Trial Outcomes & Findings for Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder (NCT NCT04745026)

Last Updated: 2025-03-30

Results Overview

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 items for each subscale range from 0-45 (irritability), 0-48 (social withdrawal and hyperactivity/noncompliance), 0-21 (stereotypic behavior), and 0-12 (inappropriate speech) 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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

103 participants

Primary outcome timeframe

Baseline up to Week 12

Results posted on

2025-03-30

Participant Flow

A total of 191 patients were screened for this study. Of those 191 patients, 103 patients met all inclusion criteria and no exclusion criteria were enrolled in the study and randomized to treatment at 24 centers in the United States, Canada, Spain, United Kingdom, and Australia.

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).

Participant milestones

Participant milestones
Measure	GWP42003-P 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.	Placebo Patients who were randomized to placebo for 12 weeks.
Overall Study STARTED	49	54
Overall Study COMPLETED	36	41
Overall Study NOT COMPLETED	13	13

Reasons for withdrawal

Reasons for withdrawal
Measure	GWP42003-P 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.	Placebo Patients who were randomized to placebo for 12 weeks.
Overall Study Physician Decision	1	0
Overall Study Adverse Event	2	2
Overall Study Protocol Violation	1	3
Overall Study Lost to Follow-up	4	3
Overall Study Withdrawal by Subject	5	5

