Safety and Tolerability of Cannabidivarin (CBDV) in Children and Young Adults With Autism Spectrum Disorder

NCT03849456 | Terminated Phase 2 FDA Drug

• 1 other identifier: GWND18089
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 3

Brief Summary

To determine the safety and tolerability of GWP42006 (cannabidivarin, CBDV) in children and young adults with autism spectrum disorder (ASD) and to examine the effect of GWP42006 on communication, social interactions, sleep, behavior, and cognition profiles.

Conditions

Autism Spectrum Disorder

Keywords

Cannabidivarin; CBDV; GWP42006

Outcome Measures

Primary Outcomes (1)

• Number of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)

  A TEAE was defined as an adverse event (AE) with an onset date on or after the first dose of GWP42006. If an AE had a partial onset date and it was unclear from the partial date (or the stop date) whether the AE started prior to or following the first dose of GWP42006, then the AE was considered a TEAE. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through to the Safety Follow-up will be presented.

  Day 1 to Day 403

Secondary Outcomes (8)

• Change from Baseline in Children's Communication Checklist-2 (CCC-2)

  Baseline to End of Treatment (Day 365) or Early Termination (ET)

• Change from Baseline in Social Responsiveness Scale-2 (SRS-2)

  Baseline to End of Treatment (Day 365) or ET

• Change from Baseline in Vineland Adaptive Behavior Scales, 3rd Edition (Vineland-3)

  Baseline to End of Treatment (Day 365) or ET

• Change from Baseline in National Institutes of Health (NIH) Toolbox Cognition Battery

  Baseline to End of Treatment (Day 365) or ET

• Change from Baseline in Repetitive Behavioral Scale - Revised (RBS-R)

  Baseline to End of Treatment (Day 365) or ET

Study Arms (1)

GWP42006

EXPERIMENTAL

Oral solution taken twice daily with food for 52 weeks.

Drug: GWP42006

Interventions

GWP42006 DRUG

Oral solution containing cannabidivarin 50 milligrams per milliliter (mg/mL) in sesame oil with anhydrous ethanol, sucralose, strawberry flavoring, and β-carotene.

Also known as: Cannabidivarin, CBDV

GWP42006

Eligibility Criteria

• Age: 4 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of Autism Spectrum Disorder (ASD) as defined by the Autism Diagnostic Observation Schedule, 2nd Edition and The Diagnostic and Statistical Manual of Mental Disorders, 5th Edition
• Intelligence quotient (IQ) of 40-120 (inclusive)
• Participant and their caregiver are English speaking.
• In the opinion of the investigator, the participant presents with ASD symptoms that warrant a therapeutic trial with GWP42006.

You may not qualify if:

• Known single gene neurogenetic disorder with high rates of epilepsy/autism (e.g., fragile X, tuberous sclerosis complex), structural brain lesion (prior stroke or hemispheric brain malformations), or history of any other epileptic encephalopathy, including infantile spasms, before the diagnosis of ASD
• More than 2 epileptic seizures per month within the 6 months prior to screening
• Initiation of a behavioral therapy program, new psychotropic medication, or therapeutic diet within the 2 months prior to screening, or plan to change or start any of the above during the trial
• Presence of a significant untreated medical problem (obstructive sleep apnoea, restless legs syndrome, gastroesophageal reflux disease, etc.) which may have significant impact on sleep study measures
• Behavioral management issues (e.g., self-injury, aggression) severe enough to be of safety concerns (to participant and/or staff)
• Clinically significant electrocardiogram abnormality or postural drop in systolic blood pressure at screening
• Any known or suspected hypersensitivity to cannabinoids or any of the excipients of GWP42006, such as sesame oil
• Known history of psychiatric disorder (defined as schizophrenia, bipolar disorder, or other psychiatric disease with a known history of hallucinations or delusions)
• History of any inborn errors of metabolism
• Significantly impaired hepatic function at screening
• Received an investigational product within the 3 months prior to screening
• Participant has been taking felbamate for less than 1 year prior to screening
• History of substance use disorders or positive drug of abuse dipstick test at screening (unless the positive result is due to a known concomitant medication)
• Currently using or has used recreational or medicinal cannabis or cannabinoid-based medications within the 3 months prior to screening and is unwilling to abstain for the duration of the trial
• Any history of suicidal behavior or any suicidal ideation within the month prior to or at screening

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

Study Sites (3)

Clinical Trial Site

Louisville, Kentucky, 40202, United States

Location

Clinical Trial Site

Lexington, Massachusetts, 02421, United States

Location

Clinical Trial Site

Columbus, Ohio, 43205, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

cannabidivarin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2019
• First Posted: February 21, 2019
• Study Start: January 7, 2020
• Primary Completion: May 26, 2020
• Study Completion: May 26, 2020
• Last Updated: September 2, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)