Epilepsy, Focal Onset Seizure
Conditions
Keywords
epilepsy, FOS, cenobamate, retrospective, Early Access Program, Europe
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.
Interventions
None listed
Sponsors
Hippocrates Research
Aziende Chimiche Riunite Angelini Francesco S.p.A
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Change in Seizures Frequency | 1 and 3 months after start of cenobamate therapy | Change in Seizures Frequency at 1 and 3 months after start of cenobamate therapy, |
| Assessment of quality of life | 1 and 3 months after start of cenobamate therapy | The quality of life was assessed through the Questionnaire Quality of Life in Epilepsy Inventory - 31 items |
| No. of Adverse Reactions (ADRs), | Through study completion, an average of 2 years | Adverse Reactions (ADRs), including DRESS, rash/hypersensitivity occurred during the EAP. |
| Portion of seizure free | 1 and 3 months after start of cenobamate therapy | Portion of seizure free (100% reduction from screening/baseline) 1 and 3 months after start of cenobamate therapy |
| Retention rate | 1 and 3 months after start of cenobamate therapy | Retention rate measured as percentage of patients remaining in the study and on adjunctive therapy at: 1 and 3 months after start of cenobamate therapy |
| Portion of responders | 1 and 3 months after start of cenobamate therapy | Portion of responders (defined as a ≥50% and \<100% reduction from screening/baseline in focal onset seizure frequency) at 1 and 3 months after start of cenobamate therapy |
Countries
France, Germany, United Kingdom
Outcome results
None listed