Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   40977   clinical trials with a EudraCT protocol, of which   6698   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled Trial to Investigate the Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients with Rett Syndrome.

    Summary
    EudraCT number
    2018-003370-27
    Trial protocol
    GB   IT  
    Global end of trial date
    21 Jan 2021

    Results information
    Results version number
    v1(current)
    This version publication date
    05 Aug 2021
    First version publication date
    05 Aug 2021
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    GWND18064
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03848832
    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-PIP02-19
    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
    21 Jan 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Jan 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Jan 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the effect of GWP42003-P (Cannabidiol; CBD), compared with placebo, at the end of 24 weeks’ treatment in reducing symptom severity in subjects with Rett syndrome using the Rett Syndrome Behaviour Questionnaire (RSBQ).
    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 The International Council for Harmonisation (ICH) E6 Guideline for Good Clinical Practice (EMA/CHMP/ICH/135/1995) and the European Clinical Trial Directive 2001/20/EC. The ICH 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 British, Spanish and United States laws.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    26 Jul 2019
    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: 15
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    United Kingdom: 5
    Worldwide total number of subjects
    29
    EEA total number of subjects
    9
    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)
    18
    Adolescents (12-17 years)
    8
    Adults (18-64 years)
    3
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    A total of 41 subjects were screened for eligibility; 29 were randomized to the study treatments, and 12 were screen failures.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    5 mg/kg/day GWP42003-P
    Arm description
    Subjects received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).
    Arm type
    Experimental

    Investigational medicinal product name
    GWP42003-P
    Investigational medicinal product code
    Other name
    cannabidiol; CBD
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    GWP42003-P was administered orally in a fed state.

    Arm title
    15 mg/kg/day GWP42003-P
    Arm description
    Subjects received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.
    Arm type
    Experimental

    Investigational medicinal product name
    GWP42003-P
    Investigational medicinal product code
    Other name
    cannabidiol; CBD
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    GWP42003-P was administered orally in a fed state.

    Arm title
    Placebo
    Arm description
    Subjects received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    The matching placebo oral solution was administered in a fed state.

    Number of subjects in period 1
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Started
    10
    9
    10
    Completed
    7
    6
    4
    Not completed
    3
    3
    6
         Physician decision
    2
    1
    3
         Withdrawal Of Consent Due To Covid-19
    -
    -
    1
         Sponsor Decision - Covid-19 Precaution
    -
    -
    1
         Withdrawal (Covid-19)
    -
    2
    -
         Adverse event, non-fatal
    1
    -
    -
         Consent withdrawn by subject
    -
    -
    1

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Period 1
    Reporting group description
    -

    Reporting group values
    Period 1 Total
    Number of subjects
    29 29
    Age categorical
    Units: subjects
        <=18 years
    29 29
        Between 18 and 65 years
    0 0
        >=65 years
    0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    9.7 ± 5.11 -
    Gender categorical
    Units: subjects
        Female
    29 29
        Male
    0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    1 1
        Asian
    0 0
        Native Hawaiian or Other Pacific Islander
    0 0
        Black or African American
    0 0
        White
    28 28
        More than one race
    0 0
        Unknown or Not Reported
    0 0
    Subject analysis sets

    Subject analysis set title
    5 mg/kg/day GWP42003-P
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Analysis Set: All subjects who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received. One subject who was assigned to the placebo group received GWP42003-P 5 mg/kg. For baseline analyses, this subject was assigned to the GWP42003-P 5 mg/kg group.

    Subject analysis set title
    15 mg/kg/day GWP42003-P
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Analysis Set: All subjects who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received.

    Subject analysis set title
    Placebo
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Analysis Set: All subjects who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received. One subject who was assigned to the placebo group received GWP42003-P 5 mg/kg. For baseline analyses, this subject was assigned to the GWP42003-P 5 mg/kg group.

    Subject analysis sets values
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Number of subjects
    11
    9
    9
    Age categorical
    Units: subjects
        <=18 years
    11
    9
    9
        Between 18 and 65 years
    0
    0
    0
        >=65 years
    0
    0
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    9.7 ± 6.02
    10.7 ± 3.64
    8.6 ± 5.53
    Gender categorical
    Units: subjects
        Female
    11
    9
    9
        Male
    0
    0
    0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    1
    0
    0
        Asian
    0
    0
    0
        Native Hawaiian or Other Pacific Islander
    0
    0
    0
        Black or African American
    0
    0
    0
        White
    10
    9
    9
        More than one race
    0
    0
    0
        Unknown or Not Reported
    0
    0
    0

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    5 mg/kg/day GWP42003-P
    Reporting group description
    Subjects received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).

