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
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|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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
- Clinical Global Impression Scale -Improvement (CGI-I) [ Time Frame: Baseline, 6 Weeks ]This is a 7-point scale measuring symptom change from baseline.
- 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).
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
|Contact: Paige Cervantes||646-754-7243||Paige.Cervantes@nyulangone.org|
|Contact: Greta Conlonfirstname.lastname@example.org|
|United States, New York|
|NYU Langone Health||Recruiting|
|New York, New York, United States, 10016|
|Contact: Greta Conlon 212-404-3770 email@example.com|
|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, PhD|
|Sub-Investigator: Yuliya Yoncheva, PhD|
|Sub-Investigator: Paige Cervantes, PhD|
|Principal Investigator:||Francisco Castellanos, MD||New York Langone Health|