Clinical Trials
Cannabidivarin (CBDV) vs. Placebo in Children with Autism Spectrum Disorder (ASD)
Principal Investigator: Dr. Eric Hollander
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.
Funded by the DOD CDMRP Autism Research Program. 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
Sensory integration therapy
Principal Investigators: Dr. Sophie Molholm & Dr. Roseann Schaaf from Jefferson University
Additional Investigators: Dr. Elizabeth Ridgway, Dr. Emily A. Jones, Queens College, CUNY, and Dr. John Foxe, University of Rochester
Up to 90% of individuals with autism present with difficulties processing and integrating sensory information and these have been shown to be a significant factor impacting functional skills. Intervention for these sensory issues is one of the most highly requested treatments. We have showed that children with autism and sensory issues who were randomized to a 30-session Sensory Integration Treatment (SIT) made significant gains in functional skills, showed reduced sensory-related maladaptive behaviors, and improved their participation in daily activities compared to controls. Now that we have evidence in support of SIT for improvement of functional skills in this group of children with sensory issues, we seek to compare the effectiveness of SIT to focused behavioral strategies on improvement of functional skills for children with autism and sensory issues ages 6-9.5 years of age. After a comprehensive phenotypic assessment, eligible participants will be randomized to 30 one-hour sessions of SIT, ABA or No Treatment (NT). Outcome assessment, which includes behavioral and electrophysiological measures, will occur at baseline, post treatment and at 12 weeks following treatment to assess sustainability. The specific aims of the study are to: 1) compare 30 session SIT to the same dosage of ABA to evaluate immediate and sustained changes in functional skills and maladaptive behaviors; 2) to assess whether the treatment regimen has differential effects on sensory processing and multisensory integration functions; and 3) to determine whether autism severity, cognitive level or severity of sensory symptoms moderate intervention outcomes.
Gabaergic modulation to treat social communication deficits and restricted and repetitive behaviors in autism (A Phase II Multicenter, Randomized, Double-Blind Study)
Principal Investigator: Dr. Eric Hollander
This project evaluates 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). 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).
Funded by Roche Pharmaceuticals. 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).
Long-term Antipsychotic Pediatric Safety Trial (LAPS)
Principal Investigator: Dr. Eric Hollander
The purpose of this study is to evaluate the long-term pathologic weight changes associated with multi-year risperidone or aripiprazole therapy in 3 -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.
Funded by the Kennedy Shriver National Institute of Child Health and Human Development (NICHD). 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.