    Reporting group title
    15 mg/kg/day GWP42003-P
    Reporting group description
    Subjects received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.

    Reporting group title
    Placebo
    Reporting group description
    Subjects received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.

    Subject analysis set title
    5 mg/kg/day GWP42003-P
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Analysis Set: All subjects who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received. One subject who was assigned to the placebo group received GWP42003-P 5 mg/kg. For baseline analyses, this subject was assigned to the GWP42003-P 5 mg/kg group.

    Subject analysis set title
    15 mg/kg/day GWP42003-P
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Analysis Set: All subjects who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received.

    Subject analysis set title
    Placebo
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Analysis Set: All subjects who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received. One subject who was assigned to the placebo group received GWP42003-P 5 mg/kg. For baseline analyses, this subject was assigned to the GWP42003-P 5 mg/kg group.

    Primary: Change from Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/day GWP42003-P Dose Level Compared with Placebo

    Close Top of page
    End point title
    Change from Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/day GWP42003-P Dose Level Compared with Placebo [1] [2]
    End point description
    RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Higher scores represent greater severity. Except for item 31 ("Uses eye gaze to convey feelings, needs and wishes"), which is reverse-scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true" and 2 indicating "not true as far as you know"). Analysis was conducted in members of the ITT Set, defined as all randomized subjects who received at least 1 dose of drug in the trial, and had Baseline efficacy data. CFB = Change from Baseline.
    End point type
    Primary
    End point timeframe
    Baseline; Week 24
    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: Only descriptive statistical analysis was performed for this primary end point. The planned inferential statistical analysis was not carried out due to the limited sample size compared to that planned, because of the trial termination.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Only the comparison between GWP42003-P 15 mg/kg group with placebo was evaluated in this primary end point.
    End point values
    15 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    9 [3]
    10 [4]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline, n=9, 10
    45.9 ± 16.77
    50.0 ± 7.56
        CFB at Week 24, n=7, 7
    -12.1 ± 13.63
    -6.1 ± 7.22
    Notes
    [3] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [4] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Secondary: Change from Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/day GWP42003-P Dose Level Compared with Placebo

    Close Top of page
    End point title
    Change from Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/day GWP42003-P Dose Level Compared with Placebo [5]
    End point description
    RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Higher scores represent greater severity. Except for item 31 ("Uses eye gaze to convey feelings, needs and wishes"), which is reverse-scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true" and 2 indicating "not true as far as you know"). CFB = Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Only the comparison between GWP42003-P 5 mg/kg group with placebo was evaluated in this secondary end point.
    End point values
    5 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    10 [6]
    10 [7]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline, n=10, 10
    39.8 ± 12.80
    50.0 ± 7.56
        CFB at Week 24, n=9, 7
    0.4 ± 12.51
    -6.1 ± 7.22
    Notes
    [6] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [7] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Secondary: Mean Clinical Global Impressions-Improvement (CGI-I) Score at Week 24

    Close Top of page
    End point title
    Mean Clinical Global Impressions-Improvement (CGI-I) Score at Week 24
    End point description
    CGI-I is a 7-point scale that requires the clinician to assess how much a subject's illness has improved or worsened relative to a Baseline state at the beginning of the intervention. 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.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    9 [8]
    7 [9]
    7 [10]
    Units: units on a scale
        arithmetic mean (standard deviation)
    3.2 ± 1.20
    2.9 ± 1.07
    3.1 ± 0.38
    Notes
    [8] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [9] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [10] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Mean Clinician Global Impressions - Severity (CGI-S) Score at Week 24

    Close Top of page
    End point title
    Change from Baseline in Mean Clinician Global Impressions - Severity (CGI-S) Score at Week 24
    End point description
    CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's illness at the time of assessment relative to the clinician's experience with subjects who had the same diagnosis. 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. CFB = Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    10 [11]
    9 [12]
    10 [13]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline, n=10, 9, 10
    4.3 ± 1.25
    4.2 ± 0.67
    4.4 ± 0.84
        CFB at Week 24, n=9, 7, 7
    -0.1 ± 0.33
    -0.1 ± 0.38
    0.0 ± 0.00
    Notes
    [11] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [12] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [13] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Mean RSBQ Subscale Scores at Week 24

