XEN496 (Ezogabine) in Children With KCNQ2 Developmental and Epileptic Encephalopathy (EPIK)
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|ClinicalTrials.gov Identifier: NCT04639310|
Recruitment Status : Recruiting
First Posted : November 20, 2020
Last Update Posted : November 10, 2022
|Condition or disease||Intervention/treatment||Phase|
|Epilepsy Epilepsy in Children Epilepsy; Seizure Disease Brain Diseases Central Nervous System Diseases Nervous System Diseases Epileptic Syndromes||Drug: XEN496 Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects With KCNQ2 Developmental and Epileptic Encephalopathy|
|Actual Study Start Date :||March 29, 2021|
|Estimated Primary Completion Date :||November 2024|
|Estimated Study Completion Date :||December 2024|
24-day dose titration period to a top dose of 21 mg/kg/day. Subjects continue at the top dose, or the highest tolerated dose up to the top dose, for 12-week maintenance period. If the subject does not immediately enter into the separate open-label extension (OLE) study, the maintenance period will be followed by a 15-day taper period.
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.
Placebo Comparator: Placebo
To maintain the blinded aspect of the study, subjects will be titrated on placebo over the 24-day period and remain at this dose for the 12-week maintenance period. If the subject does not immediately enter into the separate OLE study, the maintenance period will be followed by a 15-day taper period.
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.
- Percent change from baseline in monthly (28 day) countable motor seizure frequency during the blinded treatment period [ Time Frame: From baseline to the end of the double-blind, 12 week treatment period (maintenance) ]Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity
- Proportion of subjects with ≥50 percent reduction in monthly (28 day) seizure frequency [ Time Frame: From baseline to the end of the double-blind, 12 week treatment period (maintenance) ]Parent/caregiver seizure diary record will be used to assess frequency, type and duration
- Caregiver Global Impression of Change (CaGI-C) scores for the subject's overall condition and for seizures [ Time Frame: Study Days 24, 67, 88 and 109 ]CaGI-C scale is a caregiver-reported assessment for the subject's overall condition and for seizures. Responses to the CaGI-C questionnaire are to be rated on a 7 item Likert scale ranging from very much improved to very much worse.
- Change from baseline in the Caregiver Global Impression of Severity (CaGI-S) for the subject's overall condition and for seizures [ Time Frame: Study Days 1, 24, 67, 88 and 109 ]CaGI-S scale is Caregiver-reported assessment of the severity of the subject's seizures and overall condition over the previous 7 days. Responses to the CaGI-S questionnaire are to be rated on a 5 item Likert scale ranging from none to very severe.
- Assess the safety and tolerability of XEN496 (e.g., adverse events) in pediatric subjects with KCNQ2-DEE [ Time Frame: From screening through to the end of the study (maintenance phase for those continuing into the OLE) or Day 151 for those exiting the study ]To assess adverse events as criteria for safety and tolerability
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04639310
|Contact: Xenon Pharmaceuticals Inc.||1-604-484-3300||XenonCares@xenon-pharma.com|
|Study Director:||Study Director||Xenon Pharmaceuticals Inc.|