Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruiting
Participants planned
131
Countries
5
Sites
12
Primary Generalized Tonic-Clonic Seizures
To evaluate the safety and tolerability of cenobamate in subjects with PGTC seizures
Key facts
- Sponsor
- Sk Life Science Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 9 Oct 2019 → ongoing
- Decision date (initial)
- 2024-05-14
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- SK Life Science, Inc.
External identifiers
- EU CT number
- 2023-506688-32-00
- EudraCT number
- 2018-002981-37
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Therapy
To evaluate the safety and tolerability of cenobamate in subjects with PGTC seizures
Secondary objectives 1
- N/A
Conditions and MedDRA coding
Primary Generalized Tonic-Clonic Seizures
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 26.1 | PT | 10018100 | Generalised tonic-clonic seizure | 100000004852 |
Study design 3 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Double-blind Conversion Period Subjects who have completed the Double-blind Treatment Period in the Core study, choose to participate in this OLE study and meet eligibility criteria for this study, will sign the informed consent form (ICF) for this study. After signing the ICF for this study, subjects will enter a 12 week Double-blind Conversion Period before starting the Open-label treatment. During the Double-blind Conversion Period adult subjects will receive study drug in tablet form and adolescent subjects, 12 to <18 years of age when they entered the core study will receive study drug as an oral suspension based on body weight to provide adolescent equivalent doses.
|
Not Applicable | Double | [{"id":157002,"code":4,"name":"Analyst"},{"id":157001,"code":2,"name":"Investigator"},{"id":157004,"code":5,"name":"Carer"},{"id":157005,"code":3,"name":"Monitor"},{"id":157003,"code":1,"name":"Subject"}] | |
| 2 | Open-label Maintenance Period "During this period, adult subjects will receive cenobamate starting at 200mg/day and adolescent subjects will receive the adolescent equivalent based on weight via oral suspension in an Openlabel manner.
The duration is 40 weeks and subjects will have 7 study visits during this period."
|
Not Applicable | None | ||
| 3 | Follow-up Period "After successful completion of the Open-label Maintenance Period, the Principal Investigator at his or her discretion may offer the opportunity to subjects to enroll in an expanded access study. For eligible subjects willing to enroll in the expanded access study, they or their legal guardians (if subject is unable to sign ICF) will be asked to review and sign a separate ICF for the expanded access study at the Visit 13 (End of Treatment) of this study. They will not enter the Follow-up Period for this study.
Subjects who successfully complete the Open-label Maintenance Period but do not enroll in the
expanded access study will enter the 3- week Follow-up Period."
|
Not Applicable | None |
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-002563-PIP02-19
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- "In addition to satisfying all the inclusion criterion established in YKP3089C025, each subject must meet the following inclusion criteria to be enrolled in the study: 1. The subject must have successfully completed the Double-blind Treatment Period in the Core study. 2. Written informed consent signed by the subject or legal guardian, prior to entering the study, in accordance with the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) guidelines. If the written informed consent is provided by the legal guardian because the subject is unable to do so, a written or verbal assent from the subject must also be obtained. As required by country-specific regulations, only the subject may sign the ICF in accordance with ICH guidelines."
Exclusion criteria 1
- "In addition to all the exclusion criterion established in YKP3089C025, subjects meeting any of the following criteria will be excluded from the study: 1. Subject tests positive, via urine drug screen at Visit 14 of the Core study, for illicit drugs except for tetrahydrocannabinol and Cannabinoids. 2. Any significant changes to the subject's medical history that, in the opinion of the Principal Investigator, could affect the subject's safety or conduct of the study. Any potential exception to the inclusion as well as exclusion criteria allowing de minimis (clinically trivial and meaningless) variations must be approved by the Medical Monitor."
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The primary endpoints are safety measures: the incidences of adverse events (AEs) and serious adverse events (SAEs); summary statistics for clinical laboratory test results and vital signs; and physical examination, neurologic examination, and electrocardiogram (ECG) findings.
Secondary endpoints 1
- N/A
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 5
PRD10986001 · Product
- Active substance
- Cenobamate
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 200 mg milligram(s)
- Max total dose
- 77000 mg milligram(s)
- Max treatment duration
- 55 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- SK LIFE SCIENCE, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD4240496 · Product
- Active substance
- Cenobamate
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 200 mg milligram(s)
- Max total dose
- 77000 mg milligram(s)
- Max treatment duration
- 55 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- SK LIFE SCIENCE, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10986003 · Product
- Active substance
- Cenobamate
- Pharmaceutical form
- ORAL SUSPENSION
- Route of administration
- ORAL USE
- Max daily dose
- 3 mg/kg milligram(s)/kilogram
- Max total dose
- 77000 mg milligram(s)
- Max treatment duration
- 55 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- SK LIFE SCIENCE, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10986000 · Product
- Active substance
- Cenobamate
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 200 mg milligram(s)
- Max total dose
- 77000 mg milligram(s)
- Max treatment duration
- 55 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- SK LIFE SCIENCE, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10986002 · Product
- Active substance
- Cenobamate
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 200 mg milligram(s)
- Max total dose
- 77000 mg milligram(s)
- Max treatment duration
- 55 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- SK LIFE SCIENCE, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Sk Life Science Inc.
- Sponsor organisation
- Sk Life Science Inc.
- Address
- 461 From Road Fl 5
- City
- Paramus
- Postcode
- 07652-3524
- Country
- United States
Scientific contact point
- Organisation
- Sk Life Science Inc.
- Contact name
- Sunita Misra, MD
Public contact point
- Organisation
- Sk Life Science Inc.
