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Trial record 2 of 2 for:    cannabinoids | Autism Spectrum Disorder

Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

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ClinicalTrials.gov Identifier: NCT03202303
Recruitment Status : Recruiting
First Posted : June 28, 2017
Last Update Posted : February 18, 2021
Sponsor:
Collaborators:
United States Department of Defense
GW Pharmaceuticals Ltd.
Information provided by (Responsible Party):
Eric Hollander, Montefiore Medical Center

Tracking Information
First Submitted Date  ICMJE June 27, 2017
First Posted Date  ICMJE June 28, 2017
Last Update Posted Date February 18, 2021
Actual Study Start Date  ICMJE April 12, 2019
Estimated Primary Completion Date June 30, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 27, 2017)
Aberrant Behavior Checklist-Irritability Subscale (ABC-I) [ Time Frame: Change in ABC-I from Baseline to Week 12 (Change over 12 weeks) ]
Change in ABC-I from Baseline to Endpoint
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 21, 2020)
  • Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: Change in RBS-R from Baseline to Week 12 (Change over 12 weeks) ]
    Change in RBS-R from Baseline to Endpoint
  • Aberrant Behavior Checklist-Social Withdrawal Subscale (ABC-SW) [ Time Frame: Change in ABC-SW from Baseline to Week 12 (Change over 12 weeks) ]
    Change in ABC-SW from Baseline to Endpoint
  • Pediatric Quality of Life Inventory (PedsQL) Family Impact Module [ Time Frame: Change in PedsQL from Baseline to Week 12 (Change over 12 weeks) ]
    Change in PedsQL from Baseline to Endpoint
  • Vineland Adaptive Behavior Scale-3 (Vineland 3) [ Time Frame: Change in Vineland-3 from Baseline to Week 12 (Change over 12 weeks) ]
    Change in Vineland-3 from Baseline to Endpoint
  • Clinical Global Impressions-Improvement (CGI-I) [ Time Frame: Change in CGI-I from Baseline to Week 12 (Change over 12 weeks) ]
    Change in CGI-I from Baseline to Endpoint
  • Montefiore Einstein Rigidity Scale-Revised (MERS-R) [ Time Frame: Change in MERS-R from Baseline to Week 12 (Change over 12 weeks) ]
    The MERS-R is designed to assess three domains of rigid behavior in children and adults with ASD: 1. Behavioral Rigidity (e.g., insistence on sameness, things must be done in his/her way, etc.) 2. Cognitive Rigidity (e.g., special interests, inflexible adherence to rules, etc.) 3. Protest (in response to deviation from rigidity; e.g., verbal objection, tantrum, physical aggression).
Original Secondary Outcome Measures  ICMJE
 (submitted: June 27, 2017)
  • Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: Change in RBS-R from Baseline to Week 12 (Change over 12 weeks) ]
    Change in RBS-R from Baseline to Endpoint
  • Aberrant Behavior Checklist-Social Withdrawal Subscale (ABC-SW) [ Time Frame: Change in ABC-SW from Baseline to Week 12 (Change over 12 weeks) ]
    Change in ABC-SW from Baseline to Endpoint
  • Pediatric Quality of Life Inventory (PedsQL) Family Impact Module [ Time Frame: Change in PedsQL from Baseline to Week 12 (Change over 12 weeks) ]
    Change in PedsQL from Baseline to Endpoint
  • Vineland Adaptive Behavior Scale-II (VABS-II) [ Time Frame: Change in VABS-II from Baseline to Week 12 (Change over 12 weeks) ]
    Change in VABS-II from Baseline to Endpoint
  • Clinical Global Impressions-Improvement (CGI-I) [ Time Frame: Change in CGI-I from Baseline to Week 12 (Change over 12 weeks) ]
    Change in CGI-I from Baseline to Endpoint
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)
Official Title  ICMJE Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)
Brief Summary This trial aims to study the efficacy and safety of cannabidivarin (CBDV) in children with ASD.
Detailed Description

There is a clear unmet need for new therapeutics to treat irritability in children with ASD that do not have the metabolic and weight adverse event profiles of the currently approved treatments. Cannabidivarin (CBDV) is a nonpsychoactive phytocannabinoid and a safe variant of Cannabidiol (CBD). It has no appreciable tetrahydrocannabinol (THC) [less than 0.01%], has been shown to have no impact on weight or metabolism, and improves both social and cognitive functioning in animal models of idiopathic and syndromal autism (Fragile X, Rett Syndrome, Angelman Syndrome). The CDC currently estimates 1 in 59 children have ASD. ASD is characterized by deficits in social communication, irritability, repetitive behaviors, impulsivity, temper tantrums, and high caregiver burden. Currently, the only FDA-approved medications for symptoms of ASD are aripiprazole and risperidone, both of which are indicated for irritability in pediatric ASD. These medications are effective but are associated with considerable side effects with long term treatment in this chronic developmental disorder, including weight gain, metabolic syndrome and the risk of type 2 diabetes, prolactin elevation and growth of breast tissue, extrapyramidal symptoms and the risk of tardive dyskinesia. The anticonvulsant divalproex sodium (valproate/VPA) also significantly reduces irritability and repetitive behaviors in individuals with ASD. Although VPA is efficacious for pediatric epilepsy and some symptoms of ASD, it also has significant side effects, including weight gain, sedation and nausea. CBDV, like VPA, is effective in the treatment of pediatric epilepsy, and ASD mouse models demonstrate potential mechanisms for treatment with CBDV, including potential therapeutic effects on repetitive behaviors, irritability, sociability, and quality of life, and the capacity to reduce inflammation. This study aims to examine the efficacy and safety of cannabidivarin (CBDV) with a primary aim of studying its effect on irritability in children with ASD.

STUDY DESIGN: This is a 12-week randomized, double-blind study of CBDV vs. placebo in 100 child and adolescent subjects aged 5 to 18 years with a diagnosis of ASD.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Phase 2, 12-week double-blind, randomized, placebo-controlled trial
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
Double-Blind
Primary Purpose: Treatment
Condition  ICMJE Autism Spectrum Disorder
Intervention  ICMJE
  • Drug: Cannabidivarin
    Weight-based dosing of 10 mg/kg/day of CBDV
    Other Name: CBDV
  • Drug: Matched Placebo
    Weight-based dosing of 10 mg/kg/day of placebo
    Other Name: Placebo
Study Arms  ICMJE
  • Experimental: Cannabidivarin (CBDV)
    Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks
    Intervention: Drug: Cannabidivarin
  • Placebo Comparator: Matched Placebo
    Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks
    Intervention: Drug: Matched Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 27, 2017)
100
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 30, 2021
Estimated Primary Completion Date June 30, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria

  1. Male or Female pediatric outpatients aged between and including ages 5 to 18. Diagnosis of Autism Spectrum Disorder (ASD) confirmed by the ADOS-2 and DSM-5 criteria.*During special circumstances (e.g. COVID-19 pandemic) where the ADOS-2 cannot be performed due to site restrictions (e.g. mandatory use of face masks), eligibility can be confirmed using the Autism Diagnostic Interview, Revised (ADI-R)
  2. Aberrant Behavior Checklist (ABC) - Irritability Subscale (ABC-I) score of 18 or greater at screening visit.
  3. Social Responsiveness Scale (SRS) score of 66T or higher at screening visit.
  4. Clinical Global Impression Scale - Severity (CGI-S) score of 4 or higher at screening.
  5. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to randomization and for the duration of the study.
  6. Physical exam and laboratory results that are within normal range for individuals with ASD.
  7. 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.

Exclusion Criteria

  1. Exposure to any investigational agent in the 30 days prior to randomization.
  2. Prior chronic treatment with CBD, CBDV or an endocannabinoid 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. Recent history of drug abuse including marijuana/cannabis use in the past 3 months.
  5. Diagnosis of a known genetic disorder (ie. Prader-Willi Syndrome, Angelman Syndrome etc.).
  6. A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, Post-Traumatic Stress Disorder (PTSD) or Major Depressive Disorder (MDD). These patients will be excluded due to potential confounding results.
  7. 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.
  8. A known diagnosis of Rett Syndrome or Childhood Disintegrative Disorder, or marked sensory impairment such as deafness or blindness.
  9. Subjects who have had changes in allied health therapies, behavioral or educational interventions within four weeks prior to randomization other than those associated with school holidays.
  10. Subjects who have had changes in medications or medication doses within four weeks of randomization. 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
  11. Liver dysfunction manifested by > 2 X UNL values of AST or ALT
  12. ECG abnormality at baseline screening or clinically significant postural drop in systolic blood pressure at screening. If the initial screening ECG show 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 to 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.
  13. Known allergy to sesame oil
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Bonnie P Taylor, PhD 718-839-7530 botaylor@montefiore.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03202303
Other Study ID Numbers  ICMJE 2017-8538
AR160104 ( Other Grant/Funding Number: Department of Defense (DOD) )
G32379 ( Other Grant/Funding Number: GW Pharmaceuticals )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Responsible Party Eric Hollander, Montefiore Medical Center
Study Sponsor  ICMJE Eric Hollander
Collaborators  ICMJE
  • United States Department of Defense
  • GW Pharmaceuticals Ltd.
Investigators  ICMJE
Principal Investigator: Eric Hollander, MD Montefiore Medical Center/Albert Einstein College of Medicine
PRS Account Montefiore Medical Center
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP