Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults

NCT ID: NCT05219617

Last Updated: 2025-07-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

252 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-28

Study Completion Date

2028-12-31

Brief Summary

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The primary objective is to evaluate the efficacy of carisbamate (YKP509) as adjunctive treatment in reducing the number of drop seizures (tonic, atonic, and tonic-clonic) compared with placebo in pediatric and adult subjects (age 4-55 years) diagnosed with Lennox Gastaut Syndrome (LGS).

Detailed Description

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The secondary objectives are:

* To evaluate the efficacy of carisbamate (YKP509) as adjunctive treatment in reducing the total number of seizures compared with placebo in pediatric and adult subjects diagnosed with Lennox Gastaut Syndrome (LGS)
* Evaluate the safety, tolerability of carisbamate in the LGS population
* Evaluate steady-state pharmacokinetics of carisbamate in subjects with Lennox Gastaut.

Conditions

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Seizures Lennox Gastaut Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This double-blind, randomized, placebo-controlled study will evaluate the efficacy of carisbamate 200 mg BID or the pediatric equivalent dose and 300 mg BID or the pediatric equivalent dose for the treatment of seizures associated with Lennox Gastaut syndrome in subjects 4 to 55 years of age.
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Caregivers
Double-blind study

Study Groups

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Carisbamate 200 mg BID arm

Age: 4 to \<12y\* Titration: 2 mg/kg BID Maintenance: 4 mg/kg BID

Age: ≥12 y Titration: 100 mg BID Maintenance: 200 mg BID

Group Type EXPERIMENTAL

Carisbamate

Intervention Type DRUG

Adolescent subjects 12 to 18 years old will receive the same dose as adults. Subjects 4 to \< 12 years old in the carisbamate 200 mg BID arm will receive 4 mg/kg BID (not to exceed 200 mg BID \[or a total of 400 mg per day\]).

Subjects 4 to \< 12 years old in the carisbamate 300 mg BID arm will receive 5.5 mg/kg BID (not to exceed 300 mg BID \[or a total of 600 mg per day\]).

Carisbamate 300 mg BID arm

Age: 4 to \<12y\* Titration: 2.75 mg/kg BID Maintenance: 5.5 mg/kg BID

Age: ≥12 y Titration: 150 mg BID Maintenance: 300 mg BID

Group Type EXPERIMENTAL

Carisbamate

Intervention Type DRUG

Adolescent subjects 12 to 18 years old will receive the same dose as adults. Subjects 4 to \< 12 years old in the carisbamate 200 mg BID arm will receive 4 mg/kg BID (not to exceed 200 mg BID \[or a total of 400 mg per day\]).

Subjects 4 to \< 12 years old in the carisbamate 300 mg BID arm will receive 5.5 mg/kg BID (not to exceed 300 mg BID \[or a total of 600 mg per day\]).

Placebo matched to 200 mg BID arm

Age: 4 to \<12y\* Titration: Volume equivalent to 2 mg/kg BID Maintenance: Volume equivalent to 4 mg/kg BID

Age: ≥12 y Titration: Volume equivalent to 100 mg BID Maintenance: Volume equivalent to 200 mg BID

Group Type PLACEBO_COMPARATOR

Carisbamate

Intervention Type DRUG

Adolescent subjects 12 to 18 years old will receive the same dose as adults. Subjects 4 to \< 12 years old in the carisbamate 200 mg BID arm will receive 4 mg/kg BID (not to exceed 200 mg BID \[or a total of 400 mg per day\]).

Subjects 4 to \< 12 years old in the carisbamate 300 mg BID arm will receive 5.5 mg/kg BID (not to exceed 300 mg BID \[or a total of 600 mg per day\]).

Placebo matched to 300 mg BID arm

Age: 4 to \<12y\* Titration: Volume equivalent to 2.75 mg/kg BID Maintenance: Volume equivalent to 5.5 mg/kg BID

Age: ≥12 y Titration: Volume equivalent to 150 mg BID Maintenance: Volume equivalent to 300 mg BID

Group Type PLACEBO_COMPARATOR

Carisbamate

Intervention Type DRUG

Adolescent subjects 12 to 18 years old will receive the same dose as adults. Subjects 4 to \< 12 years old in the carisbamate 200 mg BID arm will receive 4 mg/kg BID (not to exceed 200 mg BID \[or a total of 400 mg per day\]).

Subjects 4 to \< 12 years old in the carisbamate 300 mg BID arm will receive 5.5 mg/kg BID (not to exceed 300 mg BID \[or a total of 600 mg per day\]).

Interventions

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Carisbamate

Adolescent subjects 12 to 18 years old will receive the same dose as adults. Subjects 4 to \< 12 years old in the carisbamate 200 mg BID arm will receive 4 mg/kg BID (not to exceed 200 mg BID \[or a total of 400 mg per day\]).

Subjects 4 to \< 12 years old in the carisbamate 300 mg BID arm will receive 5.5 mg/kg BID (not to exceed 300 mg BID \[or a total of 600 mg per day\]).

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Subject must have a documented history of Lennox-Gastaut syndrome by:

1. Evidence of more than one type of seizure, of which at least one should be an atonic or tonic seizure
2. History of an electroencephalogram (EEG) reporting diagnostic criteria for LGS (abnormal background activity accompanied by slow, spike and wave pattern \<3.0 Hz)
3. History of developmental delay
2. Male or female subjects
3. Subjects must be age 4-55 years at the time of consent/assent
4. Must have been \<11 years old at the onset of LGS
5. Subjects must have experienced at least 2 drop seizures with potential to fall (tonic, atonic, tonic-clonic) during the 4-week Baseline period preceding randomization (minimum of 4 drop seizures in the first two weeks and 4 in the last two weeks). Drop seizures are defined as a seizure involving the entire body, trunk, or head that led or could have led to a fall, injury, slumping in a chair, or hitting the subject's head on a surface. All drop seizure types must be countable (either as isolated seizures or as countable isolated seizures in a cluster).
6. Subjects must have been receiving 1 to 4 concomitant anti-seizure medications (ASMs) at a stable dose for at least 4 weeks before Visit 1
7. If not taking Epidiolex, subjects may take other approved cannabidiol or over the counter cannabidiol products. If taking cannabidiol other than Epidiolex, consult Medical Monitor to determine if it counts as a concomitant ASM.
8. Dietary therapy and any CNS stimulator settings must be stable for 4 weeks prior to baseline and maintain stable regimen throughout the study. The dietary therapy and CNS stimulators are not counted as an ASM.
9. Parents or caregivers must be able to keep accurate seizure diaries
10. Subject is either not of childbearing potential, defined as premenarchal, postmenopausal for at least 1 year or surgically sterile (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy), if of childbearing potential, must comply with an acceptable method of birth control during the study, for at least 4 weeks prior to study entry and for 4 weeks following completion of the study, if able.
11. Subject and/or caregiver(s)/legal representative must be willing and able to give informed assent/consent for participation in the study
12. Subject and their caregiver must be willing and able (in the investigator's opinion) to comply with all study requirements
13. History of COVID-19 vaccination is permitted

Exclusion Criteria

1. Etiology of subject's seizures is a progressive neurologic disease. Subjects with tuberous sclerosis will not be excluded from study participation, unless there is a progressive brain tumor
2. Evidence of clinically significant disease (e.g., cardiac, respiratory, gastrointestinal, renal disease, hepatic disease) that in the opinion of the investigator(s) could affect the subject's safety or study conduct
3. Subjects who were on adrenocorticotropic hormone (ACTH) therapy in the 6 months prior to baseline
4. Subject on dietary therapy for less than 4 weeks prior to screening visit (Visit 1) or suffers from frequent stooling
5. Current use of felbamate with less than 18 months of continuous exposure
6. Concomitant use of vigabatrin: subjects who took vigabatrin in the past must be discontinued for at least 5 months before Visit 1 and must have documentation showing no evidence of a vigabatrin-associated clinically significant abnormality in an automated visual perimetry test, if able.
7. Subject who had a history of hypoxia which needed emergency resuscitation within 12 months prior to baseline
8. Status epilepticus within 12 weeks prior to Visit 1
9. Any clinically significant illness (including COVID-19) in the 4 weeks prior to Visit 1, as evaluated by the Investigator
10. Subject has clinically significant abnormal laboratory values, in the investigator's opinion, at Visit 1 or time of randomization (Visit 2)
11. Subject has a history of any serious drug-induced hypersensitivity, e.g., toxic epidermal necrolysis, or Drug Reaction with Eosinophilia and Systemic Symptoms \[DRESS\]) or any drug-related rash requiring hospitalization
12. Vagus Nerve Stimulation (VNS), Deep Brain Stimulation (DBS), Responsive Neurostimulator System (RNS) or other neurostimulation for epilepsy device implanted or activated \<5 months year prior to enrollment. Stimulation parameters that have been stable for \<4 weeks, or Battery life of unit not anticipated to extend for duration of trial.
13. Subject is pregnant, may be pregnant, lactating or planning to be pregnant
14. 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 in the Suicidal Ideation section of the age- specific Columbia-Suicide Severity Rating Scale (C-SSRS) in subjects aged 6 and above who are able to be evaluated
15. Any suicidal behavior within 2 years before Visit 2 (i.e., answering YES to any question in the Suicidal behavior section of the age-specific Columbia-Suicide Severity Rating Scale (C-SSRS) in subjects aged 6 and above who are able to be evaluated.
16. Evidence of significant active hepatic disease. Stable elevations of liver enzymes (alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) due to concomitant medication(s) will be allowed if they are \<3 x ULN
17. Subject with total bilirubin \[TBL\] \>2 x ULN (except for Gilbert's syndrome).
18. Active viral hepatitis (B or C) as demonstrated by positive serology at the Screening visit (Visit 1)
19. History of positive antibody/antigen test for human immunodeficiency virus (HIV)
20. If taking Epidiolex, subject may not use other approved cannabidiol or over the counter cannabidiol products
21. Scheduled for epilepsy-related surgery, VNS insertion, or any other stimulators/surgery during the projected course of the study
22. Subject who has taken or used any investigational drug or device in the 4 weeks prior to the screening visit (Visit 1)
23. Concomitant use of medications known to be strong inducers of cytochrome P450 (CYP3A) including, but not limited to: phenobarbital, phenytoin, carbamazepine, primidone, rifampin, troglitazone, St. John's Wort, efavirenz, nevirapine, glucocorticoids (other than topical usage), modafinil, pioglitazone, and rifabutin
24. Evidence of cardiac disease, including unstable angina, myocardial infarction, within the past 2 years, uncontrolled heart failure, major arrhythmias, congenital short QT syndrome
25. Subject with a short QTc interval (\<340 msec) or long QTc interval (\>460 msec) as confirmed by a repeated electrocardiogram (ECG)
26. Benzodiazepine rescue administered on average more than once a week in the month before Visit 1
27. Previous exposure to carisbamate or sensitivity/allergy to components of the oral suspension.
Minimum Eligible Age

