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    Summary
    EudraCT Number:2022-001256-42
    Sponsor's Protocol Code Number:YKP509C003
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2023-01-17
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGreece - EOF
    A.2EudraCT number2022-001256-42
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Carisbamate (YKP509) as Adjunctive Treatment for Seizures Associated with Lennox-Gastaut Syndrome in Children and Adults, with Optional Open- Label Extension
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to evaluate safety and efficacy of Caribasmate
    A.4.1Sponsor's protocol code numberYKP509C003
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05219617
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSK Life Science, Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSK Life Science, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSK Life Science, Inc
    B.5.2Functional name of contact pointExecutive Director Clinical Develop
    B.5.3 Address:
    B.5.3.1Street Address461 From Road, 5th Floor Paramus
    B.5.3.2Town/ cityNJ
    B.5.3.3Post code07652
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1503212-4795
    B.5.6E-mailanasser@sklsi.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCarisbamate
    D.3.2Product code YKP509
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCARISBAMATE
    D.3.9.2Current sponsor codeYKP509
    D.3.9.3Other descriptive nameCarisbamate (S)-2-O-carbamoyl-1-o-chlorophenyl-ethanol
    D.3.9.4EV Substance CodeSUB130483
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Lennox-Gastaut syndrome (LGS)
    E.1.1.1Medical condition in easily understood language
    Lennox-Gastaut syndrome (LGS)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10048816
    E.1.2Term Lennox-Gastaut syndrome
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of carisbamate (YKP509) as adjunctive treatment in reducing the number of drop seizures (tonic, atonic, tonic-clonic) compared with placebo in pediatric and adult subjects (age 4-55 years) diagnosed with Lennox Gastaut Syndrome (LGS)
    E.2.2Secondary objectives of the trial
    • 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.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must have a documented history of Lennox-Gastaut syndrome by:
    a. Evidence of more than one type of seizure, of which at least one should be an atonic or tonic seizure
    b. History of an electroencephalogram (EEG) reporting diagnostic criteria for LGS (abnormal background activity accompanied by slow, spike and wave pattern <3.0 Hz)
    c. 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 8 drop seizures with
    potential to fall (tonic, atonic, tonic-clonic) during the 4-week Baseline period preceding randomization (minumim of 4 drop seizures in the first two weeks and 4 in the last two weeks). Drop seizures are defined as a countable 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 2 weeks after dose of study drug.
    11. Subject and/or caregiver/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.
    E.4Principal 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 12 months of continuous exposure
    6. Subjects who took vigabatrin in the past must have discontinued it at least 5 months before Visit 1. Subjects taking vigabatrin should have documentation showing no evidence of a vigabatrin-associated visual field abnormality.
    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 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 agespecific Columbia-Suicide Severity Rating Scale (C-SSRS) in subjects aged 6 years 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 participated in any clinical study of an investigational product or device in the 30 days prior to the screening visit (Visit 1)
    23. Concomitant use of medications known to be strong inducers of cytochrome UGT enzymes 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 QTcF interval (<340 msec) or long QTcF interval (>460 msec) as confirmed by a repeated electrocardiogram (ECG)
    26. Intermittent benzodiazepine rescue administered more than four times in the 28 days prior to Visit 1 (1 to 2 doses in a 24-hour period is considered as one rescue).
    27. Previous exposure to carisbamate or sensitivity/allergy to components of the oral suspension.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome will be the percentage change from baseline in the total frequency (average per 28 days) of countable drop seizures with potential to fall (tonic, atonic, tonic-clonic) during thethe double-blind treatment period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the double-blind treatment period.
    E.5.2Secondary end point(s)
    1. The percentage of subjects with at least a 50% reduction from baseline in the total frequency of countable drop seizures (tonic, atonic, tonic-clonic) during the double-blind treatment period.
    2. Percentage change from baseline in the frequency of all types of seizures (total seizures) during the double-blind treatment period.
    3. Subject/Caregiver Global Impression of Change in overall condition (S/CGIC) score
    at the last visit.

    Other Secondary Endpoints:
    1. Percentage change from baseline in the 28-day frequency of
    a. drop seizures (tonic, atonic, tonic-clonic),
    b. non-drop seizures (myoclonic seizures, atypical absence),
    c. total seizures during the maintenance phase of the double-blind
    treatment period.
    2. Percentage change from baseline in non-drop seizure frequency
    (myoclonic seizures, atypical absence) per 28 days during the double-blind treatment period.
    3. The percentage of subjects with at least a 50% reduction from
    baseline in the total frequency of drop seizures (tonic, atonic, tonic-clonic) during the maintenance phase of the double-blind treatment period.
    4. Proportion of subjects with a 75%, 90% and 100% response rate for
    total frequency of drop seizures (tonic, atonic, tonic-clonic), non-drop
    seizures, and total seizures during the maintenance phase the double-blind treatment period.
    Proportion of subjects with a 75%, 90% and 100% response rate for for total frequency of drop seizures (tonic, atonic, tonic-clonic), non-drop seizures, and total seizures during the double-blind treatment period.

    Exploratory Endpoints:
    1. Time to event: The time to experience the same total number of
    countable drop seizures in the double-blind phase that was observed
    during the baseline phase (per 28 days) for each subject.
    2. Quality of Life (QOL) based on standardized measures such as:
    a. Vineland Adaptive Behavior Scale, for pediatric and adult subjects,
    when able to be assessed and only in English.
    b. Behavior Rating Inventory of Executive Function scale (BRIEF)
    Version 2 – aged 5 to 18nyears old, when able to be assessed and only in English.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. During the double-blind treatment period
    2. During the double-blind treatment period
    3. At last visit

    Other Secondary Endpoints:
    1. During the maintenance phase of the double-blind treatment period
    2. During the double-blind treatment period
    3. During the maintenance phase of the double-blind treatment period
    4. During the double-blind treatment period
    5. During the maintenance phase of the double-blind treatment period
    6. Screening, maintenance phase and EoT/EoS
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Quality of life
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Optional open-Label extension
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned Information not present in EudraCT
    E.8.4 The trial involves multiple sites in the Member State concerned Information not present in EudraCT
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Colombia
    Taiwan
    Australia
    Korea, Republic of
    Mexico
    Serbia
    United States
    Germany
    Greece
    Hungary
    Italy
    Poland
    Portugal
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 168
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 102
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 66
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 84
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 252
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Expanded Access Programs will be provided following the Open-label Extension.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2023-04-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-05-30
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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