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    Clinical Trial Results:
    An exploratory, Phase 2, randomized, double-blind, placebo-controlled trial to investigate the safety and efficacy of cannabidiol oral solution (GWP42003-P; CBD-OS) in children and adolescents with Autism Spectrum Disorder.

    Summary
    EudraCT number
    		 2020-002819-21
    Trial protocol
    		 DE   ES  
    Global end of trial date
    21 Dec 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Jul 2024
    First version publication date
    07 Jul 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GWND19189
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04745026
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND Number: 150775
    Sponsors
    Sponsor organisation name
    Jazz Pharmaceuticals Research UK Limited
    Sponsor organisation address
    Building 730, Kent Science Park, Sittingbourne, Kent, United Kingdom, ME9 8AG
    Public contact
    Director Clinical Trial Disclosure & Transparency, Jazz Pharmaceuticals Research UK Limited, 1 215 8323750, ClinicalTrialDisclosure@JazzPharma.com
    Scientific contact
    Director of Clinical Trial Disclosure & Transparency, Jazz Pharmaceuticals Research UK Limited, 1 2158323750, ClinicalTrialDisclosure@JazzPharma.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
    09 Feb 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Dec 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy and safety of GWP42003-P in reducing symptom severity in children with Autism Spectrum Disorder (ASD).
    Protection of trial subjects
    A copy of the protocol, proposed ICF, master ICF, other participant information material, any proposed advertising material, and any further documentation requested was submitted to the IRB/EC for written approval. This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines including the Declaration of Helsinki and the International Ethical Guidelines, applicable ICH GCP Guidelines, and other applicable laws and regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    04 May 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    Canada: 19
    Country: Number of subjects enrolled
    United Kingdom: 10
    Country: Number of subjects enrolled
    Australia: 3
    Country: Number of subjects enrolled
    United States: 67
    Worldwide total number of subjects
    103
    EEA total number of subjects
    4
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    64
    Adolescents (12-17 years)
    39
    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
    		 A total of 191 patients were enrolled in the study. Of those 191 patients, 103 patients met all inclusion criteria and no exclusion criteria were randomized to treatment at 24 centers in the United States, Canada, Spain, United Kingdom, and Australia.

    Pre-assignment
    Screening details
    		 Eligible participants were randomized 7 to 14 days after the screening visit (Visit 1), once all required assessments were completed and laboratory results were reviewed. If required, screening assessments were permitted to be split over 2 visits; however, both visits were conducted within 7- to 14-day window prior to randomization (Visit 2).

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 GWP42003-P
    Arm description
    		 Patients who were randomized to 5 mg/kg/day GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P for 11 weeks. At the end of treatment, participants tapered off the medication over a period of 1 week.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GWP42003-P
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension in pre-filled oral applicator
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally twice daily (morning and evening)

    Arm title
    		 Placebo
    Arm description
    		 Patients who were randomized to placebo for 12 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension in pre-filled oral applicator
    Routes of administration
    Oral use
    Dosage and administration details
    Administered twice daily (morning and evening)

    Number of subjects in period 1
    		GWP42003-P Placebo
    Started
    49
    54
    Completed
    36
    41
    Not completed
    13
    13
         Physician decision
    1
    -
         Adverse event, non-fatal
    2
    2
         Protocol violation
    1
    3
         Lost to follow-up
    4
    3
         Withdrawal by subject
    5
    5

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    GWP42003-P
    Reporting group description
    		 Patients who were randomized to 5 mg/kg/day GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P for 11 weeks. At the end of treatment, participants tapered off the medication over a period of 1 week.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients who were randomized to placebo for 12 weeks.

