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Cannabidiol for ASD Open Trial

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ClinicalTrials.gov Identifier: NCT03900923
Recruitment Status : Recruiting
First Posted : April 3, 2019
Last Update Posted : June 4, 2019
Sponsor:
Information provided by (Responsible Party):
NYU Langone Health

Tracking Information
First Submitted Date  ICMJE April 1, 2019
First Posted Date  ICMJE April 3, 2019
Last Update Posted Date June 4, 2019
Actual Study Start Date  ICMJE March 12, 2019
Estimated Primary Completion Date January 31, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 15, 2019)
Clinical Global Impression Scale -Improvement (CGI-I) [ Time Frame: Baseline, 6 Weeks ]
This is a 7-point scale measuring symptom change from baseline.
Original Primary Outcome Measures  ICMJE
 (submitted: April 2, 2019)
Clinical Global Impression Scale -Improvement (CGI-I) [ Time Frame: Baseline, 6 Weeks ]
7-point scale where: 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse,and 7=very much worse.
Change History Complete list of historical versions of study NCT03900923 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 17, 2019)
  • Brief Observation of Social Communication - Change (BOSCC) [ Time Frame: Baseline, Week 6 ]
    The BOSCC consists of 16 items rated across two 5-minute segments; 10 items address social communication abilities, three items address RRBs, and three items address symptoms that often co-occur with ASD (e.g., hyperactive, disruptive, and anxious behaviors). Each item is coded on a 6-point scale using a decision tree, which relates to information about specific behaviors. BOSCC coding is completed using two 5-minute videos of interactions.
  • Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: Baseline, Week 6 ]
    The RBS-R consists of six subscales covering a range of RRB subtypes including: Stereotyped Behavior, Self-injurious Behavior, Compulsive Behavior, Routine Behavior, Sameness Behavior, and Restricted Behavior. The presence and frequency of behaviors are rated on a 4-point scale (ranging from 0=behavior does not occur to 3=behavior occurs and is a severe problem).
  • Social Responsiveness Scale, 2nd Edition (SRS-2), School-Age Form [ Time Frame: Baseline, Week 6 ]
    Five domains are assessed including: Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests and Repetitive Behavior. Items are scored on a 4-point scale (ranging from 1=not true to 4=almost always true).
  • Aberrant Behavior Checklist (ABC) - Irritability and Social Withdrawal Subscales [ Time Frame: Baseline, Week 6 ]
    The subscales of the ABC include: Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactivity/Noncompliance, and Inappropriate Speech. Items are rated on a 4-point scale (ranging from 0=no problem at all to 3 = problem is severe in degree).
  • Anxiety, Depression and Mood Scale (ADAMS) [ Time Frame: Baseline, Week 6 ]
    28 symptom items that resolve into five subscales labeled: Manic/Hyperactive Behavior, Depressed Mood, Social Avoidance, General Anxiety, and Compulsive Behavior. Items are rated on 4-point scale ranging from 0=not a problem to 3=severe problem.
  • Children's Communication Checklist-2 (CCC-2) [ Time Frame: Baseline, Week 6 ]
    A caregiver-report measure of language designed for use with children ages 4-16 years old who speak in sentences. The measures consists of 70 items making up 10 domains: Speech, Syntax, Semantics, Coherence, Inappropriate Initiation, Scripted Stereotyped Language, Use of Context, Nonverbal Communication, Social Relations, and Interests.
  • Screen for Child Anxiety Related Disorders (SCARED), Parent Version [ Time Frame: Baseline, Week 6 ]
    The SCARED, Parent Version is a parent report measure developed to measure child anxiety symptoms. Subscales include Somatic Symptoms/Panic Disorder, Generalized Anxiety Disorder, Separation Anxiety, Social Phobia, and School Phobia. Items are rated on a 3-point scale ranging from 0=not true or hardly ever true to 2=very true or often true.
  • Sleep Disturbance Scale for Children (SDSC) [ Time Frame: Baseline, Week 6 ]
    Six subscales including Disorders of Initiating and Maintaining Sleep, Sleep Breathing Disorders, Disorders of Arousal, Sleep Wake Transition Disorders, Disorders of Excessive Somnolence, and Sleep Hyperhydrosis. Items are rated on 5-point scale where 1=never and 5=always (daily). Subscale scores sum to equal a total score
  • 10. Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) [ Time Frame: Baseline, Week 6 ]
    Parent/Caregiver Form: Used to measure adaptive functioning. The parent/caregiver is given a questionnaire with three core domains (Communication, Daily Living Skills, and Socialization), and two optional domains (Motor Skills and Maladaptive Behavior); items are rated on a 3-point scale (0=never; 1=sometimes; 2=usually or often). The core domains sum to a total Adaptive Behavior Composite.
  • Clinical Global Impression-Severity(CGI-S): [ Time Frame: Screening, Baseline, Week 6 ]
    Reflects clinicians impression of severity of illness on a 7 point scale ranging from 1=not at all to 7=among the most extremely ill.
  • Pediatric Quality of Life Inventory (PedsQL) Family Impact Module [ Time Frame: Baseline, Week 6 ]