Baseline Characteristics

The WASI-II raw score is converted into T-scores and then summed to derive the composite score, which ranges from 70 (worst outcome) to 160 (best outcome). Composite score performance categories are: Profoundly high=150-160, Superior= 130-160, Very high=120-129, Bright normal=110-119, Average=90-109, Low average=80-89, and Borderline=70-79.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GWP42003-P n=49 Participants 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.	Placebo n=54 Participants Patients who were randomized to placebo for 12 weeks.	Total n=103 Participants Total of all reporting groups
Age, Categorical <=18 years	49 Participants n=49 Participants	54 Participants n=54 Participants	103 Participants n=103 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=49 Participants	0 Participants n=54 Participants	0 Participants n=103 Participants
Age, Categorical >=65 years	0 Participants n=49 Participants	0 Participants n=54 Participants	0 Participants n=103 Participants
Age, Continuous	11.1 years STANDARD_DEVIATION 3.02 • n=49 Participants	10.8 years STANDARD_DEVIATION 3.01 • n=54 Participants	10.9 years STANDARD_DEVIATION 3 • n=103 Participants
Sex: Female, Male Female	13 Participants n=49 Participants	7 Participants n=54 Participants	20 Participants n=103 Participants
Sex: Female, Male Male	36 Participants n=49 Participants	47 Participants n=54 Participants	83 Participants n=103 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	1 Participants n=49 Participants	2 Participants n=54 Participants	3 Participants n=103 Participants
Race/Ethnicity, Customized Asian	1 Participants n=49 Participants	2 Participants n=54 Participants	3 Participants n=103 Participants
Race/Ethnicity, Customized Black or African American	5 Participants n=49 Participants	2 Participants n=54 Participants	7 Participants n=103 Participants
Race/Ethnicity, Customized White	32 Participants n=49 Participants	42 Participants n=54 Participants	74 Participants n=103 Participants
Race/Ethnicity, Customized Multiple	5 Participants n=49 Participants	2 Participants n=54 Participants	7 Participants n=103 Participants
Race/Ethnicity, Customized Unknown	2 Participants n=49 Participants	1 Participants n=54 Participants	3 Participants n=103 Participants
Race/Ethnicity, Customized Not Reported	1 Participants n=49 Participants	2 Participants n=54 Participants	3 Participants n=103 Participants
Race/Ethnicity, Customized Other	2 Participants n=49 Participants	1 Participants n=54 Participants	3 Participants n=103 Participants
WASI-II Intelligent Quotient Score	99.42 score on a scale STANDARD_DEVIATION 18.03 • n=49 Participants • The WASI-II raw score is converted into T-scores and then summed to derive the composite score, which ranges from 70 (worst outcome) to 160 (best outcome). Composite score performance categories are: Profoundly high=150-160, Superior= 130-160, Very high=120-129, Bright normal=110-119, Average=90-109, Low average=80-89, and Borderline=70-79.	100.89 score on a scale STANDARD_DEVIATION 17.16 • n=54 Participants • The WASI-II raw score is converted into T-scores and then summed to derive the composite score, which ranges from 70 (worst outcome) to 160 (best outcome). Composite score performance categories are: Profoundly high=150-160, Superior= 130-160, Very high=120-129, Bright normal=110-119, Average=90-109, Low average=80-89, and Borderline=70-79.	100.19 score on a scale STANDARD_DEVIATION 17.49 • n=103 Participants • The WASI-II raw score is converted into T-scores and then summed to derive the composite score, which ranges from 70 (worst outcome) to 160 (best outcome). Composite score performance categories are: Profoundly high=150-160, Superior= 130-160, Very high=120-129, Bright normal=110-119, Average=90-109, Low average=80-89, and Borderline=70-79.
Aberrant Behavior Checklist Irritability	23.2 score on a scale STANDARD_DEVIATION 7.36 • n=48 Participants • Aberrant Behavior Checklist Irritability was assessed in participants with available data in the Full Analysis Set.	22.5 score on a scale STANDARD_DEVIATION 8.74 • n=54 Participants • Aberrant Behavior Checklist Irritability was assessed in participants with available data in the Full Analysis Set.	22.8 score on a scale STANDARD_DEVIATION 8.09 • n=102 Participants • Aberrant Behavior Checklist Irritability was assessed in participants with available data in the Full Analysis Set.
Aberrant Behavior Checklist Social Withdrawal	12.6 score on a scale STANDARD_DEVIATION 8.63 • n=48 Participants • Aberrant Behavior Checklist Social Withdrawal was assessed in participants with available data in the Full Analysis Set.	12.7 score on a scale STANDARD_DEVIATION 9.11 • n=54 Participants • Aberrant Behavior Checklist Social Withdrawal was assessed in participants with available data in the Full Analysis Set.	12.7 score on a scale STANDARD_DEVIATION 8.85 • n=102 Participants • Aberrant Behavior Checklist Social Withdrawal was assessed in participants with available data in the Full Analysis Set.
Aberrant Behavior Checklist Stereotypic Behavior	6.7 score on a scale STANDARD_DEVIATION 5.08 • n=48 Participants • Aberrant Behavior Checklist Stereotypic Behavior was assessed in participants with available data in the Full Analysis Set.	6.7 score on a scale STANDARD_DEVIATION 4.83 • n=54 Participants • Aberrant Behavior Checklist Stereotypic Behavior was assessed in participants with available data in the Full Analysis Set.	6.7 score on a scale STANDARD_DEVIATION 4.92 • n=102 Participants • Aberrant Behavior Checklist Stereotypic Behavior was assessed in participants with available data in the Full Analysis Set.
Aberrant Behavior Checklist Hyperactivity/Noncompliance	25.2 score on a scale STANDARD_DEVIATION 12.27 • n=48 Participants • Aberrant Behavior Checklist Hyperactivity/Noncompliance was assessed in participants with available data in the Full Analysis Set.	26.9 score on a scale STANDARD_DEVIATION 9.63 • n=54 Participants • Aberrant Behavior Checklist Hyperactivity/Noncompliance was assessed in participants with available data in the Full Analysis Set.	26.1 score on a scale STANDARD_DEVIATION 10.93 • n=102 Participants • Aberrant Behavior Checklist Hyperactivity/Noncompliance was assessed in participants with available data in the Full Analysis Set.
Aberrant Behavior Checklist Inappropriate Speech	5.7 score on a scale STANDARD_DEVIATION 3.22 • n=48 Participants • Aberrant Behavior Checklist Inappropriate Speech was assessed in participants with available data in the Full Analysis Set.	5.6 score on a scale STANDARD_DEVIATION 3.08 • n=54 Participants • Aberrant Behavior Checklist Inappropriate Speech was assessed in participants with available data in the Full Analysis Set.	5.6 score on a scale STANDARD_DEVIATION 3.13 • n=102 Participants • Aberrant Behavior Checklist Inappropriate Speech was assessed in participants with available data in the Full Analysis Set.
Vineland Adaptive Behavior Scales Social and Communication Composite Score	66.6 score on a scale STANDARD_DEVIATION 19.61 • n=48 Participants • Vineland Adaptive Behavior Scales Social and Communication Composite Score was assessed in participants with available data in the Full Analysis Set.	67.7 score on a scale STANDARD_DEVIATION 15.18 • n=50 Participants • Vineland Adaptive Behavior Scales Social and Communication Composite Score was assessed in participants with available data in the Full Analysis Set.	67.2 score on a scale STANDARD_DEVIATION 17.41 • n=98 Participants • Vineland Adaptive Behavior Scales Social and Communication Composite Score was assessed in participants with available data in the Full Analysis Set.
Clinical Global Impression - Severity (CGI-S)	4.76 score on a scale STANDARD_DEVIATION 0.630 • n=49 Participants • Clinical Global Impression - Severity (CGI-S) was assessed in participants with available data in the Full Analysis Set.	4.82 score on a scale STANDARD_DEVIATION 0.684 • n=51 Participants • Clinical Global Impression - Severity (CGI-S) was assessed in participants with available data in the Full Analysis Set.	4.79 score on a scale STANDARD_DEVIATION 0.656 • n=100 Participants • Clinical Global Impression - Severity (CGI-S) was assessed in participants with available data in the Full Analysis Set.

