Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

NCT03202303 | Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: 2017-8538AR160104G32379
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 2

Brief Summary

This trial aims to study the efficacy and safety of cannabidivarin (CBDV) in children with ASD.

Conditions

Autism Spectrum Disorder

Keywords

Autism; Irritability; Cannabinoids

Outcome Measures

Primary Outcomes (1)

• Aberrant Behavior Checklist-Irritability (ABC-I) Subscale

  Change in aberrant behavior from baseline will be assessed using the irritability subscale of the ABC. The ABC is an informative rating instrument that was empirically derived by principal component analysis to measure behavior in those with developmental disability and autism spectrum disorder (ASD). The ABC-I will be completed by any adult who knows the patient well, such as a parent/caregiver. The ABC-I subscale consists of 15 items that address the presence of aggression, tantrums, and/or self-injury. Scores for each item on the subscale range from 0 (no problem at all) to 3 (problem is severe in degree), yielding an overall possible score for the subscale from 0-45, such that higher scores are indicative of increased severity of irritability. Scores will be summarized by study arm using basic descriptive statistics.

  Change from Baseline to Week 12 (Change over 12 weeks)

Secondary Outcomes (6)

• Repetitive Behavior Scale-Revised (RBS-R)

  Change in RBS-R from Baseline to Week 12 (Change over 12 weeks)

• Montefiore Einstein Rigidity Scale-Revised (MERS-R)

  Change in MERS-R from Baseline to Week 12 (Change over 12 weeks)

• Aberrant Behavior Checklist-Social Withdrawal (ABC-SW) Subscale

  Change in ABC-SW from Baseline to Week 12 (Change over 12 weeks)

• Pediatric Quality of Life Inventory (PedsQL) Family Impact Module

  Change in PedsQL from Baseline to Week 12 (Change over 12 weeks)

• Vineland Adaptive Behavior Scale, Third Edition (VABS-3)

  Change in VABS-3 from Baseline to Week 12 (Change over 12 weeks)

Study Arms (2)

Cannabidivarin (CBDV)

EXPERIMENTAL

Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks

Drug: Cannabidivarin

Matched Placebo

PLACEBO COMPARATOR

Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks

Drug: Matched Placebo

Interventions

Cannabidivarin DRUG

Weight-based dosing of 10 mg/kg/day of CBDV

Also known as: CBDV

Cannabidivarin (CBDV)

Matched Placebo DRUG

Weight-based dosing of 10 mg/kg/day of placebo

Also known as: Placebo

Matched Placebo

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or Female pediatric outpatients aged between and including ages 5 to 18. Diagnosis of Autism Spectrum Disorder (ASD) confirmed by the ADOS-2 and DSM-5 criteria.\*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), eligibility can be confirmed using the Autism Diagnostic Interview, Revised (ADI-R)
• Aberrant Behavior Checklist (ABC) - Irritability Subscale (ABC-I) score of 18 or greater at screening visit.
• Social Responsiveness Scale (SRS) score of 66T or higher at screening visit.
• Clinical Global Impression Scale - Severity (CGI-S) score of 4 or higher at screening.
• Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to randomization and for the duration of the study.
• Physical exam and laboratory results that are within normal range for individuals with ASD.
• Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior throughout the study. Child Assent will be obtained if the subject is 7 years of age or older and has the mental capacity to understand and sign a written assent form and/or give verbal assent.

You may not qualify if:

• Exposure to any investigational agent in the 30 days prior to randomization.
• Prior chronic treatment with CBD, CBDV or an endocannabinoid treatment.
• Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.
• Recent history of drug abuse including marijuana/cannabis use in the past 3 months.
• Diagnosis of a known genetic disorder (ie. Prader-Willi Syndrome, Angelman Syndrome etc.).
• A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, Post-Traumatic Stress Disorder (PTSD) or Major Depressive Disorder (MDD). These patients will be excluded due to potential confounding results.
• A medical condition that severely impacts the subject's ability to participate in the study, interferes with the conduct of the study, confounds interpretation of study results or endangers the subject's well-being.
• A known diagnosis of Rett Syndrome or Childhood Disintegrative Disorder, or marked sensory impairment such as deafness or blindness.
• Subjects who have had changes in allied health therapies, behavioral or educational interventions within four weeks prior to randomization other than those associated with school holidays.
• Subjects who have had changes in medications or medication doses within four weeks of randomization. Renal, pancreatic, or hematologic dysfunction as evidenced by values above upper limits of normal for BUN/creatinine, or values twice the upper limit of normal for serum lipase and amylase, platelets \<80,000 /mcL, or WBC\<3.0 103 /mcL
• Liver dysfunction manifested by \> 2 X UNL values of AST or ALT
• Known allergy to sesame oil

Sponsors & Collaborators

• Montefiore Medical Center: lead
• United States Department of Defense: collaborator
• GW Pharmaceuticals Ltd: collaborator
• Jazz Pharmaceuticals: collaborator

Study Sites (2)

New York University (NYU) Langone

New York, New York, 10016, United States

COMPLETED

Montefiore Medical Center

The Bronx, New York, 10467, United States

RECRUITING

Related Publications (1)

• Ferretti CJ, Cook BL, Mahant AM, Chu P, Zhao Y, Taylor BP, Herold BC, Hollander E. Cognitive inflexibility and immunome biomarkers in children with autism spectrum disorder. Neurosci Appl. 2024 May 4;3:104071. doi: 10.1016/j.nsa.2024.104071. eCollection 2024.

  PMID: 40656115 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

cannabidivarin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Eric Hollander, MD

  Montefiore Medical Center/Albert Einstein College of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Casara Ferretti, MS, MA

CONTACT

914-315-4236; casara.ferretti@einsteinmed.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-Blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Phase 2, 12-week double-blind, randomized, placebo-controlled trial

Study Record Dates

• First Submitted: June 27, 2017
• First Posted: June 28, 2017
• Study Start: April 12, 2019
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: October 22, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)