    Close Top of page
    End point title
    Change from Baseline in Mean RSBQ Subscale Scores at Week 24
    End point description
    RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Higher scores represent greater severity. Except for item 31 ("Uses eye gaze to convey feelings, needs and wishes"), which is reverse-scored (0 indicating "very true or often true", 1 indicating 'somewhat or sometimes true" and 2 indicating "not true as far as you know"). The 45-item RSBQ is comprised of 8 subscales: 1) general mood, 2) breathing problems, 3) hand behaviors, 4) face movements, 5) body rocking (BR)/expressionless face, 6) night-time behaviors, 7) anxiety/fear, 8) walking/standing. CFB = Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    10 [14]
    9 [15]
    10 [16]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline General Mood Score, n=10, 9, 10
    6.0 ± 3.65
    8.2 ± 4.58
    8.9 ± 3.14
        CFB General Mood Score, n=9, 7, 7
    -0.6 ± 2.70
    -4.9 ± 2.67
    -2.6 ± 2.57
        Baseline Breathing Problems Score, n=10, 9, 10
    4.7 ± 2.45
    5.0 ± 3.08
    5.0 ± 3.43
        CFB Breathing Problems Score, n=9, 7, 7
    0.2 ± 1.86
    -1.0 ± 1.53
    -0.3 ± 1.25
        Baseline Hand Behaviors Score, n=10, 9, 10
    7.8 ± 2.15
    7.6 ± 3.54
    9.1 ± 2.08
        CFB Hand Behaviors Score, n=9, 7, 7
    0.7 ± 2.24
    -0.9 ± 3.29
    0.1 ± 0.90
        Baseline Face Movements Score, n=10, 9, 10
    2.3 ± 2.45
    4.6 ± 2.13
    4.3 ± 2.21
        CFB Face Movements Score, n=9, 7, 7
    0.0 ± 1.12
    -1.4 ± 1.72
    -0.1 ± 1.07
        Baseline BR/Expressionless Face Score, n=10, 9, 10
    4.5 ± 2.59
    5.1 ± 1.96
    5.4 ± 2.59
        CFB BR/Expressionless Face Score, n=9, 7, 7
    0.6 ± 2.46
    -0.6 ± 1.13
    -0.1 ± 1.86
        Baseline Night-time Behaviors Score, n=10, 9, 10
    0.8 ± 1.14
    0.8 ± 0.97
    1.8 ± 1.69
        CFB Night-time Behaviors Score, n=9, 7, 7
    0.2 ± 0.97
    -0.1 ± 1.46
    -0.3 ± 1.11
        Baseline Anxiety/Fear Score, n=10, 9, 10
    4.0 ± 1.89
    4.6 ± 2.46
    5.1 ± 1.45
        CFB Anxiety/Fear Score, n=9, 7, 7
    -0.2 ± 1.92
    -1.3 ± 2.29
    -1.7 ± 0.95
        Baseline Walking/Standing Score, n=10, 9, 10
    2.7 ± 1.64
    2.1 ± 1.36
    2.9 ± 1.29
        CFB Walking/Standing Score, n=9, 7, 7
    0.9 ± 2.09
    0.0 ± 0.58
    0.1 ± 1.07
    Notes
    [14] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [15] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [16] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Mean 9-item Motor Behavioral Assessment (MBA-9) Total Score and Subscale scores at Week 24