PRIMARY outcome

Timeframe: Baseline up to Week 12

Population: Aberrant behavior checklist subscale total scores were assessed in participants with available data in the Full Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=40 Participants 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.	Placebo n=51 Participants Patients who were randomized to placebo for 12 weeks.
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Irritability, Week 4	-6.35 score on a scale Standard Deviation 7.98	-5.41 score on a scale Standard Deviation 7.16
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Irritability, Week 8	-6.67 score on a scale Standard Deviation 8.63	-8.05 score on a scale Standard Deviation 9.12
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Irritability, Week 12	-6.97 score on a scale Standard Deviation 9.68	-8.57 score on a scale Standard Deviation 10.10
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Social Withdrawal, Week 4	-2.53 score on a scale Standard Deviation 5.61	-2.92 score on a scale Standard Deviation 5.24
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Social Withdrawal, Week 8	-3.81 score on a scale Standard Deviation 6.76	-4.74 score on a scale Standard Deviation 6.25
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Social Withdrawal, Week 12	-3.74 score on a scale Standard Deviation 6.21	-4.51 score on a scale Standard Deviation 5.95
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Stereotypic Behavior, Week 4	-2.10 score on a scale Standard Deviation 4.24	-1.39 score on a scale Standard Deviation 3.24
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Stereotypic Behavior, Week 8	-1.92 score on a scale Standard Deviation 3.99	-1.98 score on a scale Standard Deviation 4.05
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Stereotypic Behavior, Week 12	-1.88 score on a scale Standard Deviation 3.63	-2.31 score on a scale Standard Deviation 4.03
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Hyperactivity/Noncompliance, Week 4	-5.73 score on a scale Standard Deviation 8.08	-4.55 score on a scale Standard Deviation 8.15
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Hyperactivity/Noncompliance, Week 8	-6.39 score on a scale Standard Deviation 9.15	-7.42 score on a scale Standard Deviation 9.06
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Hyperactivity/Noncompliance, Week 12	-6.74 score on a scale Standard Deviation 10.62	-8.77 score on a scale Standard Deviation 9.94
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Inappropriate Speech, Week 4	-2.13 score on a scale Standard Deviation 2.88	-0.88 score on a scale Standard Deviation 2.07
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Inappropriate Speech, Week 8	-1.58 score on a scale Standard Deviation 2.74	-1.44 score on a scale Standard Deviation 2.73
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores ABC-Inappropriate Speech, Week 12	-1.94 score on a scale Standard Deviation 2.93	-1.51 score on a scale Standard Deviation 2.97

PRIMARY outcome

Timeframe: Baseline up to Week 12

Population: Vineland Adaptive Behavior Scales-3 (VABS-3) scores were assessed in participants with available data in the Full Analysis Set.

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). The adaptive behavior composite score is calculated as arithmetic mean of all 3 domain scores. The total score range is 20 to 140, where low scores indicate low (worst) clinical outcome and high scores indicate high (best) clinical outcome. The change from baseline in VABS-3 is being reported with positive values indicating a positive improvement in adaptive behavior.

