This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.
This clinical research trial aims to study the feasibility and safety of cannabidivarin (CBDV), 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 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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
14
CBDV is obtained from the Cannabis sativa L. plant and contains a negligible quantity (less than 0.2%) of THC
Placebo oral solution contains matching excipients.
Montefiore Medical Center, Albert Einstein College of Medicine
The Bronx, New York, United States
Aberrant Behavior Checklist-Irritability Subscale (ABC-I)
Change in the ABC-I score. The ABC-I is a well-characterized outcome that is accepted by the FDA for the purpose of labeling, and is one of the best and most validated outcome measures in the developmental disabilities. An inclusion cutoff of 18 or higher on the ABC-I at screening was chosen based on multiple medication trials with irritability as the primary target.
Time frame: from Baseline to Week 12
Repetative Behavior Scale- Revised (RBS-R)
Change in RBS-R score (a 44-item self-report questionnaire that is used to measure the breadth or repetitive behaviors)
Time frame: from Baseline to Week 12
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Change in the CY-BOCS score. The CY-BOCS is 10-item clinician measure designed to assess the severity of obsessive compulsive symptoms in children and adolescents over the previous week. It consists of four primary sections including Obsessions checklist, Severity items for Obsessions, Compulsions checklist and severity items for Compulsions and a set of investigational items. Improvement on this scale was associated with improvements in caregiver quality of life in our 8-week study of intranasal oxytocin.
Time frame: from Baseline to Week 12
Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
Change in HQ-CT score. The HQ-CT is a 9-item caregiver-reported measure of food-seeking behaviors that was used in the phase 3 beloranib trial.
Time frame: from Baseline to Week 12
ActiGraph GT9X-BT activity monitors
Change in Sleep Behaviors. Actigraph activity monitors are a well validated activity sleep monitoring device that has been utilized widely in clinical trials and health research, measuring sleep latency, total sleep time, and sleep efficiency.
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Time frame: from Baseline to Week 12
Clinical Global Impression Scale - Improvement (CGI-I)
The CGI-I will be used as a measure of improvement and contains a 7 point scale as follows: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse. The CGI-I is a clinician rated global measure of improvement and has been used as a measure in previous clinical psychopharmacology trials.
Time frame: from Baseline to Week 12
Caregiver Strain Questionnaire (CSQ)
The Caregiver Strain questionnaire is a 21-item self-report questionnaire that was developed to assess caregiver strain for families with a child living with an emotional or behavioral disorder.
Time frame: from Baseline to Week 12
Montefiore Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS)
The Montefiore-Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS) is designed to assess three domains of rigid behavior in individuals with PWS: 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., tantrum, irritability, arguing) The MERS-R-PWS is a clinician-rated scale and takes about 20 minutes to complete. It will be completed only for subjects who display rigid behaviors at baseline, week 4, week 8 and week 12.
Time frame: from Baseline to Week 12
Aberrant Behavior Checklist (ABC) subscales in lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech
The ABC is an informative rating instrument that was empirically derived by principal component analysis to measure behavior in those with developmental disability and ASD. It contains 58 items that resolve into 5 subscales. The subscales and the respective number of items are as follows: (a) irritability - 15 items, (b) lethargy/social withdrawal - 16 items, (c) stereotypic behavior - 7 items, (d) hyperactivity/noncompliance - 16 items, and (e) inappropriate speech - 4 items. The ABC was designed to be completed by any adult who knows the patient well, such as a parent/caregiver or teacher. This instrument measures behavior on a four-point severity scale where 0 = no problem at all, 1 = behavior is a problem but in a slight degree, 2 = problem is moderately serious, and 3 = problem is severe in degree.
Time frame: from Baseline to Week 12