Index of Current Studies:
- Cannabidivarin (CBDV) vs. Placebo in Children with Autism Spectrum Disorder (ASD)
- Cannabidivarin (CBDV) vs. Placebo in Children and Adults up to age 30 with Prader-Willi Syndrome (PWS).
- Caregivers’ perspectives and usage of ketogenic diet for Autism Spectrum Disorder, Prader Willi Syndrome and Rett Syndrome (Survey)
- A Phase 3 Efficacy and Safety Study of Pitolisant in Patients with Prader-Willi Syndrome
- Intranasal Oxytocin vs. Placebo in Children with Prader-Willi Syndrome (PWS)
- A Phase 3, Randomized, Double-Blind, Dose-Ranging Evaluation of Transcutaneous Vagus Nerve Stimulation (tVNS) to Reduce Temper Outbursts in People with Prader-Willi Syndrome (PWS)
Details on Current Studies:
Cannabidivarin (CBDV) vs. Placebo in Children with Autism Spectrum Disorder (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
Goal: Cannabidivarin (CBDV) is a non-psychoactive 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.
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
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.
Caregivers’ perspectives and usage of ketogenic diet for Autism Spectrum Disorder, Prader Willi Syndrome and Rett Syndrome
Description of Project: We are conducting a survey to examine the use of the ketogenic diet in children and adults with Autism Spectrum Disorder, Prader Willi Syndrome, and/or Rett Syndrome. The ketogenic diet (KD) is gaining attention for its potential anti-inflammatory, antioxidant, and neuroprotective properties. Noteworthy mechanisms, including decreased neuronal excitability and improved mitochondrial function, make the KD an intriguing candidate. The rationale is grounded in limited but promising evidence from pilot studies, case reports, and trials showing positive impacts on core symptoms and weight control in patients with neurodevelopmental disorders.
Goal: The goal of this study is to examine perspectives and usage of the ketogenic diet among caregivers of children and adults with Autism Spectrum disorders, Prader Willi syndrome, and Rett syndrome. Some research has highlighted challenges with introducing and sustaining the ketogenic diet. Our study is hoping to further investigate this finding and identify potential barriers and facilitators for successful diet completion by surveying caregivers. You will also be asked to report on symptom severity through validated questionnaires.
A Phase 3 Efficacy and Safety Study of Pitolisant in Patients with Prader-Willi Syndrome
Sponsored by Harmony Biosciences, LLC.
Description of Project: This is a Phase 3, placebo-controlled, multicenter, global clinical study to assess efficacy and safety of pitolisant in patients with Prader-Willi Syndrome, ages >6 years (adults with Prader-Willi syndrome may be eligible as well).
Goal: The primary aim of this study is to evaluate the impact of pitolisant on excessive daytime sleepiness in patients with Prader-Willi syndrome. Other measures that are being assessed include the impact of pitolisant on irritability, disruptive behaviors, eating habits, and social behaviors. Pitolisant is a first-in-class compound that acts as a highly selective antagonist/inverse agonist of the H3R histamine receptor. Previous non-clinical studies have indicated that pitolisant may improve excessive daytime sleepiness in patients with Prader-Willi syndrome. Children with Prader-Willi syndrome who received pitolisant in Europe showed improved energy, alertness, engagement with surroundings, and more. Based on this data, this study is aiming to further evaluate pitolisant for the treatment of these symptoms in Prader-Willi syndrome.
Intranasal Oxytocin vs. Placebo in Children with Prader-Willi Syndrome (PWS)
Funded by the FDA
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.
A Phase 3, Randomized, Double-Blind, Dose-Ranging Evaluation of Transcutaneous Vagus Nerve Stimulation (tVNS) to Reduce Temper Outbursts in People with Prader-Willi Syndrome (PWS)
Funded by the Foundation for Prader-Willi Research (FPWR)
Description of Project: A Phase 3, Randomized, Double-Blind, Dose-Ranging, Evaluation of Transcutaneous Vagus Nerve Stimulation (tVNS) to Reduce Temper
Outbursts in People with Prader-Willi Syndrome (PWS). The main objectives of this trial are to determine the acceptability, safety, and effectiveness of tVNS to treat the severe behavior problems, specifically, temper outbursts, that affect people with PWS. https://clinicaltrials.gov/study/NCT06144645
Goal: The Primary Efficacy Objective: To determine the efficacy of tVNS in reducing temper outbursts in people with PWS aged 10 years and to 40 years.
Secondary Efficacy Objectives: To evaluate the potential benefit of tVNS beyond the temper outbursts, including the impact on hyperphagia, anxiousness, caregiver burden and quality of life.
For more information about any of our studies, please contact us using the information below:
Autism and Obsessive Compulsive Spectrum Program
Psychiatry Research Institute Montefiore-Einstein (PRIME)
1225 Morris Park Ave, Bronx, NY 10461
EinsteinAutismOCD@einsteinmed.edu
Phone: 718-839-7525
Recent/Other Studies:
Long-term Antipsychotic Pediatric Safety Trial (LAPS)
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.
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 - <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)
aV1ation: A Study to Investigate the Efficacy and Safety of Balovaptan in Participants with Autism Spectrum Disorder (ASD)
Sponsored by Hoffmann-La Roche
Description of Project: There is no pharmacological treatment available for the social and communication deficits in individuals with ASD and as a consequence there is a high unmet medical need for effective pharmacological therapies in the treatment of core symptoms in ASD. Balovaptan is a drug that blocks a hormone receptor (vasopressin 1a) in the brain that is linked to socialization, stress, anxiety, and aggression. Balovaptan has the potential to be the first pharmacotherapy to help improve core socialization and communication symptoms of Autism Spectrum Disorder, and it has been granted Breakthrough Therapy Designation by the FDA. https://clinicaltrials.gov/ct2/show/NCT02901431
Goal: This is a multi-center, randomized, double-blind, 24-week study to investigate the efficacy, safety, and pharmacokinetics of Balovaptan in children and adolescents aged 5-17 years with ASD who are high functioning (IQ >=70). After the participant completes the 24-week
double-blind phase of the study, he/she will have the option to receive balovaptan in open treatment for 52 weeks.
oRBiting: A Study to Evaluate Scales for Repetitive and Restricted Behaviors in Children, Adolescents, and Adults With Autism Spectrum Disorder (ASD)
Sponsored by Hoffmann-La Roche.
Description of Project: Restricted and repetitive behaviors (RRBs) are core diagnostic features of Autism Spectrum Disorder (ASD). RRBs constitute a broad range of behaviors, including simple motor stereotypies as well as more complex ritualized and rigid behaviors, compulsions, and restricted interests that vary in frequency, intensity, and duration. This study is observational and seeks to characterize different scales to measure repetitive and restricted behaviors in different ASD sub-populations over time.
https://clinicaltrials.gov/ct2/show/NCT03611075
Goal: This is a 12-week study that includes 4 in-person visits to our program. The participant will also receive a smart watch and a smartphone. The smart watch will be used to monitor repetitive movements that the participant may exhibit. The smartphone will be used at home to play games that assess cognition, social skills and speech.