    Close Top of page
    End point title
    Change from Baseline in Mean 9-item Motor Behavioral Assessment (MBA-9) Total Score and Subscale scores at Week 24
    End point description
    MBA-9 is derived from the full MBA scale (37 Rett syndrome symptoms) by selecting the items deemed amenable to change and which reflected areas of meaningful clinical change. The severity of current symptoms is rated by the investigator on a 5-point numerical scale; (0 = normal or never; 1 = mild or rare; 2 = moderate or occasional; 3 = marked or frequent; 4 = very severe or constant). The MBA-9 score is calculated by summing the scores of the individual items. The maximum score is 36; higher total scores represent greater severity. CFB = Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    10 [17]
    9 [18]
    10 [19]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline Regression of Motor Skills, n=10, 9, 10
    2.2 ± 1.23
    2.6 ± 0.88
    2.6 ± 0.84
        CFB Regression of Motor Skills, n=9, 7, 7
    0.3 ± 1.58
    -0.3 ± 0.49
    0.0 ± 0.58
        Baseline Poor Eye/Social Contact, n=10, 9, 10
    1.7 ± 0.82
    1.6 ± 1.01
    1.5 ± 0.97
        CFB Poor Eye/Social Contact, n=9, 7, 7
    0.0 ± 0.87
    -0.1 ± 0.69
    -0.9 ± 1.07
        Baseline Lack of Sustained Interest, n=10, 9, 10
    1.8 ± 0.79
    2.1 ± 1.17
    1.5 ± 0.71
        Lack of Sustained Interest, n=9, 7, 7
    0.0 ± 0.71
    -0.3 ± 1.11
    -0.1 ± 0.38
        Baseline DNR for Objects or People, n=10, 9, 10
    2.0 ± 1.25
    1.9 ± 1.05
    2.3 ± 1.25
        CFB DNR for Objects or People, n=9, 7, 7
    -0.4 ± 2.24
    -0.4 ± 0.98
    0.1 ± 1.21
        Baseline Chewing Difficulties, n=10, 9, 10
    1.3 ± 1.34
    1.3 ± 0.50
    1.1 ± 0.74
        Chewing Difficulties, n=9, 7, 7
    -0.2 ± 0.67
    0.0 ± 0.00
    0.4 ± 0.53
        Baseline Speech Disturbance, n=10, 9, 10
    3.0 ± 0.47
    2.8 ± 0.83
    2.7 ± 0.48
        CFB Speech Disturbance, n=9, 7, 7
    0.0 ± 0.00
    0.0 ± 0.58
    0.3 ± 0.49
        Baseline Hand Clumsiness, n-10, 9, 10
    2.8 ± 1.14
    2.8 ± 1.56
    3.1 ± 1.10
        CFB Hand Clumsiness, n=9, 7, 7
    -0.6 ± 1.33
    -0.4 ± 1.13
    0.0 ± 0.58
        Baseline Dysonia, n=10, 9, 10
    1.0 ± 1.41
    1.2 ± 1.56
    1.0 ± 1.41
        CFB Dysonia, n=9, 7, 7
    0.4 ± 1.42
    -0.1 ± 0.90
    0.1 ± 1.46
        Baseline Hypertonia/Rigidity, n=10, 9, 10
    1.4 ± 1.51
    1.1 ± 1.45
    1.0 ± 1.41
        CFB Hypertonia/Rigidity, n=9, 7, 7
    0.4 ± 0.73
    -0.1 ± 0.38
    0.0 ± 1.00
        Baseline MBA-9 Total Score, n=10, 9, 10
    17.2 ± 5.81
    17.3 ± 6.78
    16.8 ± 5.96
        CFB MBA-9 Total Score, n=9, 7, 7
    0.0 ± 4.87
    -1.9 ± 4.06
    0.0 ± 3.92
    Notes
    [17] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [18] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [19] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Mean Children's Sleep Habits Questionnaire (CSHQ) Total Score and Subscale Scores at Week 24

