Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age With Partial-onset (Focal) Seizures

NCT05067634 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: YKP3089C040
• Study Type: interventional
• Target: 140
• Locations: 7 countries
• Sites: 54

Brief Summary

Primary objective: To evaluate the safety and tolerability of cenobamate in pediatric subjects 2-17 years of age with partial-onset (focal) seizures

Conditions

Partial Epilepsy

Keywords

Partial-onset (focal) seizures; Pediatrics

Outcome Measures

Primary Outcomes (1)

• Incidence of Adverse Events and SAEs

  Summary statistics for clinical laboratory test results and vital signs; and physical examination, neurologic examination and electrocardiogram (ECG) finding.of age with partial-onset (focal) seizures

  3 Years

Secondary Outcomes (2)

• To collect plasma samples of cenobamate to support the evaluation of the pharmacokinetics of cenobamate tablets and suspension in pediatric subjects with partial onset (focal) seizures

  3 Years

• Acceptability and palatability assessment (determined by a 5-point Hedonic Scale) of the oral formulation and tablets

  3 Years

Study Arms (4)

12 to < 18 year olds

EXPERIMENTAL

Drug: Xcopri

6 to <12 years old

EXPERIMENTAL

Drug: Xcopri

4 to <6 years old

EXPERIMENTAL

Drug: Xcopri

2 to <4 years old

EXPERIMENTAL

Drug: Xcopri

Interventions

Xcopri DRUG

Age groups 12 to \<18 will enroll once dosing data is received from Cohort I of the PK analysis of YKP3089C039 study. Age groups 6 to \<12 will enroll once dosing data is received from Cohort IIa of the PK analysis of YKP3089C039 study. Age groups 4 to \<6 will enroll once dosing data is received from Cohort IIb of the PK analysis of YKP3089C039 study. Age groups 2 to \<4 will enroll once dosing data is received from Cohort III of the PK analysis of YKP3089C039 study.

12 to < 18 year olds; 2 to <4 years old; 4 to <6 years old; 6 to <12 years old

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Have a diagnosis of epilepsy with partial-onset (focal) seizures (POS) with or without secondarily generalized seizures according to the International League Against Epilepsy's (ILAE) Classification of Epileptic Seizures. A diagnosis should have been established at least 12 months prior to Visit 1 (Screening) by clinical history and an electroencephalogram (EEG) that is consistent with the diagnosis; normal interictal EEGs will be allowed provided that the participant meets the other diagnosis criterion (i.e., clinical history)
• Male or female participant, from age 2 to less than 18 years at the time of informed consent/assent (dates including informed consent in YKP3089C039)
• Have a minimum weight of 10.0 kilograms (kg) (22.0 pounds \[lb\])
• Have had a brain imaging (e.g., magnetic resonance imaging \[MRI\] scan or computed tomography (CT) within 10 years before Visit 1 (Screening) that ruled out a progressive cause of epilepsy.
• Are currently being treated with stable doses of 1 to a maximum of 3 approved antiepileptic drugs (AEDs). Doses must be stable for at least 4 weeks before to Visit 1 (Screening). A vagal nerve stimulator \[VNS\] will not be counted as one of the 3 allowed AEDs but the settings should be stable for at least 4 weeks prior to Visit 1 (Screening).
• Investigator believes subject could benefit from new or continued exposure to study drug
• Subjects receiving felbamate as a concomitant AED must meet the following criteria:
• Have a 12-month history of felbamate use and a history of a fixed dosing regimen for a minimum of 60 days prior to Visit 1 (Screening).
• No prior or known history of hepatotoxicity or hematologic disorder due to felbamate.
• Subjects following a ketogenic diet will be allowed as long as the diet has been stable for at least 30 days prior to Visit 1 (Screening) and will remain stable for the duration of the study

You may not qualify if:

• Females who are breastfeeding or pregnant at Screening or Baseline.
• Current or history of pseudo-seizures (psychogenic nonepileptic seizures) within approximately 2 years before Visit 1 (Screening).
• Have a history of status epilepticus that required hospitalization during the 6 months before Visit 1 (Screening).
• Have an unstable psychiatric diagnosis that may confound participants' ability to participate in the study or that may prevent completion of the protocol-specified tests (e.g., significant suicide risk, including suicidal behavior and ideation within 6 months before Visit 1 (Screening), current psychotic disorder, acute mania).
• Any suicidal ideation with intent, with or without a plan within 6 months before Visit 2 \[i.e., answering "Yes" to questions 4 or 5 on the Suicidal Ideation section of the Columbia-Suicide Severity Rating Scale (C-SSRS) in participants aged 6 and above, if able\].
• Are scheduled and/or confirmed to have epilepsy surgery within 6 months after Visit 1 (Screening); however, those who have previously documented "failed" epilepsy surgery will be allowed.
• Evidence of clinically significant disease (e.g., cardiac, respiratory, gastrointestinal, renal disease) that in the opinion of the investigator(s) could affect the participant's safety or interfere with the study assessments.
• Presence of only nonmotor simple partial seizures or primary generalized epilepsies.
• Evidence of moderate or severe renal insufficiency as defined by estimated glomerular filtration rates (eGFRs) of 31 to \< 60 "milliliters per minute (mL/min)" and \< 30 mL/min, respectively.
• Evidence of significant active hepatic disease. Stable elevation of liver enzymes, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) due to concomitant medication(s), will be allowed if they are less than 3 times the upper limit of normal (ULN).
• Evidence of significant active hematological disease; white blood cell (WBC) count equal or less than 2500/µL (2.50 1E+09/liter \[L\]) or an absolute neutrophil count equal or less than 1000/µL (1.00 1E+09/L).
• Subjects with Familial short QT syndrome.
• Clinically significant electrocardiogram (ECG) abnormality, including prolonged corrected QT interval (QTc) defined as greater than 450 milliseconds (msec) or shortened corrected QT interval (QTc) defined as less than 340 msec.
• Have a progressive central nervous system (CNS) disease, including degenerative CNS diseases and progressive tumors.
• Subject has a history of any serious drug-induced hypersensitivity reaction (including, but not limited to, Stevens Johnson syndrome, toxic epidermal necrolysis, or DRESS) or any drug-related rash requiring hospitalization.

Sponsors & Collaborators

• SK Life Science, Inc.: lead

Study Sites (54)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Lucile Packard Children's Hospital Stanford

Palo Alto, California, 94304, United States

Location

University of California Davis Health

Sacramento, California, 95817, United States

Location

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

Augusta University Medical Center

Augusta, Georgia, 30912, United States

Location

Clinical Integrative Research Center of Atlanta

Sandy Springs, Georgia, 30328, United States

Location

Meridian Clinical Research - Savannah Neurology Specialists

Savannah, Georgia, 31406, United States

Location

Kentucky Clinic

Lexington, Kentucky, 40536, United States

Location

Mid-Atlantic Epilepsy and Sleep Center

Bethesda, Maryland, 20817, United States

Location

Spectrum Health Hospitals Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

University of Missouri Health Care - Women's and Children's Hospital

Columbia, Missouri, 65212, United States

Location

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

Location

Northeast Regional Epilepsy Group - Morristown

Morristown, New Jersey, 07960, United States

Location

Boston Children's Health Physicians - Neurology at Hawthorne

Hawthorne, New York, 10532, United States

Location

Duke University Hospital

Durham, North Carolina, 27710, United States

Location

Akron Children's Hospital NeuroDevelopmental Science Center/Pediatric Neurology

Akron, Ohio, 44308, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Cleveland Clinic Main Campus

Cleveland, Ohio, 44195, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

Child Neurology Consultants of Austin

Austin, Texas, 78731, United States

Location

Scottish Rite for Children

Dallas, Texas, 75219, United States

Location

MultiCare Institute - Mary Bridge Children's Neurology

Tacoma, Washington, 98405, United States

Location

Austin Health

Heidelberg, Australia

Location

Royal Children's Hospital Melbourne

Parkville, Australia

Location

Sydney Children's Hospital - Randwick

Randwick, Australia

Location

Children's Health Queensland Hospital and Health Service

South Brisbane, Australia

Location

Charite University Hospital

Berlin, Germany

Location

Diakonie Kork

Kehl, Germany

Location

Universitätsklinikum Schleswig-Holstein - Campus Kiel

Kiel, Germany

Location

Neurologische Klinik und Poliklinik Interdisziplinäres Epilepsiezentrum München

Munich, Germany

Location

Universitätsklinikum Tübingen

Tübingen, Germany

Location

Bethesda Gyermekkorhaz

Budapest, Hungary

Location

Országos Klinikai Idegtudományi Intézet, Neurológiai Osztály

Budapest, Hungary

Location

Semmelweis University Dept. Of Paediatrics

Budapest, Hungary

Location

Servus Salvus Egészségügyi Szolgáltató Kft.

Budapest, Hungary

Location

Debreceni Egyetem Klinikai Központ

Debrecen, Hungary

Location

Niepubliczny Zaklad Opieki Zdrowotnej - Centrum Neurologii Dzieciecej i Leczenia Padaczki

Kielce, Poland

Location

Centrum Medyczne Plejady

Krakow, Poland

Location

Wojewódzki Specjalistyczny Szpital Dziecięcy im. św. Ludwika w Krakowie

Krakow, Poland

Location

Chungbuk National University Hospital

Cheonju, South Korea

Location

Korea University Guro Hospital

Seoul, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Severance Hospital

Seoul, South Korea

Location

SMG-SNU Boramae Medical Center

Seoul, South Korea

Location

Ajou University Hospital

Suwon, South Korea

Location

Hospital Sant Joan de Déu Barcelona

Barcelona, Spain

Location

Hospital Universitari Vall d'Hebrón

Barcelona, Spain

Location

Hospital Infantil Universitario Niño Jesús

Madrid, Spain

Location

Hospital Universitario La Paz

Madrid, Spain

Location

Clinica Universidad de Navarra - Pamplona

Pamplona, Spain

Location

Instituto de Investigación Sanitaria de la Fundación Ramón Domínguez

Santiago de Compostela, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Spain

Location

MeSH Terms

Conditions

Epilepsies, Partial; Seizures

Interventions

Cenobamate

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Marc Kamin, MD

  SK Life Science, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2021
• First Posted: October 5, 2021
• Study Start: January 14, 2022
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: March 25, 2026

Record last verified: 2025-09

Locations

ES (7); PL (3); DE (5); HU (5); US (24); AU (4); KR (6)