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Cannabidiol for ASD Open Trial

Primary Purpose

ASD, Autism Spectrum Disorder

Status

Active

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

98% pure CBD

About this trial

This is an interventional treatment trial for ASD focused on measuring cannabidiol (CBD)

Eligibility Criteria

7 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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
Diagnosis of ADHD confirmed by clinician review of K-SADS-COMP and DSM-5 Criteria
SRS-2 Total T-score of 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 or current evidence of significantly impaired liver function, defined as 1) Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 5 × upper limit of normal (ULN); 2) ALT or AST > 3 × ULN with concomitant total bilirubin > 2.0 × ULN; or 3) ALT or AST ≥ 3 × ULN with the appearance of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia
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
Exposure to any investigational agent in the 30 days prior to initiation of trial
Treatment with CBD or other cannabinoid within the previous two months
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 drug screening indicating presence of the following drugs: THC, opiates, methamphetamine, or cocaine
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

Sites / Locations

NYU Langone Health

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

98% pure CBD

Arm Description

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

Outcomes

Primary Outcome Measures

Clinical Global Impression Scale -Improvement (CGI-I)

This is a 7-point scale measuring symptom change from baseline.

Secondary Outcome Measures

Repetitive Behavior Scale-Revised (RBS-R)

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

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

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)

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

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)

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

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):

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)

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)

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

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

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

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)

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)

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)

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.

Full Information

NCT ID

NCT03900923

First Posted

April 1, 2019

Last Updated

June 26, 2023

Sponsor

NYU Langone Health

1. Study Identification

Unique Protocol Identification Number

NCT03900923

Brief Title

Cannabidiol for ASD Open Trial

Official Title

A Phase 2 Study of Cannabidiol as a New Treatment for Autism Spectrum Disorder in Children and Adolescents

Study Type

Interventional

2. Study Status

Record Verification Date

June 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

March 12, 2019 (Actual)

Primary Completion Date

May 16, 2023 (Actual)

Study Completion Date

May 16, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

NYU Langone Health

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

ASD, Autism Spectrum Disorder

Keywords

cannabidiol (CBD)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

29 (Actual)

8. Arms, Groups, and Interventions

Arm Title

98% pure CBD

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

98% pure CBD

Intervention Description

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.

Primary Outcome Measure Information:

Title

Clinical Global Impression Scale -Improvement (CGI-I)

Description

This is a 7-point scale measuring symptom change from baseline.

Time Frame

6 Weeks

Secondary Outcome Measure Information:

Title

Repetitive Behavior Scale-Revised (RBS-R)

Description

Time Frame

Baseline, Week 6

Title

Social Responsiveness Scale, 2nd Edition (SRS-2), School-Age Form

Description

Time Frame

Baseline, Week 6

Title

Aberrant Behavior Checklist (ABC) - Irritability and Social Withdrawal Subscales

Description

Time Frame

Baseline, Week 6

Title

Anxiety, Depression and Mood Scale (ADAMS)

Description

Time Frame

Baseline, Week 6

Title

Screen for Child Anxiety Related Disorders (SCARED), Parent Version

Description

Time Frame

Baseline, Week 6

Title

Sleep Disturbance Scale for Children (SDSC)

Description

Time Frame

Baseline, Week 6

Title

Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Parent/Caregiver Form

Description

Time Frame

Baseline, Week 6

Title

Clinical Global Impression-Severity (CGI-S):

Description

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.

Time Frame

Screening, Baseline, Week 6

Title

Autism Family Experience Questionnaire (AFEQ)

Description

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.

Time Frame

Baseline, Week 6

Title

Anxiety Scale for Children - Autism Spectrum Disorder - Parent Versions (ASC-ASD-P)

Description

Time Frame

Baseline, Week 6

Title

Anxiety Scale for Children - Autism Spectrum Disorder (ASC-ASD-C) - Child Versions

Description

Time Frame

Baseline, Week 6

Title

OSU Autism Clinical Global Impressions: Severity

Description

Reflects clinicians impression of severity of illness on a 7-point scale ranging from 1=not present to 7="classic" autism.

Time Frame

Screening, Baseline, Week 6

Title

OSU Autism Clinical Global Impressions: Improvement

Description

Reflects clinicians impression of improvement on a 7-point scale ranging from 1=very much improved to 7=very much worse.

Time Frame

Week 6

Title

Behavioral Inflexibility Scale (BIS)

Description

Time Frame

Baseline, Week 6

Title

Home Situations Questionnaire - Modified for ASD (HSQ-ASD)

Description

Time Frame

Baseline, Week 6

Title

16. Strengths and Weaknesses of Attention-Deficit/Hyperactivity Disorder Symptoms and Normal Behavior (SWAN)

Description

Time Frame

Baseline, Week 6

10. Eligibility

Sex

All

Minimum Age & Unit of Time

7 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility 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 Diagnosis of ADHD confirmed by clinician review of K-SADS-COMP and DSM-5 Criteria SRS-2 Total T-score of 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 or current evidence of significantly impaired liver function, defined as 1) Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 5 × upper limit of normal (ULN); 2) ALT or AST > 3 × ULN with concomitant total bilirubin > 2.0 × ULN; or 3) ALT or AST ≥ 3 × ULN with the appearance of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia 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 Exposure to any investigational agent in the 30 days prior to initiation of trial Treatment with CBD or other cannabinoid within the previous two months 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 drug screening indicating presence of the following drugs: THC, opiates, methamphetamine, or cocaine 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Francisco X Castellanos, MD

Organizational Affiliation

New York Langone Health

Official's Role

Principal Investigator

Facility Information:

Facility Name

NYU Langone Health

City

New York

State/Province

New York

ZIP/Postal Code

10016

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices).

IPD Sharing 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).

IPD Sharing Access Criteria

Data will be available for any purpose.

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Cannabidiol for ASD Open Trial

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