4 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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SK Life Science, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc Kamin, MD

Role: STUDY_DIRECTOR

SK Life Science, Inc.

Locations

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Stanford University Hospital

Palo Alto, California, United States

Site Status RECRUITING

University of Florida Health Science Center

Jacksonville, Florida, United States

Site Status COMPLETED

AdventHealth

Orlando, Florida, United States

Site Status COMPLETED

Pediatric Epilepsy and Neurology Specialists

Tampa, Florida, United States

Site Status RECRUITING

University of South Florida

Tampa, Florida, United States

Site Status COMPLETED

Axcess Medical Research

Wellington, Florida, United States

Site Status COMPLETED

Consultants in Epilepsy and Neurology PLLC

Boise, Idaho, United States

Site Status COMPLETED

Bluegrass Epilepsy Research, LLC

Lexington, Kentucky, United States

Site Status COMPLETED

University Medical Center New Orleans

New Orleans, Louisiana, United States

Site Status COMPLETED

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status RECRUITING

Mid-Atlantic Epilepsy and Sleep Center

Bethesda, Maryland, United States

Site Status RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

Site Status COMPLETED

University of Missouri School of Medicine

Columbia, Missouri, United States

Site Status COMPLETED

Northeast Regional Epilepsy Group

Hackensack, New Jersey, United States

Site Status RECRUITING

St. Peters Hospital

New Brunswick, New Jersey, United States

Site Status COMPLETED

Montefiore

The Bronx, New York, United States

Site Status COMPLETED

Duke University Clinical Research at Pickett Road

Durham, North Carolina, United States

Site Status RECRUITING

Wake Forest University - School of Medicine

Winston-Salem, North Carolina, United States

Site Status COMPLETED

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Austin Epilepsy Care Center - Clinic/Outpatient Facility

