Cenobamate in Adults With Focal-Onset Seizures: a Real-World Multicenter Observational Italian
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 936
- 试验地点
- 10
- 主要终点
- Relative frequency of patients achieving a ≥50% (50% or greater) reduction in the seizure frequency (stratified)
研究概览
简要总结
The BLESS Study contributes to filling this information gap by collecting data from the Italian clinical practice and the Compassionate Use Program, to better characterize the clinical profile of cenobamate describing its effectiveness, safety and tolerability in adult patients diagnosed with uncontrolled focal epilepsy despite the use of at least two antiepileptic medicinal products.
详细描述
The main objective of the study is to describe the effectiveness of adjunctive cenobamate treatment in adult patients with uncontrolled focal epilepsy in Italy, overall and according to age class, setting of cenobamate treatment, cenobamate final target daily dose prescribed, and number of concomitant Anti Seizure Medications (ASMs). This will be assessed by intra-patient percent change and achievement of a ≥50% reduction in the seizure frequency from the pre-treatment baseline over a period of 52 weeks.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years at the time of cenobamate treatment initiation
- •Male or female patients
- •Patients diagnosed with focal epilepsy uncontrolled despite a history of treatment with at least two antiepileptic medicinal products at the time of cenobamate treatment initiation in agreement with the Summary of Product Characteristics (SmPC)
- •Patients who at enrolment had received at least 12 weeks (titration period up to the initial recommended target dose of 200 mg daily completed) but no more than 52 weeks of cenobamate as adjunctive treatment of focal-onset seizures with or without secondary generalization
- •Patients with available retrospective data in medical charts and seizure diaries, including information about baseline seizure frequency prior to cenobamate treatment initiation
- •Patients who gave written informed consent to take part into the study and personal data processing consent following local regulation.
- •Patients who received adjunctive cenobamate for at least 12 weeks and discontinued permanently treatment before enrolment will also be included in the study-
排除标准
- •Patients diagnosed with familial short-QT syndrome
- •Patients affected by hypersensitivity to the active substance cenobamate or to any of the excipients (e.g., lactose monohydrate)
- •Patients with history of severe drug-induced hypersensitivity reaction, including (but not limited to) drug reaction with eosinophilia and systemic symptoms (DRESS), and Stevens Johnson syndrome
- •Patients enrolled in a clinical trial in which treatments for epilepsy are managed through a study protocol
- •Patient unable to read and write in Italian language and to autonomously fill in questionnaires and scales
- •Patients with a known pregnancy or who are breast-feeding from cenobamate treatment initiation till enrolment visit.
结局指标
主要结局
Relative frequency of patients achieving a ≥50% (50% or greater) reduction in the seizure frequency (stratified)
时间窗: 12, 24 and 52 weeks of cenobamate treatment initiation
Seizure frequency during the baseline and post-baseline assessments was calculated by summing the total number of seizures (all types of seizures) reported in each considered period and dividing by the duration of that period (number of days), excluding days with no available diary data, and multiplying by 28 to normalize to a monthly rate ("monthly seizure frequency"). Population will be stratified according to the following: * Age class of patient at the time of cenobamate treatment initiation (i.e., \<65 years; ≥65 years). * Setting of cenobamate treatment initiation (i.e., CUP; current clinical practice). * Cenobamate final target daily dose prescribed (i.e., 200 mg; \>200 mg). * Number of ASMs concomitant with cenobamate (i.e., ≤2 ASMs; \>2 ASMs).
Intra-patient percent change in the seizure frequency (stratified)
时间窗: 12, 24 and 52 weeks of cenobamate treatment initiation
Intra-patient percent change from baseline will be defined as \[(monthly seizure frequency at post-baseline assessments - monthly seizure frequency at baseline), divided by the monthly seizure frequency at baseline\] multiplied by 100. Population will be stratified according to the following: * Age class of patient at the time of cenobamate treatment initiation (i.e., \<65 years; ≥65 years). * Setting of cenobamate treatment initiation (i.e., CUP; current clinical practice). * Cenobamate final target daily dose prescribed (i.e., 200 mg; \>200 mg). * Number of ASMs concomitant with cenobamate (i.e., ≤2 ASMs; \>2 ASMs).