    Reporting group values
    		 GWP42003-P Placebo Total
    Number of subjects
    		 49 54 103
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 30 34 64
        Adolescents (12-17 years)
    		 19 20 39
        Adults (18-64 years)
    		 0 0 0
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 11.1 ( 3.02 ) 10.8 ( 3.01 ) -
    Gender categorical
    Units: Subjects
        Female
    		 13 7 20
        Male
    		 36 47 83
    WASI-II Intelligent Quotient Score
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 99.42 ( 18.03 ) 100.89 ( 17.16 ) -
    Aberrant Behavior Checklist Irritability
    GWP42003-P n=48; Placebo n=54
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 23.2 ( 7.36 ) 22.5 ( 8.74 ) -
    Aberrant Behavior Checklist Social Withdrawal
    GWP42003-P n=48; Placebo n=54
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 12.6 ( 8.63 ) 12.7 ( 9.11 ) -
    Aberrant Behavior Checklist Stereotypic Behavior
    GWP42003-P n=48; Placebo n=54
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 6.7 ( 5.08 ) 6.7 ( 4.83 ) -
    Aberrant Behavior Checklist Hyperactivity/Noncompliance
    GWP42003-P n=48; Placebo n=54
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 25.2 ( 12.27 ) 26.9 ( 9.63 ) -
    Aberrant Behavior Checklist Inappropriate Speech
    GWP42003-P n=48; Placebo n=54
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 5.7 ( 3.22 ) 5.6 ( 3.08 ) -
    Vineland Adaptive Behavior Scales Social and Communication Composite Score
    GWP42003-P n=48; Placebo n=50
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 66.6 ( 19.61 ) 67.7 ( 15.18 ) -
    Clinical Global Impression – Severity (CGI-S)
    GWP42003-P n=49; Placebo n=51
    Units: score on a scale
        arithmetic mean (standard deviation)
    		 4.76 ( 0.630 ) 4.82 ( 0.684 ) -

    End points

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    End points reporting groups
    Reporting group title
    GWP42003-P
    Reporting group description
    		 Patients who were randomized to 5 mg/kg/day GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P for 11 weeks. At the end of treatment, participants tapered off the medication over a period of 1 week.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients who were randomized to placebo for 12 weeks.

