NCT04639310

XEN496 (Ezogabine) in Children With KCNQ2 Developmental and Epileptic Encephalopathy

A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects With KCNQ2 Developmental and Epileptic Encephalopathy

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Xenon Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 1 Month – 6 Years
• Healthy volunteers: Not accepted

Summary

To investigate the potential antiseizure effects of adjunctive XEN496 (ezogabine) compared with placebo in children with KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE).

Detailed description

The EPIK Phase 3 clinical trial is designed as a randomized, double-blind, placebo-controlled, multicenter study targeting to enroll approximately 40 pediatric subjects (aged from 1 month to less than 6 years) with documented genetic evidence consistent with a diagnosis of KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE). After screening, subjects will enter a baseline period before being randomized to receive either XEN496 (ezogabine) or placebo, added to their existing antiseizure medications (ASMs), for 12 weeks (maintenance), once a titration period of up to 24 days is complete. At the end of the maintenance phase, eligible subjects will have the opportunity to qualify for and participate in the separate open-label extension (OLE) study and receive XEN496 or, should they choose to exit the study, will undergo a dose taper period of up to 15 days and 4-week follow-up.

Conditions

• Epilepsy
• Epilepsy in Children
• Epilepsy; Seizure
• Disease
• Brain Diseases
• Central Nervous System Diseases
• Nervous System Diseases
• Epileptic Syndromes

Interventions

Type	Name	Description
DRUG	XEN496	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 TID.
DRUG	Placebo	Placebo 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 TID.

Timeline

• Start date: 2021-03-29
• Primary completion: 2023-05-16
• Completion: 2023-05-16
• First posted: 2020-11-20
• Last updated: 2024-08-23
• Results posted: 2024-08-23

Locations

20 sites across 5 countries: United States, Australia, Belgium, Italy, Spain

Regulatory

• FDA-regulated drug study