Adults 7 to 17, any sex, with ASD or Autism Spectrum Disorder. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Clinical Global Impression Scale-Improvement (CGI-I)Primary· Week 6
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
3.17
± 0.75
6 mg/kg 98% Pure CBD
2.56
± 0.88
9 mg/kg 98% Pure CBD
2.25
± 0.71
Change in Repetitive Behavior Scale-Revised (RBS-R) ScoreSecondary· Baseline, Week 6
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 dec
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-0.5
± 9.52
6 mg/kg 98% Pure CBD
-6.67
± 9.41
9 mg/kg 98% Pure CBD
-4.38
± 8.26
Change in Social Responsiveness Scale, 2nd Edition (SRS-2), School-Age Form ScoreSecondary· Baseline, Week 6
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
* Betwee
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-5.5
± 5.4
6 mg/kg 98% Pure CBD
-5.89
± 7.51
9 mg/kg 98% Pure CBD
-1.4
± 3.1
Change in Screen for Child Anxiety Related Disorders (SCARED), Parent Version ScoreSecondary· Baseline, Week 6
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-6.33
± 13.7
6 mg/kg 98% Pure CBD
-16
± 17.12
9 mg/kg 98% Pure CBD
-3.25
± 12.6
Change in Sleep Disturbance Scale for Children (SDSC) ScoreSecondary· Baseline, Week 6
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-7.5
± 12.65
6 mg/kg 98% Pure CBD
-6.89
± 5.44
9 mg/kg 98% Pure CBD
-0.13
± 4.82
Change in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Parent/Caregiver Form ScoreSecondary· 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. The range of possible composite scores ranges from 20-140, with a higher score indicating higher levels of adaptive functioning.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-0.5
± 3.89
6 mg/kg 98% Pure CBD
4.33
± 5.72
9 mg/kg 98% Pure CBD
8.88
± 7.32
Change in Clinical Global Impression-Severity (CGI-S) ScoreSecondary· Baseline, Week 6
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-0.5
± 0.84
6 mg/kg 98% Pure CBD
-1
± 0.87
9 mg/kg 98% Pure CBD
-0.5
± 0.53
Change in Autism Family Experience Questionnaire (AFEQ) ScoreSecondary· 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. The total score is the sum of responses and ranges from 48 - 240; lower scores indicate better outcomes.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-3.17
± 14.5
6 mg/kg 98% Pure CBD
-14
± 20.1
9 mg/kg 98% Pure CBD
-8.88
± 8.37
Change in Anxiety Scale for Children - Autism Spectrum Disorder - Parent Versions (ASC-ASD-P) ScoreSecondary· Baseline, Week 6
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-2.33
± 4.76
6 mg/kg 98% Pure CBD
-6.56
± 9.84
9 mg/kg 98% Pure CBD
-3.13
± 6.15
Anxiety Scale for Children - Autism Spectrum Disorder (ASC-ASD-C) - Child VersionSecondary· Baseline, Week 6
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.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
-4
± 6.81
6 mg/kg 98% Pure CBD
-4.78
± 5.61
9 mg/kg 98% Pure CBD
-6.5
± 11.2
Change in Ohio State University (OSU) Autism Clinical Global Impressions: SeveritySecondary· Baseline, Week 6
Reflects clinicians impression of severity of illness on a 7-point scale ranging from 1=not present to 7="classic" autism.
Group
Value
95% CI
3 mg/kg 98% Pure CBD
0
± 0
6 mg/kg 98% Pure CBD
-0.22
± 0.67
9 mg/kg 98% Pure CBD
-0.38
± 0.52
Adverse events — posted to ClinicalTrials.gov
Time frame: 6 weeks after participant received first dose..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by NYU Langone Health
Last refreshed: 31 May 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03900923.