Safety and Local Tolerability of Diazepam Nasal Spray for Children With Epilepsy Aged 2-5 Years: Final Results of a Phase 1/2a Trial.

Diazepam nasal spray is approved to treat seizure clusters in patients aged ≥2 years. A long-term safety study in patients aged 6-65 years found a safety profile similar to diazepam rectal gel. We conducted an open-label, phase 1/2a trial of diazepam nasal spray in children with epilepsy aged 2-5 years with motor seizures or seizures with clear alteration of awareness. After a pharmacokinetic period, caregivers were instructed to use diazepam nasal spray (0.5 mg/kg) as needed for frequent seizures or seizure clusters during 180-day safety and optional extension periods. Treatment-emergent adverse events (TEAEs) were assessed. Of 36 enrolled patients, 27 (86.1%) completed both treatment periods (mean duration 15.45 months). Treatment-related TEAEs occurred in 7 patients in the 180-day period and 1 in the optional extension period. No serious treatment-related TEAEs or TEAEs leading to discontinuation/death occurred. The safety profile aligns with patients aged ≥6 years, supporting use in this population.(ClinicalTrials.gov identifier: NCT05076838).