CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)

NCT03848481 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019-9914
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.

Conditions

Prader-Willi Syndrome

Keywords

PWS; Prader- Willi Syndrome; Irritability; CBDV; Cannabinoids

Outcome Measures

Primary Outcomes (1)

• Irritability Based on Aberrant Behavior Checklist-Irritability (ABC-I) Subscale

  Irritability will be assessed using the Aberrant Behavior Checklist-Irritability Subscale (ABC-I). 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. The ABC-Irritability subscale consists of 15 questions that address the presence of irritability, aggression, tantrums and/or self-injury. Each item is rated on a scale ranging from 0 ("Not at all a problem") to 3 ("Severe problem"), resulting in a total score range of 0-45, such that higher ABC-I scores are indicative of more severe behavioral problems. Subjects must score an 18 or higher at screening to be included in the study. ABC-I scores for Week 4, Week 8, and Week 12 are summarized in the table by study arm using descriptive statistics. Baseline results for this outcome can be found in the Baseline Characteristics module.

  Baseline, Week 4, Week 8, Week 12

Secondary Outcomes (8)

• Repetitive Behavior Based on the Repetitive Behavior Scale-Revised (RBS-R).

  Baseline, Week 4, Week 8, Week 12

• Repetitive Behaviors Based on Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)

  Baseline, Week 4, Week 8, Week 12

• Hyperphagia

  Baseline, Week 4, Week 8, Week 12

• Global Functioning

  Week 4, Week 8, Week 12

• Caregiver Strain

  Baseline, Week 4, Week 8, Week 12

Study Arms (2)

Cannabidivarin (CBDV)

EXPERIMENTAL

Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks

Drug: CBDV Compound

Matched Placebo

PLACEBO COMPARATOR

Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks

Drug: Placebo

Interventions

CBDV Compound DRUG

CBDV is obtained from the Cannabis sativa L. plant and contains a negligible quantity (less than 0.2%) of Tetrahydrocannabinol (THC).

Cannabidivarin (CBDV)

Placebo DRUG

Placebo oral solution contains matching excipients.

Matched Placebo

Eligibility Criteria

• Age: 5 Years - 30 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or Female outpatients aged 5 to 30 years.
• Diagnosis of PWS confirmed by genetic testing and patient medical records and history.
• Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
• Have a physical exam and laboratory results that are within the norms for PWS
• Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the patient's development and behavior throughout the study. Child Assent will be obtained if the subject is 7 years of age or older and has the mental capacity to understand and sign a written assent form and/or give verbal assent.
• Score on the Clinical Global Impression Scale Severity (CGI-S) ≥ 4 (moderate severity) at baseline.
• Score of ≥18 on the Aberrant Behavior Checklist-Irritability (ABC-I) at baseline.
• Agree not to drive or operate machinery.

You may not qualify if:

• Exposure to any investigational agent in the 30 days prior to randomization.
• Prior chronic treatment with CBD or CBDV.
• Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.
• History of Drug Abuse Disorder including Cannabis Use Disorder
• A primary psychiatric diagnosis other than PWS, including bipolar disorder, psychosis, schizophrenia, Post-Traumatic Stress Disorder (PTSD), or Major Depressive Disorder (MDD). These patients will be excluded due to potential confounding results.
• A medical condition that severely impacts the subject's ability to participate in the study, interferes with the conduct of the study, confounds interpretation of study results or endangers the subject's well-being (including but not limited to hepatic or renal impairment and cardiovascular disease).
• Known or suspected allergy to CBDV or excipients used in the formulation (i.e. sesame).
• Clinical indications of renal, pancreatic, or hematologic dysfunction as evidenced by values above upper limits of normal for Blood Urea Nitrogen (BUN)/creatinine, values twice the upper limit of normal for serum lipase and amylase, platelets \<80,000 /microliter, white blood cell (WBC)\<3.0 103 /microliter or \> 2x Upper Limit of Normal (UNL) values of aspartate aminotransferase (AST) or alanine aminotransferase (ALT).
• Female subjects who are pregnant will be excluded from the study. If a female subject is able to become pregnant, she will be given a pregnancy test before entry into the study. Female subjects will be informed not become pregnant while taking CBDV. Female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if they become pregnant during the study.

Sponsors & Collaborators

• Eric Hollander: lead
• Foundation for Prader-Willi Research: collaborator
• GW Pharmaceuticals Ltd: collaborator

Study Sites (1)

Montefiore Medical Center, Albert Einstein College of Medicine

The Bronx, New York, 10467, United States

Location

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Related Links

• Research FfP-W. PWS Research Plan 2017-2021. 2017

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

cannabidivarin

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Limitations and Caveats

The study was terminated early and powered statistical analyses were not conducted with exception of effect size determinations for the MERS-R-PWS outcome measure notwithstanding.

Results Point of Contact

• Title: Eric Hollander
• Organization: Albert Einstein College of Medicine

Eholland@montefiore.org

718-839-7516

Study Officials

• Eric Hollander, MD

  Montefiore Medical Center/Albert Einstein College of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director, Autism and Obsessive Compulsive Spectrum Program, Anxiety and Depression Program

Study Record Dates

• First Submitted: February 19, 2019
• First Posted: February 20, 2019
• Study Start: November 23, 2020
• Primary Completion: October 31, 2024
• Study Completion: October 31, 2024
• Last Updated: May 7, 2026
• Results First Posted: May 7, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Following publication for an indefinite period.
• Access Criteria: Contact Principal Investigator for availability of IPD data.

Locations

US (1)