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    Clinical Trial Results:
    A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol (CBD; GWP42003-P) in Infants with Infantile Spasms Following an Initial Open-label Pilot Study

    Summary
    EudraCT number
    		 2015-004904-50
    Trial protocol
    		 PL  
    Global end of trial date
    13 Jun 2019

    Results information
    Results version number
    		 v2(current)
    This version publication date
    15 Mar 2020
    First version publication date
    29 Dec 2019
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    Correction of one data point.

    Trial information

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    Trial identification
    Sponsor protocol code
    GWEP15100
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    GW Research Ltd
    Sponsor organisation address
    Sovereign House, Vision Park, Chivers Way, Histon, Cambridge, United Kingdom, CB24 9BZ
    Public contact
    Medical Enquiries, GW Research Ltd, medinfo@gwpharm.com
    Scientific contact
    Medical Enquiries, GW Research Ltd, medinfo@gwpharm.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001964-PIP01-16
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Jun 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    13 Jun 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jun 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This study evaluated the efficacy and safety of CBD (GWP42003-P) in subjects with infantile spasms (IS) who have failed to become spasm free following treatment with 1 or more approved IS therapies.
    Protection of trial subjects
    This trial was conducted in accordance with the ethical principles that have their origin in the World Medical Association (WMA) Declaration of Helsinki, adopted by the 18th WMA General Assembly, Helsinki, Finland, June 1964, and subsequent amendments. This trial was also designed to comply with ICH E6 Guideline for good clinical practice (EMA/CHMP/ICH/135/1995) and the European Clinical Trial Directive 2001/20/EC. The International Council for Harmonisation adopted guidelines and other relevant international guidelines, recommendations and requirements were taken into account as comprehensively as possible, as long as they did not violate Polish or US law.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    25 Apr 2017
    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
    United States: 8
    Country: Number of subjects enrolled
    Poland: 1
    Worldwide total number of subjects
    9
    EEA total number of subjects
    1
    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)
    9
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    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
    		 -

    Pre-assignment
    Screening details
    		 Subjects were screened to assess their eligibility to enter the trial within 7 days prior to the first dose administration.

    Period 1
    Period 1 title
    Pilot
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Pilot, Cohort 1: GWP42003-P
    Arm description
    		 Subjects 6 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GWP42003-P
    Investigational medicinal product code
    GWP42003-P
    Other name
    EPIDIOLEX, cannabidiol, CBD-OS
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were administered GWP42003-P twice daily or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal a total dose of 40mg/kg/day. Oral liquid formulation that is clear and colorless to yellow in appearance (100 mg/mL), in sesame oil with anhydrous ethanol added sweetener (sucralose) and strawberry flavoring. The oral liquid formulation was administered with a syringe.

    Arm title
    		 Pilot, Cohort 2: GWP42003-P
    Arm description
    		 Subjects 1 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GWP42003-P
    Investigational medicinal product code
    GWP42003-P
    Other name
    EPIDIOLEX, cannabidiol, CBD-OS
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were administered GWP42003-P, orally, twice daily or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose. Oral liquid formulation that is clear and colorless to yellow in appearance (100 mg/mL), in sesame oil with anhydrous ethanol added sweetener (sucralose) and strawberry flavoring. The oral liquid formulation was administered with a syringe.

    Number of subjects in period 1
    		Pilot, Cohort 1: GWP42003-P Pilot, Cohort 2: GWP42003-P
    Started
    5
    4
    Completed
    5
    4
    Period 2
    Period 2 title
    Open Label Extension (OLE)
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 OLE: GWP42003-P
    Arm description
    		 Following completion of the pilot period, subjects were eligible to participate in the OLE period. Subjects continued their same dose administered in the pilot period for a maximum of 1 year and completed a 10 day taper after completing the study or withdrawing.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GWP42003-P
    Investigational medicinal product code
    GWP42003-P
    Other name
    EPIDIOLEX, cannabidiol, CBD-OS
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were administered GWP42003-P, orally, twice daily or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose. Oral liquid formulation that is clear and colorless to yellow in appearance (100 mg/mL), in sesame oil with anhydrous ethanol added sweetener (sucralose) and strawberry flavoring. The oral liquid formulation was administered with a syringe.

    Number of subjects in period 2
    		OLE: GWP42003-P
    Started
    9
    Completed
    2
    Not completed
    7
         Not specified
    1
         Subject not receiving any benefit
    3
         Withdrawn consent by subject or caregiver
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pilot
    Reporting group description
    		 -

    Reporting group values
    		 Pilot Total
    Number of subjects
    		 9 9
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 9 9
    Age continuous
    Units: months
        arithmetic mean (standard deviation)
    		 12.2 ( 5.56 ) -
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 3 3
    Race
    Units: Subjects
        White
    		 8 8
        Other, not specified
    		 1 1

    End points

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    End points reporting groups
    Reporting group title
    Pilot, Cohort 1: GWP42003-P
    Reporting group description
    		 Subjects 6 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.

    Reporting group title
    Pilot, Cohort 2: GWP42003-P
    Reporting group description
    		 Subjects 1 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.
    Reporting group title
    OLE: GWP42003-P
    Reporting group description
    		 Following completion of the pilot period, subjects were eligible to participate in the OLE period. Subjects continued their same dose administered in the pilot period for a maximum of 1 year and completed a 10 day taper after completing the study or withdrawing.

    Subject analysis set title
    Pilot, All Subjects
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    This analysis set includes all subjects from both Cohort 1 and Cohort 2 in the Pilot Period.

    Primary: Pilot and OLE: Number of subjects with clinically significant electrocardiogram findings

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    End point title
    		 Pilot and OLE: Number of subjects with clinically significant electrocardiogram findings [1]
    End point description
    Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
    End point type
    Primary
    End point timeframe
    Pilot, up to Day 15 OLE, up to Day 389
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [2]
    9 [3]
    Units: subjects
    0
    0
    Notes
    [2] - Safety Analysis Set
    [3] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot and OLE: Number of subjects with clinically significant physical examination findings

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    End point title
    		 Pilot and OLE: Number of subjects with clinically significant physical examination findings [4]
    End point description
    Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
    End point type
    Primary
    End point timeframe
    Pilot, up to Day 15 OLE, up to Day 389
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [5]
    9 [6]
    Units: subjects
    0
    0
    Notes
    [5] - Safety Analysis Set
    [6] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot and OLE: Number of subjects with clinically significant vital sign findings

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    End point title
    		 Pilot and OLE: Number of subjects with clinically significant vital sign findings [7]
    End point description
    Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
    End point type
    Primary
    End point timeframe
    Pilot, up to Day 15 OLE, up to Day 389
    Notes
    [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [8]
    9 [9]
    Units: subjects
    0
    0
    Notes
    [8] - Safety Analysis Set
    [9] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot and OLE: Number of subjects with severe treatment-emergent adverse events (TEAEs)

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    End point title
    		 Pilot and OLE: Number of subjects with severe treatment-emergent adverse events (TEAEs) [10]
    End point description
    TEAEs are defined as AEs not present prior to the first investigational medicinal product (IMP) or placebo administration or any event already present that worsened in severity or frequency following IMP exposure. A TEAE occurred if the event was continuous from Baseline and was serious; IMP related; or resulted in death, discontinuation, interruption, or reduction of IMP. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
    End point type
    Primary
    End point timeframe
    Pilot, up to Day 15 OLE, up to Day 417
    Notes
    [10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [11]
    9 [12]
    Units: subjects
    7
    6
    Notes
    [11] - Safety Analysis Set
    [12] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot: Number of responders

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    End point title
    		 Pilot: Number of responders [13]
    End point description
    A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
    End point type
    Primary
    End point timeframe
    Pilot, Baseline to Day 15
    Notes
    [13] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 Pilot, Cohort 1: GWP42003-P Pilot, Cohort 2: GWP42003-P
    Number of subjects analysed
    5 [14]
    4 [15]
    Units: subjects
    0
    0
    Notes
    [14] - Safety Analysis Set
    [15] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot: Percentage of responders

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    End point title
    		 Pilot: Percentage of responders [16]
    End point description
    A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
    End point type
    Primary
    End point timeframe
    Pilot, Baseline to Day 15
    Notes
    [16] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 Pilot, Cohort 1: GWP42003-P Pilot, Cohort 2: GWP42003-P
    Number of subjects analysed
    5 [17]
    4 [18]
    Units: percentage
        number (not applicable)
    0
    0
    Notes
    [17] - Safety Analysis Set
    [18] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot and OLE: Number of subjects with any low or high hematology laboratory parameter value

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    End point title
    		 Pilot and OLE: Number of subjects with any low or high hematology laboratory parameter value [19]
    End point description
    Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Primary
    End point timeframe
    Pilot, Day 1 to OLE, End of Taper, Day 389
    Notes
    [19] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [20]
    9 [21]
    Units: subjects
        Pilot, Day 4, Low, n=9
    999
    4
        Pilot, Day 4, High, n=9
    999
    4
        Pilot, Day 15, Low, n=8
    999
    1
        Pilot, Day 15, High, n=8
    999
    3
        OLE, Day 19, Low, n=8
    3
    999
        OLE, Day 19, High, n=8
    4
    999
        OLE, Day 29, Low, n=8
    3
    999
        OLE, Day 29, High, n=8
    4
    999
        OLE, Day 43, Low, n=6
    1
    999
        OLE, Day 43, High, n=6
    2
    999
        OLE, Day 71, Low, n=5
    1
    999
        OLE, Day 71, High, n=5
    3
    999
        OLE, Day 127, Low, n=5
    1
    999
        OLE, Day 127, High, n=5
    3
    999
        OLE, Day 211, Low, n=3
    1
    999
        OLE, Day 211, High, n=3
    2
    999
        OLE, Day 295, Low, n=3
    2
    999
        OLE, Day 295, High, n=3
    0
    999
        OLE, Day 379, Low, n=7
    4
    999
        OLE, Day 379, High, n=7
    4
    999
        OLE, Day 389, Low, n=6
    1
    999
        OLE, Day 389, High, n=6
    4
    999
    Notes
    [20] - Safety Analysis Set
    [21] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot and OLE: Number of subjects with any low or high biochemistry laboratory parameter value