    Self-report forms to measure caregiver and family functioning. Consists of six scales measuring caregiver functioning (Physical Functioning, Emotional Functioning, Social Functioning, Cognitive Functioning, Communication, Worry) and two scales measuring family functioning (Daily Activities and Family Relationships). A 5-point response scale is utilized and items are reverse scored and transformed to a 0-100 scale, so the higher scores indicate better functioning (less negative impact).
Original Secondary Outcome Measures  ICMJE
 (submitted: April 2, 2019)
  • Brief Observation of Social Communication - Change (BOSCC) [ Time Frame: Baseline, Week 6 ]
    The BOSCC consists of 16 items rated across two 5-minute segments; 10 items address social communication abilities, three items address RRBs, and three items address symptoms that often co-occur with ASD (e.g., hyperactive, disruptive, and anxious behaviors). Each item is coded on a 6-point scale using a decision tree, which brelates to information about specific behaviors. BOSCC coding is completed using two 5-minute videos of interactions.
  • Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: Baseline, Week 6 ]
    RBS-R consists of six subscales covering a range of RRB subtypes including: Stereotyped Behavior, Self-injurious Behavior, Compulsive Behavior, Routine Behavior, Sameness Behavior, and Restricted Behavior. The presence and frequency of behaviors are rated on a 4-point scale (ranging from 0=behavior does not occur to 3=behavior occurs and is a severe problem)
  • Social Responsiveness Scale, 2nd Edition (SRS-2), School-Age Form [ Time Frame: Baseline, Week 6 ]
    Five domains are assessed including: Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests and Repetitive Behavior. Items are scored on a 4-point scale (ranging from 1=not true to 4=almost always true). Excellent psychometric properties have been previously established for the SRS for use in children and adults
  • Aberrant Behavior Checklist (ABC) - Irritability and Social Withdrawal Subscales [ Time Frame: Baseline, Week 6 ]
    The subscales of the ABC include: Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactivity/Noncompliance, and Inappropriate Speech. Items are rated on a 4-point scale (ranging from 0=no problem at all to 3 = problem is severe in degree)
  • Anxiety, Depression and Mood Scale (ADAMS) [ Time Frame: Baseline, Week 6 ]
    Contains 55 symptom items that resolve into five subscales labeled: Manic/Hyperactive Behavior, Depressed Mood, Social Avoidance, General Anxiety, and Compulsive Behavior
  • Children's Communication Checklist-2 (CCC-2) [ Time Frame: Baseline, Week 6 ]
    A caregiver-report measure of language designed for use with children ages 4-16 years old who speak in sentences. The measures consists of 70 items 10 domains: Speech, Syntax,Semantics,Coherence, Initiation, Scripted Language, Context, Nonverbal Communication, Social Relations, and Interests. The CCC-2 takes approximately 10-15 minutes to complete. Psychometrics are strong, with test- retest reliability estimates between 0.86-0.96
  • Computerized Kiddie Schedule for Affective Disorders and Schizophrenia (KSADS-COMP) [ Time Frame: Baseline, Week 6 ]
    The KSADS-COMP is a DSM-5 based instrument administered by a secure web-based computer program to parents/guardians derived from the KSADS (83). The responses and the algorithmically determined presumptive diagnoses are then immediately provided for follow- u p and clarification, as needed, by a licensed clinician.
  • Screen for Child Anxiety Related Disorders (SCARED), Parent Version [ Time Frame: Baseline, Week 6 ]
    The SCARED, Parent Version is a parent report measure with strong psychometrics developed to measure child anxiety symptoms. Subscales include Somatic Symptoms/Panic Disorder, Generalized Anxiety Disorder, Separation Anxiety, Social Phobia, and School Phobia.
  • Sleep Disturbance Scale for Children [ Time Frame: Baseline, Week 6 ]
    Six subscales including Disorders of Initiating and Maintaining Sleep, Sleep Breathing Disorders, Disorders of Arousal, Sleep Wake Transition Disorders, Disorders of Excessive Somnolence, and Sleep Hyperhydrosis
  • Vineland Adaptive BehaviorScales, Third Edition (Vineland-3), [ Time Frame: Baseline, Week 6 ]
    Used to measure adaptive functioning across the areas of communicatio, daily living skills, socialization, motor skills and maladaptive behavior. The parent/caregiver is given a questionnaire with three core domains (communication, daily living skills, and socialization), and two optional domains (Motor Skills and Maladaptive Behavior). The core domains sum to a total Adaptive Behavior Composite.
  • Clinical Global Impression-Severity(CGI-S): [ Time Frame: Screening, Baseline, Week 6 ]
    Reflects clinicians impression of severity of illness on a 7 point scale ranging from 1=not at all to 7=among the most extremely ill.
  • Pediatric Quality of Life Inventory (PedsQL) Family Impact Module [ Time Frame: Baseline, Week 6 ]
    Self report forms to measure caregiver and family functioning. Consists of 6 scales measuring caregiver functioning (physical functioning, emotional functioning, social functioning, cognitive functioning, communication, Worry) and two scales measuring family functioning (Daily Activities and Family Relationships). 5-point response scale is utilized and items are reverse scored and transformed to a 0-100 scale, so the higher scores indicate better functioning (less negative impact).
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Cannabidiol for ASD Open Trial
Official Title  ICMJE A Phase 2 Study of Cannabidiol as a New Treatment for Autism Spectrum Disorder in Children and Adolescents
Brief Summary