    Primary: Change from Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores

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    End point title
    		 Change from Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores
    End point description
    The caregiver-assessed ABC was designed to assess the presence and severity of various problem behaviors commonly observed in individuals diagnosed with intellectual and developmental disability. The checklist contains 5 subscales: Irritability (15 items); Social Withdrawal (16 items); Stereotypic Behavior (7 items); Hyperactivity/Noncompliance (16 items); and Inappropriate Speech (4 items). Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). The total score of all subscales ranges from 0 to 174 where higher scores indicate worse clinical outcome. The change from baseline to Week 4, Week 8, and Week 12 is reported with lower scores indicating better clinical outcome.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 12
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49 [1]
    54 [2]
    Units: score on a scale
    arithmetic mean (standard deviation)
        ABC-Irritability, Week 4
    -6.35 ( 7.98 )
    -5.41 ( 7.16 )
        ABC-Irritability, Week 8
    -6.67 ( 8.63 )
    -8.05 ( 9.12 )
        ABC-Irritability, Week 12
    -6.97 ( 9.68 )
    -8.57 ( 10.10 )
        ABC-Social Withdrawal, Week 4
    -2.53 ( 5.61 )
    -2.92 ( 5.24 )
        ABC-Social Withdrawal, Week 8
    -3.81 ( 6.76 )
    -4.74 ( 6.25 )
        ABC-Social Withdrawal, Week 12
    -3.74 ( 6.21 )
    -4.51 ( 5.95 )
        ABC-Stereotypic Behavior, Week 4
    -2.10 ( 4.24 )
    -1.39 ( 3.24 )
        ABC-Stereotypic Behavior, Week 8
    -1.92 ( 3.99 )
    -1.98 ( 4.05 )
        ABC-Stereotypic Behavior, Week 12
    -1.88 ( 3.63 )
    -2.31 ( 4.03 )
        ABC-Hyperactivity/Noncompliance, Week 4
    -5.73 ( 8.08 )
    -4.55 ( 8.15 )
        ABC-Hyperactivity/Noncompliance, Week 8
    -6.39 ( 9.15 )
    -7.42 ( 9.06 )
        ABC-Hyperactivity/Noncompliance, Week 12
    -6.74 ( 10.62 )
    -8.77 ( 9.94 )
        ABC-Inappropriate Speech, Week 4
    -2.13 ( 2.88 )
    -0.88 ( 2.07 )
        ABC-Inappropriate Speech, Week 8
    -1.58 ( 2.74 )
    -1.44 ( 2.73 )
        ABC-Inappropriate Speech, Week 12
    -1.94 ( 2.93 )
    -1.51 ( 2.97 )
    Notes
    [1] - All subscales WK4:n=40; WK8: n=36; WK12:n=34
    [2] - All subscales WK4:n=51; WK8:n=43; WK12:n=35
    Statistical analysis title
    		 Irritability: GWP42003-P vs Placebo (Week 12)
    Statistical analysis description
    Superiority analysis
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.085 [3]
    Method
    		 Mixed models for repeated measures
    Parameter type
    		 Least square mean difference
    Point estimate
    3.37
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.48
         upper limit
    		 7.21
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.931
    Notes
    [3] - Mixed models for repeated measures (MMRM) model includes randomization stratification factors, baseline score, visit, treatment arm, visit by treatment arm interaction as fixed effects, and visit repeated within each patient as a repeated effect.
    Statistical analysis title
    		 Social Withdrawal: GWP42003-P vs Placebo (Week 12)
    Statistical analysis description
    Superiority analysis
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3107 [4]
    Method
    		 Mixed models for repeated measures
    Parameter type
    		 Least square mean difference
    Point estimate
    1.14
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.08
         upper limit
    		 3.36
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.16
    Notes
    [4] - Mixed models for repeated measures (MMRM) model includes randomization stratification factors, baseline score, visit, treatment arm, visit by treatment arm interaction as fixed effects, and visit repeated within each patient as a repeated effect.
    Statistical analysis title
    		 Stereotypic Behavior: GWP42003-P vs Placebo (WK12)
    Statistical analysis description
    Superiority analysis
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.9513 [5]
    Method
    		 Mixed models for repeated measures
    Parameter type
    		 Least square mean difference
    Point estimate
    0.04
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.34
         upper limit
    		 1.42
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.695
    Notes
    [5] - Mixed models for repeated measures (MMRM) model includes randomization stratification factors, baseline score, visit, treatment arm, visit by treatment arm interaction as fixed effects, and visit repeated within each patient as a repeated effect.
    Statistical analysis title
    		 Hyperactivity/Noncompliance: GWP42003-P vs Placebo
    Statistical analysis description
    Superiority analysis at Week 12
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.305 [6]
    Method
    		 Mixed models for repeated measures
    Parameter type
    		 Least square mean difference
    Point estimate
    2.01
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.86
         upper limit
    		 5.88
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.943
    Notes
    [6] - Mixed models for repeated measures (MMRM) model includes randomization stratification factors, baseline score, visit, treatment arm, visit by treatment arm interaction as fixed effects, and visit repeated within each patient as a repeated effect.
    Statistical analysis title
    		 Inappropriate Speech: GWP42003-P vs Placebo (WK12)
    Statistical analysis description
    Superiority analysis
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4754 [7]
    Method
    		 Mixed models for repeated measures
    Parameter type
    		 Least square mean difference
    Point estimate
    -0.37
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.4
         upper limit
    		 0.66
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.517
    Notes
    [7] - Mixed models for repeated measures (MMRM) model includes randomization stratification factors, baseline score, visit, treatment arm, visit by treatment arm interaction as fixed effects, and visit repeated within each patient as a repeated effect.

    Primary: Change from Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores

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    End point title
    		 Change from Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores
    End point description
    The VABS-3 scales assess what a person does, rather than what he or she can do. The Vineland-3 assesses adaptive behavior in 3 domains: Communication, Daily Living Skills, and Socialization. Each domain is comprised of 3 subdomains: receptive expression and written (communication); personal, domestic and community (daily living skills); Interpersonal relationships, play and leisure and copying skills (socialization).
    End point type
    Primary
    End point timeframe
    Baseline up to Week 12
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49 [8]
    54 [9]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 4
    2.97 ( 6.70 )
    0.99 ( 9.29 )
        Week 8
    3.69 ( 10.82 )
    3.54 ( 8.83 )
        Week 12
    4.85 ( 8.83 )
    5.55 ( 10.42 )
    Notes
    [8] - WK4: n=37; WK8: n=36; WK12: n=31
    [9] - WK4: n=45; WK8: n=40; WK12: n=32
    Statistical analysis title
    		 GWP42003-P vs Placebo (Week 12)
    Statistical analysis description
    Superiority analysis
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8506 [10]
    Method
    		 Mixed models for repeated measures
    Parameter type
    		 Least square mean difference
    Point estimate
    0.45
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -4.26
         upper limit
    		 5.15
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.36
    Notes
    [10] - Mixed models for repeated measures (MMRM) model includes randomization stratification factors, baseline score, visit, treatment arm, visit by treatment arm interaction as fixed effects, and visit repeated within each patient as a repeated effect.

    Primary: Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category

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    End point title
    		 Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category
    End point description
    The CGI-I is a 7-point scale that requires the clinician to assess how much the patient’s illness has improved or worsened relative to a baseline state at the beginning of the intervention. The clinician is asked: Compared to the patient’s condition at admission to the project, how much has the patient changed? This is rated as: 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; or 7 = very much worse. Higher scores indicate worse clinical outcome. The number of patients in each CGI-I category is reported.
    End point type
    Primary
    End point timeframe
    Day 85
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    34
    38
    Units: number of patients
    number (not applicable)
        Minimally improved
    13
    14
        Much improved
    12
    11
        No change
    9
    11
        Much worse
    0
    1
        Very much improved
    0
    1
        Minimally worse
    0
    0
        Very much worse
    0
    0
    Statistical analysis title
    		 GWP42003-P vs Placebo (Week 12)
    Statistical analysis description
    Responders with 'Very Much Improved' or 'Much Improved' response at week 12
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    72
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7087 [11]
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.22
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.43
         upper limit
    		 3.51
    Notes
    [11] - Logistic regression model includes treatment arm, randomization stratification variables and baseline score (CGI-S) as covariates. Responders are those who achieved a score of 1 or 2 (’Very Much Improved’ or ‘Much Improved’) at post-baseline visits.

    Primary: Change from Baseline in Clinical Global Impression Severity (CGI-S) Scores

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    End point title
    		 Change from Baseline in Clinical Global Impression Severity (CGI-S) Scores
    End point description
    The CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician’s experience with patients who have the same diagnosis. The clinician is asked: Considering your total clinical experience with this particular population, how ill is the patient at this time? This is rated as: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; or 7 = extremely ill. Higher scores indicate worse outcome. The change from baseline in CGI-S scores is reported and lower mean scores indicate better outcome.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 12
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49 [12]
    54 [13]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 4
    -0.33 ( 0.61 )
    -0.31 ( 0.69 )
        Week 8
    -0.50 ( 0.68 )
    -0.48 ( 0.92 )
        Week 12
    -0.55 ( 0.85 )
    -0.63 ( 1.10 )
    Notes
    [12] - WK4: n=43; WK8: n=40; WK12: n=31
    [13] - WK4: n=51; WK8: n=42; WK12: n=32
    Statistical analysis title
    		 GWP42003-P vs Placebo (Week 12)
    Statistical analysis description
    Superior analysis
    Comparison groups
    GWP42003-P v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6108
    Method
    		 Cochran-Mantel-Haenszel
    Confidence interval

    Secondary: Number of Patients Reporting Treatment-emergent Adverse Events

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    End point title
    		 Number of Patients Reporting Treatment-emergent Adverse Events
    End point description
    A TEAE is one that started, or worsened in severity or seriousness, following the first dose of IMP. AEs were coded according to the Medical Dictionary for Regulatory Activities v24.0 dictionary.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 106
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49
    54
    Units: number of patients
    number (not applicable)
        TEAE
    27
    24
        Non-TEAE
    25
    32
        Treatment-related TEAE
    6
    10
        Serious TEAE
    2
    1
        Serious treatment-related TEAE
    0
    0
        TEAE leading to study intervention withdrawal
    2
    1
        Treatment-related TEAE leading to drug withdrawal
    1
    1
        TEAE leading to withdrawal
    2
    2
        TEAE leading to death
    0
    0
    No statistical analyses for this end point

