Dose-Escalation Study of Cenobamate (YKP3089) in Pediatric Subjects With Partial-Onset Seizures

NCT04903314 | Completed Phase 1 FDA Drug

• 1 other identifier: YKP3089C039
• Study Type: interventional
• Target: 26
• Locations: 3 countries
• Sites: 13

Brief Summary

The primary objective of this study is to assess the pharmacokinetics of cenobamate (YKP3089) in pediatric subjects with partial-onset (focal) seizures following single and multiple-dosing.

Conditions

Partial Epilepsy

Keywords

Partial-onset (focal) seizures; Pediatrics

Outcome Measures

Primary Outcomes (2)

• The area under the curve (AUC) of Xcopri after a single and multiple doses of Xcopri

  Safety Assessment

  18 Months

• The maximum plasma concentration (Cmax) after a single and multiple doses of Xcopri

  Safety Assessment

  18 Months

Secondary Outcomes (1)

• Safety - adverse events (AEs) reporting after a single and multiple doses of Xcopri

  18 Months

Study Arms (4)

Cohort I

EXPERIMENTAL

Xcopri to be administered to ages 12 to \< 18 years not to exceed 400 mg/day.

Drug: Xcopri

Cohort IIa

EXPERIMENTAL

Xcopri to be administered to ages 6 to \< 12 years not to exceed 400 mg/day.

Drug: Xcopri

Cohort IIb

EXPERIMENTAL

Xcopri to be administered to ages 4 to \< 6 years not to exceed 400 mg/day.

Drug: Xcopri

Cohort III

EXPERIMENTAL

Xcopri to be administered to ages 2 to \< 4 years not to exceed 400 mg/day.

Drug: Xcopri

Interventions

Xcopri DRUG

Xcopri will be administered orally not to exceed 400mg/day adult equivalent

Cohort I; Cohort III; Cohort IIa; Cohort IIb

Eligibility Criteria

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

You may qualify if:

• Diagnosis of epilepsy with partial-onset 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 6 months prior to Visit 1 by clinical history and an electroencephalogram (EEG) that is consistent with the diagnosis; normal interictal EEGs will be allows provided that the participant meets the other diagnosis criterion (i.e., clinical history, including a history of treatment failure with at least 2 AEDs)
• Male or female subjects, from age 2 to less than 18 years at the time of informed consent
• Have a minimum weight of 10.0 kilograms (kg) (22.0 pounds \[lb\])
• Written informed consent signed by the subject, legal guardian, or legally authorized representative (LAR) prior to entering the study in accordance with the ICH GCP guidelines. Age appropriate assent will be obtained for children and adolescents. If the written informed consent is provided by the legal guardian or LAR because the subject is unable to do so, a written or verbal assent from the subject must also be obtained
• Are currently being treated with stable doses of 1 to a maximum of 2 approved antiepileptic drugs (AEDs). Doses must be stable for at least 4 weeks before to Visit 1; in the case where a new AED regimen has been initiated for a participant, the dose must be stable for at least 8 weeks prior to Visit 1. A vagal nerve stimulator (VNS) will not be counted as one of the 2 allowable AEDs
• In the Investigator's opinion, parents or caregivers must be able to report accurate seizure assessments during the screening and study periods and subjects must be able to ingest study drug
• Subjects with an implanted vagal nerve stimulator will be allowed if the vagal nerve stimulator was implanted at least 5 months prior to Visit 1 (Screening) and the stimulator parameters have not been changed for 30 days prior to Visit 1 and for the duration of the study
• 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:

• Progressive neurological disease, including degenerative CNS diseases and progressive tumors
• Evidence of clinically significant disease or any medical condition that would compromise the subject's ability to safely complete the study including, but not limited to, hepatic or renal failure, ischemic disease, human immunodeficiency virus (HIV) infection, active sexually transmitted disease (STD), active viral hepatitis, or malignancy
• Positive urine screen of drugs of abuse (if not due to concomitant medication, e.g., benzodiazepines as hypnotics) for Cohort 1 subjects.
• History of anoxic episodes require resuscitation within 6 months before Visit 1, drug or alcohol dependency or abuse within approximately the last 2 years or use of illegal recreational drugs.
• Any surgical or medical condition that may interfere with the absorption, distribution, metabolism, or excretion of the investigational product
• Consumption of any caffeine-containing products (e.g., coffee, tea, chocolate, or soda) or alcoholic beverages within 72 hours before Day 1 and 72 hours before the day of multiple dose PK sampling (Day 59 for Cohort I)
• Consumption of grapefruit or grapefruit-containing products within 72 hours before Day 1 and 72 hours before the day of multiple dose PK sampling (Day 59 for Cohort I)
• Significant clinical laboratory abnormalities, including elevation of serum AST or ALT more than 2 times the upper limit or normal (ULN) for each age group.
• Acute disease state (e.g., nausea, vomiting, fever, or diarrhea) within 7 days before Day 1
• Scheduled for surgery during the study
• Ketogenic diet or vagal nerve stimulation that has undergone alteration within 30 days of Visit 1
• Treatment with an investigational drug or device (other than VNS) ≤ 30 days before Visit 1
• Females who are breastfeeding or pregnant at Screening or Baseline or who are of reproductive age and do not agree to be abstinent or to use highly effective methods of contraception
• Current or history of pseudo-seizures (psychogenic nonepileptic seizures) within approximately 5 years before Visit 1
• Have a history of status epilepticus that required hospitalization during the 6 months before Visit 1

Sponsors & Collaborators

• SK Life Science, Inc.: lead

Study Sites (13)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Mid-Atlantic Epilepsy and Sleep Center

Bethesda, Maryland, 20817, United States

Location

Missouri University Pediatric and Adolescent Specialty Clinic

Columbia, Missouri, 65201, United States

Location

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

MultiCare Institute - Mary Bridge Children's Neurology

Tacoma, Washington, 98405, United States

Location

I. Sz. Gyermekgyógyászati Klinika

Budapest, Hungary

Location

Chungbuk National University Hospital

Cheonju, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Severance Hospital

Seoul, South Korea

Location

Ajou University Hospital

Suwon, South Korea

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

• Sunita Misra, MD

  SK Life Science, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Cohort I will enroll 6 subjects. Upon review of Cohort I's PK analysis, a dose will be determined for Cohort IIa. Upon review of Cohot IIa's PK analysis, a dose will be determined for Cohort 11b and Cohort III.

Study Record Dates

• First Submitted: May 21, 2021
• First Posted: May 26, 2021
• Study Start: May 27, 2021
• Primary Completion: November 6, 2025
• Study Completion: November 6, 2025
• Last Updated: March 31, 2026

Record last verified: 2025-09

Locations

KR (4); HU (1); US (8)