CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
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|ClinicalTrials.gov Identifier: NCT03848481|
Recruitment Status : Recruiting
First Posted : February 20, 2019
Last Update Posted : December 24, 2020
|Condition or disease||Intervention/treatment||Phase|
|Prader-Willi Syndrome||Drug: CBDV Compound Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||26 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Cannabidivarin (CBDV) vs. Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)|
|Actual Study Start Date :||November 23, 2020|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
Experimental: Cannabidivarin (CBDV)
Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks
Drug: CBDV Compound
CBDV is obtained from the Cannabis sativa L. plant and contains a negligible quantity (less than 0.2%) of THC
Placebo Comparator: Matched Placebo
Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks
Placebo oral solution contains matching excipients.
- Aberrant Behavior Checklist-Irritability Subscale (ABC-I) [ Time Frame: from Baseline to Week 12 ]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.
- Repetative Behavior Scale- Revised (RBS-R) [ Time Frame: from Baseline to Week 12 ]Change in RBS-R score (a 44-item self-report questionnaire that is used to measure the breadth or repetitive behaviors)
- Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: from Baseline to Week 12 ]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.
- Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [ Time Frame: from Baseline to Week 12 ]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.
- ActiGraph GT9X-BT activity monitors [ Time Frame: from Baseline to Week 12 ]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.
- Clinical Global Impression Scale - Improvement (CGI-I) [ Time Frame: from Baseline to Week 12 ]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.
- Caregiver Strain Questionnaire (CSQ) [ Time Frame: from Baseline to Week 12 ]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.
- Montefiore Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS) [ Time Frame: from Baseline to Week 12 ]
The Montefiore-Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS) is designed to assess three domains of rigid behavior in individuals with PWS:
- Behavioral Rigidity (e.g., Insistence on sameness, things must be done in his/her way, etc.)
- Cognitive Rigidity (e.g., Special interests, inflexible adherence to rules, etc.)
- 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.
- Aberrant Behavior Checklist (ABC) subscales in lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech [ Time Frame: from Baseline to Week 12 ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03848481
|Contact: Bonnie Taylor, PhDfirstname.lastname@example.org|
|Contact: Vera Nezgovorova, MDemail@example.com|
|United States, New York|
|Montefiore Medical Center, Albert Einstein College of Medicine||Recruiting|
|Bronx, New York, United States, 10467|
|Contact: Vera Nezgovorova, MD 718-839-7510 firstname.lastname@example.org|
|Contact: Bonnie Taylor, PhD 718-839-7530 email@example.com|
|Principal Investigator: Eric Hollander, MD|
|Principal Investigator:||Eric Hollander, MD||Montefiore Medical Center/Albert Einstein College of Medicine|