An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE

(EPIK-OLE Trial)

Recruiting at 12 trial locations

Overseen By Xenon Pharmaceuticals Inc.

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Xenon Pharmaceuticals Inc.

Disqualifiers: Adverse events, Cardiac issues, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

8 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing XEN496, a medication for children with a rare form of epilepsy called KCNQ2-DEE. The goal is to see if it can safely help control their seizures by stabilizing brain activity. KCNQ channel openers merit further study as potential treatments for seizures in infants and children.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify if you need to stop taking your current medications, but it mentions that you cannot participate if you need to take any disallowed medications during the study.

Research Team

Study Director

Principal Investigator

Xenon Pharmaceuticals Inc.

Eligibility Criteria

Inclusion Criteria

The caregiver is willing and able to be compliant with diary completion, visit schedule, and study drug administration.

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.

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.

Exclusion Criteria

Any conditions that were specified as exclusion criteria in the primary study, XPF-009-301.

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 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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Blinded Transition/Titration

A 24-day blinded transition/titration period where subjects transition from the primary study to the open-label extension, maintaining blinding to treatment allocation.

24 days

1 visit (in-person)

Open-label Treatment

Participants receive the open-label study drug at their optimal dose for long-term safety and tolerability assessment.

35 months

Regular visits every 3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment completion.

4 weeks

Treatment Details

Interventions

• XEN496

Participant Groups

Group I: Group 2: Placebo to XEN496 Experimental Treatment 1 Intervention

24-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.

Group II: Group 1: XEN496 only Experimental Treatment 1 Intervention

24-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

Who Is Running the Clinical Trial?

Xenon Pharmaceuticals Inc.

Lead Sponsor

Trials

19

Recruited

3,400+