    Close Top of page
    End point title
    Change from Baseline in Mean Children's Sleep Habits Questionnaire (CSHQ) Total Score and Subscale Scores at Week 24
    End point description
    The CSHQ is a caregiver-completed sleep screening instrument designed for school-aged children, including 33 items within 8 subscales reflecting the following sleep domains: 1) bedtime resistance, 2) sleep onset delay, 3) sleep duration, 4) sleep anxiety, 5) night wakings, 6) parasomnias, 7) sleep-disordered breathing, 8) daytime sleepiness. Each item is answered with 1 of 3 markers: "usually," for 5 or more times a week, "sometimes," for 2-4 times a week, and "rarely," for never or 1 time a week. Higher scores reflect more disturbed sleep behavior. CFB = Change from Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    5 mg/kg/day GWP42003-P 15 mg/kg/day GWP42003-P Placebo
    Number of subjects analysed
    10 [20]
    9 [21]
    10 [22]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline Total Score, n=10, 9, 10
    49.8 ± 9.75
    46.7 ± 4.90
    49.4 ± 5.42
        CFB Total Score, n=9, 7, 7
    -1.6 ± 6.17
    -5.0 ± 6.14
    -3.7 ± 4.07
        Baseline Bedtime resistance, n=10, 9, 10
    9.2 ± 3.43
    7.3 ± 2.12
    7.0 ± 1.25
        CFB Bed-time resistance, n=9, 7, 7
    0.0 ± 1.00
    -0.4 ± 1.27
    -0.1 ± 1.68
        Baseline Sleep onset delay, n=10, 9, 10
    1.5 ± 0.71
    1.4 ± 0.53
    1.8 ± 0.79
        CFB Sleep onset delay, n=9, 7, 7
    0.1 ± 1.27
    -0.1 ± 0.69
    -0.6 ± 0.53
        Baseline Sleep duration, n-10, 9, 10
    4.7 ± 2.41
    3.4 ± 0.73
    4.5 ± 1.58
        CFB Sleep duration, n=9, 7, 7
    0.4 ± 1.13
    -0.3 ± 0.95
    -0.4 ± 1.72
        Baseline Sleep anxiety, n=10, 9, 10
    5.6 ± 1.65
    4.6 ± 0.73
    4.9 ± 1.10
        CFB Sleep anxiety, n=9, 7, 7
    -0.1 ± 0.78
    -0.1 ± 0.38
    0.0 ± 1.15
        Baseline Night wakings, n=10, 9, 10
    5.3 ± 2.36
    4.4 ± 1.33
    4.6 ± 1.43
        CFB Night wakings, n=9, 7, 7
    -0.4 ± 0.88
    -0.6 ± 1.40
    0.1 ± 0.69
        Baseline Parasomnias, n=10, 9, 10
    11.0 ± 1.49
    10.4 ± 1.42
    11.7 ± 2.63
        CFB Parasomnias, n=9, 7, 7
    -0.6 ± 1.51
    -1.1 ± 1.68
    -1.4 ± 2.30
        Baseline Sleep disordered breathing, n=10, 9, 10
    4.2 ± 1.69
    4.0 ± 1.22
    3.7 ± 1.06
        CFB Sleep disordered breathing, n=9, 7, 7
    -0.4 ± 1.74
    -0.9 ± 1.07
    -0.1 ± 0.38
        Baseline Daytime sleepiness, n=10, 9, 10
    11.6 ± 3.20
    13.2 ± 2.39
    13.7 ± 2.50
        CFB Daytime sleepiness, n=9, 7, 7
    -0.7 ± 3.64
    -1.4 ± 2.07
    -1.3 ± 2.56
    Notes
    [20] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [21] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    [22] - ITT Set. Subjects with non-missing data (n) were included for analysis.
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Up to approximately 207 days
    Adverse event reporting additional description
    Safety Analysis Set: Subjects who received at least 1 dose of IMP in the trial were included and analyzed according to the treatment received. One subject who was assigned to the placebo group received GWP42003-P 5 milligram (mg)/kilogram (kg). For safety analyses, this subject was assigned to the GWP42003-P 5 mg/kg.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    5 mg/kg/day GWP42003-P
    Reporting group description
    Subjects received 5 mg/kg/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID). One subject who was assigned to the placebo group received GWP42003-P 5 mg/kg. For safety analyses, this subject was assigned to the GWP42003-P 5 mg/kg group.

    Reporting group title
    Placebo
    Reporting group description
    Subjects received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID. One subject who was assigned to the placebo group received GWP42003-P 5 mg/kg. For safety analyses, this subject was assigned to the GWP42003-P 5 mg/kg group.

    Reporting group title
    15 mg/kg/day GWP42003-P
    Reporting group description
    Subjects received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.

