Clinical Study of Synthetic Cannabidiol in Children and Adolescents with 22q11.2 Deletion Syndrome

This is an open-label single-center study, to assess the safety, tolerability and efficacy of ZYN002 (a synthetic cannabidiol) administered as a transdermal gel, for the treatment of child and adolescent patients with 22q11.2 Deletion Syndrome. Approximately 20 male and female patients ages 6 to < 18 years will be treated for 14 weeks. The study gel will be applied to clean, dry, intact skin of the upper arms and shoulders. All participants will undergo a screening process to assess eligibility. Eligible participants will then participate in a 14 week treatment period, where all participants will received ZYN002. Parents/caregivers will apply the study gel twice daily for the treatment period. Participants who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002 (125 mg of cannabidiol), applied every 12 hours (± 2 hours). Participants who weigh more than 35 kg will receive 2 sachets of ZYN002 (250 mg of cannabidiol), applied every 12 hours (± 2 hours). Participants who are taking anti-epileptic drugs may have an additional one or two weeks of treatment to taper off study treatment. The taper period is part of the clinical trial and will be overseen by the Principal Investigator. These patients will taper their study dose in the following manner beginning at Visit 4 or at Early Termination: (a) For those patients weighing less than or equal to 35 kg (250 mg daily dose), the dose of study drug will be reduced to a total daily dose of 125 mg ZYN002 each day for one week (one sachet each evening), after which time the patients will discontinue from the study at Week 16. (b) For those patients weighing greater than 35 kg (500 mg daily dose), the dose of study drug will be reduced over two weeks: during the first week of taper the dose will be reduced to 125 mg applied every 12 hours; (±2 hours); total daily dose of 250 mg, followed by a second week of taper from 250 mg total daily dose to a total daily dose of 125 mg ZYN002 each day (one sachet each evening). After the taper, patients will discontinue from the study Week 17. Patients tapering their dose will have all Visit 4 End Of Study/Early Termination assessments completed. At Week 14, patients will have their original dose in the morning and will have their evening dose as their new tapered dose. Patients will return on Week 16 or 17 and have their final concomitant medication review and safety assessments completed. Patient compliance with the intervention will be monitored by the study coordinator through drug accountability at each study visit.