Phase 2
Completed N=103
Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder
Source: ClinicalTrials.gov NCT04745026 ↗Enrolled (actual)
103
Serious AEs
2.9%
Results posted
Mar 2025
Primary outcomePrimary: Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores — -6.35; -5.41; -6.67; -8.05 score on a scale — p==0.085
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores |
-6.35; -5.41; -6.67; -8.05; -6.97; -8.57 | =0.085 |
| PRIMARY Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores |
2.97; 0.99; 3.69; 3.54; 4.85; 5.55 | =0.8506 |
| PRIMARY Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category |
13; 14; 12; 11; 9; 11 | =0.7087 |
| PRIMARY Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores |
-0.33; -0.31; -0.50; -0.48; -0.55; -0.63 | =0.6108 |
| SECONDARY Number of Participants Reporting Treatment-emergent Adverse Events |
27; 24; 25; 32; 6; 10 | — |
| SECONDARY Mean Change From Baseline in Hematology Clinical Laboratory Levels |
-0.01; 0; -0.09; 0; 0.04; -0.20 | — |
| SECONDARY Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels |
-0.07; -0.03; -1.33; 0.22; -0.02; 0.43 | — |
| SECONDARY Mean Change From Baseline in Hemoglobin Levels |
-0.06; 0.08 | — |
| SECONDARY Mean Change From Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels |
0.13; -0.03 | — |
| SECONDARY Mean Change From Baseline in Erythrocyte Mean Corpuscular Volume Levels |
0.52; 0.03 | — |
| SECONDARY Number of Patients With Clinically Significant Vital Sign Values |
7; 13; 2; 2; 4; 6 | — |
| SECONDARY Number of Patients With Clinically Significant Physical Examination Procedure Findings |
2; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Patients With Clinically Significant 12-lead Electrocardiogram Findings |
7; 4; 2; 0; 1; 0 | — |
| SECONDARY Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) |
4; 3; 3; 3; 2; 1 | — |
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 pube
Data sourced from ClinicalTrials.gov (NCT04745026). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.