Cenobamate in Adults With Focal-Onset Seizures

N/AActive Not RecruitingNCT05859854

Aziende Chimiche Riunite Angelini Francesco S.p.A936 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

936

Conditions

Epilepsy Focal-Onset Seizure Neurological Disorder

Eligibility

Sex: ALLMin age: 18 YearsMax age: 99 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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- Exclusion Criteria: * 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.

Locations (10)

Policlinico di Bari

Bari, Bari, Italy

Università degli Studi di Catanzaro "Magna Graecia"

Catanzaro, Catanzaro, Italy

IRCCS Neuromed

Pozzilli, Isernia, Italy

Fondazione Istituto Neurologico Casimiro Mondino

Outcomes

Primary Outcomes

Absolute frequency of patients achieving a 50 % or greater reduction in the seizure frequency (overall)

Time frame: 12, 24 and 52 weeks of cenobamate treatment initiation

Relative frequency of patients achieving a ≥50% (50% or greater) reduction in the seizure frequency (overall)

Time frame: 12, 24 and 52 weeks of cenobamate treatment initiation

Intra-patient percent change in the seizure frequency (overall)

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.

Time frame: 12, 24 and 52 weeks of cenobamate treatment initiation

Absolute frequency of patients achieving a 50 % or greater reduction in the seizure (stratified)

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).

Data from ClinicalTrials.gov

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Secondary Outcomes

Absolute frequency of patients achieving a ≥50%/≥75%/≥90%/=100% sustained reduction in the seizure frequency

Sustained seizure frequency reduction will be defined as a ≥50%/≥75%/≥90%/=100% reduction in baseline seizure frequency achieved at 12 weeks of cenobamate treatment initiation, that continues (is sustained) without interruption and without cenobamate permanent discontinuation through the observation period until 24 and 52 weeks of cenobamate treatment initiation, respectively.

Time frame: 24 and 52 weeks of cenobamate treatment initiation.

Absolute frequency of patients achieving a ≥75%/≥90%/=100% reduction in the seizure frequency

Seizure frequency reduction will be defined as a ≥75%/≥90%/=100% reduction in baseline seizure frequency achieved at 12, 24 and 52 weeks of cenobamate treatment initiation, respectively.

Time frame: 12, 24 and 52 weeks of cenobamate treatment initiation.

Relative frequencies of patients achieving a ≥50%/≥75%/≥90%/=100% sustained reduction in the seizure frequency

Time frame: 24 and 52 weeks of cenobamate treatment initiation.

Relative frequencies of patients achieving a ≥75%/≥90%/=100% reduction in the seizure frequency

Seizure frequency reduction will be defined as a ≥75%/≥90%/=100% reduction in baseline seizure frequency achieved at 12, 24 and 52 weeks of cenobamate treatment initiation.

Time frame: 12, 24 and 52 weeks of cenobamate treatment initiation.

Absolute frequency of patients with at least one AE

Absolute frequency of patients treated with cenobamate who experienced at least one AE during the applicable observation period

Time frame: Through study completion, an average of 1 year

Relative frequency of patients with at least one AE

Relative frequency of patients treated with cenobamate who experienced at least one AE during the applicable observation period

Time frame: Through study completion, an average of 1 year

Absolute frequency of patients with at least one ADR

Absolute frequency of patients treated with cenobamate who experienced at least one ADR during the applicable observation period

Time frame: Through study completion, an average of 1 year

Relative frequency of patients with at least one ADR

Relative frequency of patients treated with cenobamate who experienced at least one ADR during the applicable observation period

Time frame: Through study completion, an average of 1 year

Absolute frequency of patients treated with at least one SAE

Absolute frequency of patients treated with at least one SAE during the applicable observation period

Time frame: Through study completion, an average of 1 year

Relative frequency of patients treated with at least one SAE

Relative frequency of patients treated with at least one SAE during the applicable observation period

Time frame: Through study completion, an average of 1 year

Health-Related Quality of life (HRQoL)

The Health-Related Quality of life (HRQoL) of patients will be assessed by means of the 31-item Quality Of Life In Epilepsy inventory (QOLIE-31). The 31-item Quality Of Life In Epilepsy inventory (QOLIE-31) is a patient self-reported instrument that contains 31 items grouped in seven multi-item scales ( "seizure worry", "overall QoL", "emotional well-being", "energy fatigue", "cognitive functioning", "medication effects" and "social functioning"). Different items in the QOLIE-31 have different ranges of precoded numeric values, so the scoring procedure requires conversion from raw, precoded numeric values to scores of 0-100 points, with higher scores reflecting better quality of life. The QOLIE-31 overall score is calculated by summing the product of each scale score times its weight and summing over all scales, according to the instructions obtained from copyright holders.