Outcome measures

Outcome measures
Measure	GWP42003-P n=37 Participants 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.	Placebo n=45 Participants Patients who were randomized to placebo for 12 weeks.
Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores Week 4	2.97 score on a scale Standard Deviation 6.70	0.99 score on a scale Standard Deviation 9.29
Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores Week 8	3.69 score on a scale Standard Deviation 10.82	3.54 score on a scale Standard Deviation 8.83
Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores Week 12	4.85 score on a scale Standard Deviation 8.83	5.55 score on a scale Standard Deviation 10.42

PRIMARY outcome

Timeframe: Day 85

Population: Patients per CGI-I category were assessed in participants with available data in the Full Analysis Set.

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.

Outcome measures

Outcome measures
Measure	GWP42003-P n=34 Participants 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.	Placebo n=38 Participants Patients who were randomized to placebo for 12 weeks.
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category Minimally improved	13 Participants	14 Participants
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category Much improved	12 Participants	11 Participants
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category No change	9 Participants	11 Participants
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category Much worse	0 Participants	1 Participants
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category Very much improved	0 Participants	1 Participants
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category Minimally worse	0 Participants	0 Participants
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category Very much worse	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 12

Population: CGI-S was assessed in participants with available data in the Full Analysis Set.

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.

Outcome measures

Outcome measures
Measure	GWP42003-P n=43 Participants 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.	Placebo n=51 Participants Patients who were randomized to placebo for 12 weeks.
Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores Week 4	-0.33 score on a scale Standard Deviation 0.61	-0.31 score on a scale Standard Deviation 0.69
Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores Week 8	-0.50 score on a scale Standard Deviation 0.68	-0.48 score on a scale Standard Deviation 0.92
Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores Week 12	-0.55 score on a scale Standard Deviation 0.85	-0.63 score on a scale Standard Deviation 1.10

SECONDARY outcome

Timeframe: Baseline up to Week 12

Population: Adverse events were assessed in the Safety Analysis Set.

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.

Outcome measures

Outcome measures
Measure	GWP42003-P n=49 Participants 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.	Placebo n=54 Participants Patients who were randomized to placebo for 12 weeks.
Number of Participants Reporting Treatment-emergent Adverse Events TEAE	27 Participants	24 Participants
Number of Participants Reporting Treatment-emergent Adverse Events Non-TEAE	25 Participants	32 Participants
Number of Participants Reporting Treatment-emergent Adverse Events Treatment-related TEAE	6 Participants	10 Participants
Number of Participants Reporting Treatment-emergent Adverse Events Serious TEAE	2 Participants	1 Participants
Number of Participants Reporting Treatment-emergent Adverse Events Serious Treatment-related TEAE	0 Participants	0 Participants
Number of Participants Reporting Treatment-emergent Adverse Events TEAE leading to study intervention withdrawal	2 Participants	1 Participants
Number of Participants Reporting Treatment-emergent Adverse Events Treatment-related TEAE leading to drug withdrawal	1 Participants	1 Participants
Number of Participants Reporting Treatment-emergent Adverse Events TEAE leading to withdrawal	2 Participants	2 Participants
Number of Participants Reporting Treatment-emergent Adverse Events TEAE leading to death	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Week 9 (end of taper/withdrawal)

Population: Hematology clinical laboratory levels were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=30 Participants 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.	Placebo n=37 Participants Patients who were randomized to placebo for 12 weeks.
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Basophils	-0.01 10^9 cells/L Standard Deviation 0.03	0 10^9 cells/L Standard Deviation 0.03
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Eosinophils	-0.09 10^9 cells/L Standard Deviation 0.311	0 10^9 cells/L Standard Deviation 0.40
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Lymphocytes	0.04 10^9 cells/L Standard Deviation 0.70	-0.20 10^9 cells/L Standard Deviation 0.68
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Monocytes	-0.01 10^9 cells/L Standard Deviation 0.15	-0.01 10^9 cells/L Standard Deviation 0.19
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Neutrophils	0.04 10^9 cells/L Standard Deviation 1.80	-0.01 10^9 cells/L Standard Deviation 1.26
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Platelets	-9.43 10^9 cells/L Standard Deviation 34.50	8.43 10^9 cells/L Standard Deviation 52.13
Mean Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Leukocytes	-0.02 10^9 cells/L Standard Deviation 1.87	-0.23 10^9 cells/L Standard Deviation 1.79