    Secondary: Mean Change from Baseline in Hematology Clinical Laboratory Levels

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    End point title
    		 Mean Change from Baseline in Hematology Clinical Laboratory Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 9 (end of taper/withdrawal)
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    30
    37
    Units: 10^9/L
    arithmetic mean (standard deviation)
        Week 9: Basophils
    -0.01 ( 0.03 )
    0 ( 0.03 )
        Week 9: Eosinophils
    -0.09 ( 0.311 )
    0 ( 0.40 )
        Week 9: Lymphocytes
    0.04 ( 0.70 )
    -0.20 ( 0.68 )
        Week 9: Monocytes
    -0.01 ( 0.15 )
    -0.01 ( 0.19 )
        Week 9: Neutrophils
    0.04 ( 1.80 )
    -0.01 ( 1.26 )
        Week 9: Platelets
    -9.43 ( 34.50 )
    8.43 ( 52.13 )
        Week 9: Leukocytes
    -0.02 ( 1.87 )
    -0.23 ( 1.79 )
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Hematology Clinical Laboratory Levels

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    End point title
    		 Mean Percentage Change from Baseline in Hematology Clinical Laboratory Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 9 (end of taper/withdrawal)
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    30 [14]
    37 [15]
    Units: percentage change from baseline
    arithmetic mean (standard deviation)
        Week 9: Basophils/Leukocytes
    -0.07 ( 0.45 )
    -0.03 ( 0.42 )
        Week 9: Eosinophils/Leukocytes
    -1.33 ( 5.37 )
    0.22 ( 4.88 )
        Week 9: Hematocrit
    -0.02 ( 2.19 )
    0.43 ( 2.30 )
        Week 9: Lymphocytes/Leukocytes
    1.05 ( 11.25 )
    -2.32 ( 8.26 )
        Week 9: Monocytes/Leukocytes
    -0.16 ( 2.04 )
    0.05 ( 1.86 )
        Week 9: Neutrophils/Leukocytes
    0.49 ( 12.29 )
    2.08 ( 8.89 )
    Notes
    [14] - Hematocrit: n=31
    [15] - Hematocrit: n=38
    No statistical analyses for this end point

    Secondary: Mean Change from Baseline in Hemoglobin Levels

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    End point title
    		 Mean Change from Baseline in Hemoglobin Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Week 9 (end of taper/withdrawal)
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    31
    38
    Units: g/dL
    arithmetic mean (standard deviation)
        Week 9
    -0.06 ( 0.55 )
    0.08 ( 0.65 )
    No statistical analyses for this end point

    Secondary: Mean Change from Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels

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    End point title
    		 Mean Change from Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Week 9 (end of taper/withdrawal)
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    31
    38
    Units: pg/cell
    arithmetic mean (standard deviation)
        Week 9
    0.13 ( 0.67 )
    -0.03 ( 0.72 )
    No statistical analyses for this end point

    Secondary: Mean Change from Baseline in Erythrocyte Mean Corpuscular Volume Levels

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    End point title
    		 Mean Change from Baseline in Erythrocyte Mean Corpuscular Volume Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Week 9 (end of taper/withdrawal)
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    31
    38
    Units: fL
    arithmetic mean (standard deviation)
        Week 9
    0.52 ( 2.32 )
    0.03 ( 2.39 )
    No statistical analyses for this end point

    Secondary: Number of Patients with Clinically Significant Vital Sign Values

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    End point title
    		 Number of Patients with Clinically Significant Vital Sign Values
    End point description
    End point type
    Secondary
    End point timeframe
    Post-baseline
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    45 [16]
    52 [17]
    Units: patients
    number (not applicable)
        Increase in weight from baseline >7%
    7
    13
        Decrease in weight from baseline >7%
    2
    2
        Body temperature <36 degrees Celsius
    4
    6
        Body temperature >38 degrees Celsius
    1
    0
        Pulse rate <60 beats/min
    1
    3
        Pulse rate >100 beats/min
    7
    4
        Respiratory rate <12 breaths/min
    0
    0
        Respiratory rate >20 breaths/min
    13
    17
        Systolic blood pressure <90 mmhg
    5
    3
        Systolic blood pressure >160 mmhg
    0
    0
        Diastolic blood pressure <50 mmhg
    3
    2
        Diastolic blood pressure >120 mmhg
    0
    0
    Notes
    [16] - Weight: n=41; Respiratory rate: n=43
    [17] - Weight: n=49
    No statistical analyses for this end point

