Trial · NCT04745026

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

NCT04745026, Phase 2, Completed, Jazz Pharmaceuticals; results first posted 2025-03-30

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder. Trials, record NCT04745026. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04745026

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder
NCT number: NCT04745026
Status: Completed
Phases: Phase 2
Enrollment: 103 participants, actual
Lead sponsor: Jazz Pharmaceuticals
Conditions: Autism Spectrum Disorder
Interventions: GWP42003-P, Placebo
First posted: 2021-02-09
Last updated: 2025-03-30
Other registries' IDs: eudract:2020-002819-21
Results first posted: 2025-03-30

Source ClinicalTrials.gov

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Baseline up to Week 12	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.
Primary	Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores	Baseline up to Week 12	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.
Primary	Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category	Day 85	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.
Primary	Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores	Baseline up to Week 12	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.
Secondary	Number of Participants Reporting Treatment-emergent Adverse Events	Baseline up to Week 12	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.
Secondary	Mean Change From Baseline in Hematology Clinical Laboratory Levels	Baseline up to Week 9 (end of taper/withdrawal)
Secondary	Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels	Baseline up to Week 9 (end of taper/withdrawal)
Secondary	Mean Change From Baseline in Hemoglobin Levels	Week 9 (end of taper/withdrawal)
Secondary	Mean Change From Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels	Week 9 (end of taper/withdrawal)
Secondary	Mean Change From Baseline in Erythrocyte Mean Corpuscular Volume Levels	Week 9 (end of taper/withdrawal)

1 to 10 of 14Source ClinicalTrials.gov

See all 14 registered outcomes

Outcomes14

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Baseline up to Week 12	Aberrant behavior checklist subscale total scores were assessed in participants with available data in the Full Analysis Set.	score on a scale	MEAN
PRIMARY	Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores	Baseline up to Week 12	Vineland Adaptive Behavior Scales-3 (VABS-3) scores were assessed in participants with available data in the Full Analysis Set.	score on a scale	MEAN
PRIMARY	Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category	Day 85	Patients per CGI-I category were assessed in participants with available data in the Full Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores	Baseline up to Week 12	CGI-S was assessed in participants with available data in the Full Analysis Set.	score on a scale	MEAN
SECONDARY	Number of Participants Reporting Treatment-emergent Adverse Events	Baseline up to Week 12	Adverse events were assessed in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Mean Change From Baseline in Hematology Clinical Laboratory Levels	Baseline up to Week 9 (end of taper/withdrawal)	Hematology clinical laboratory levels were assessed in participants with available data in the Safety Analysis Set.	10^9 cells/L	MEAN
SECONDARY	Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels	Baseline up to Week 9 (end of taper/withdrawal)	Hematology clinical laboratory levels were assessed in participants with available data in the Safety Analysis Set.	percentage change from baseline	MEAN
SECONDARY	Mean Change From Baseline in Hemoglobin Levels	Week 9 (end of taper/withdrawal)	Hemoglobin levels were assessed in participants with available data in the Safety Analysis Set.	g/dL	MEAN
SECONDARY	Mean Change From Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels	Week 9 (end of taper/withdrawal)	Erythrocyte mean corpuscular hemoglobin levels were assessed in participants with available data in the Safety Analysis Set.	pg/cell	MEAN
SECONDARY	Mean Change From Baseline in Erythrocyte Mean Corpuscular Volume Levels	Week 9 (end of taper/withdrawal)	Erythrocyte mean corpuscular volume levels were assessed in participants with available data in the Safety Analysis Set.	fL	MEAN

1 to 10 of 14Source ClinicalTrials.gov

See all 14 outcomes

Results by group190

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	GWP42003-P	ABC-Irritability, Week 4	-6.35	Standard Deviation 7.98	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Placebo	ABC-Irritability, Week 4	-5.41	Standard Deviation 7.16	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	GWP42003-P	ABC-Irritability, Week 8	-6.67	Standard Deviation 8.63	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Placebo	ABC-Irritability, Week 8	-8.05	Standard Deviation 9.12	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	GWP42003-P	ABC-Irritability, Week 12	-6.97	Standard Deviation 9.68	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Placebo	ABC-Irritability, Week 12	-8.57	Standard Deviation 10.10	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	GWP42003-P	ABC-Social Withdrawal, Week 4	-2.53	Standard Deviation 5.61	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Placebo	ABC-Social Withdrawal, Week 4	-2.92	Standard Deviation 5.24	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	GWP42003-P	ABC-Social Withdrawal, Week 8	-3.81	Standard Deviation 6.76	score on a scale
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores	Placebo	ABC-Social Withdrawal, Week 8	-4.74	Standard Deviation 6.25	score on a scale

1 to 10 of 190Source ClinicalTrials.gov

See all 190 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	GWP42003-P	49
Overall Study	STARTED	Placebo	54
Overall Study	COMPLETED	GWP42003-P	36
Overall Study	COMPLETED	Placebo	41
Overall Study	NOT COMPLETED	GWP42003-P	13
Overall Study	NOT COMPLETED	Placebo	13

Source ClinicalTrials.gov

Adverse events20

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Headache	Nervous system disorders	GWP42003-P	3	49
other	Headache	Nervous system disorders	Placebo	4	54
other	Pyrexia	General disorders	GWP42003-P	0	49
other	Pyrexia	General disorders	Placebo	5	54
other	Fatigue	General disorders	GWP42003-P	0	49
other	Fatigue	General disorders	Placebo	3	54
other	Vomiting	Gastrointestinal disorders	GWP42003-P	7	49
other	Vomiting	Gastrointestinal disorders	Placebo	4	54
other	Cough	Respiratory, thoracic and mediastinal disorders	GWP42003-P	3	49
other	Cough	Respiratory, thoracic and mediastinal disorders	Placebo	0	54

1 to 10 of 20Source ClinicalTrials.gov

See all 20 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	GWP42003-P	0	49
Total, all-cause mortality	Placebo	0	54
Total, other adverse events	GWP42003-P	14	49
Total, other adverse events	Placebo	20	54
Total, serious adverse events	GWP42003-P	2	49
Total, serious adverse events	Placebo	1	54

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
GWP42003-P vs Placebo: deaths from any cause	Trial comparisons
GWP42003-P vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04745026

GET https://conductscience.org/api/trials/v1/NCT04745026/results?page=2

API reference For AI assistants