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

March 12, 2025 updated by: Jazz Pharmaceuticals

An Exploratory, Phase 2, Randomized, Double-blind, Placebo-controlled Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder

This study will be conducted to evaluate the efficacy of GWP42003-P, compared with placebo, in reducing symptom severity in children with Autism Spectrum Disorder (ASD).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
103

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
      • Clayton, Australia, 03168
        • Monash Medical Centre
      • South Brisbane, Australia, 4101
        • Queensland Children's Hospital
  • Canada
    • Ontario
      • Ajax, Ontario, Canada, L1Z0M1
        • The Kids Clinic
      • Ottawa, Ontario, Canada, K2G1W2
        • Center for Pediatric Excellence
  • Germany
      • Freiburg, Germany, 79104
        • Klinik fur Psychiatrie, Psychotherapie und Psychosomatik im Kindes und Jugendalter
      • Mannheim, Germany, 68159
        • Zentralinstitut fuer Seelische Gesundheit
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d'Hebron
      • Barcelona, Spain, 08007
        • Instituto Global de Atencion Integral del Neurodesarrollo (IGAIN)
      • Sabadell, Spain, 08208
        • Corporacio Sanitaria Parc Tauli
  • United Kingdom
      • Glasgow, United Kingdom, G128QQ
        • University of Glasgow Institute of Health and Wellbeing
      • London, United Kingdom
        • Institute of Psychiatry, King's College London
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85006
        • Southwest Autism Research and Resource Center (SARRRC)
    • California
      • La Jolla, California, United States, 92093
        • UCSD School of Medicine
      • Los Angeles, California, United States, 90024
        • UCLA Neuropsychiatric Institute
      • San Francisco, California, United States, 94143
        • University of California San Francisco
    • Florida
      • Orlando, Florida, United States, 32803
        • Apg Research, Llc
    • Kentucky
      • Louisville, Kentucky, United States, 40292
        • University of Louisville
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
      • Lexington, Massachusetts, United States, 02421
        • Massachusetts General Hospital (Lurie Center for Autism)
    • New York
      • Staten Island, New York, United States, 10312
        • Richmond Behavioral Associates
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Texas
      • Houston, Texas, United States, 77090
        • Red Oak Psychiatry Associates, PA
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

6 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Participant weight is at least 12 kilograms (kg).
  • Participants (if possessing adequate understanding, in the investigator's opinion) and their parent(s)/legal representative are willing and able to give informed assent and consent for participation in the trial.
  • Participant and their caregiver are willing and able (in the investigator's opinion) to comply with all trial requirements.
  • Participant has a diagnosis of Autism Spectrum Disorder (ASD) as per Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria for ASD, confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria (conducted within 2 years at the trial site or at screening by a qualified assessor). Note: During special circumstances (e.g., COVID-19 pandemic) where the ADOS-2 cannot be performed due to site restrictions (e.g., mandatory use of face masks) and an ADOS- 2 conducted within 2 years at the trial site by a qualified assessor is not available, eligibility can be confirmed using: 1) an ADOS-2 performed within 2 years by a qualified assessor (external to the site); 2) if 1) is not available, eligibility may be confirmed using the Autism Diagnostic Interview, Revised (ADI-R) at screening.
  • Clinical Global Impressions - Improvement Scale (CGI-S) ≥ 4 (moderately ill) at screening and randomization.
  • Intelligence quotient (IQ) ≥ 70 at screening, or measured within 1 year of screening, using Wechsler Abbreviated Scale of Intelligence Scale Second Edition (WASI-II).
  • All medications or interventions (including psychosocial interventions, dietary supplements, probiotics, speech therapy, etc.) for ASD related symptoms must have been stable for 4 weeks prior to screening and randomization, and the patient/caregiver should be willing to maintain a stable regimen throughout the trial.
  • Participants must have the ability to swallow the investigational medicinal product (IMP), provided as a liquid solution.
  • Participant and/or parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
  • Participant and/or parent(s)/legal representative is/are willing to allow the participant's primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.

