@uza.be
    Scientific contact
    Cognitive Genetics Group, Antwerp University Hospital, +32 32759756, medische.genetica@uza.be
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    16 Oct 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    16 Oct 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Nov 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the safety, tolerability and efficacy of ganaxolone for treatment of anxiety and attention in subjects with fragile X syndrome.
    Protection of trial subjects
    All families signed an informed consent that was reviewed by the UCD Institutional Review Boards (IRBs).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Nov 2012
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 11
    Country: Number of subjects enrolled
    United States: 48
    Worldwide total number of subjects
    59
    EEA total number of subjects
    11
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    29
    Adolescents (12-17 years)
    30
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patients were selected from existing contacts and though patient organisations. Potential participants were screened by telephone using a questionnaire. Those who met all criteria were scheduled for a baseline visit. All families signed an informed consent that was review ed by the UCD Institutional Review Boards.

    Pre-assignment
    Screening details
    		 Baseline assessments included intelligence testing , Autism Diagnostic Observation Schedule (ADOS); Diagnostic and Statistical Manual of Mental Disorders, Text Revision IV (DSM-IV) checklist; and Clinical Global ImpressionSeverity (CGI-S)).

    Pre-assignment period milestones
    Number of subjects started
    		 59
    Number of subjects completed
    		 59

    Period 1
    Period 1 title
    Arm 1 (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Monitor, Data analyst, Carer, Assessor, Subject
    Blinding implementation details
    Drugs and placebo were identical and the code was only known to the pharmacist.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Drug - Placebo
    Arm description
    		 2-armed trial with cross-over (drug/placebo): first treatment with drug, second with placebo
    Arm type
    		 Experimental

    Investigational medicinal product name
    ganaxolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    3 mg/kg up to 12 mg/kg, with maximum of 1500 mg/day

    Arm title
    		 Placebo - Drug
    Arm description
    		 2-armed trial with cross-over (drug/placebo): first treatment with placebo, second with drug
    Arm type
    		 Placebo

    Investigational medicinal product name
    ganaxolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    3 mg/kg up to 12 mg/kg, with maximum of 1500 mg/day

    Number of subjects in period 1
    		Drug - Placebo Placebo - Drug
    Started
    30
    29
    Completed
    25
    26
    Not completed
    5
    3
         study medication compromised
    -
    1
         Adverse event, non-fatal
    4
    1
         No perceived benefit
    -
    1
         Lost to follow-up
    1
    -

    Baseline characteristics

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    End points

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    End points reporting groups
    Reporting group title
    Drug - Placebo
    Reporting group description
    		 2-armed trial with cross-over (drug/placebo): first treatment with drug, second with placebo

    Reporting group title
    Placebo - Drug
    Reporting group description
    		 2-armed trial with cross-over (drug/placebo): first treatment with placebo, second with drug

    Primary: Primary end point

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    End point title
    		 Primary end point
    End point description
    Clinical Global Impression-Improvement (CGI-I) scale was used as primary end point
    End point type
    Primary
    End point timeframe
    Measured at the end of each treatment arm
    End point values
    		 Drug - Placebo Placebo - Drug
    Number of subjects analysed
    30
    29
    Units: 3.4-3.5
        least squares mean (standard error)
    3.4 ± 0.13
    3.5 ± 0.13
    Statistical analysis title
    		 Linear mixed-effect (LME) model
    Statistical analysis description
    Efficacy was assessed via a linear mixed-effect (LME) model for repeated measures in a ganaxolone/placebo, 2-period crossover trial with primary endpoint at the end of the period.
    Comparison groups
    Drug - Placebo v Placebo - Drug
    Number of subjects included in analysis
    59
    Analysis specification
    Pre-specified
    Analysis type
    		 other [1]
    P-value
    		 < 0.05
    Method
    		 Mixed models analysis
    Confidence interval
    Notes
    [1] - LMEM

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    After the finalisation of the entire study.
    Adverse event reporting additional description
    334 adverse events were reported. The top three types of adverse event were upper respiratory infection, fatigue, and drowsiness.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 GP
    Dictionary version
    1
    Reporting groups
    Reporting group title
    Drug - Placebo
    Reporting group description
    		 first treatment with drug, second with placebo

    Reporting group title
    Placebo - Drug
    Reporting group description
    		 first treatment with placebo, second with drug

    Serious adverse events
    		 Drug - Placebo Placebo - Drug
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 30 (0.00%)
    0 / 29 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Drug - Placebo Placebo - Drug
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 30 (13.33%)
    1 / 29 (3.45%)
    Nervous system disorders
    Fatigue
    Additional description: Transient fatigue
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    upper respiratory infection
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Rash / Itchiness
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 29 (3.45%)
         occurrences all number
    1
    1
    Psychiatric disorders
    Aggresion/Agitation
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/28764646
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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