SECONDARY outcome

Timeframe: Baseline up to Week 9 (end of taper/withdrawal)

Population: Hematology clinical laboratory levels were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=31 Participants 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.	Placebo n=38 Participants Patients who were randomized to placebo for 12 weeks.
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Basophils/Leukocytes	-0.07 percentage change from baseline Standard Deviation 0.45	-0.03 percentage change from baseline Standard Deviation 0.42
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Eosinophils/Leukocytes	-1.33 percentage change from baseline Standard Deviation 5.37	0.22 percentage change from baseline Standard Deviation 4.88
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Hematocrit	-0.02 percentage change from baseline Standard Deviation 2.19	0.43 percentage change from baseline Standard Deviation 2.30
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Lymphocytes/Leukocytes	1.05 percentage change from baseline Standard Deviation 11.25	-2.32 percentage change from baseline Standard Deviation 8.26
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Monocytes/Leukocytes	-0.16 percentage change from baseline Standard Deviation 2.04	0.05 percentage change from baseline Standard Deviation 1.86
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels Week 9: Neutrophils/Leukocytes	0.49 percentage change from baseline Standard Deviation 12.29	2.08 percentage change from baseline Standard Deviation 8.89

SECONDARY outcome

Timeframe: Week 9 (end of taper/withdrawal)

Population: Hemoglobin levels were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=31 Participants 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.	Placebo n=38 Participants Patients who were randomized to placebo for 12 weeks.
Mean Change From Baseline in Hemoglobin Levels	-0.06 g/dL Standard Deviation 0.55	0.08 g/dL Standard Deviation 0.65

SECONDARY outcome

Timeframe: Week 9 (end of taper/withdrawal)

Population: Erythrocyte mean corpuscular hemoglobin levels were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=31 Participants 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.	Placebo n=38 Participants Patients who were randomized to placebo for 12 weeks.
Mean Change From Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels	0.13 pg/cell Standard Deviation 0.67	-0.03 pg/cell Standard Deviation 0.72

SECONDARY outcome

Timeframe: Week 9 (end of taper/withdrawal)

Population: Erythrocyte mean corpuscular volume levels were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=31 Participants 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.	Placebo n=38 Participants Patients who were randomized to placebo for 12 weeks.
Mean Change From Baseline in Erythrocyte Mean Corpuscular Volume Levels	0.52 fL Standard Deviation 2.32	0.03 fL Standard Deviation 2.39

SECONDARY outcome

Timeframe: Post-baseline up to 12 weeks

Population: Vital signs were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=45 Participants 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.	Placebo n=52 Participants Patients who were randomized to placebo for 12 weeks.
Number of Patients With Clinically Significant Vital Sign Values Increase in weight from baseline >7%	7 Participants	13 Participants
Number of Patients With Clinically Significant Vital Sign Values Decrease in weight from baseline >7%	2 Participants	2 Participants
Number of Patients With Clinically Significant Vital Sign Values Body temperature <36 degrees Celsius	4 Participants	6 Participants
Number of Patients With Clinically Significant Vital Sign Values Body temperature >38 degrees Celsius	1 Participants	0 Participants
Number of Patients With Clinically Significant Vital Sign Values Pulse rate <60 beats/min	1 Participants	3 Participants
Number of Patients With Clinically Significant Vital Sign Values Pulse rate >100 beats/min	7 Participants	4 Participants
Number of Patients With Clinically Significant Vital Sign Values Respiratory rate <12 breaths/min	0 Participants	0 Participants
Number of Patients With Clinically Significant Vital Sign Values Respiratory rate >20 breaths/min	13 Participants	17 Participants
Number of Patients With Clinically Significant Vital Sign Values Systolic blood pressure <90 mmhg	5 Participants	3 Participants
Number of Patients With Clinically Significant Vital Sign Values Systolic blood pressure >160 mmhg	0 Participants	0 Participants
Number of Patients With Clinically Significant Vital Sign Values Diastolic blood pressure <50 mmhg	3 Participants	2 Participants
Number of Patients With Clinically Significant Vital Sign Values Diastolic blood pressure >120 mmhg	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 85 post-baseline

Population: Abnormal physical exam findings were assessed in participants with available data in the Safety Analysis Set.

