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
- Conditions
- Focal Onset SeizureEpilepsy
- Registration Number
- NCT05747001
- Lead Sponsor
- Aziende Chimiche Riunite Angelini Francesco S.p.A
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 319
- 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
- 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)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Responder rate (%) at 3 months from the start of maintenance 3 months from the start of maintenance Percentage of responder rate (defined as a ≥50% reduction from screening/baseline in focal onset seizure frequency) after 3 months of maintenance phase.
- Secondary Outcome Measures
Name Time Method Retention rate 3, 6 and 12 months after the completion of the titration Retention rate measured as percentage of patients remaining in the study and on adjunctive therapy at 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Portion of responders 3, 6 and 12 months after the completion of the titration and its relation with the dosage. Portion of responders (defined as a ≥50% and \<100% reduction from screening/baseline in focal onset seizure frequency) at 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Assessment of quality of life 3, 6 and 12 months after the completion of the titration The quality of life was assessed through the Questionnaire Quality of Life in Epilepsy Inventory - 31 items
Portion of seizure free 3, 6 and 12 months after the completion of the titration and its relation with the dosage. Portion of seizure free (100% reduction from screening/baseline)3, 6 and 12 months after the completion of the titration and its relation with the dosage.
No. of Adverse Reactions (ADRs), Through study completion, an average of 2 years Adverse Reactions (ADRs), including DRESS, rash/hypersensitivity occurred during the EAP.
Change in Seizures Frequency 3, 6 and 12 months after the completion of the titration Change in Seizures Frequency at 3, 6 and 12 months after the completion of the titration
Trial Locations
- Locations (23)
Hôpital Pierre Wertheimer - Hopsices Civils de Lyon
🇫🇷Bron, France
CHRU de Lille - Hôpital Roger Salengro (LILLE)
🇫🇷Lille Cedex, France
Hôpital de la Pitié-Salpêtrière
🇫🇷Paris, France
Centre Hospitalier Universitaire (CHU) de Marseille - Hopital de la Timone
🇫🇷Marseille, France
CHRU de Nancy -Hopital Central, Service de Neurologie
🇫🇷Nancy, France
CHU Rennes - Pontchaillou Hospital
🇫🇷Rennes, France
CHU de Rouen Hôpital Charles-NicolleService de Neurophysiologie
🇫🇷Rouen, France
CHU de Strasbourg - Hôpital de Hautepierre
🇫🇷Strasbourg, France
Neurologie - Stroke Unit - Zentrum für Epilepsie
🇩🇪Berlin-Reinickendorf, Germany
Epilepsieklinik Tabor
🇩🇪Bernau bei Berlin, Germany
Epilepsy Center Frankfurt Rhine-Main Neurocenter
🇩🇪Frankfurt am main, Germany
Klinik und Poliklinik für Epileptologie, Universitätsklinikum Bonn
🇩🇪Bonn, Germany
Epilepsy Center Bethel hospital Mara
🇩🇪Bielefeld, Germany
Neurologische Klinik
🇩🇪Erlangen, Germany
Klinik für Neurochirurgie Uniklinik Freiburg -
🇩🇪Freiburg, Germany
Epilepsiezentrum Hessen, Klinik für Neurologie, Philipps Universität Marburg - Standort Marburg
🇩🇪Marburg, Germany
Evangelische Krankenhaus Alsterdorf
🇩🇪Hamburg, Germany
Diakonie Kork Epilepsiezentrum
🇩🇪Kehl, Germany
Epilepsiezentrum Kleinwachau gGmbH
🇩🇪Radeberg, Germany
Universitätsklinikum Tubingen
🇩🇪Tübingen, Germany
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
UCLH NHS Trust Epilepsy Department
🇬🇧London, United Kingdom
The Newcastle upon Tyne Hospitals
🇬🇧Newcastle, United Kingdom