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    Clinical Trial Results:
    A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC)

    Summary
    EudraCT number
    		 2021-003441-38
    Trial protocol
    		 FR   DE   ES   IT   BE  
    Global end of trial date
    14 Oct 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    10 Jul 2025
    First version publication date
    10 Jul 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    1042-TSC-3001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05323734
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND Number: 155634
    Sponsors
    Sponsor organisation name
    Marinus Pharmaceuticals, Inc.
    Sponsor organisation address
    5 Radnor Corporate Center, 100 Matsonford Road, Suite 500, Radnor, PA, India, 19087
    Public contact
    Global Integrated Evidence Generation, Marinus Pharmaceuticals, Inc., +46 853339500, clinical@immedica.com
    Scientific contact
    Global Integrated Evidence Generation, Marinus Pharmaceuticals, Inc., +46 853339500, clinical@immedica.com
    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
    14 Oct 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Sep 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Oct 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the safety and efficacy of ganaxolone compared to placebo as adjunctive therapy for seizures associated with TSC in children and adults as assessed by the change from baselinea in the frequency of countable major motor and focal seizures (primary endpoint seizures) during the double-blind phase.
    Protection of trial subjects
    Not Applicable
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Mar 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 8
    Country: Number of subjects enrolled
    Canada: 10
    Country: Number of subjects enrolled
    China: 21
    Country: Number of subjects enrolled
    Germany: 10
    Country: Number of subjects enrolled
    Spain: 19
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    United States: 42
    Worldwide total number of subjects
    129
    EEA total number of subjects
    38
    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)
    3
    Children (2-11 years)
    59
    Adolescents (12-17 years)
    21
    Adults (18-64 years)
    46
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This was a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive ganaxolone treatment in children and adults with TSC-related epilepsy.

    Pre-assignment
    Screening details
    		 A total of 129 participants were enrolled in the study.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Ganaxolone
    Arm description
    		 Participants were randomized to receive an oral suspension of ganaxolone based on their body weight. ganaxolone 63 milligrams/kilograms/day (mg/kg/day) was administered orally three times a day (TID) to participants weighing 28 kg or less. ganaxolone 1800 mg/day TID was administered orally to participants weighing more than 28 kg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ganaxolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Ganaxolone was administered as oral suspension, 3 times a day (TID)

    Arm title
    		 Placebo
    Arm description
    		 Participants were administered with matching placebo as oral suspension TID based on the body weight.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo was administered as oral suspension TID.

    Number of subjects in period 1
    		Ganaxolone Placebo
    Started
    64
    65
    Completed
    55
    60
    Not completed
    9
    5
         Adverse event, serious fatal
    5
    2
         Consent withdrawn by subject
    2
    -
         Physician decision
    1
    -
         Participant Withdrawn Due
    -
    1
         Lack of efficacy
    1
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ganaxolone
    Reporting group description
    		 Participants were randomized to receive an oral suspension of ganaxolone based on their body weight. ganaxolone 63 milligrams/kilograms/day (mg/kg/day) was administered orally three times a day (TID) to participants weighing 28 kg or less. ganaxolone 1800 mg/day TID was administered orally to participants weighing more than 28 kg.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants were administered with matching placebo as oral suspension TID based on the body weight.

    Reporting group values
    		 Ganaxolone Placebo Total
    Number of subjects
    		 64 65 129
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 2 1 3
        Children (2-11 years)
    		 31 28 59
        Adolescents (12-17 years)
    		 11 10 21
        Adults (18-64 years)
    		 20 26 46
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 14.7 ( 11.0 ) 16.1 ( 11.2 ) -
    Gender categorical
    Units: Subjects
        Female
    		 36 29 65
        Male
    		 28 36 64

    End points

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    End points reporting groups
    Reporting group title
    Ganaxolone
    Reporting group description
    		 Participants were randomized to receive an oral suspension of ganaxolone based on their body weight. ganaxolone 63 milligrams/kilograms/day (mg/kg/day) was administered orally three times a day (TID) to participants weighing 28 kg or less. ganaxolone 1800 mg/day TID was administered orally to participants weighing more than 28 kg.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants were administered with matching placebo as oral suspension TID based on the body weight.

