To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) who had participated in the primary study (XPF-009-301).
This is an open-label, long-term extension study of XEN496 for the treatment of seizures in subjects with KCNQ2-DEE, that will be open to eligible subjects who participated in the primary study, XPF-009-301. The primary objective is to assess the long-term safety of XEN496. A double-blind transition/titration period will be used to maintain blinding to the treatment allocation in the primary study (XPF-009-301). After completion of the blinded transition/titration period, subjects will receive the open label study drug at their optimal dose for approximately 35 months.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
8
XEN496 sprinkle capsules. Parents / caregivers will be instructed to sprinkle and mix the contents of the capsules into soft foods or liquids and feed it to the child.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
MultiCare Health System - Mary Bridge Pediatrics - Tacoma
Tacoma, Washington, United States
Sydney Children's Hospital
Sydney, New South Wales, Australia
Universitair Ziekenhuis Antwerpen - Dienst Kinderneurologie
Edegem, Antwerpen, Belgium
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Intervention
Safety and tolerability of XEN496 as assessed by incidence and severity of AEs and SAEs
Time frame: From Screening/Baseline through to 4 weeks post last dose
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.