Number of patients with abnormal physical exam findings are reported.

Outcome measures

Outcome measures
Measure	GWP42003-P n=49 Participants 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.	Placebo n=54 Participants Patients who were randomized to placebo for 12 weeks.
Number of Patients With Clinically Significant Physical Examination Procedure Findings Abdomen	2 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Cardiovascular	0 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Chest/lungs	0 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Dermatological	3 Participants	3 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Endocrine system	0 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Gastrointestinal	0 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings General appearance	0 Participants	2 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings HEENT	0 Participants	1 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Lymphatic	0 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Musculoskeletal/Extremities	0 Participants	1 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Neurological	2 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Other	0 Participants	0 Participants
Number of Patients With Clinically Significant Physical Examination Procedure Findings Genitourinary/Reproductive	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 85 post-baseline

Population: Clinically significant 12-lead electrocardiogram were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Outcome measures
Measure	GWP42003-P n=49 Participants 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.	Placebo n=54 Participants Patients who were randomized to placebo for 12 weeks.
Number of Patients With Clinically Significant 12-lead Electrocardiogram Findings QTcB >480 msec	2 Participants	0 Participants
Number of Patients With Clinically Significant 12-lead Electrocardiogram Findings QTcB >450 msec	7 Participants	4 Participants
Number of Patients With Clinically Significant 12-lead Electrocardiogram Findings QTcB >500 msec	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 92

Population: Suicidal ideation or behavior was assessed in participants with available data in the Safety Analysis Set.

C-SSRS rating scale results since last visit is reported. The presence/absence of any suicidal ideation or behavior is scored based on yes/no responses. The overall number of participants reporting suicidal ideation or behavior is being reported.

Outcome measures

Outcome measures
Measure	GWP42003-P n=49 Participants 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.	Placebo n=54 Participants Patients who were randomized to placebo for 12 weeks.
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Suicidal ideation	4 Participants	3 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Wish to be dead	3 Participants	3 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Non-specific active suicidal thoughts	2 Participants	1 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Active suicidal ideation, no intent to act	1 Participants	0 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Active suicidal ideation, some intent to act	0 Participants	0 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Active suicidal ideation, specific plan/intent	0 Participants	0 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Suicidal behavior	3 Participants	6 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Actual attempt	0 Participants	1 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Interrupted attempt	0 Participants	1 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Aborted attempt	0 Participants	0 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Preparatory acts or behavior	0 Participants	1 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Suicidal behavior item	0 Participants	1 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Self-injurious behavior without suicidal intent	3 Participants	5 Participants
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) Completed suicide	0 Participants	0 Participants

Adverse Events

GWP42003-P

Serious events: 2 serious events

Other events: 14 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	GWP42003-P n=49 participants at risk 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.	Placebo n=54 participants at risk Patients who were randomized to placebo for 12 weeks.
Psychiatric disorders Suicidal ideation	2.0% 1/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	0.00% 0/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
Infections and infestations COVID-19	2.0% 1/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	1.9% 1/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.

Other adverse events

Other adverse events
Measure	GWP42003-P n=49 participants at risk 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.	Placebo n=54 participants at risk Patients who were randomized to placebo for 12 weeks.
Nervous system disorders Headache	6.1% 3/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	7.4% 4/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
General disorders Pyrexia	0.00% 0/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	9.3% 5/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
General disorders Fatigue	0.00% 0/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	5.6% 3/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
Gastrointestinal disorders Vomiting	14.3% 7/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	7.4% 4/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
Respiratory, thoracic and mediastinal disorders Cough	6.1% 3/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	0.00% 0/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
Psychiatric disorders Irritability	0.00% 0/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	9.3% 5/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
Infections and infestations Nasopharyngitis	8.2% 4/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	0.00% 0/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.
Infections and infestations Upper respiratory tract infection	0.00% 0/49 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.	5.6% 3/54 • Adverse event data were collected from baseline up to 14 days after last dose, up to approximately 12 weeks.

Additional Information

Clinical Trial Disclosure & Transparency

Jazz Pharmaceuticals

Phone: 215-832-3750

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place