This is a 6-week open trial to identify the optimal dosing of cannabidiol (CBD) in youth with autism spectrum disorder (ASD) and to identify primary and secondary outcomes for future controlled studies. This study will evaluate change in symptoms commonly associated with ASD, as evidence suggests that CBD may be effective in addressing difficulties such as irritability and anxiety, while maintaining a benign adverse effect (AE) profile in children and adolescents.

30 male and female participants with ASD between the ages of 7 and 17 years old will be recruited. Participants will have fluent speech and an estimated IQ greater than or equal to 80. Study intervention will be 98% pure CBD. The CBD will be Greenwich Biosciences, Inc.'s 100mg/mL oral solution, brand name EPIDIOLEX. A Bayesian optimal interval (BOIN) design will be used, such that participants will be assigned to cohorts of size 3 receiving doses of 3, 6, or 9 mg/kg/day, depending on the treatment response of participants in prior cohorts.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • ASD
  • Autism Spectrum Disorder
Intervention  ICMJE Drug: 98% pure CBD
A Bayesian optimal interval (BOIN) design will be used, such that participants will be assigned to cohorts of size 3 receiving doses of 3, 6, or 9 mg/kg/day, depending on the treatment response of participants in prior cohorts
Study Arms  ICMJE Experimental: 98% pure CBD
98% pure CBD. The CBD will be 100mg/mL oral solution provided by Greenwich Biosciences, Inc.
Intervention: Drug: 98% pure CBD
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: April 2, 2019)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 31, 2021
Estimated Primary Completion Date January 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male or female pediatric outpatients aged between and including 7 to 17.9 years old
  • Diagnosis of ASD confirmed by the ADOS-2 and DSM-5 criteria
  • SRS-2 Total T-score of 65 66 or higher
  • CGI-S score of 4 or higher
  • Physical exam and laboratory results that are within normal range for their age
  • Fluent speech
  • Estimated IQ of at least 80
  • Presence of a parent/legal guardian who is able to consent for their participation and complete assessments regarding the child's development and behavior throughout the study

Exclusion Criteria:

  • History of active seizure disorder or epilepsy; patients seizure free for >5 years off of antiepileptic drugs and other than uncomplicated febrile seizures are not excluded
  • History or current evidence of significantly impaired liver function
  • Exposure to any investigational agent in the 30 days prior to initiation of trial
  • Prior chronic treatment with CBD or other cannabinoid
  • Current use of medications metabolized primarily by CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP3A4, or CYP2D6 isoenzymes. Methylphenidate is not contraindicated.
  • History of drug abuse including marijuana/cannabis use in the past 3 months
  • Positive urine sample results from initial and confirmatory drug screening indicating presence of the following drugs: cannabinoids, opiates, methadone, cocaine, and PCP
  • Diagnosis of a known genetic disorder (e.g., Prader-Willi Syndrome, Angelman Syndrome, etc.).
  • Active suicidality (ideation and plan) is present
  • A current psychiatric diagnosis of bipolar disorder, major depressive disorder (MDD), psychosis, schizophrenia, or post-traumatic stress disorder (PTSD)
  • Pregnant or lactating patients or patients who will not agree to be abstinent or use contraception
  • 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
  • Diagnosis of Rett Syndrome or Childhood Disintegrative Disorder or marked sensory impairment such as deafness or blindness
  • Subjects who have had changes in allied health therapies, behavioral or educational interventions within 4 weeks prior to initiation of trial, other than those associated with school holidays
  • Subjects who have had changes in non-exclusionary psychotropic medications within 4 weeks of initiation of trial
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 7 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Paige Cervantes 646-754-7243 Paige.Cervantes@nyulangone.org
Contact: Greta Conlon 2124043770 greta.conlon@nyulangone.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03900923
Other Study ID Numbers  ICMJE 18-00250
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
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices).
Supporting Materials: Study Protocol
Supporting Materials: Informed Consent Form (ICF)
Time Frame: Proposals may be submitted up to 36 months following article publication. After 36 months the data will be available in the investigator's University's data warehouse but without investigator support other than deposited metadata. Information regarding submitting requests and accessing data may be found at (Link to be provided).
Access Criteria: Data will be available for any purpose.
Responsible Party NYU Langone Health
Study Sponsor  ICMJE NYU Langone Health
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Francisco Castellanos, MD New York Langone Health
PRS Account NYU Langone Health
Verification Date May 2019

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