22q11.2 deletion syndrome (22qDS) is a genetic disorder affecting various body systems and associated with behavioral and psychiatric conditions, with no currently approved pharmacological treatments. ZYN002, a transdermal gel containing synthetic cannabidiol, has been demonstrated to be well tolerated, showing potential benefit in related neurodevelopmental disorders. The INSPIRE study evaluated the safety, tolerability, and effectiveness of ZYN002 in children and adolescents with 22qDS. INSPIRE was a phase 2, open-label, multicenter trial comprising a 14-week treatment period (Period 1) followed by an optional 24-week, open-label extension (Period 2) for participants with ≥35% improvement on the Aberrant Behavior Checklist-Community (ABC-C) irritability subscale upon Period 1 completion. Participants aged 4 to 80% of caregivers reported improvement in ≥1 problem during each period. Anxiety and irritability scores from Baseline through Period 2 maintained a similar reduction to Period 1. In this interventional, open-label trial of pediatric participants with 22qDS, ZYN002 was considered safe and well tolerated, and was associated with reductions in anxiety-related and behavioral symptoms, supporting further investigation of ZYN002 in a phase 3 randomized controlled trial. Clinicaltrials.gov (NCT05149898) registered on October 5, 2021.
Heussler et al. (Thu,) studied this question.