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

NCT ID: NCT04745026

Last Updated: 2025-03-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 103 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-05-31
• Study Completion Date: 2023-12-21

Brief Summary

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).

Conditions

Autism Spectrum Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: EXPERIMENTAL

GWP42003-P

Intervention Type: DRUG

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)

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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)

Interventions

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)

Intervention Type: 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)

Intervention Type: DRUG

Other Intervention Names

Epidiolex®; cannabidiol; CBD-OS

Eligibility Criteria

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jazz Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Southwest Autism Research and Resource Center (SARRRC)

Phoenix, Arizona, United States

UCSD School of Medicine

La Jolla, California, United States

UCLA Neuropsychiatric Institute

Los Angeles, California, United States

University of California San Francisco

San Francisco, California, United States

APG Research, LLC

Orlando, Florida, United States

University of Louisville

Louisville, Kentucky, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Massachusetts General Hospital (Lurie Center for Autism)

Lexington, Massachusetts, United States

Richmond Behavioral Associates

Staten Island, New York, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Red Oak Psychiatry Associates, PA

Houston, Texas, United States

Seattle Children's Research Institute

Seattle, Washington, United States

Monash Medical Centre

Clayton, , Australia

Queensland Children's Hospital

South Brisbane, , Australia

The Kids Clinic

Ajax, Ontario, Canada

Center for Pediatric Excellence

Ottawa, Ontario, Canada

Klinik fur Psychiatrie, Psychotherapie und Psychosomatik im Kindes und Jugendalter

Freiburg im Breisgau, , Germany

Zentralinstitut fuer Seelische Gesundheit

Mannheim, , Germany

Instituto Global de Atencion Integral del Neurodesarrollo (IGAIN)

Barcelona, , Spain

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Corporacio Sanitaria Parc Tauli

Sabadell, , Spain

University of Glasgow Institute of Health and Wellbeing

Glasgow, , United Kingdom

Institute of Psychiatry, King's College London

London, , United Kingdom

Countries

United States; Australia; Canada; Germany; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2020-002819-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GWND19189

Identifier Type: -

Identifier Source: org_study_id