AN OPEN-LABEL, SINGLE-DOSE, PHARMACOKINETICS STUDY OF VALTOCO® WITH OPEN-LABEL SAFETY PERIOD IN PEDIATRIC SUBJECTS WITH EPILEPSY
Study ID: STU-2021-0874
This is a Phase 1/2a, open-label, single-dose, PK study of VaLToCo with a 180 day open-label safety period in pediatric subjects with epilepsy age 2 to 5 years. a single 5 mg, 10 mg or 15 mg dose of intranasal VaLToCo will be administered based on the subject's body weight in a clinical setting followed by PK sampling. in the single-dose period, VaLToCo will be administered during an interictal period to subjects with epilepsy 2 to 5 years of age. The pre-dose PK blood sample will be collected at baseline after all pre-dose safety assessments are completed. once the pre-dose PK sample has been collected, a single dose of VaLToCo will be administered and the PK collection will continue for up to 6 hours after dosing to determine the diazepam plasma concentration versus time profile. For the open-label safety portion of this study, subjects will be supplied with VaLToCo to use as rescue medication as needed for frequent breakthrough seizures or aRS (as per the VaLToCo prescribing information). Subjects and caregivers will be provided an electronic diary on their own device to record any usage of VaLToCo for seizures including, but not limited to, the date of use, time of seizure onset, time of dosing, and time of seizure resolution.
Primary objective: * To assess the pharmacokinetics (PK) of diazepam after a single intranasal dose of VaLToCo administered to subjects with epilepsy 2-5 years of age. The primary PK variables to determine bioavailability will be the maximum plasma concentration (Cmax) and the area under the curve through 6 hours (auC(0-6)). Secondary objectives: * To assess the safety and tolerability of diazepam after intranasal administration of VaLToCo in the subject population. * To assess the long-term safety and tolerability of diazepam after repeated exposure to intranasal administration of VaLToCo in the subject population VaLToCo was approved by the uS FDa in 2020 for marketing in patients with epilepsy 6 years of age and older. This study will provide information on the PK, safety and tolerability (including local tolerability) of VaLToCo in the 2 to 5 years old pediatric epilepsy population.