    Primary: Percent Change From Baseline in 28-day Seizure Frequency for Primary Seizure Type During Double Blind Period

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    End point title
    		 Percent Change From Baseline in 28-day Seizure Frequency for Primary Seizure Type During Double Blind Period
    End point description
    Primary seizures include atonic/drop, bilateral clonic, bilateral tonic, focal motor with altered awareness, focal motor with intact awareness, focal to bilateral tonic-clonic seizures, focal with hypotonia impaired awareness, and generalized tonic-clonic. Seizure frequency was calculated as the total number of seizures divided by the number of days with seizure data in the period, multiplied by 28. Percent change from Baseline in 28-day seizure frequency was calculated as follows for each participant: post-baseline 28-day seizure frequency minus baseline 28-day seizure frequency whole divided by baseline 28-day seizure frequency and multiplied by 100. Intent-to-treat (ITT) Set comprises of randomized participants who dosed and had at least one post-baseline efficacy assessment.
    End point type
    Primary
    End point timeframe
    Baseline (Day 1), Day 28
    End point values
    		 Ganaxolone Placebo
    Number of subjects analysed
    64
    65
    Units: Percent change
        arithmetic mean (standard deviation)
    -7.50 ( 60.430 )
    13.57 ( 77.374 )
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Ganaxolone v Placebo
    Number of subjects included in analysis
    129
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0904 [1]
    Method
    		 Wilcoxon Rank-Sum
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -14.53
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -32.04
         upper limit
    		 2.48
    Notes
    [1] - Wilcoxon Rank-Sum statistic is applied using a 2-sided significance level of 0.05.

    Secondary: Number of Participants Who Were Considered as Treatment Responders During Double Blind Period

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    End point title
    		 Number of Participants Who Were Considered as Treatment Responders During Double Blind Period
    End point description
    Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in primary seizure type frequency during the given period. The analysis was performed in ITT population.
    End point type
    Secondary
    End point timeframe
    Up to 16 weeks
    End point values
    		 Ganaxolone Placebo
    Number of subjects analysed
    64
    65
    Units: Participants
        number (not applicable)
    12
    8
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v Ganaxolone
    Number of subjects included in analysis
    129
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.3407
    Method
    		 Fisher exact
    Parameter type
    		 Difference in percentage
    Point estimate
    6.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -6.4
         upper limit
    		 20.1

    Secondary: Number of Responders to Clinical Global Impression of Improvement (CGI-I) Scale as Assessed by Parent/Caregiver

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    End point title
    		 Number of Responders to Clinical Global Impression of Improvement (CGI-I) Scale as Assessed by Parent/Caregiver
    End point description
    The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/ legally authorized representative (LAR) and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the investigational product (IP) relative to baseline (prior to treatment with the IP). The participant was rated as follows: 1 – very much improved, 2 – much improved, 3 – minimally improved, 4 – no change, 5 – minimally worse, 6 – much worse, and 7 – very much worse. Higher scores indicated worse condition. Number of responders to each score on the scale has been presented. The estimated odds ratio of the ganaxolone group compared to the placebo group based on proportional odds logistic regression with treatment as a factor. The participants in ITT Set who responded to CGI-I scale has been presented. The estimated odds ratio of the ganaxolone group compared to the placebo group based on proportional odds logistic regression with treatment as a factor.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through 16 weeks
    End point values
    		 Ganaxolone Placebo
    Number of subjects analysed
    58
    58
    Units: Participants
    number (not applicable)
        Very Much Improved
    5
    8
        Much Improved
    16
    15
        Minimally Improved
    14
    12
        No Change
    19
    19
        Minimally Worse
    2
    4
        Much Worse
    2
    0
        Very Much Worse
    0
    0
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Ganaxolone v Placebo
    Number of subjects included in analysis
    116
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.7069
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.88
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.46
         upper limit
    		 1.7

    Secondary: Number of Responders to Clinical Global Impression of Improvement (CGI-I) Scale as Assessed by Clinician

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    End point title
    		 Number of Responders to Clinical Global Impression of Improvement (CGI-I) Scale as Assessed by Clinician
    End point description
    The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/ LAR and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the IP relative to baseline (prior to treatment with the IP). The participant was rated as follows: 1 – very much improved, 2 – much improved, 3 – minimally improved, 4 – no change, 5 – minimally worse, 6 – much worse, and 7 – very much worse. Higher scores indicated worse outcomes. The participants in ITT Set and who responded to each score on the scale has been presented.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through 16 weeks
    End point values
    		 Ganaxolone Placebo
    Number of subjects analysed
    51
    53
    Units: Participants
    number (not applicable)
        Very Much Improved
    0
    3
        Much Improved
    14
    13
        Minimally Improved
    12
    11
        No Change
    22
    25
        Minimally Worse
    2
    1
        Much Worse
    1
    0
        Very Much Worse
    0
    0
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Ganaxolone v Placebo
    Number of subjects included in analysis
    104
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.6434
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.85
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.42
         upper limit
    		 1.72

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 16 Weeks
    Adverse event reporting additional description
    Serious treatment emergent adverse events and treatment emergent adverse events were collected in Safety Analysis Set which comprises of all randomized participants who received at least 1 dose of the IP.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.0
    Reporting groups
    Reporting group title
    Ganaxolone
    Reporting group description
    		 Participants were randomized to receive an oral suspension of ganaxolone based on their body weight. ganaxolone 63 milligrams/kilograms/day (mg/kg/day) was administered orally three times a day (TID) to participants weighing 28 kg or less. ganaxolone 1800 mg/day TID was administered orally to participants weighing more than 28 kg.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants were administered with matching placebo as oral suspension TID based on the body weight.

    Serious adverse events
    		 Ganaxolone Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 64 (7.81%)
    6 / 65 (9.23%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Craniocerebral Injury
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Nervous system disorders
    Seizure
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    Respiratory, thoracic and mediastinal disorders
    Respiratory Failure
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Erysipelas
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    Pneumonia Aspiration
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    Pneumonia
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 3%
    Non-serious adverse events
    		 Ganaxolone Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    58 / 64 (90.63%)
    49 / 65 (75.38%)
    Investigations
    Weight Decreased
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    Weight Increased
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 65 (0.00%)
         occurrences all number
    3
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 64 (1.56%)
    2 / 65 (3.08%)
         occurrences all number
    1
    2
    Fall
         subjects affected / exposed
    3 / 64 (4.69%)
    3 / 65 (4.62%)
         occurrences all number
    4
    4
    Skin Abrasion
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    Upper Limb Fracture
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 65 (1.54%)
         occurrences all number
    2
    1
    Nervous system disorders
    Balance Disorder
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 65 (0.00%)
         occurrences all number
    3
    0
    Dizziness
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 65 (1.54%)
         occurrences all number
    2
    1
    Headache
         subjects affected / exposed
    6 / 64 (9.38%)
    2 / 65 (3.08%)
         occurrences all number
    7
    3
    Lethargy
         subjects affected / exposed
    2 / 64 (3.13%)
    2 / 65 (3.08%)
         occurrences all number
    2
    2
    Psychomotor Hyperactivity
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 65 (0.00%)
         occurrences all number
    7
    0
    Sedation
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 65 (0.00%)
         occurrences all number
    3
    0
    Seizure
         subjects affected / exposed
    9 / 64 (14.06%)
    7 / 65 (10.77%)
         occurrences all number
    21
    13
    Somnolence
         subjects affected / exposed
    18 / 64 (28.13%)
    11 / 65 (16.92%)
         occurrences all number
    25
    14
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 65 (0.00%)
         occurrences all number
    3
    0
    Fatigue
         subjects affected / exposed
    9 / 64 (14.06%)
    6 / 65 (9.23%)
         occurrences all number
    9
    8
    Pyrexia
         subjects affected / exposed
    10 / 64 (15.63%)
    11 / 65 (16.92%)
         occurrences all number
    14
    11
    Gastrointestinal disorders
    Abdominal Pain Upper
         subjects affected / exposed
    0 / 64 (0.00%)
    2 / 65 (3.08%)
         occurrences all number
    0
    2
    Constipation
         subjects affected / exposed
    4 / 64 (6.25%)
    4 / 65 (6.15%)
         occurrences all number
    4
    5
    Dental Caries
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 65 (1.54%)
         occurrences all number
    2
    1
    Diarrhoea
         subjects affected / exposed
    4 / 64 (6.25%)
    2 / 65 (3.08%)
         occurrences all number
    4
    3
    Mouth Ulceration
         subjects affected / exposed
    3 / 64 (4.69%)
    1 / 65 (1.54%)
         occurrences all number
    3
    2
    Toothache
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 65 (1.54%)
         occurrences all number
    2
    1
    Vomiting
         subjects affected / exposed
    5 / 64 (7.81%)
    4 / 65 (6.15%)
         occurrences all number
    7
    4
    Reproductive system and breast disorders
    Menstruation Irregular
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Adenoidal Hypertrophy
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    Cough
         subjects affected / exposed
    6 / 64 (9.38%)
    3 / 65 (4.62%)
         occurrences all number
    6
    3
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    3 / 64 (4.69%)
    1 / 65 (1.54%)
         occurrences all number
    3
    1
    Psychiatric disorders
    Affect Lability
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    Aggression
         subjects affected / exposed
    1 / 64 (1.56%)
    2 / 65 (3.08%)
         occurrences all number
    1
    2
    Insomnia
         subjects affected / exposed
    1 / 64 (1.56%)
    2 / 65 (3.08%)
         occurrences all number
    1
    2
    Irritability
         subjects affected / exposed
    2 / 64 (3.13%)
    4 / 65 (6.15%)
         occurrences all number
    2
    7
    Sleep Disorder
         subjects affected / exposed
    2 / 64 (3.13%)
    3 / 65 (4.62%)
         occurrences all number
    2
    3
    Infections and infestations
    Conjunctivitis
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 65 (1.54%)
         occurrences all number
    2
    1
    Covid-19
         subjects affected / exposed
    2 / 64 (3.13%)
    3 / 65 (4.62%)
         occurrences all number
    2
    3
    Gastrointestinal Infection
         subjects affected / exposed
    0 / 64 (0.00%)
    2 / 65 (3.08%)
         occurrences all number
    0
    2
    Influenza
         subjects affected / exposed
    4 / 64 (6.25%)
    1 / 65 (1.54%)
         occurrences all number
    4
    1
    Nasopharyngitis
         subjects affected / exposed
    5 / 64 (7.81%)
    4 / 65 (6.15%)
         occurrences all number
    6
    5
    Respiratory Tract Infection
         subjects affected / exposed
    2 / 64 (3.13%)
    2 / 65 (3.08%)
         occurrences all number
    2
    2
    Upper Respiratory Tract Infection
         subjects affected / exposed
    3 / 64 (4.69%)
    6 / 65 (9.23%)
         occurrences all number
    3
    7
    Urinary Tract Infection
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 65 (1.54%)
         occurrences all number
    2
    1
    Metabolism and nutrition disorders
    Decreased Appetite
         subjects affected / exposed
    6 / 64 (9.38%)
    4 / 65 (6.15%)
         occurrences all number
    7
    7
    Increased Appetite
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Feb 2022
    The rationale for the major changes in this protocol amendment are as follows: • The availability of new Phase 2 data raised the question of a cannabidiol (CBD) interaction. As a result, participants taking CBD will be monitored closely for adverse events (AEs), and specifically sedation-related AEs throughout the study. Participants were also stratified according to concomitant CBD use. • The allowance of an additional 2 weeks of titration after the 4 weeks titration period at the start of the maintenance period was removed as were specific dosing paradigms for participants taking Epidiolex > 10 milligrams (mg)/kg/day. This change will ensure that all participants will have the same target dose regardless of their background CBD therapy.
    07 Dec 2022
    The following changes are made in this global protocol amendment from version 2.0 to version 3.0 to: • Revise inclusion criteria to include participants aged 2 to 65 years of age. • Add a section on contraception use to include acceptable barrier methods and donation of sperm and ova. • Add details on pregnancy testing. • Elaborate on dose adjustments and rescue medications. • Update endpoint analyses to incorporate analyses related to European Medicines Agency (EMA). • Add details on blood volumes approval by investigator for participants < 15 kilograms (kg) weight. • The dosing instructions for the oral suspension were updated to match the package insert.
    08 Dec 2022
    The following changes are made in this protocol amendment from version 3.0 (applicable to Europe [EU], Middle East and North Africa [MENA] and Oceania [OC]) to version 3.1 (applicable to North America [United States and Canada] and China) to: • Revise inclusion criteria to include participants aged 1 to 65 years of age. • Specify that genetic testing will not be permitted under this protocol in China. • Specified that investigational product will be stored in accordance with applicable requirements under the Controlled Substance Act and Drug Enforcement Administration regulations.
    12 Sep 2023
    The following changes are made in this global protocol amendment from version 3.0 to version 4.0 to: • Include updated information regarding completed and ongoing clinical studies. • Editorial updates throughout. • Sample size, participant age, and inclusion criteria were updated. • References to interim analysis were removed as no interim analysis was performed. • Serious adverse event (SAE) contact details were updated. • Country-specific amendment for China/North America has been combined with the global protocol.

    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 reported
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