Clinical trial NCT05747001: This is a Retrospective Study on the Use of CENOBAMATE as Adjunctive Treatment in Patients Suffering From Epilepsy in Early Access Program in Germany, France and UK
Official title: Retrospective Study on the Use of CENOBAMATE as Adjunctive Treatment in a Cohort of Patients Suffering From Epilepsy With Focal Onset Seizure (FOS) and Enrolled Into the Early Access Program (EAP) in Germany, France and UK
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Focal Onset Seizure
Epilepsy
Keywords: epilepsy
FOS
cenobamate
retrospective
Early Access Program
Europe
Lead sponsor: Aziende Chimiche Riunite Angelini Francesco S.p.A
Sponsor class: INDUSTRY
Collaborators: name: Hippocrates Research
First posted: 2023-02-28
Enrollment: 319
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population includes adult patients suffering from epilepsy with FOS who have been treated with cenobamate as adjunctive Anti-seizure medication (ASM) during the EAP in Germany, France and UK.

Brief summary: Cenobamate is a newly-FDA and EMA approved drug used to treat -focal-onset seizures in adult patients.

The aim of the current study is to analyse retrospectively the overall effectiveness and tolerability of cenobamate from real-world data collected in patients who partecipated in the Early Access Program (EAP) and were treated with cenobamate as adjunctive ASM.

Detailed description: Cenobamate is a new approved drug used to treat -focal-onset seizures in adult patients. This novel tetrazole-derived carbamate seems to act primarily by two mechanisms that are commonly associated with epilepsy: cenobamate acts as a positive allosteric modulator of the GABAA ion channels and is effective in reducing repetitive neuronal firing by inhibition of voltage-gated sodium channels, although the complete mechanism of action is currently unknown.

In clinical trials, cenobamate showed also low toxicity and adverse drug reaction profile.

In European Union (EU), cenobamate received the marketing authorisation, valid throughout the EU, in March 2021. Starting from September 2020 an EAP was initiated with cenobamate as adjunctive ASM in several EU Countries such as Germany, France, and UK.

Real-world data are of importance to understand and confirm the efficacy and safety profile of drugs outside of the clinical trial setting. The aim of the current study is to analyse the overall effectiveness and tolerability of cenobamate from real-world data in a large series of patients treated with cenobamate as adjunctive ASM.

As a consequence, a retrospective collection and analysis of the data of the patients who participated in the EAP, according to the authorization received from the local regulatory or ethic authorities, was conducted.

Eligibility criteria: Inclusion Criteria:

* Data from adult patients diagnosed with epilepsy with FOS participating in the EAP with cenobamate as adjunctive treatment, according to the authorization received from the local regulatory or ethic authorities will be collected and analyzed.
* Available data will be collected after obtaining consent from patient/legal representative to the processing of personal data according to the General Data Protection Regulation (GDPR) and applicable local regulation

Exclusion Criteria:

* Patient enrolled in other clinical trial during the EAP.
* Patient aged less than 18 years old.
* Patient with specific syndrome (e.g. LGS and Dravet)

Primary outcomes:
measure: Responder rate (%) at 3 months from the start of maintenance
timeFrame: 3 months from the start of maintenance
Secondary outcomes:
measure: Portion of responders
timeFrame: 1 and 3 months after start of cenobamate therapy
measure: Portion of responders
timeFrame: 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
measure: Portion of seizure free
timeFrame: 1 and 3 months after start of cenobamate therapy
measure: Portion of seizure free
timeFrame: 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
measure: Retention rate
timeFrame: 1 and 3 months after start of cenobamate therapy
measure: Retention rate
timeFrame: 3, 6 and 12 months after the completion of the titration
measure: No. of Adverse Reactions (ADRs),
timeFrame: Through study completion, an average of 2 years
measure: Change in Seizures Frequency
timeFrame: 1 and 3 months after start of cenobamate therapy
measure: Change in Seizures Frequency
timeFrame: 3, 6 and 12 months after the completion of the titration
measure: Assessment of quality of life
timeFrame: 1 and 3 months after start of cenobamate therapy
measure: Assessment of quality of life
timeFrame: 3, 6 and 12 months after the completion of the titration
Other outcomes:
Locations:
city: Bron
country: France
facility: Hôpital Pierre Wertheimer - Hopsices Civils de Lyon
city: Lille Cedex
country: France
facility: CHRU de Lille - Hôpital Roger Salengro (LILLE)
city: Marseille
country: France
facility: Centre Hospitalier Universitaire (CHU) de Marseille - Hopital de la Timone
city: Nancy
country: France
facility: CHRU de Nancy -Hopital Central, Service de Neurologie
city: Paris
country: France
facility: Hôpital de la Pitié-Salpêtrière
city: Rennes
country: France
facility: CHU Rennes - Pontchaillou Hospital
city: Rouen
country: France
facility: CHU de Rouen Hôpital Charles-NicolleService de Neurophysiologie
city: Strasbourg
country: France
facility: CHU de Strasbourg - Hôpital de Hautepierre
city: Berlin-Reinickendorf
country: Germany
facility: Neurologie - Stroke Unit - Zentrum für Epilepsie
city: Bernau bei Berlin
country: Germany
facility: Epilepsieklinik Tabor
city: Bielefeld
country: Germany
facility: Epilepsy Center Bethel hospital Mara
city: Bonn
country: Germany
facility: Klinik und Poliklinik für Epileptologie, Universitätsklinikum Bonn
city: Erlangen
country: Germany
facility: Neurologische Klinik
city: Frankfurt am main
country: Germany
facility: Epilepsy Center Frankfurt Rhine-Main Neurocenter
city: Freiburg
country: Germany
facility: Klinik für Neurochirurgie Uniklinik Freiburg -
city: Hamburg
country: Germany
facility: Evangelische Krankenhaus Alsterdorf
city: Kehl
country: Germany
facility: Diakonie Kork Epilepsiezentrum
city: Marburg
country: Germany
facility: Epilepsiezentrum Hessen, Klinik für Neurologie, Philipps Universität Marburg - Standort Marburg
city: Radeberg
country: Germany
facility: Epilepsiezentrum Kleinwachau gGmbH
city: Tübingen
country: Germany
facility: Universitätsklinikum Tubingen
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: UCLH NHS Trust Epilepsy Department
city: Newcastle
country: United Kingdom
facility: The Newcastle upon Tyne Hospitals
Study officials:
affiliation: Hôpital Neurologique Pierre Wertheimer - Hospices Civils de Lyon
name: Sylvain Rheims
role: PRINCIPAL_INVESTIGATOR
affiliation: The Newcastle upon Tyne Hospitals NHS Trust Victoria Road, Newcastle, NE1 4LP
name: Rhys Thoma
role: PRINCIPAL_INVESTIGATOR
affiliation: Epilepsy Center Frankfurt Rhine-Main Neurocenter Schleusenweg 2 - 16 (Haus 95) 60528 Frankfurt am Main
name: Felix Rosenow
role: PRINCIPAL_INVESTIGATOR
References: