Purpose

This trial aims to study the efficacy and safety of cannabidivarin (CBDV) as a treatment for children with PWS.

Condition

Eligibility

Eligible Ages
Between 5 Years and 30 Years
Eligible Genders
All
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  1. Male or Female child outpatients aged 5 to 30 years.
  2. Diagnosis of PWS confirmed by genetic testing and patient medical records and history.
  3. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
  4. Have a physical exam and laboratory results that are within the norms for PWS5. Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child'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.
  5. Score on the Clinical Global Impression Scale Severity (CGI-S) ≥ 4 (moderate severity) at baseline.
  6. Score of ≥18 on the Aberrant Behavior Checklist-Irritability (ABC-I) at baseline.
  7. Agree not to drive or operate machinery.

Exclusion Criteria

  1. Exposure to any investigational agent in the 30 days prior to randomization.
  2. Prior chronic treatment with CBD, CBDV or an endo-cannabinoid treatment.
  3. Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.
  4. Lifetime history of drug abuse including marijuana/cannabis use.
  5. 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.
  6. 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).
  7. Known or suspected allergy to CBDV or excipients used in the formulation (i.e. sesame).
  8. Renal, pancreatic, or hematologic dysfunction as evidenced by values above upper limits of normal for BUN/creatinine, or values twice the upper limit of normal for serum lipase and amylase, platelets <80,000 /mcL, or WBC<3.0 103 /mcL.
  9. Liver dysfunction manifested by > 3 X UNL values of AST or ALT
  10. ECG abnormality at baseline screening or clinically significant postural drop in systolic blood pressure at screening. If the initial screening ECG shows a QTcB of greater than 460 msec, then 2 additional ECGs will be conducted in the same sitting, 5 minutes apart. If not recognized at screening, then a full triplicate repeat showing an average QTcB of 460 msec or less to meet all inclusion/exclusion criteria
  11. 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 serum 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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
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
  • Drug: Placebo
    Placebo oral solution contains matching excipients.

Recruiting Locations

Montefiore Medical Center, Albert Einstein College of Medicine
Bronx, New York 10467
Contact:
Vera Nezgovorova, MD
718-839-7510
vnezgovo@montefiore.org

More Details

Status
Recruiting
Sponsor
Montefiore Medical Center

Study Contact

Bonnie Taylor, PhD
718-839-7530
botaylor@montefiore.org

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.