Intra-patient percent change in the seizure frequency (overall)
时间窗: 12, 24 and 52 weeks of cenobamate treatment initiation
Intra-patient percent change from baseline will be defined as \[(monthly seizure frequency at post-baseline assessments - monthly seizure frequency at baseline), divided by the monthly seizure frequency at baseline\] multiplied by 100.
Absolute frequency of patients achieving a 50 % or greater reduction in the seizure (stratified)
时间窗: 12, 24 and 52 weeks of cenobamate treatment initiation
Seizure frequency during the baseline and post-baseline assessments was calculated by summing the total number of seizures (all types of seizures) reported in each considered period and dividing by the duration of that period (number of days), excluding days with no available diary data, and multiplying by 28 to normalize to a monthly rate ("monthly seizure frequency"). Population will be stratified according to the following: * Age class of patient at the time of cenobamate treatment initiation (i.e., \<65 years; ≥65 years). * Setting of cenobamate treatment initiation (i.e., CUP; current clinical practice). * Cenobamate final target daily dose prescribed (i.e., 200 mg; \>200 mg). * Number of ASMs concomitant with cenobamate (i.e., ≤2 ASMs; \>2 ASMs).
Absolute frequency of patients achieving a 50 % or greater reduction in the seizure frequency (overall)
时间窗: 12, 24 and 52 weeks of cenobamate treatment initiation
Seizure frequency during the baseline and post-baseline assessments was calculated by summing the total number of seizures (all types of seizures) reported in each considered period and dividing by the duration of that period (number of days), excluding days with no available diary data, and multiplying by 28 to normalize to a monthly rate ("monthly seizure frequency").
Relative frequency of patients achieving a ≥50% (50% or greater) reduction in the seizure frequency (overall)
时间窗: 12, 24 and 52 weeks of cenobamate treatment initiation
Seizure frequency during the baseline and post-baseline assessments was calculated by summing the total number of seizures (all types of seizures) reported in each considered period and dividing by the duration of that period (number of days), excluding days with no available diary data, and multiplying by 28 to normalize to a monthly rate ("monthly seizure frequency").
次要结局
- Relative frequencies of patients achieving a ≥75%/≥90%/=100% reduction in the seizure frequency(12, 24 and 52 weeks of cenobamate treatment initiation.)
- Relative frequency of patients with at least one AE(Through study completion, an average of 1 year)
- Patient Global functioning (CGI-E)(12, 24 and 52 weeks)
- Absolute frequency of patients achieving a ≥75%/≥90%/=100% reduction in the seizure frequency(12, 24 and 52 weeks of cenobamate treatment initiation.)
- Hospital Anxiety and Depression(12, 24 and 52 weeks)
- Absolute frequency of patients achieving a ≥50%/≥75%/≥90%/=100% sustained reduction in the seizure frequency(24 and 52 weeks of cenobamate treatment initiation.)
- Relative frequencies of patients achieving a ≥50%/≥75%/≥90%/=100% sustained reduction in the seizure frequency(24 and 52 weeks of cenobamate treatment initiation.)
- Absolute frequency of patients with at least one AE(Through study completion, an average of 1 year)
- Absolute frequency of patients treated with at least one SAE(Through study completion, an average of 1 year)
- Relative frequency of patients with at least one ADR(Through study completion, an average of 1 year)
- Health-Related Quality of life (HRQoL)(12, 24 and 52 weeks)
- Daytime Sleepiness(12, 24 and 52 weeks)
- Absolute frequency of patients with at least one ADR(Through study completion, an average of 1 year)
- Relative frequency of patients treated with at least one SAE(Through study completion, an average of 1 year)
- Patient Global functioning (CGI-S)(12, 24 and 52 weeks)
- Patient Global functioning (CGI-I)(12, 24 and 52 weeks)
- Retention with cenobamate(52 weeks)