Exclusion Criteria:

  • Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or major depression (participants with depression in remission may be included)
  • Has a diagnosis other than ASD that dominates the clinical presentation (e.g., Attention Deficit Hyperactivity Disorder [ADHD])
  • Has a progressive neurological condition
  • Seizures in the past 24 weeks
  • Changes in anticonvulsive therapy within the last 12 weeks
  • Currently taking more than 2 anti-epileptic drugs (AEDs)
  • Taking sirolimus, everolimus, temsirolimus, or tacrolimus
  • Taking clobazam
  • Taking omeprazole, lansoprazole, tolbutamide, or warfarin
  • Taking repaglinide, pioglitazone, rosiglitazone, montelukast, bupropion, or efavirenz
  • Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®, or Epidiolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial
  • Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP, such as sesame oil.
  • Participant has moderately impaired hepatic function at screening, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN) or total bilirubin (TBL) > 2 × ULN. This criterion can only be confirmed once the laboratory results are available; participants enrolled into the trial who are later found to meet this criterion must be screen-failed.
  • Participant is male and fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) unless willing to ensure that they use male contraception (condom) or remain sexually abstinent during the trial and for 12 weeks thereafter.
  • Participant is female and of childbearing potential (i.e., following menarche and until becoming postmenopausal for ≥ 12 consecutive months unless permanently sterile by hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) unless willing to ensure that they use a highly effective method of birth control (e.g., hormonal contraception, intrauterine device/hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 12 weeks thereafter.
  • Female participant who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the trial or within 12 weeks thereafter.
  • Participant has received an IMP within the 12 weeks prior to the screening visit.
  • Participant had brain surgery or traumatic brain injury within 1 year of screening.
  • Participant has any other significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the participant's ability to take part in the trial.
  • Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the trial
  • Any history of suicidal behavior (lifelong) or any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) in the last 4 weeks or at screening or randomization
  • Participant has donated blood during the past 12 weeks and is unwilling to abstain from donation of blood during the trial.
  • Participant has any known or suspected history of alcohol or substance abuse or positive drugs of abuse test at screening (not justified by a known concurrent medication).
  • Participant has previously been randomized into this trial.
  • Participant has plans to travel outside their country of residence during the trial, unless the participant has confirmation that the IMP is permitted in the destination country/state

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: GWP42003-P 10 mg/kg/day
Participants will be stratified based on their age (6 to 11 years old, 12 to 17 years old), use of antipsychotics (on versus off), and region (North America versus Rest of the World) and will be randomized to receive 5 milligrams per kilogram per day (mg/kg/day) GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P for 11 weeks.
Drug: GWP42003-P
GWP42003-P oral solution (100 milligrams per milliliter [mg/mL] cannabidiol [CBD] in sesame oil with anhydrous ethanol, ethanol [10% v/v] sweetener [sucralose], and strawberry flavoring), administered twice a day (morning and evening)
Other Names:
  • Epidiolex®
  • cannabidiol
  • CBD-OS
Placebo Comparator: Placebo
Participants will be stratified based on their age (6 to 11 years old, 12 to 17 years old), use of antipsychotics (on versus off), and region (North America versus Rest of the World) and will be randomized to receive matching placebo for 12 weeks.
Drug: Placebo
Oral placebo to match GWP42003-P oral solution containing sesame oil with anhydrous ethanol, sweetener (sucralose), strawberry flavoring, and beta carotene, administered twice a day (morning and evening)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores
Time Frame: 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.
Baseline up to Week 12
Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores
Time Frame: 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.
Baseline up to Week 12
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category
Time Frame: 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.
Day 85
Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores
Time Frame: 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.
Baseline up to Week 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of Participants Reporting Treatment-emergent Adverse Events
Time Frame: 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.
Baseline up to Week 12
Mean Change From Baseline in Hematology Clinical Laboratory Levels
Time Frame: Baseline up to Week 9 (end of taper/withdrawal)
Baseline up to Week 9 (end of taper/withdrawal)
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels
Time Frame: Baseline up to Week 9 (end of taper/withdrawal)
Baseline up to Week 9 (end of taper/withdrawal)
Mean Change From Baseline in Hemoglobin Levels
Time Frame: Week 9 (end of taper/withdrawal)
Week 9 (end of taper/withdrawal)
Mean Change From Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels
Time Frame: Week 9 (end of taper/withdrawal)
Week 9 (end of taper/withdrawal)
Mean Change From Baseline in Erythrocyte Mean Corpuscular Volume Levels
Time Frame: Week 9 (end of taper/withdrawal)
Week 9 (end of taper/withdrawal)
Number of Patients With Clinically Significant Vital Sign Values
Time Frame: Post-baseline up to 12 weeks
Post-baseline up to 12 weeks
Number of Patients With Clinically Significant Physical Examination Procedure Findings
Time Frame: Baseline up to Day 85 post-baseline
Number of patients with abnormal physical exam findings are reported.
Baseline up to Day 85 post-baseline
Number of Patients With Clinically Significant 12-lead Electrocardiogram Findings
Time Frame: Baseline up to Day 85 post-baseline
Baseline up to Day 85 post-baseline
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS)
Time Frame: Baseline up to Day 92
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.
Baseline up to Day 92

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jazz Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 31, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 21, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 21, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 4, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 8, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 9, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 30, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 12, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GWND19189
  • 2020-002819-21 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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