Austin, Texas, United States

Site Status COMPLETED

Neurology Consultants of Dallas, PA - Hospital

Dallas, Texas, United States

Site Status COMPLETED

Virginia Epilepsy and Neurodevelopmental Clinic at WNC

Winchester, Virginia, United States

Site Status COMPLETED

Hospital de Ninos de La Santisma Trinidad

Córdoba, Córdoba Province, Argentina

Site Status COMPLETED

Resolution Psychopharmacology Research Institute

Mendoza, Mendoza Province, Argentina

Site Status COMPLETED

Austin Hosptial

Heidelberg, , Australia

Site Status RECRUITING

Alfred Health

Melbourne, , Australia

Site Status RECRUITING

Perth's Children Hospital

Nedlands, , Australia

Site Status COMPLETED

Queensland Children's Hospital

South Brisbane, , Australia

Site Status COMPLETED

Fundacion Hospital Universidad del Norte

Barranquilla, , Colombia

Site Status WITHDRAWN

Fundacion Valle del Lili/Clinic - Outpatient

Cali, , Colombia

Site Status WITHDRAWN

CliniSalud del Sur S.A.S - Centro de Investigación

Envigado, , Colombia

Site Status COMPLETED

Hospital Pabloe Tubon Uribe

Medellín, , Colombia

Site Status COMPLETED

Institutio Neurologico de Colombia

Medellín, , Colombia

Site Status RECRUITING

Universitatsklinikum Erangen

Erlangen, Bavaria, Germany

Site Status RECRUITING

Kleinwachau Sächsisches Epilepsiezentrum

Radeberg, Saxony, Germany

Site Status RECRUITING

Iaso Children's Hospital

Marousi, Attica, Greece

Site Status RECRUITING

Orszagos Mentalis, Ideggyogyaszati es Idegsebezeti Intezet

Budapest, , Hungary

Site Status COMPLETED

Semmelweis Egyetem Idegsebeszeti es Neurointervencios Klinika

Budapest, , Hungary

Site Status RECRUITING

Tela Viv Sourlasky Medical Center

Tel Aviv, Tel Aviv, Israel

Site Status COMPLETED

Soroka University Medical Centre

Beersheba, , Israel

Site Status COMPLETED

Hadassah Medical Center

Jerusalem, , Israel

Site Status COMPLETED

Sheba Medical Center

Ramat Gan, , Israel

Site Status COMPLETED

Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

Genoa, Liguria, Italy

Site Status RECRUITING

ASST Fatebenefratelli Sacco - Ospedale dei Bambini Vittore Buzzi

Milan, Lombardy, Italy

Site Status RECRUITING

Fondazione IRCCS Di Rilievo Nazionale Instituto

Milan, Lombardy, Italy

Site Status RECRUITING

Azienda Ospedaliera Universitaria Integrata Di Verona

Verona, Verona, Italy

Site Status RECRUITING

Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN

Florence, , Italy

Site Status RECRUITING

ASST Santi Paolo E Carlo - Azienda Universitaria-Polo Universitaria - San Paolo

Milan, , Italy

Site Status COMPLETED

Hospital Civil Fray Antonio Alcalde

Guadalajara, Jalisco, Mexico

Site Status RECRUITING

Neurociencias Estudios Clinicos S.C.

Culiacán, , Mexico

Site Status RECRUITING

Clinstile, S.A. de C.V.

Mexico City, , Mexico

Site Status RECRUITING

Szpital Kliniczny im.H.Swiecickiego Uniwersytetu Medycznego im.K.Marcinkowskiego w Poznaniu-Dluga1/2

Poznan, Greater Poland Voivodeship, Poland

Site Status COMPLETED

Centrum Medyczne Plejady

Krakow, , Poland

Site Status RECRUITING

Centro Hospitalar de Lisboa Norte, EPE

Lisbon, Lisbon District, Portugal

Site Status RECRUITING

Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Sao Francisco Xavier

Lisbon, Lisbon District, Portugal

Site Status COMPLETED

Centro Hospitalar de Sao Joao, EPE

Porto, Porto District, Portugal

Site Status RECRUITING

Hospital Garcia de Orta

Almada, Setúbal District, Portugal

Site Status COMPLETED

Childrens University Hospital

Belgrade, Belgrade, Serbia

Site Status RECRUITING

University Clinical Center of Serbia - PPDS

Belgrade, , Serbia

Site Status RECRUITING

University Clinical Center Kragujevac

Kragujevac, , Serbia

Site Status RECRUITING

University Clinical Center Nis

Niš, , Serbia

Site Status RECRUITING

Children and Youth Health Care Institute of Vojvodina

Novi Sad, , Serbia

Site Status RECRUITING

Kyungpook National University Chilgok Hospital

Daegu, , South Korea

Site Status RECRUITING

Samsung Medical Center

Seoul, , South Korea

Site Status RECRUITING

Seoul National University Hospital

Seoul, , South Korea

Site Status RECRUITING

Hospital Sant Joan de Deu - PIN

Esplugues de Llobregat, Barcelona, Spain

Site Status COMPLETED

Hospital Infantil Universitario Niño Jesus - PIN

Madrid, , Spain

Site Status RECRUITING

Hospital Ruber Internacional (Grupo Quironsalud)

Madrid, , Spain

Site Status RECRUITING

National Taiwan University Hospital

Taipei, , Taiwan

Site Status RECRUITING

Taipei Veterans General Hospital

Taipei, , Taiwan

Site Status RECRUITING

Chang Gung Memorial Hospital

Taoyuan District, , Taiwan

Site Status COMPLETED

Countries

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United States Argentina Australia Colombia Germany Greece Hungary Israel Italy Mexico Poland Portugal Serbia South Korea Spain Taiwan

Central Contacts

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Barbara Remes

Role: CONTACT

201-421-3810

Facility Contacts

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Arkady Barber

Role: primary

Anna Sakovics, MD

Role: primary

Tatjana Redzek Mudrinic, MD

Role: primary

Other Identifiers

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YKP509C003

Identifier Type: -

Identifier Source: org_study_id

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