Status:
COMPLETED
CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
Lead Sponsor:
Montefiore Medical Center
Collaborating Sponsors:
Foundation for Prader-Willi Research
GW Pharmaceuticals Ltd
Conditions:
Prader-Willi Syndrome
Eligibility:
All Genders
5-30 years
Phase:
PHASE2
Brief Summary
This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.
Detailed Description
This clinical research trial aims to study the feasibility and safety of cannabidivarin (CBDV), in children and young adults with Prader-Willi Syndrome (PWS). CBDV has effects independent of CB1 and C...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- 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.
- Exclusion Criteria
- 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.
- 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 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.
Exclusion
Key Trial Info
Start Date :
November 23 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 31 2024
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT03848481
Start Date
November 23 2020
End Date
October 31 2024
Last Update
November 20 2024
Active Locations (1)
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1
Montefiore Medical Center, Albert Einstein College of Medicine
The Bronx, New York, United States, 10467