Phase 2 bright (an open-label tolerability and efficacy study of ZYN002 administered as a transdermal gel to children and adolescents with autism spectrum disorder): Baseline characteristics

Abstract

Autism spectrum disorder (ASD) is a complex neurodevelopmental disorder characterized by difficulties with behaviors, communication and reciprocal social interaction. ZYN002 is a pharmaceutically manufactured transdermal cannabidiol (CBD) gel currently in clinical development for the treatment of behavioral symptoms in ASD. BRIGHT is a 14 week open-label single-center study (ZYN2-CL-030) to evaluate the safety and tolerability in 37 pediatric and adolescent patients diagnosed with ASD. The efficacy assessments include the Aberrant Behavior Checklist (ABC-C), Parent Rated Anxiety Scale - Autism Spectrum Disorder (PRAS-ASD), Autism Impact Measure (AIM), the Children's Sleep Habit Questionnaire (CSHQ), Clinical Global Impression - Severity and Improvement (CGI-S, CGI-I), Autism Parenting Stress Index, and the Qualitative Caregiver Reported Behavioral Problems Survey. The Autism Diagnostic Observation Schedule Second Edition (ADOS-2) was also used to assess the severity of ASD symptoms at baseline. The majority of patients enrolled were male, n=34 (91.9%), the mean age was 9.2 years old (range 3-16 years), and 75.7% were white, while 5.4, 8.1, 10.8% were Aboriginal, Asian, or other, respectively. Ninety-four percent of enrolled patients were categorized in the moderate-to-severe symptom range as guided by the ADOS-2 criteria. Similarly, the mean baseline ABC-C Irritability subscale score was 30.0 (range: 18-41) which further describes the increased severity of the enrolled patient population. In conclusion, this study was enriched for disease severity in order to avoid floor effects on outcome measures, while still enrolling a broad and inclusive patient population with a spectrum of behavioral symptomology and an enduring high unmet need.L6358204122021-08-31 <br />