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
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 319
- 试验地点
- 23
- 主要终点
- Responder rate (%) at 3 months from the start of maintenance
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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)
结局指标
主要结局
Responder rate (%) at 3 months from the start of maintenance
时间窗: 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.
次要结局
- Retention rate(3, 6 and 12 months after the completion of the titration)
- Portion of responders(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)
- Portion of seizure free(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)
- Change in Seizures Frequency(3, 6 and 12 months after the completion of the titration)