Summary

Eligibility
for people ages 1 month to 6 years (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion

Description

Summary

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).

Official Title

An Open-Label Extension of the Study XEN496 in Children With KCNQ2 Developmental and Epileptic Encephalopathy

Details

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.

Keywords

Epilepsy Epilepsy in Children Epilepsy; Seizure Disease Brain Diseases Central Nervous System Diseases Nervous System Diseases Epileptic Syndromes XEN496 Ezogabine Retigabine Encephalopathy Seizure KCNQ2 EPIK Seizures

Eligibility

You can join if…

Open to people ages 1 month to 6 years

  • Subject completed participation in the primary study, XPF-009-301. A subject who withdraws from the primary study due to meeting protocol-specified worsening criteria will be considered as having completed participation in the primary study.
  • The caregiver is willing and able to be compliant with diary completion, visit schedule, and study drug administration.
  • Subject's caregiver achieved a minimum of 85% compliance with daily diary completion during both baseline and the double-blind period of the primary study.

You CAN'T join if...

  • Any adverse event(s) or serious adverse event(s) during the primary study XPF-009-301, which in the opinion of the investigator and sponsor's medical monitor, would preclude the subject's entry into the OLE study.
  • A clinically significant condition or illness, or symptoms other than those resulting from KCNQ2-DEE, present at screening/baseline that, in the opinion of the investigator, would pose a risk to the subject if s/he were to enter the study.
  • Any conditions that were specified as exclusion criteria in the primary study, XPF-009-301.
  • It is anticipated that the subject will require treatment with at least 1 of the disallowed medications during the study.
  • Any change in cardiac rhythm or atrioventricular conduction in the primary study that, in the investigator's opinion, is a significant risk to subject safety.

Locations

  • UCSF Benioff Children's Hospital accepting new patients
    San Francisco California 94158 United States
  • Oregon Health and Science University accepting new patients
    Portland Oregon 97239 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Xenon Pharmaceuticals Inc.
ID
NCT04912856
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 40 study participants
Last Updated