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

NCT03848481 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: 2019-9914
• Study Type: interventional
• Target: 14
• 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)

• 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.

  from Baseline to Week 12

Secondary Outcomes (8)

• Repetative Behavior Scale- Revised (RBS-R)

  from Baseline to Week 12

• Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)

  from Baseline to Week 12

• Hyperphagia Questionnaire for Clinical Trials (HQ-CT)

  from Baseline to Week 12

• ActiGraph GT9X-BT activity monitors

  from Baseline to Week 12

• Clinical Global Impression Scale - Improvement (CGI-I)

  from Baseline to 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 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, PTSD or 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 BUN/creatinine, values twice the upper limit of normal for serum lipase and amylase, platelets \<80,000 /mcL, WBC\<3.0 103 /mcL. or \> 2 X UNL values of AST or 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

• Montefiore Medical Center: 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

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

Study Officials

• Eric Hollander, MD

  Montefiore Medical Center/Albert Einstein College of Medicine

  PRINCIPAL INVESTIGATOR

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

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: November 20, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share

Locations

US (1)