Overview

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

Status:
Not yet recruiting
Trial end date:
2024-09-01
Target enrollment:
0
Participant gender:
All
Summary
Primary objective: To evaluate the safety and tolerability of cenobamate in pediatric subjects 2-17 years of age with partial-onset (focal) seizures
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
SK Life Science, Inc.
Treatments:
Cenobamate
Criteria
Inclusion Criteria:

1. 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 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)

2. 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)

3. Have a minimum weight of 12.5 kilograms (kg) (27.5 pounds [lb])

4. Have had a brain imaging (e.g., magnetic resonance imaging [MRI] scan or computed
tomography (CT) within 5 years before Visit 1 that ruled out a progressive cause of
epilepsy

5. For subjects new to Study YKP3089C040 during the 8 weeks prior to Visit 1,
participants must have had at least 1 POS seizure. Only simple POS with motor signs,
complex POS, and complex POS with secondary generalization are counted toward this
inclusion for POS

6. 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; 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 be counted as one of the 3 allowed AEDs, with the settings stable for at least 4
weeks prior to screening and maintain stable throughout the study.

7. Investigator believes subject could benefit from new or continued exposure to study
drug

8. Subjects must continue to meet all of the inclusion criteria from the YKP3089C039
study

9. Subjects receiving felbamate as a concomitant AED must meet the following criteria:

1. Have an 18-month history of felbamate use and a history of a fixed dosing
regimenfor a minimum of 60 days prior to Visit 1 (Screening/Baseline).

2. No prior or known history of hepatotoxicity or hematologic disorder due to
felbamate.

3. 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

Exclusion Criteria:

1. Females who are breastfeeding or pregnant at Screening or Baseline

2. Current or history of pseudo-seizures (psychogenic nonepileptic seizures) within
approximately 2 years before Visit 1.

3. Have a history of status epilepticus that required hospitalization during the 6 months
before Visit 1.

4. 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, current psychotic disorder, acute mania).

5. 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.

6. Are scheduled and/or confirmed to have epilepsy surgery within 6 months after Visit 1;
however, those who have previously documented "failed" epilepsy surgery will be
allowed.

7. 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.

8. 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.

9. 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).

10. 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).

11. Subjects with Familial short QT syndrome

12. 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 ms.

13. Have a progressive central nervous system (CNS) disease, including degenerative CNS
diseases and progressive tumors.

14. 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.

15. History of AED-associated rash that involved conjunctiva or mucosae.

16. History of more than one non-serious drug-related hypersensitivity reaction that
required discontinuation of the medication.

17. Concomitant use of vigabatrin. Participants who took vigabatrin in the past must be
off vigabatrin for at least 5 months before Visit 1 and with documentation showing no
evidence of a vigabatrin-associated clinically significant abnormality in a visual
perimetry test.

18. A history of intermittent use of rescue benzodiazepines (i.e., 1 to 2 doses over a
24-hourperiod is considered a 1- time rescue) more than once within the 30 days prior
to Visit1 (Screening/Baseline).

19. A VNS implanted less than 5 months before Visit 1 or changes in parameter less than 4
weeks before Visit 1 (or thereafter during the study)

20. History of or a concomitant medical condition that in the opinion of the
investigator(s) would preclude the participant's participation in a clinical study or
compromise the participant's ability to safely complete the study.

21. Have participated in a study involving administration of an investigational drug or
device within 4 weeks before Visit 1, or within approximately 5 half-lives of the
previous investigational compound, whichever is longer.

22. Participants with rare hereditary problems of galactose intolerance, the Lapp lactase
deficiency or glucose-galactose malabsorption.