NCT05149898

Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)

An Open-Label Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Harmony Biosciences Management, Inc. · Industry
• Sex: All
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to \<18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).

Detailed description

This was an open-label study that assessed the safety, tolerability and efficacy of cannabidiol (CBD) administered as ZYN002, a transdermal gel, for the treatment of child and adolescent participants with 22qDS. Male and female participants with 22qDS were treated in Period 1 for 14 weeks. Participants that met study criteria were allowed to continue to Period 2 for an additional 24 weeks of treatment. At the end of the study, participants taking antiepileptic drug (AED) medication(s) had an additional one- or two-week Taper Period. Approximately 20 male and female participants, ages 4 to \< 18 years, received ZYN002.

Conditions

• 22Q11.2 Deletion Syndrome

Interventions

Type	Name	Description
DRUG	ZYN002	Synthetic CBD (sCBD) Transdermal Gel pharmaceutically manufactured. sCBD formulated as a clear gel (transdermal delivery). Dose received is based on weight. 1. Participants who weigh ≤ 35 kilogram (kg) will receive 125 mg CBD Q12H (every 12 hours ± 2 hours); for a total daily dose of 250 mg CBD. 2. Participants who weigh \> 35 kg will receive 250 mg CBD Q12H (±2 hours) for a total daily dose of 500 mg CBD. Patients in both weight ranges ≤ 35 kg or \> 35 kg may receive an increased daily dose of 500 mg sCBDor 750 mg sCBD, respectively.

Timeline

• Start date: 2020-02-19
• Primary completion: 2022-11-09
• Completion: 2022-11-09
• First posted: 2021-12-08
• Last updated: 2026-03-09
• Results posted: 2026-03-09

Locations

3 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study