- Contact name
- Sunita Misra, MD
Third parties 8
| Organisation | City, country | Duties |
|---|---|---|
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | On site monitoring, Code 11, Code 12, Code 2, Interactive response technologies (IRT), Data management, E-data capture, Code 9 |
| Opt-X-Pense Kft. ORG-100047138
|
Budaors, Hungary | Other |
| Njs Associates Company ORG-100045907
|
Bridgewater, United States | Code 10 |
| Scout Clinical ORG-100042228
|
Dallas, United States | Other |
| PCI Pharma Services Germany GmbH ORG-100031981
|
Großbeeren, Germany | Code 14, Other |
| Iqvia Rds Ireland Limited ORG-100009589
|
Dublin 3, Ireland | Code 8 |
| MEDPACE LABORATORIES ORG-100042942
|
Leuven, Belgium | Laboratory analysis |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
Locations
5 EU/EEA countries · 12 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ended | 10 | 4 |
| Hungary | Ongoing, recruitment ended | 10 | 1 |
| Poland | Ended | 24 | 2 |
| Slovakia | Ended | 25 | 2 |
| Spain | Ended | 8 | 3 |
| Rest of world
Australia, Ukraine, Korea, Republic of, United States
|
— | 54 | — |
Investigational sites
Universitaetsklinikum Jena KöR
Klinik für Neuropädiatrie, Am Klinikum 1, Lobeda, Jena
Epilepsiezentrum Kleinwachau gGmbH
Fachklinik für Neurologie, Wachauer Strasse 30, Liegau-Augustusbad, Radeberg
Charite Universitaetsmedizin Berlin KöR
Department of Pediatric Neurology, Augustenburger Platz 1, Wedding, Berlin
Universitaetsklinikum Schleswig-Holstein AöR
Klinik für Kinder- und Jugendmedizin II, Abteilung fuer Neuropädiatrie und Sozialpädiatrie, Arnold-Heller-Strasse 3, Brunswik, Kiel
Semmelweis University
Gyermekgyógyászati Klinika, Bókay utcai részleg, Tuzolto Utca 7-9, 1094, Budapest
Niepubliczny Zakład Opieki Zdrowotnej - Centrum Neurologii Dziecięcej i Leczenia Padaczki
NZOZ, Centrum Neurologii Dziecięcej i Leczenia Padaczki, ulica Generała Tadeusza Kościuszki 52/012, 25-316 Kielce, Kielce
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.
Centrum Medyczne Plejady, U2 U3 U4 U5, Ul. Tadeusza Szafrana 5d, Cracow
In Medic s.r.o
Neurology, Novy Sad 930/17, 085 01, Bardejov
Konzilium s.r.o.
Neurology, Puskinova 795/8, 018 51, Nova Dubnica
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Hungary | 2020-05-27 | 2020-06-16 | 2025-05-26 | ||
| Poland | 2019-10-09 | 2025-09-10 | 2019-10-16 | 2025-05-26 | |
| Slovakia | 2019-10-15 | 2026-05-20 | 2019-10-29 | 2025-05-26 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 32 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol _2023-506688-32-00_redacted | 3.0 |
| Protocol (for publication) | D1_Protocol Admin Letter_2023-506688-32-00_redacted | 1.0 |
| Recruitment arrangements (for publication) | K_HU_Recruitment Arrangements_Placeholder document | 1 |
| Recruitment arrangements (for publication) | K1_PL_Recruitment Procedure_Polish | 1.0 |
| Recruitment arrangements (for publication) | K1_SK_Recruitment Procedure | 1.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Adult-Parent ICF_Hungarian | 4.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Adult-Parent SIS_Hungarian_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Adult-Parent_Hungarian_redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Assent 12-17 ICF_Hungarian | 2.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Assent 12-17 SIS_Hungarian | 2.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Assent 12-17_Hungarian | 3.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Assent 18 plus ICF_Hungarian | 2.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Assent 18 plus PIS_Hungarian | 2.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_DTP ICF Addendum_Hungarian | 1.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_DTP IS Addendum_Hungarian | 1.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Pregnant Participant ICF_Hungarian | 2.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Pregnant Participant IS_Hungarian_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_HU_SIS-ICF_Pregnant Participant_Hungarian | 3.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_ Assent 12-17_Polish | 3.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_ Assent 18 plus_Polish | 3.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Addendum-DtP Drug Supply_Polish | 1.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Adult-Parent_Polish_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Pregnant Participant_Polish | 4.0 |
| Subject information and informed consent form (for publication) | L1_SK_SIS-ICF_Addendum-DtP Drug Supply _Slovak | 1.0 |
| Subject information and informed consent form (for publication) | L1_SK_SIS-ICF_Adult-Parent_Slovak | 5.1 |
| Subject information and informed consent form (for publication) | L1_SK_SIS-ICF_Assent 12-17_Slovak | 3.1 |
| Subject information and informed consent form (for publication) | L1_SK_SIS-ICF_Pregnant Participant_Slovak | 3.1 |
| Subject information and informed consent form (for publication) | L2_HU_Other Subject Material_Patient Card_Hungarian | 2.0 |
| Synopsis of the protocol (for publication) | D1_ Protocol Synopsis_2023-506688-32-00 | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2023-506688-32-00_Hungarian | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2023-506688-32-00_Polish | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol_Synopsis_2023-506688-32-00 _Slovak | 3.0 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-04-18 | Spain | Acceptable 2024-05-10
|
2024-05-10 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-02-14 | Acceptable 2025-04-30
|
2025-05-05 | |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-12-10 | Spain | Acceptable 2025-04-30
|
2025-12-10 |