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ASDPhase 2NCT03900923

Cannabidiol for ASD Open Trial

A Phase 2 study testing 98% pure CBD in people with ASD and Autism Spectrum Disorder, run by NYU Langone Health at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 7 to 17 years
Conditions the study is for
ASD, Autism Spectrum Disorder
What is being tested?
98% pure CBD
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
1 siteUnited States
Study dates
12 Mar 2019to 4 May 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 7 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site
  • NYU Langone Health

    New York, New York, 10016

Other trials in United States

About this study

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

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.

Being tested
98% pure CBD
Keywords
cannabidiol (CBD)
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 98% pure CBDDrug

    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.

Study groups in NCT03900923
Study groupRoleWhat they receive
98% pure CBDExperimental98% pure CBD. The CBD will be 100mg/mL oral solution provided by Greenwich Biosciences, Inc.
Receives: Drug: 98% pure CBD

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinical Global Impression Scale-Improvement (CGI-I)

    The physician-rated CGI-I comprises one question asking how much a participant's condition has changed since baseline. The item is answered using a 7-point Likert 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. The total score is the item response; lower scores indicate greater improvement.

    Measured: Week 6

Secondary outcomes (15)
  1. Change in Repetitive Behavior Scale-Revised (RBS-R) Score

    The RBS-R is a 43-item self report used to measure the breadth of repetitive behavior in children. For each item, behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. On the last question, participants are asked to "lump together" all of the behaviors described in the questionnaire, and provide a rating for how much of a problem these repetitive behaviors are overall, on a scale from 1-100: 1-Not a problem at all, to 100-As bad as you can imagine. A decrease in scores indicates the repetitive behaviors became less of a problem overall.

    The total score is the sum of all items and ranges from 0 to 229.

    Measured: Baseline, Week 6

  2. Change in Social Responsiveness Scale, 2nd Edition (SRS-2), School-Age Form Score

    The SRS-2 is a 65-item questionnaire assessing 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). The raw score is the sum of responses and is converted to a T-score; the final T-score has a mean of 50 points with a standard deviation of 10 points, where lower scores indicate greater social responsiveness:

    • Less than or equal to 59 = Low-to-no symptom impacts
    • Between 60-65 = Mild-to-moderate defcits in social interaction
    • Between 66-75 = Moderate defcit in social interaction
    • Greater than or equal to 76 = Severe

    Measured: Baseline, Week 6

  3. Change in Aberrant Behavior Checklist (ABC) - Irritability Subscale Score

    The ABC Irritability subscale comprises 15 items rated on a 4-point scale (ranging from 0=no problem at all to 3 = problem is severe in degree). The total score is the sum of responses and ranges from 0 to 45; higher scores indicate greater presences of irritability related behavior.

    Measured: Baseline, Week 6

  4. Change in Screen for Child Anxiety Related Disorders (SCARED), Parent Version Score

    The SCARED, Parent Version is a 41-item 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. The total score is the sum of responses and ranges from 0 to 82; lower scores indicate lesser child anxiety symptoms.

    Measured: Baseline, Week 6

  5. Change in Sleep Disturbance Scale for Children (SDSC) Score

    The SDSC is a 26-item parent-completed assessment of their child's sleep-wake rhythm. The questionnaire measures 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). The total score is the sum of responses and ranges from 26 to 130; lower scores indicate healthier sleep-wake rhythms.

    Measured: Baseline, Week 6

  6. Change in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Parent/Caregiver Form Score

    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. The range of possible composite scores ranges from 20-140, with a higher score indicating higher levels of adaptive functioning.

    Measured: Baseline, Week 6

  7. Change in Clinical Global Impression-Severity (CGI-S) Score

    1-item assessment of clinicians impression of severity of illness. Illness severity is rated on a 7-point scale ranging from 1=not at all to 7=among the most extremely ill.

    Measured: Baseline, Week 6

  8. Change in Autism Family Experience Questionnaire (AFEQ) Score

    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. The total score is the sum of responses and ranges from 48 - 240; lower scores indicate better outcomes.

    Measured: Baseline, Week 6

  9. Change in Anxiety Scale for Children - Autism Spectrum Disorder - Parent Versions (ASC-ASD-P) Score

    24-item Parent/Caregiver-completed form developed to detect symptoms of anxiety in youth with ASD. Items are rated on a 4-point scale (0=never and 3=always). The total score is the sum of responses and ranges from 0 to 72; lower scores indicate lesser symptoms of anxiety.

    Measured: Baseline, Week 6

  10. Anxiety Scale for Children - Autism Spectrum Disorder (ASC-ASD-C) - Child Version

    24-item Child-completed form developed to detect symptoms of anxiety in youth with ASD. Items are rated on a 4-point scale (0=never and 3=always). The total score is the sum of responses and ranges from 0 to 72; lower scores indicate lesser symptoms of anxiety.

    Measured: Baseline, Week 6

  11. Change in Ohio State University (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.

    Measured: Baseline, Week 6

  12. 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.

    Measured: Week 6

  13. Change in Behavioral Inflexibility Scale (BIS) Score

    38-item Parent/Caregiver-completed measure designed to assess rigid patterns of behavior commonly associated with ASD. Items are rated on a 6-point Likert scale where 0=Not at all a problem and 5=Very severe or extreme problem. The total score is the sum of responses and ranges from 0 to 228; higher scores indicate greater behavioral inflexibility.

    Measured: Baseline, Week 6

  14. Change in Home Situations Questionnaire - Modified for ASD (HSQ-ASD) Score

    24-item Parent/Caregiver-completed questionnaire measuring behavioral noncompliance in everyday settings over the past four weeks. Severity of non-compliance is rated on a 9-point Likert scale (where 1 = no interruption, and 9 = frequent/consistent interruption). The total score is the sum of responses and ranges from 24 to 216; higher scores indicate greater behavioral noncompliance.

    Measured: Baseline, Week 6

  15. Change in Strengths and Weaknesses of Attention-Deficit/Hyperactivity Disorder Symptoms and Normal Behavior (SWAN) Score

    SWAN is an 18-item Parent/Caregiver-completed measure designed to assess symptoms of ADHD in children and adolescents. Items are rated on a 7-point Likert scale, where Far Below Average = 3, Below Average = 2, Somewhat Below Average = 1, Average = 0, Somewhat Above Average = -1, Above Average = -2, and Far Above Average = -3. The total score is the sum of responses and ranges from -54 to 54; the lower the score, the higher the severity of ADHD symptoms.

    Measured: Baseline, Week 6

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Apr 2019
  2. Study start12 Mar 2019
  3. Primary completion4 May 2023
  4. Study completion4 May 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03900923
Sponsor's protocol ID
18-00250
Status last verified
May 2024
First submitted
1 Apr 2019
Last updated
31 May 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices).

Publications

  • Lawson J, Robinson L, Conlon GR, Shalev RA, Cervantes PE, Yoncheva Y, Hirsch GS, Troxel AB, Friedman D, Devinsky O, Castellanos FX. A Phase-2 Open-Label Trial of Cannabidiol to Treat Core and Associated Symptoms of Autism in Children and Adolescents Without Intellectual Disability. J Child Adolesc Psychopharmacol. 2026 Aug;36(6):350-358. doi: 10.1177/10445463261452514. Epub 2026 May 28.

    PubMed 42204954

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing 98% pure CBD in people with ASD and Autism Spectrum Disorder, run by NYU Langone Health at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 31 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Clinical Global Impression Scale-Improvement (CGI-I) (time frame: Week 6). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03900923, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 May 2024.

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