Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)
- Conditions
- Autism Spectrum Disorder
- Interventions
- Drug: Matched Placebo
- Registration Number
- NCT03202303
- Lead Sponsor
- Montefiore Medical Center
- Brief Summary
This trial aims to study the efficacy and safety of cannabidivarin (CBDV) in children with ASD.
- Detailed Description
There is a clear unmet need for new therapeutics to treat irritability in children with ASD that do not have the metabolic and weight adverse event profiles of the currently approved treatments. Cannabidivarin (CBDV) is a nonpsychoactive phytocannabinoid and a safe variant of Cannabidiol (CBD). It has no appreciable tetrahydrocannabinol (THC) \[less than 0.01%\], has been shown to have no impact on weight or metabolism, and improves both social and cognitive functioning in animal models of idiopathic and syndromal autism (Fragile X, Rett Syndrome, Angelman Syndrome). The CDC currently estimates 1 in 59 children have ASD. ASD is characterized by deficits in social communication, irritability, repetitive behaviors, impulsivity, temper tantrums, and high caregiver burden. Currently, the only FDA-approved medications for symptoms of ASD are aripiprazole and risperidone, both of which are indicated for irritability in pediatric ASD. These medications are effective but are associated with considerable side effects with long term treatment in this chronic developmental disorder, including weight gain, metabolic syndrome and the risk of type 2 diabetes, prolactin elevation and growth of breast tissue, extrapyramidal symptoms and the risk of tardive dyskinesia. The anticonvulsant divalproex sodium (valproate/VPA) also significantly reduces irritability and repetitive behaviors in individuals with ASD. Although VPA is efficacious for pediatric epilepsy and some symptoms of ASD, it also has significant side effects, including weight gain, sedation and nausea. CBDV, like VPA, is effective in the treatment of pediatric epilepsy, and ASD mouse models demonstrate potential mechanisms for treatment with CBDV, including potential therapeutic effects on repetitive behaviors, irritability, sociability, and quality of life, and the capacity to reduce inflammation. This study aims to examine the efficacy and safety of cannabidivarin (CBDV) with a primary aim of studying its effect on irritability in children with ASD.
STUDY DESIGN: This is a 12-week randomized, double-blind study of CBDV vs. placebo in 100 child and adolescent subjects aged 5 to 18 years with a diagnosis of ASD.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Cannabidivarin (CBDV) Cannabidivarin Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks Matched Placebo Matched Placebo Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks
- Primary Outcome Measures
Name Time Method Aberrant Behavior Checklist-Irritability Subscale (ABC-I) Change in ABC-I from Baseline to Week 12 (Change over 12 weeks) Change in ABC-I from Baseline to Endpoint
- Secondary Outcome Measures
Name Time Method Repetitive Behavior Scale-Revised (RBS-R) Change in RBS-R from Baseline to Week 12 (Change over 12 weeks) Change in RBS-R from Baseline to Endpoint
Montefiore Einstein Rigidity Scale-Revised (MERS-R) Change in MERS-R from Baseline to Week 12 (Change over 12 weeks) The MERS-R is designed to assess three domains of rigid behavior in children and adults with ASD: 1. Behavioral Rigidity (e.g., insistence on sameness, things must be done in his/her way, etc.) 2. Cognitive Rigidity (e.g., special interests, inflexible adherence to rules, etc.) 3. Protest (in response to deviation from rigidity; e.g., verbal objection, tantrum, physical aggression).
Aberrant Behavior Checklist-Social Withdrawal Subscale (ABC-SW) Change in ABC-SW from Baseline to Week 12 (Change over 12 weeks) Change in ABC-SW from Baseline to Endpoint
Pediatric Quality of Life Inventory (PedsQL) Family Impact Module Change in PedsQL from Baseline to Week 12 (Change over 12 weeks) Change in PedsQL from Baseline to Endpoint
Vineland Adaptive Behavior Scale-3 (Vineland 3) Change in Vineland-3 from Baseline to Week 12 (Change over 12 weeks) Change in Vineland-3 from Baseline to Endpoint
Clinical Global Impressions-Improvement (CGI-I) Change in CGI-I from Baseline to Week 12 (Change over 12 weeks) Change in CGI-I from Baseline to Endpoint
Trial Locations
- Locations (2)
New York University (NYU) Langone
🇺🇸New York, New York, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States