This phase 2, randomized, double blind 12-week treatment trial of Cannabidivarin (CBDV) will assess the affect of CBDV on behavior in people with PWS.
The study is recruiting patients ages 5 to 30 years with PWS and will take place at the Montefiore Medical Center in Bronx, NY. This trial only requires 2 on-site visits.
Learn more about the study in this short recorded webinar.
Study Type: Randomized, double-blind, placebo-controlled
Eligible Ages: 5 to 30 years
Trial Id: NCT03848481
Duration: 12 weeks
Status: opening soon
Lead Sponsor: Montefiore Medical Center
Countries: United States
- Male or Female child 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 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.
- 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.
- Exposure to any investigational agent in the 30 days prior to randomization.
- Prior chronic treatment with CBD, CBDV or an endo-cannabinoid treatment.
- Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.
- Lifetime history of drug abuse including marijuana/cannabis use.
- 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).
- 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.
- Liver dysfunction manifested by > 3 X UNL values of AST or ALT
- 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
- 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.
This study is available at the following sites:
Montefiore Medical Center, Albert Einstein College of Medicine
Bronx, New York, United States, 10467
Principal Investigator: Eric Hollander, MD
Contact: Bonnie Taylor
If you are interested in learning more about this trial, please contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you. More information on this study is available on the study information page at clincaltrials.gov.