An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE
Key facts
- Who runs it
- Xenon Pharmaceuticals Inc.
- Where
- Philadelphia and Tacoma, United StatesSee the 2 sitesAlso in 2 other countries, 2 sites
- Trial code
- Status
- Terminated
- Phase
- Phase 3
- People taking part
- 8 people enrolled
- Started
- 17 Aug 2021
- Ended
- 17 Nov 2023
- Type of study
- Interventional, 2 groups
- Treatments tested
- XEN496
- Record updated
- 14 February 2025
- Why it stopped
- Sponsor decision; not a safety decision
Who can take part
- Age
- 1 month to 6 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 8 criteriaShow less
Who can take part
- 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
Who cannot take part
- It is anticipated that the subject will require treatment with at least 1 of the disallowed medications during the 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
- 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
- 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
Where in United States
Also in 2 other countries, 2 sites
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesNo longer taking part
- MultiCare Health System - Mary Bridge Pediatrics - TacomaTacoma, United StatesNo longer taking part
About the trial
What the trial wants to find out
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).
The 2 groups in the trial
- Group 1: XEN496 onlyTreatment being tested
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
Treatment: XEN496
- Group 2: Placebo to XEN496Treatment being tested
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.
Treatment: XEN496
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- An Open-Label Extension of the Study XEN496 in Children With KCNQ2 Developmental and Epileptic Encephalopathy
- Record updated
- 14 February 2025