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Trial record 32 of 56 for:    Recruiting, Not yet recruiting, Available Studies | NOT (Use Disorders OR Marijuana Use OR Dependence OR Abuse OR Drug Use) | cannabinoids

CBDV vs Placebo in Children With Prader-Willi Syndrome (PWS)

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ClinicalTrials.gov Identifier: NCT03848481
Recruitment Status : Not yet recruiting
First Posted : February 20, 2019
Last Update Posted : April 2, 2019
Sponsor:
Collaborator:
Foundation for Prader-Willi Research
Information provided by (Responsible Party):
Eric Hollander, Montefiore Medical Center

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

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: CBDV Compound Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 26 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Cannabidivarin (CBDV) vs Placebo in Children With Prader-Willi Syndrome (PWS)
Estimated Study Start Date : September 2019
Estimated Primary Completion Date : October 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. Aberrant Behavior Checklist-Irritability Subscale (ABC-I) [ Time Frame: from Baseline to Week 12 ]
    Change in ABC-I score (from 0-45) From Baseline to Endpoint


Secondary Outcome Measures :
  1. Repetative Behavior Scale- Revised [ Time Frame: from Baseline to Week 12 ]
    Change in RBS-R score (a 44-item self-report questionnaire that is used to measure the breadth or repetitive behaviors)



Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03848481


Contacts
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Contact: Bonnie Taylor, PhD 718-839-7530 botaylor@montefiore.org
Contact: Vera Nezgovorova, MD 718-839-7510 vnezgovo@montefiore.org

Locations
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United States, New York
Montefiore Medical Center, Albert Einstein College of Medicine Not yet recruiting
Bronx, New York, United States, 10467
Contact: Vera Nezgovorova, MD    718-839-7510    vnezgovo@montefiore.org   
Contact: Bonnie Taylor, PhD    718-839-7530    botaylor@montefiore.org   
Principal Investigator: Eric Hollander, MD         
Sponsors and Collaborators
Montefiore Medical Center
Foundation for Prader-Willi Research
Investigators
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Principal Investigator: Eric Hollander, MD Montefiore Medical Center/Albert Einstein College of Medicine

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Responsible Party: Eric Hollander, Director, Autism and Obsessive Compulsive Spectrum Program, Anxiety and Depression Program, Montefiore Medical Center
ClinicalTrials.gov Identifier: NCT03848481     History of Changes
Other Study ID Numbers: 2019-9914
First Posted: February 20, 2019    Key Record Dates
Last Update Posted: April 2, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Eric Hollander, Montefiore Medical Center:
PWS
Prader- Willi Syndrome
Irritability
CBDV
Cannabinoids
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders