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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 : May 17, 2019
Sponsor:
Information provided by (Responsible Party):
New York University School of Medicine

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.


Condition or disease Intervention/treatment Phase
ASD Autism Spectrum Disorder Drug: 98% pure CBD Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study of Cannabidiol as a New Treatment for Autism Spectrum Disorder in Children and Adolescents
Actual Study Start Date : March 12, 2019
Estimated Primary Completion Date : January 31, 2021
Estimated Study Completion Date : January 31, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 98% pure CBD
98% pure CBD. The CBD will be 100mg/mL oral solution provided by Greenwich Biosciences, Inc.
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




Primary Outcome Measures :
  1. Clinical Global Impression Scale -Improvement (CGI-I) [ Time Frame: Baseline, 6 Weeks ]
    This is a 7-point scale measuring symptom change from baseline.


Secondary Outcome Measures :
  1. 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.

  2. 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).

  3. 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

  4. 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).

  5. Anxiety, Depression and Mood Scale (ADAMS) [ Time Frame: Baseline, Week 6 ]
    Contains 55 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.

  6. 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. The CCC-2 takes approximately 10-15 minutes to complete. Psychometrics are strong, with test- retest reliability estimates between 0.86-0.96Items are rated on a 4-point scale from 0=less than once a week to 3= several times a day.

  7. Computerized Kiddie Schedule for Affective Disorders and Schizophrenia (KSADS-COMP) [ 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. Items are rated on a 3-point scale ranging from 0=not true or hardly ever true to 2=very true or often true.

  8. 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.

  9. Sleep Disturbance Scale for Children [ 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 (Ccommunication, Ddaily Lliving Sskills, and Ssocialization), 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.

  10. Vineland Adaptive BehaviorScales, Third Edition (Vineland-3), [ Time Frame: 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.

  11. 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.

  12. Pediatric Quality of Life Inventory (PedsQL) Family Impact Module [ Time Frame: Baseline, Week 6 ]
    Self Self-report forms to measure caregiver and family functioning. Consists of six6 scales measuring caregiver functioning (physical Physical Ffunctioning, emotional Emotional Ffunctioning, Ssocial Ffunctioning, Ccognitive Ffunctioning, Ccommunication, 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).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   7 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  • Treatment with valproate or clobazam or other antiepileptic medicationCurrent 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

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): NCT03900923


Contacts
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Contact: Greta Conlon 212-404-3770 greta.conlon@nyulangone.org
Contact: Paige Cervantes, PhD 646-754-7243 Paige.Cervantes@nyulangone.org

Locations
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United States, New York
NYU Langone Health Recruiting
New York, New York, United States, 10016
Contact: Greta Conlon    212-404-3770    greta.conlon@nyulangone.org   
Contact: Paige Conlon, PhD    646-754-7243    Paige.Cervantes@nyulangone.org   
Principal Investigator: Francisco Castellanos, MD         
Principal Investigator: Orrin Devinsky, MD         
Sub-Investigator: Daniel Friedman, MD         
Sub-Investigator: Melissa Nishawala, MD         
Sub-Investigator: Glenn Hirsh, MD         
Sub-Investigator: Judith Bluvstein, MD         
Sub-Investigator: Rebecca Shalev, MD         
Sub-Investigator: Yuliya Yoncheva, MD         
Sub-Investigator: Paige Cervantes, MD         
Sponsors and Collaborators
New York University School of Medicine
Investigators
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Principal Investigator: Francisco Castellanos, MD New York Langone Medical Center

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Responsible Party: New York University School of Medicine
ClinicalTrials.gov Identifier: NCT03900923     History of Changes
Other Study ID Numbers: 18-00250
First Posted: April 3, 2019    Key Record Dates
Last Update Posted: May 17, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
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
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.

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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

Keywords provided by New York University School of Medicine:
cannabidiol (CBD)

Additional relevant MeSH terms:
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Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Epidiolex
Anticonvulsants