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    End point title
    		 Pilot and OLE: Number of subjects with any low or high biochemistry laboratory parameter value [22]
    End point description
    Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Primary
    End point timeframe
    Pilot, Day 1 to OLE, End of Taper, Day 389
    Notes
    [22] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [23]
    9 [24]
    Units: subjects
        Pilot, Day 4, Low, n=9
    999
    7
        Pilot, Day 4, High, n=9
    999
    5
        Pilot, Day 15, Low, n=8
    999
    5
        Pilot, Day 15, High, n=8
    999
    7
        OLE, Day 19, Low, n=9
    6
    999
        OLE, Day 19, High, n=9
    7
    999
        OLE, Day 29, Low, n=8
    6
    999
        OLE, Day 29, High, n=8
    6
    999
        OLE, Day 43, Low, n=6
    4
    999
        OLE, Day 43, High, n=6
    5
    999
        OLE, Day 71, Low, n=5
    2
    999
        OLE, Day 71, High, n=5
    4
    999
        OLE, Day 127, Low, n=5
    4
    999
        OLE, Day 127, High, n=5
    3
    999
        OLE, Day 211, Low, n=3
    2
    999
        OLE, Day 211, High, n=3
    3
    999
        OLE, Day 295, Low, n=3
    2
    999
        OLE, Day 295, High, n=3
    3
    999
        OLE, Day 379, Low, n=7
    5
    999
        OLE, Day 379, High, n=7
    7
    999
        OLE, Day 389, Low, n=5
    3
    999
        OLE, Day 389, High, n=5
    2
    999
    Notes
    [23] - Safety Analysis Set
    [24] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Pilot and OLE: Number of subjects with any clinically relevant urinalysis parameter value

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    End point title
    		 Pilot and OLE: Number of subjects with any clinically relevant urinalysis parameter value [25]
    End point description
    Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Primary
    End point timeframe
    Pilot, Day 1 to OLE, End of Taper, Day 389
    Notes
    [25] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No stat analysis conducted for this outcome measure.
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [26]
    9 [27]
    Units: subjects
        Pilot, Day 4, n=7
    999
    0
        Pilot, Day 15, n=9
    999
    0
        OLE, Day 19, n=7
    0
    999
        OLE, Day 29, n=7
    0
    999
        OLE, Day 43, n=6
    0
    999
        OLE, Day 71, n=5
    1
    999
        OLE, Day 127, n=4
    0
    999
        OLE, Day 211, n=3
    0
    999
        OLE, Day 295, n=2
    0
    999
        OLE, Day 379. n=6
    0
    999
        OLE, Day 389, n=3
    0
    999
    Notes
    [26] - Safety Analysis Set
    [27] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Number of subjects free of clinical spasms

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    End point title
    		 Pilot and OLE: Number of subjects free of clinical spasms
    End point description
    Clinical spasms were determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [28]
    9 [29]
    Units: subjects
        Pilot, Day 15, n=0,9
    999
    0
        OLE, Day 29, n=8,0
    1
    999
        OLE, Day 43, n=6,0
    2
    999
        OLE, Day 127, n=4,0
    1
    999
        OLE, Day 211, n=3,0
    1
    999
        OLE, Day 295, n=2,0
    1
    999
        OLE, Day 379, n=7,0
    3
    999
    Notes
    [28] - Safety Analysis Set
    [29] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Percentage of subjects free of clinical spasms

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    End point title
    		 Pilot and OLE: Percentage of subjects free of clinical spasms
    End point description
    Clinical spasms were determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [30]
    9 [31]
    Units: percent
    number (not applicable)
        Pilot, Day 15, n=0, 9
    999
    0
        OLE, Day 29, n=8,0
    11.1
    999
        OLE, Day 43, n=6,0
    22.2
    999
        OLE, Day 127, n=4,0
    11.1
    999
        OLE, Day 211, n=3,0
    11.1
    999
        OLE, Day 295, n=2,0
    11.1
    999
        OLE, Day 379, n=7,0
    33.3
    999
    Notes
    [30] - Safety Analysis Set
    [31] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Number of subjects with a resolution of hypsarrhythmia