    Secondary: Number of Patients with Clinically Significant Physical Examination Procedure Findings

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    End point title
    		 Number of Patients with Clinically Significant Physical Examination Procedure Findings
    End point description
    Number of patients with abnormal physical exam findings are reported.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 85 post-baseline
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49
    54
    Units: patients
    number (not applicable)
        Abdomen
    2
    0
        Cardiovascular
    0
    0
        Chest/lungs
    0
    0
        Dermatological
    3
    3
        Endocrine system
    0
    0
        Gastrointestinal
    0
    0
        General appearance
    0
    2
        HEENT
    0
    1
        Lymphatic
    0
    0
        Musculoskeletal/Extremities
    0
    1
        Neurological
    2
    0
        Other
    0
    0
        Genitourinary/Reproductive
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Patients with Clinically Significant 12-lead Electrocardiogram Findings

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    End point title
    		 Number of Patients with Clinically Significant 12-lead Electrocardiogram Findings
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 85 post-baseline
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49
    54
    Units: patients
    number (not applicable)
        QTcB >450 msec
    7
    4
        QTcB >480 msec
    2
    0
        QTcB >500 msec
    1
    0
    No statistical analyses for this end point

    Secondary: Number of Patients Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS)

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    End point title
    		 Number of Patients Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS)
    End point description
    C-SSRS rating scale results since last visit is reported.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 92
    End point values
    		 GWP42003-P Placebo
    Number of subjects analysed
    49
    54
    Units: patients
    number (not applicable)
        Suicidal ideation
    4
    3
        Wish to be dead
    3
    3
        Non-specific active suicidal thoughts
    2
    1
        Active suicidal ideation, no intent to act
    1
    0
        Active suicidal ideation, some intent to act
    0
    0
        Active suicidal ideation, specific plan/intent
    0
    0
        Suicidal behavior
    3
    6
        Actual attempt
    0
    1
        Interrupted attempt
    0
    1
        Aborted attempt
    0
    0
        Preparatory acts or behavior
    0
    1
        Suicidal behavior item
    0
    1
        Self-injurious behavior without suicidal intent
    3
    5
        Completed suicide
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    GWP42003-P
    Reporting group description
    		 Patients who were randomized to 5 mg/kg/day GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P. At the end of treatment, participants tapered off the medication over a period of 1 week.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients who were randomized to placebo for 12 weeks.

    Serious adverse events
    		 GWP42003-P Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 49 (4.08%)
    1 / 54 (1.85%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Psychiatric disorders
    Suicidal ideation
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 49 (2.04%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 1
    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
    		 GWP42003-P Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 49 (28.57%)
    20 / 54 (37.04%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 49 (6.12%)
    4 / 54 (7.41%)
         occurrences all number
    3
    4
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 49 (0.00%)
    5 / 54 (9.26%)
         occurrences all number
    0
    5
    Fatigue
         subjects affected / exposed
    0 / 49 (0.00%)
    3 / 54 (5.56%)
         occurrences all number
    0
    3
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    7 / 49 (14.29%)
    4 / 54 (7.41%)
         occurrences all number
    7
    4
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 49 (6.12%)
    0 / 54 (0.00%)
         occurrences all number
    3
    0
    Psychiatric disorders
    Irritability
         subjects affected / exposed
    0 / 49 (0.00%)
    5 / 54 (9.26%)
         occurrences all number
    0
    5
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    4 / 49 (8.16%)
    0 / 54 (0.00%)
         occurrences all number
    4
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 49 (0.00%)
    3 / 54 (5.56%)
         occurrences all number
    0
    3

    More information

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

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

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