Suspended
EPIK-OLEAn Open-Label Extension of the Study XEN496 in Children With KCNQ2 Developmental and Epileptic Encephalopathy
What is being tested
XEN496
Drug
Who is being recruted
Epileptic Syndromes+8
+ Brain Diseases
+ Central Nervous System Diseases
From 1 Months to 6 Years
+8 Eligibility Criteria
How is the trial designed
Treatment Study
Phase 3
Interventional
Study Start: August 2021
Summary
Principal SponsorXenon Pharmaceuticals Inc.
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner
Study start date: August 17, 2021
Actual date on which the first participant was enrolled.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.
Official TitleAn Open-Label Extension of the Study XEN496 in Children With KCNQ2 Developmental and Epileptic Encephalopathy
Principal SponsorXenon Pharmaceuticals Inc.
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.Design Details
8 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Conditions
Criteria
Any sex
Biological sex of participants that are eligible to enroll.From 1 Months to 6 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Epileptic SyndromesBrain DiseasesCentral Nervous System DiseasesDiseaseEpilepsyNervous System DiseasesNeurologic ManifestationsPathologic ProcessesSeizuresSigns and SymptomsPathological Conditions, Signs and Symptoms
Criteria
3 inclusion criteria required to participate
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.
5 exclusion criteria prevent from participating
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.
Show More Criteria
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.Treatment Groups
Study Objectives
2 intervention groups are designated in this study
This study does not include a placebo group
Treatment Groups
Group I
Experimental24-day blinded transition/titration period. Subjects who received XEN496 in the preceding study will continue to receive XEN496 at the same dose, in a blinded manner, without any further titration. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects.
Subjects who discontinue will be required to taper off study drug over a period of up to 15 days
Group II
Experimental24-day blinded transition/titration period. Subjects who were allocated to placebo in the preceding study, will be titrated to a tolerated dose up to a maximum dose of 21 mg/kg/day, with a maximum daily dose of 672 mg/day. To maintain the blinded aspect of the study, placebo will be dispensed to all subjects during the transition/titration period to ensure the total number of capsules are consistent across all subjects
Subjects who discontinue or complete the study treatment will be required to taper off study drug over a period of up to 15 days.
Study Objectives
Primary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 4 locations
Suspended
Children's Hospital of Philadelphia
Philadelphia, United StatesOpen Children's Hospital of Philadelphia in Google MapsSuspended
MultiCare Health System - Mary Bridge Pediatrics - Tacoma
Tacoma, United StatesSuspended
Sydney Children's Hospital
Sydney, AustraliaSuspended
Universitair Ziekenhuis Antwerpen - Dienst Kinderneurologie
Edegem, BelgiumSuspended4 Study Centers