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    End point title
    		 Pilot and OLE: Number of subjects with a resolution of hypsarrhythmia
    End point description
    Resolution of hypsarrhythmia was determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Secondary
    End point timeframe
    Pilot: Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [32]
    9 [33]
    Units: subjects
        Pilot, Day 15, n=0, 9
    999
    0
        OLE, Day 29, n=8,0
    1
    999
        OLE, Day 43, n=6,0
    0
    999
        OLE, Day 127, n=4,0
    1
    999
        OLE, Day 211, n=3,0
    0
    999
        OLE, Day 295, n=2,0
    1
    999
        OLE, Day 379, n=7,0
    3
    999
    Notes
    [32] - Safety Analysis Set
    [33] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Percentage of subjects with a resolution of hypsarrhythmia

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    End point title
    		 Pilot and OLE: Percentage of subjects with a resolution of hypsarrhythmia
    End point description
    Resolution of hypsarrhythmia was determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [34]
    9 [35]
    Units: percent
    number (not applicable)
        Pilot, Day 15, n=0,9
    999
    0
        OLE, Day 29, n=8,0
    11.1
    999
        OLE, Day 43, n=6,0
    0
    999
        OLE, Day 127, n=4,0
    11.1
    999
        OLE, Day 211, n=3,0
    0
    999
        OLE, Day 295, n=2,0
    11.1
    999
        OLE, Day 379, n=7,0
    33.3
    999
    Notes
    [34] - Safety Analysis Set
    [35] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Number of subjects experiencing spasms and seizures subtypes

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    End point title
    		 Pilot and OLE: Number of subjects experiencing spasms and seizures subtypes
    End point description
    Caregivers recorded subject's spasms and seizures by category in a daily diary. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point.
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [36]
    9 [37]
    Units: subjects
        Pilot, Day 4, Clonic
    999
    0
        Pilot, Day 4, Tonic-Clonic
    999
    1
        Pilot, Day 4, Atonic
    999
    0
        Pilot, Day 4, Myoclonic
    999
    0
        Pilot, Day 4, Focal
    999
    1
        Pilot, Day 4, Absence
    999
    0
        Pilot, Day 4, Not Done
    999
    0
        Pilot, Day 15, Clonic
    999
    0
        Pilot, Day 15, Tonic-Clonic
    999
    0
        Pilot, Day 15, Atonic
    999
    0
        Pilot, Day 15, Myoclonic
    999
    0
        Pilot, Day 15, Focal
    999
    2
        Pilot, Day 15, Absence
    999
    0
        Pilot, Day 15, Not Done
    999
    0
        OLE, Day 19, Clonic
    0
    999
        OLE, Day 19, Tonic-Clonic
    0
    999
        OLE, Day 19, Atonic
    0
    999
        OLE, Day 19, Myoclonic
    0
    999
        OLE, Day 19, Focal
    1
    999
        OLE, Day 19, Absence
    0
    999
        OLE, Day 19, Not Done
    0
    999
        OLE, Day 29, Clonic
    0
    999
        OLE, Day 29, Tonic-Clonic
    0
    999
        OLE, Day 29, Atonic
    0
    999
        OLE, Day 29, Myoclonic
    0
    999
        OLE, Day 29, Focal
    1
    999
        OLE, Day 29, Absence
    0
    999
        OLE, Day 29, Not Done
    0
    999
        OLE, Day 127, Clonic
    0
    999
        OLE, Day 127, Tonic-Clonic
    1
    999
        OLE, Day 127, Atonic
    0
    999
        OLE, Day 127, Myoclonic
    1
    999
        OLE, Day 127, Focal
    0
    999
        OLE, Day 127, Absence
    1
    999
        OLE, Day 127, Not Done
    0
    999
        OLE, Day 211, Clonic
    0
    999
        OLE, Day 211, Tonic-Clonic
    1
    999
        OLE, Day 211, Atonic
    0
    999
        OLE, Day 211, Myoclonic
    0
    999
        OLE, Day 211, Focal
    0
    999
        OLE, Day 211, Absence
    1
    999
        OLE, Day 211, Not Done
    0
    999
        OLE, Day 295, Clonic
    0
    999
        OLE, Day 295, Tonic-Clonic
    1
    999
        OLE, Day 295, Atonic
    1
    999
        OLE, Day 295, Myoclonic
    0
    999
        OLE, Day 295, Focal
    0
    999
        OLE, Day 295, Absence
    1
    999
        OLE, Day 295, Not Done
    0
    999
        OLE, Day 379, Clonic
    1
    999
        OLE, Day 379, Tonic-Clonic
    1
    999
        OLE, Day 379, Atonic
    0
    999
        OLE, Day 379, Myoclonic
    0
    999
        OLE, Day 379, Focal
    1
    999
        OLE, Day 379, Absence
    1
    999
        OLE, Day 379, Not Done
    0
    999
    Notes
    [36] - Safety Analysis Set
    [37] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Average time to cessation of spasms

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    End point title
    		 Pilot and OLE: Average time to cessation of spasms
    End point description
    Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9
    9
    Units: hours
        arithmetic mean (standard deviation)
    998 ( 998 )
    998 ( 998 )
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Caregiver Global Impression of Change (CGIC)

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    End point title
    		 Pilot and OLE: Caregiver Global Impression of Change (CGIC)
    End point description
    The CGIC is a single question assessment completed by the caregiver. The question assesses the status of the subject's condition since they've started treatment. The caregiver provides a rating on a 7 point scale from 1-"very much improved" to 7-"very much worse". Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point.
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [38]
    9 [39]
    Units: score on a scale
    number (not applicable)
        Pilot, Day 15, Very Much Improved
    999
    1
        Pilot, Day 15, Much Improved
    999
    0
        Pilot, Day 15, Slightly Improved
    999
    7
        Pilot, Day 15, No Change
    999
    1
        Pilot, Day 15, Slightly Worse
    999
    0
        Pilot, Day 15, Much Worse
    999
    0
        Pilot, Day 15, Very Much Worse
    999
    0
        Pilot, Day 15, Not Done
    999
    0
        OLE, Day 29, Very Much Improved
    1
    999
        OLE, Day 29, Much Improved
    1
    999
        OLE, Day 29, Slightly Improved
    5
    999
        OLE, Day 29, No Change
    1
    999
        OLE, Day 29, Slightly Worse
    0
    999
        OLE, Day 29, Much Worse
    0
    999
        OLE, Day 29, Very Much Worse
    0
    999
        OLE, Day 29, Not Done
    0
    999
        OLE, Day 43, Very Much Improved
    2
    999
        OLE, Day 43, Much Improved
    1
    999
        OLE, Day 43, Slightly Improved
    3
    999
        OLE, Day 43, No Change
    0
    999
        OLE, Day 43, Slightly Worse
    0
    999
        OLE, Day 43, Much Worse
    0
    999
        OLE, Day 43, Very Much Worse
    0
    999
        OLE, Day 43, Not Done
    0
    999
        OLE, Day 71, Very Much Improved
    3
    999
        OLE, Day 71, Much Improved
    0
    999
        OLE, Day 71, Slightly Improved
    1
    999
        OLE, Day 71, No Change
    0
    999
        OLE, Day 71, Slightly Worse
    1
    999
        OLE, Day 71, Much Worse
    0
    999
        OLE, Day 71, Very Much Worse
    0
    999
        OLE, Day 71, Not Done
    0
    999
        OLE, Day 127, Very Much Improved
    2
    999
        OLE, Day 127, Much Improved
    2
    999
        OLE, Day 127, Slightly Improved
    0
    999
        OLE, Day 127, No Change
    0
    999
        OLE, Day 127, Slightly Worse
    0
    999
        OLE, Day 127, Much Worse
    0
    999
        OLE, Day 127, Very Much Worse
    0
    999
        OLE, Day 127, Not Done
    0
    999
        OLE, Day 211, Very Much Improved
    1
    999
        OLE, Day 211, Much Improved
    2
    999
        OLE, Day 211, Slightly Improved
    0
    999
        OLE, Day 211, No Change
    0
    999
        OLE, Day 211, Slightly Worse
    0
    999
        OLE, Day 211, Much Worse
    0
    999
        OLE, Day 211, Very Much Worse
    0
    999
        OLE, Day 211, Not Done
    0
    999
        OLE, Day 295, Very Much Improved
    1
    999
        OLE, Day 295, Much Improved
    2
    999
        OLE, Day 295, Slightly Improved
    0
    999
        OLE, Day 295, No Change
    0
    999
        OLE, Day 295, Slightly Worse
    0
    999
        OLE, Day 295, Much Worse
    0
    999
        OLE, Day 295, Very Much Worse
    0
    999
        OLE, Day 295, Not Done
    0
    999
        OLE, Day 379, Very Much Improved
    2
    999
        OLE, Day 379, Much Improved
    1
    999
        OLE, Day 379, Slightly Improved
    3
    999
        OLE, Day 379, No Change
    0
    999
        OLE, Day 379, Slightly Worse
    1
    999
        OLE, Day 379, Much Worse
    1
    999
        OLE, Day 379, Very Much Worse
    0
    999
        OLE, Day 379, Not Done
    1
    999
    Notes
    [38] - Safety Analysis Set
    [39] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Pilot and OLE: Physician Global Impression of Change (PGIC)