    Serious adverse events
    5 mg/kg/day GWP42003-P Placebo 15 mg/kg/day GWP42003-P
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 9 (11.11%)
    2 / 9 (22.22%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Dyskinesia
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 9 (11.11%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    5 mg/kg/day GWP42003-P Placebo 15 mg/kg/day GWP42003-P
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 11 (81.82%)
    7 / 9 (77.78%)
    7 / 9 (77.78%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    2 / 9 (22.22%)
         occurrences all number
    0
    1
    3
    Psychiatric disorders
    Agitation
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Anxiety
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Bruxism
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Dermatillomania
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Insomnia
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 9 (22.22%)
    0 / 9 (0.00%)
         occurrences all number
    1
    2
    0
    Irritability
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Middle insomnia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Nervousness
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Sleep disorder
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Sunburn
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Blood thyroid stimulating hormone increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Breath sounds abnormal
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Mean cell volume increased
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Blood and lymphatic system disorders
    Coagulopathy
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Eosinophilia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Macrocytosis
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    1
    0
    Neutropenia
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    1
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    1
    0
    1
    Choking
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Hiccups
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinorrhoea
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Hyperventilation
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Disturbance in attention
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Dizziness
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Dizziness postural
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Drooling
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Hyperreflexia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Lethargy
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    3 / 9 (33.33%)
         occurrences all number
    0
    0
    3
    Seizure
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    2
    1
    0
    Somnolence
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Eye disorders
    Ocular hyperaemia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Constipation
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    2
    0
    2
    Faeces soft
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Tooth discolouration
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    0
    3
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Rash
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 9 (11.11%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    1
    Skin discolouration
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Joint stiffness
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Scoliosis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 9 (0.00%)
    2 / 9 (22.22%)
         occurrences all number
    0
    0
    2
    Hypoglycaemia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Selenium deficiency
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    2
    2
    0
    Otitis media
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Pharyngitis streptococcal
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 11 (9.09%)
    3 / 9 (33.33%)
    0 / 9 (0.00%)
         occurrences all number
    1
    5
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    4
    0
    0

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    04 Feb 2019
    - Exclusion criteria were updated to exclude patients with moderate hepatic impairment; added clarification to investigational medicinal product (IMP) dosing instructions and compliance documentation and information collected on pharmacokinetic sample collection days; updated the list of prohibited therapies during the trial; and added clarifications to the statistical analysis and trial monitoring. -Updated the exclusion criteria related to hepatic and cardiovascular functions, contraception and hypersensitivity to cannabinoids or excipients. -Added additional time points to the safety assessments (pregnancy, electrocardiogram, clinical laboratory). -Information on concomitant therapy and potential drug interactions and dose reduction of IMP were added. -Minimum age was lowered from 4 years to 2 years in the inclusion criteria. -Caregiver assessment of Rett symptoms (symptom diary) was added.
    18 Jul 2019
    - The protocol was updated to specify the Rett Syndrome Behaviour Questionnaire (RSBQ) as the primary end point, and the Clinical Global Impressions - Improvement (CGI-I) as the key secondary end point. - Contraceptive measures were more precisely documented within the protocol, and aligned with the Clinical Trial Facilitation Group guideline. - The protocol was updated to include a benefit-risk assessment concluding that the overall benefit-risk for the development of Cannabidiol oral solution in the Rett syndrome population is favorable. - Clarification that methyl CpG binding protein 2 (MECP2) mutation must be pathogenic; removal of option to proceed with randomization without confirmation of mutation type; this was to ensure sites confirm the genetic mutation was pathogenic prior to randomization. - Clarification on criteria requiring IMP discontinuation rather than study withdrawal, as patients/caregivers were encouraged to remain in the trial and continue to complete trial assessments/visits as per protocol even if IMP was discontinued.
    07 Jul 2020
    - Inclusion criteria were changed to allow the enrolment of males, the contraception requirements and tanner staging were updated for inclusion of males. - Wording regarding IMP administration via gastrostomy or nasogastric feeding tube was updated in the inclusion criteria and the IMP administration sections to remove reference to specific tube materials and instead required a discussion with the medical monitor to confirm suitability of tubes being used. - The number of sites expected to participate was increased to 35 from 25. The requirement of subject weight <13 kg was updated to <19 kg for the collection of blood sample at Visit 1/prior to Visit 1 for MECP2 mutation analysis and the permitted visit window for Visit 2 of +3 days was updated to +6 days from the scheduled visit date so that sufficient time was available for MECP2 mutation analysis results. Language related to meal at Visit 5 and telephone visits (Visits 3, 4 and 11) was updated. Informed consent procedure and training requirements were updated as a result of the change in policy with the Sponsor. - A new section for special circumstances was included to detect cases of suspected or confirmed COVID-19 infection (or other significant communicable infectious diseases) within a year of Screening (Visit 1). - Language updates were made to the reporting procedures for all AEs in relation to action taken with trial medication; to the trial monitoring procedure to reflect that monitoring might occur onsite or remotely; and to align with the GW Research Ltd publication policy.

    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 was terminated early due to enrollment challenges and the COVID-19 pandemic. Numbers of subjects in each treatment groups were small, which precluded the planned formal statistical inferences and limited data interpretation.
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA