Research

Index of Current Studies:

1. Cannabidivarin (CBDV) vs. Placebo in Children with Autism Spectrum Disorder (ASD) 
2. Intranasal Oxytocin vs. Placebo in Children with Prader-Willi Syndrome (PWS) 
3. Cannabidivarin (CBDV) vs. Placebo in Children and Adults up to age 30 with Prader-Willi Syndrome (PWS). 
4. Long-term Antipsychotic Pediatric Safety Trial (LAPS) 
5. BP41316: A Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety and Tolerability of R07017773 in Participants Aged 15 to 45 Years with Autism Spectrum Disorder.

Details on Current Studies:

Cannabidivarin (CBDV) vs. Placebo in Children with Autism Spectrum Disorder (ASD)
Funded by the DOD CDMRP Autism Research Program.
April 12, 2019 - June 30, 2021  

Description of Project: 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. https://clinicaltrials.gov/ct2/show/NCT03202303 

Goal: 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). 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.

Intranasal Oxytocin vs. Placebo in Children with Prader-Willi Syndrome (PWS)
Funded by the FDA
April 11, 2018 to February 11, 2020.  

Description of Project: This is an 8-week double-blind, randomized study in 50 children with PWS aged 5-17. Participation involves 5 in-person visits to our program and two telephone-call visits. Travel expenses will be reimbursed to participating families. https://clinicaltrials.gov/ct2/show/NCT03197662 

Goal: Oxytocin has been implicated in the pathophysiology of PWS and there have been small studies of intranasal oxytocin (IN-OXT) in this population. To date, however, studies have not been adequately powered to detect significance in target symptoms of hyperphagia and associated symptoms of individuals with PWS. The primary goal of this study is to examine the safety and efficacy of IN-OXT on hyperphagia, as measured by the Hyperphagia Questionnaire-Clinical Trails, from baseline to week 8.

Cannabidivarin (CBDV) vs. Placebo in Children and Adults up to age 30 with Prader-Willi Syndrome (PWS).
Funded by the Foundation for Prader-Willi Research (FPWR) and GW Pharmaceuticals
June 1, 2020 to June 1, 2021.  

Description of Project: This is a single-site 12-week double-blind placebo-controlled pilot study of CBDV manufactured by GW Pharmaceuticals as GWP42006 in 36 children and young adults aged 5 to 30 diagnosed with PWS and have a high level of irritability (≥18 on the ABC-I). https://clinicaltrials.gov/ct2/show/NCT03848481 

Goal: This clinical research trial aims to study the efficacy and safety of cannabidivarin (CBDV), a naturally occurring homolog of the phytocannabinoid cannabidiol (CBD) in children and young adults with Prader-Willi Syndrome (PWS). CBDV has effects independent of CB1 and CB2 receptor activation and a good safety profile. This proposal addresses the

Foundation for Prader Willi Research’s five year PWS Research Plan: Program 1, Clinical Care Research: seeks to evaluate treatments that aim to reduce behavioral symptoms, such as irritability, in order to improve the quality of life of both the individual with PWS and their families. GW Pharmaceuticals will provide the CBDV drug and matching placebo and additional funding to the site.

Long-term Antipsychotic Pediatric Safety Trial (LAPS)
Funded by the Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
January 10, 2019 to September 10, 2021.

Description of Project: This is a multi-center, observational study that will include approximately 350 children being treated with risperidone and 350 children being treated with aripiprazole. The participants’ personal physicians will continue to prescribe their medications over the course of the study. Assessments will occur every six months at in-person visits, for 2 years. https://clinicaltrials.gov/ct2/show/NCT03522168 

Goal: The purpose of this study is to evaluate the long-term pathologic weight changes associated with multi-year risperidone or aripiprazole therapy in 3 - &glt;18-year-old children, who have varying durations of prior antipsychotic drug exposure. This is critical because children appear to have greater vulnerability to antipsychotic-associated weight gain than adults, and obesity has significant effects on morbidity and mortality.

BP41316: A Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety and Tolerability of R07017773 in Participants Aged 15 to 45 Years with Autism Spectrum Disorder.
Sponsored by Roche Pharmaceuticals
January 2020 to January 2023  

Description of Project: This study will investigate the efficacy, safety and tolerability, and pharmacokinetics of RO7017773 in participants aged 15 to 45 years with a diagnosis of ASD and Wechsler Abbreviated Scale of Intelligence (WASI-II) score ≥50. RO7017773 is a selective gamma aminobutyric acid type A, alpha 5 (GABAA-a5) subunit-containing receptor positive allosteric modulator (PAM). RO7017773 is being developed for the treatment of the two core domains of ASD: social communication deficits and restricted and repetitive behaviors (RRBs). RO7017773 has the potential to normalize gamma-aminobutyric acid (GABA)-ergic signaling in key brain regions implicated in ASD without the side-effects of non-specific GABA modulators (e.g., benzodiazepines).

Goal: To evaluate the efficacy of 12-week treatment with RO7017773 compared with placebo in treating social communication deficits in participants with ASD. To evaluate the safety and tolerability of a 12-week treatment with RO7017773 in 15- to 45-year-old participants with ASD. To evaluate the efficacy of a 12-week treatment with RO7017773 compared to placebo on restricted and repetitive behaviors (RRBs)