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    End point title
    		 Pilot and OLE: Physician Global Impression of Change (PGIC)
    End point description
    The PGIC is a single question assessment completed by the investigator. The question assesses the status of the subject's condition since they've started treatment. The investigator provides a rating on a 7 point scale from 1-"very much improved" to 7-"very much worse". Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point.
    End point type
    Secondary
    End point timeframe
    Pilot, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [40]
    9 [41]
    Units: score on a scale
    number (not applicable)
        Pilot, Day 15, Very Much Improved
    999
    0
        Pilot, Day 15, Much Improved
    999
    1
        Pilot, Day 15, Slightly Improved
    999
    5
        Pilot, Day 15, No Change
    999
    3
        Pilot, Day 15, Slightly Worse
    999
    0
        Pilot, Day 15, Much Worse
    999
    0
        Pilot, Day 15, Very Much Worse
    999
    0
        Pilot, Day 15, Not Done
    999
    0
        OLE, Day 29, Very Much Improved
    1
    999
        OLE, Day 29, Much Improved
    1
    999
        OLE, Day 29, Slightly Improved
    2
    999
        OLE, Day 29, No Change
    4
    999
        OLE, Day 29, Slightly Worse
    0
    999
        OLE, Day 29, Much Worse
    0
    999
        OLE, Day 29, Very Much Worse
    0
    999
        OLE, Day 29, Not Done
    0
    999
        OLE, Day 43, Very Much Improved
    1
    999
        OLE, Day 43, Much Improved
    0
    999
        OLE, Day 43, Slightly Improved
    4
    999
        OLE, Day 43, No Change
    1
    999
        OLE, Day 43, Slightly Worse
    0
    999
        OLE, Day 43, Much Worse
    0
    999
        OLE, Day 43, Very Much Worse
    0
    999
        OLE, Day 43, Not Done
    0
    999
        OLE, Day 71, Very Much Improved
    1
    999
        OLE, Day 71, Much Improved
    2
    999
        OLE, Day 71, Slightly Improved
    1
    999
        OLE, Day 71, No Change
    1
    999
        OLE, Day 71, Slightly Worse
    0
    999
        OLE, Day 71, Much Worse
    0
    999
        OLE, Day 71, Very Much Worse
    0
    999
        OLE, Day 71, Not Done
    0
    999
        OLE, Day 127, Very Much Improved
    1
    999
        OLE, Day 127, Much Improved
    2
    999
        OLE, Day 127, Slightly Improved
    1
    999
        OLE, Day 127, No Change
    0
    999
        OLE, Day 127, Slightly Worse
    0
    999
        OLE, Day 127, Much Worse
    0
    999
        OLE, Day 127, Very Much Worse
    0
    999
        OLE, Day 127, Not Done
    0
    999
        OLE, Day 211, Very Much Improved
    0
    999
        OLE, Day 211, Much Improved
    1
    999
        OLE, Day 211, Slightly Improved
    1
    999
        OLE, Day 211, No Change
    1
    999
        OLE, Day 211, Slightly Worse
    0
    999
        OLE, Day 211, Much Worse
    0
    999
        OLE, Day 211, Very Much Worse
    0
    999
        OLE, Day 211, Not Done
    0
    999
        OLE, Day 295, Very Much Improved
    0
    999
        OLE, Day 295, Much Improved
    1
    999
        OLE, Day 295, Slightly Improved
    0
    999
        OLE, Day 295, No Change
    1
    999
        OLE, Day 295, Slightly Worse
    0
    999
        OLE, Day 295, Much Worse
    1
    999
        OLE, Day 295, Very Much Worse
    0
    999
        OLE, Day 295, Not Done
    0
    999
        OLE, Day 379, Very Much Improved
    0
    999
        OLE, Day 379, Much Improved
    1
    999
        OLE, Day 379, Slightly Improved
    3
    999
        OLE, Day 379, No Change
    2
    999
        OLE, Day 379, Slightly Worse
    1
    999
        OLE, Day 379, Much Worse
    1
    999
        OLE, Day 379, Very Much Worse
    0
    999
        OLE, Day 379, Not Done
    1
    999
    Notes
    [40] - Safety Analysis Set
    [41] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Number of responders

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    End point title
    		 OLE: Number of responders
    End point description
    A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. n=number of subjects with evaluable data.
    End point type
    Secondary
    End point timeframe
    OLE, Day 16 to Day 379
    End point values
    		 OLE: GWP42003-P
    Number of subjects analysed
    9 [42]
    Units: subjects
        OLE, Day 29, n=8
    1
        OLE, Day 43, n=6
    0
        OLE, Day 127, n=4
    0
        OLE, Day 211, n=3
    0
        OLE, Day 295, n=2
    1
        OLE, Day 379, n=7
    3
    Notes
    [42] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Percentage of responders

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    End point title
    		 OLE: Percentage of responders
    End point description
    A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. n=number of subjects with evaluable data.
    End point type
    Secondary
    End point timeframe
    OLE, Day 16 to Day 379
    End point values
    		 OLE: GWP42003-P
    Number of subjects analysed
    9 [43]
    Units: percent
    number (not applicable)
        OLE, Day 29, n=8
    11.1
        OLE, Day 43, n=6
    0
        OLE, Day 127, n=4
    0
        OLE, Day 211, n=3
    0
        OLE, Day 295, n=2
    11.1
        OLE, Day 379, n=7
    33.3
    Notes
    [43] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Change from Baseline in height

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    End point title
    		 OLE: Change from Baseline in height
    End point description
    A positive change indicates an increase in the average subject's height. A negative change indicates a decrease in the average subject's height. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999 = No analysis was conducted for this treatment arm at this time point. CFB = Change from Baseline
    End point type
    Secondary
    End point timeframe
    Pilot, Baseline, Day 1 to OLE, End of Taper, Day 389
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [44]
    9 [45]
    Units: centimeters
    arithmetic mean (standard deviation)
        Pilot, Baseline, Day 1, n=9
    999 ( 999 )
    75.04 ( 9.557 )
        OLE, CFB, Day 29, n=8
    0.85 ( 0.980 )
    999 ( 999 )
        OLE, CFB, Day 43, n=6
    0.92 ( 1.530 )
    999 ( 999 )
        OLE, CFB, Day 71, n=5
    3.10 ( 0.652 )
    999 ( 999 )
        OLE, CFB, Day 127, n=4
    3.38 ( 1.377 )
    999 ( 999 )
        OLE, CFB, Day 211, n=3
    5.33 ( 1.258 )
    999 ( 999 )
        OLE, CFB, Day 295, n=3
    8.17 ( 1.528 )
    999 ( 999 )
        OLE, CFB, Day 379, n=8
    5.31 ( 4.036 )
    999 ( 999 )
        OLE, CFB, Day 389, n=6
    3.55 ( 4.085 )
    999 ( 999 )
    Notes
    [44] - Safety Analysis Set
    [45] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Change from Baseline in body weight

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    End point title
    		 OLE: Change from Baseline in body weight
    End point description
    A positive change indicates an increase in the average subject's height. A negative change indicates a decrease in the average subject's height. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999 = No analysis was conducted for this treatment arm at this time point. CFB=Change from Baseline
    End point type
    Secondary
    End point timeframe
    Pilot, Baseline, Day 1 to OLE, End of Taper, Day 389
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [46]
    9 [47]
    Units: kilograms
    arithmetic mean (standard deviation)
        Pilot, Baseline, Day 1, n=9
    999 ( 999 )
    9.92 ( 3.245 )
        OLE, CFB, Day 29, n=8
    0.39 ( 0.309 )
    999 ( 999 )
        OLE, CFB, Day 43, n=6
    0.75 ( 0.809 )
    999 ( 999 )
        OLE, CFB, Day 71, n=5
    1.10 ( 0.430 )
    999 ( 999 )
        OLE, CFB, Day 127, n=4
    1.25 ( 0.420 )
    999 ( 999 )
        OLE, CFB, Day 211, n=3
    1.17 ( 0.503 )
    999 ( 999 )
        OLE, CFB, Day 295, n=3
    2.10 ( 0.889 )
    999 ( 999 )
        OLE, CFB, Day 379, n=9
    1.19 ( 0.862 )
    999 ( 999 )
        OLE, CFB, Day 389, n=6
    0.98 ( 0.637 )
    999 ( 999 )
    Notes
    [46] - Safety Analysis Set
    [47] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Change from Baseline in head circumference

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    End point title
    		 OLE: Change from Baseline in head circumference
    End point description
    A positive change indicates an increase in the average subject's head circumference. A negative change indicates a decrease in the average subject's head circumference. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999 = No analysis was conducted for this treatment arm at this time point. CFB=Change from Baseline
    End point type
    Secondary
    End point timeframe
    Pilot, Baseline, Day 1 to OLE, End of Taper, Day 389
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [48]
    9 [49]
    Units: centimeters
    arithmetic mean (standard deviation)
        Pilot, Baseline, Day 1, n=9
    999 ( 999 )
    44.71 ( 2.724 )
        OLE, CFB, Day 29, n=8
    -0.01 ( 1.229 )
    999 ( 999 )
        OLE, CFB, Day 43, n=5
    0.54 ( 0.288 )
    999 ( 999 )
        OLE, CFB, Day 71, n=5
    0.70 ( 0.570 )
    999 ( 999 )
        OLE, CFB, Day 127, n=3
    1.50 ( 0.500 )
    999 ( 999 )
        OLE, CFB, Day 211, n=2
    0.75 ( 0.354 )
    999 ( 999 )
        OLE, CFB, Day 295, n=3
    -0.3 ( 2.31 )
    999 ( 999 )
        OLE, CFB, Day 379, n=8
    1.14 ( 1.707 )
    999 ( 999 )
        OLE, CFB, Day 389, n=5
    0.90 ( 1.782 )
    999 ( 999 )
    Notes
    [48] - Safety Analysis Set
    [49] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Change from Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score

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    End point title
    		 OLE: Change from Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score
    End point description
    The Vineland-II scores were assessed by the subject’s caregiver. Caregivers were asked to score questions in following categories the subject's communication, daily living, physical activity, problem behaviors, and social skills and relationships. Scoring was slightly different for each section, but generally ranged from "usually" to "never". Higher scores represent greater levels of functioning and lower skills represent lower levels of functioning. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. n=number of subjects with evaluable data. CFB=Change from Baseline
    End point type
    Secondary
    End point timeframe
    Pilot, Baseline, Day 1 to OLE, Day 379
    End point values
    		 OLE: GWP42003-P Pilot, All Subjects
    Number of subjects analysed
    9 [50]
    9 [51]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Pilot, Baseline, Day 1, n=9
    999 ( 999 )
    33.6 ( 37.27 )
        OLE, CFB, Day 211, n=3
    6.3 ( 3.51 )
    999 ( 999 )
        OLE, CFB, Day 379, n=9
    -5.4 ( 23.42 )
    999 ( 999 )
    Notes
    [50] - Safety Analysis Set
    [51] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: OLE: Number of subjects with relapse of spasms

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    End point title
    		 OLE: Number of subjects with relapse of spasms
    End point description
    Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
    End point type
    Secondary
    End point timeframe
    OLE Period, Day 16 to Day 379
    End point values
    		 OLE: GWP42003-P
    Number of subjects analysed
    9
    Units: subjects
    998
    No statistical analyses for this end point

    Secondary: OLE: Percentage of subjects with relapse of spasms

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    End point title
    		 OLE: Percentage of subjects with relapse of spasms
    End point description
    Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
    End point type
    Secondary
    End point timeframe
    OLE Period, Day 16 to Day 379
    End point values
    		 OLE: GWP42003-P
    Number of subjects analysed
    9
    Units: percent
        number (not applicable)
    998
    No statistical analyses for this end point

    Secondary: OLE: Average time to relapse

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    End point title
    		 OLE: Average time to relapse
    End point description
    Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
    End point type
    Secondary
    End point timeframe
    OLE Period, Day 16 to Day 379
    End point values
    		 OLE: GWP42003-P
    Number of subjects analysed
    9
    Units: day(s)
        arithmetic mean (standard deviation)
    998 ( 998 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Pilot, up to Day 15 OLE, up to Day 417
    Adverse event reporting additional description
    Treatment-emergent adverse events (TEAEs) were collected in members of the Safety Population, comprised of all subjects who received at least 1 dose of GWP42003-P. TEAEs are defined as all adverse events not present prior to the first IMP or placebo administration or any event already present that worsened in severity or frequency following IMP.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Pilot
    Reporting group description
    		 -

    Reporting group title
    Open Label Extension (OLE)
    Reporting group description
    		 -

    Serious adverse events
    		 Pilot Open Label Extension (OLE)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 9 (11.11%)
    2 / 9 (22.22%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    Nervous system disorders
    Status epilepticus
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypoxia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia aspiration
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory distress
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urinary retention
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Enterovirus infection
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Rhinovirus infection
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bronchiolitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia bacterial
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia klebsiella
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Urinary tract infection bacterial
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Pilot Open Label Extension (OLE)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 9 (55.56%)
    7 / 9 (77.78%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Hypotension
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Application site erosion
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    4
    Drug tolerance
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract congestion
         subjects affected / exposed
    0 / 9 (0.00%)
    3 / 9 (33.33%)
         occurrences all number
    0
    3
    Adenoidal hypertrophy
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Irritability
         subjects affected / exposed
    1 / 9 (11.11%)
    2 / 9 (22.22%)
         occurrences all number
    1
    2
    Sleep disorder
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Investigations
    Blood triglycerides increased
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Nervous system disorders
    Somnolence
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Myoclonic epilepsy
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Petit mal epilepsy
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    2
    Ear and labyrinth disorders
    Deafness
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Middle ear effusion
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Eye disorders
    Pupils unequal
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 9 (22.22%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Constipation
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Gingival pain
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis diaper
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Rash
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Skin irritation
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Scoliosis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 9 (22.22%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    3
    Viral infection
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Bronchiolitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Ear infection
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Otitis media
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Pneumonia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Sinusitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Decreased appetite
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Feeding intolerance
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Fluid overload
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Hypokalaemia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Hyponatraemia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 May 2016
    -Revised clinical phase and overall design; -Addition of a pilot phase to confirm safety; -Clarification of video-EEG evaluation; -Clarification of cannabidiol metabolites; -Duration of Open-label Extension Phase; -Clarification of procedures following the investigator’s decision to discontinue GWP42003-P during the open label extension phase; -Revised eligibility criteria; -Clarification of withdrawal criterion; -Revised pharmacokinetic blood sampling times; -Removal of Caregiver Impression of IMP Palatability Questionnaire; -Removal of THC testing.
    18 Oct 2016
    -Extend age range in the pilot phase; second cohort; -Clarification of allowable changes in concomitant medications; -Utilization of central video electroencephalography readings; -Changes to inclusion/exclusion criteria; -Updated withdrawal criteria; -Changes to information recorded in the paper diary; -Updated statistical considerations.

    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.
    This study met No Go Criteria. Pilot Phase (PP) subjects had continued hypsarrhythmia/spasms after 2 weeks of treatment. The PP was terminated; the pivotal period was not initiated. Subjects completing the PP could roll into the OLE for up to 1 year.
    For support, Contact us.
    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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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