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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
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

GWND18064

ISRCTN number

US NCT number

NCT03848832

WHO universal trial number (UTN)

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

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?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

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

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

Independent data monitoring committee (IDMC) involvement?

Yes

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

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

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 title

Period 1 (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Investigator, Carer, Subject

Are arms mutually exclusive

Yes

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.

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.

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
Consent withdrawn by subject	-	-	1
Adverse event, non-fatal	1	-	-
Sponsor Decision - Covid-19 Precaution	-	-	1
Withdrawal Of Consent Due To Covid-19	-	-	1
Withdrawal (Covid-19)	-	2	-

Baseline characteristics

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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 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

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

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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

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

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

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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

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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

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

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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

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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

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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

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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

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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

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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 name

MedDRA

Dictionary version

23.0

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
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
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
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
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
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
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
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
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
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

More information

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Were there any global substantial amendments to the protocol? Yes

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.

Were there any global interruptions to the trial? No

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.

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Clinical trials

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.

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

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Outside EU/EEA

Clinical trials

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.

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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.