Cannabidiol for ASD Open Trial
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|ClinicalTrials.gov Identifier: NCT03900923|
Recruitment Status : Recruiting
First Posted : April 3, 2019
Last Update Posted : September 27, 2021
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 evaluates 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 are being recruited. Participants have ﬂuent speech and an estimated IQ greater than or equal to 80. Study intervention is 98% pure CBD. The CBD is Greenwich Biosciences, Inc.'s 100mg/mL oral solution, brand name EPIDIOLEX. First, a Bayesian optimal interval (BOIN) design was used, such that participants were 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.
The BOIN design ended after the fifth cohort of participants, and the two lower doses, 3 and 6 mg/kg/day, were eliminated. The highest dose, 9 mg/kg/day, was not tested. Therefore, in subsequent cohorts, we will be examining 9 mg/kg/day exclusively in up to 15 additional participants with co-occurring ASD and attention-deficit/hyperactivity disorder (ADHD) diagnoses, as this clinical profile appears to most closely resemble youth classified as responders within the BOIN design.
|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 :||December 31, 2022|
|Estimated Study Completion Date :||December 31, 2022|
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 was used, such that participants were 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. The BOIN design has ended, and two doses have been tested and eliminated (3 and 6 mg/kg/day). We are now examining 9 mg/kg/day exclusively.
- Clinical Global Impression Scale -Improvement (CGI-I) [ Time Frame: 6 Weeks ]This is a 7-point scale measuring symptom change from baseline.
- 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.
- 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
- Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Parent/Caregiver Form [ Time Frame: Baseline, Week 6 ]Used to measure adaptive functioning across 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.
- Autism Family Experience Questionnaire (AFEQ) [ Time Frame: Baseline, Week 6 ]Parent/Caregiver form used to measure impact of autism interventions on family experience and quality of life. Items are rated on a 5-point scale where 1=always and 5=never.
- Anxiety Scale for Children - Autism Spectrum Disorder - Parent Versions (ASC-ASD-P) [ Time Frame: Baseline, Week 6 ]Parent/Caregiver form developed to detect symptoms of anxiety in youth with ASD. Composed of four subscales (Performance Anxiety, Uncertainty, Anxious Arousal, and Separation Anxiety), items are rated on a 4-point scale (0=never and 3=always). Subscales sum to equal a total score.
- Anxiety Scale for Children - Autism Spectrum Disorder (ASC-ASD-C) - Child Versions [ Time Frame: Baseline, Week 6 ]Child form developed to detect symptoms of anxiety in youth with ASD. Composed of four subscales (Performance Anxiety, Uncertainty, Anxious Arousal, and Separation Anxiety), items are rated on a 4-point scale (0=never and 3=always). Subscales sum to equal a total score.
- OSU Autism Clinical Global Impressions: Severity [ Time Frame: Screening, Baseline, Week 6 ]Reflects clinicians impression of severity of illness on a 7-point scale ranging from 1=not present to 7="classic" autism.
- OSU Autism Clinical Global Impressions: Improvement [ Time Frame: Week 6 ]Reflects clinicians impression of improvement on a 7-point scale ranging from 1=very much improved to 7=very much worse.
- Behavioral Inflexibility Scale (BIS) [ Time Frame: Baseline, Week 6 ]Parent/Caregiver measure designed to assess rigid patterns of behavior commonly associated with ASD. The measure is composed of 38 items rated on a 6-point Likert scale where 0=Not at all a problem and 5=Very severe or extreme problem. Items sum to a total score.
- Home Situations Questionnaire - Modified for ASD (HSQ-ASD) [ Time Frame: Baseline, Week 6 ]Parent/Caregiver questionnaire that measures behavioral noncompliance in everyday settings. Consists of two 12-item factors: Socially Inflexible and Demand Specific. Severity of non-compliance is rated on a 1-9 Likert scale, and higher scores indicate greater non-compliance.
- 16. Strengths and Weaknesses of Attention-Deficit/Hyperactivity Disorder Symptoms and Normal Behavior (SWAN) [ Time Frame: Baseline, Week 6 ]Parent/Caregiver measure designed to assess symptoms of ADHD in children and adolescents. Includes 18 items rated on a 7-point Likert scale ranging from far-below average to far-above average. Subscales include ADHD-Inattentive and ADHD-Hyperactive/Impulsive. Total range of score is 0-126; higher score indicates greater symptoms.
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, PhD||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 Cervantes, 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|