Clinical trial NCT05859854: Cenobamate in Adults With Focal-Onset Seizures
Official title: Cenobamate in Adults With Focal-Onset Seizures: a Real-World Multicenter Observational Italian
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Epilepsy
Focal-Onset Seizure
Neurological Disorder
Keywords: cenobamate
Real-word
epilepsy
focal-onset seizure
observational
focal epilepsy uncontrolled
Lead sponsor: Aziende Chimiche Riunite Angelini Francesco S.p.A
Sponsor class: INDUSTRY
Collaborators: name: Iqvia Pty Ltd
First posted: 2023-05-16
Enrollment: 1200
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Adult patients diagnosed with focal epilepsy uncontrolled despite the use of at least two antiepileptic medicinal products, treated with adjunctive cenobamate in Italy. A single cohort of patients will be involved in the study, enrolling both subjects who initiated cenobamate treatment in accordance with the current clinical practice, and subjects previously included in the cenobamate Compassionate Use Programme in Italy, provided that they fulfil all of the eligibility criteria listed below

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Absolute frequency of patients achieving a 50 % or greater reduction in the seizure frequency (overall)
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation
measure: Relative frequency of patients achieving a ≥50% (50% or greater) reduction in the seizure frequency (overall)
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation
measure: Intra-patient percent change in the seizure frequency (overall)
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation
measure: Absolute frequency of patients achieving a 50 % or greater reduction in the seizure (stratified)
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation
measure: Relative frequency of patients achieving a ≥50% (50% or greater) reduction in the seizure frequency (stratified)
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation
measure: Intra-patient percent change in the seizure frequency (stratified)
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation
Secondary outcomes:
measure: Absolute frequency of patients achieving a ≥50%/≥75%/≥90%/=100% sustained reduction in the seizure frequency
timeFrame: 24 and 52 weeks of cenobamate treatment initiation.
measure: Absolute frequency of patients achieving a ≥75%/≥90%/=100% reduction in the seizure frequency
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation.
measure: Relative frequencies of patients achieving a ≥50%/≥75%/≥90%/=100% sustained reduction in the seizure frequency
timeFrame: 24 and 52 weeks of cenobamate treatment initiation.
measure: Relative frequencies of patients achieving a ≥75%/≥90%/=100% reduction in the seizure frequency
timeFrame: 12, 24 and 52 weeks of cenobamate treatment initiation.
measure: Absolute frequency of patients with at least one AE
timeFrame: Through study completion, an average of 1 year
measure: Relative frequency of patients with at least one AE
timeFrame: Through study completion, an average of 1 year
measure: Absolute frequency of patients with at least one ADR
timeFrame: Through study completion, an average of 1 year
measure: Relative frequency of patients with at least one ADR
timeFrame: Through study completion, an average of 1 year
measure: Absolute frequency of patients treated with at least one SAE
timeFrame: Through study completion, an average of 1 year
measure: Relative frequency of patients treated with at least one SAE
timeFrame: Through study completion, an average of 1 year
measure: Health-Related Quality of life (HRQoL)
timeFrame: 12, 24 and 52 weeks
measure: Patient Global functioning (CGI-S)
timeFrame: 12, 24 and 52 weeks
measure: Patient Global functioning (CGI-I)
timeFrame: 12, 24 and 52 weeks
measure: Patient Global functioning (CGI-E)
timeFrame: 12, 24 and 52 weeks
measure: Daytime Sleepiness
timeFrame: 12, 24 and 52 weeks
measure: Hospital Anxiety and Depression
timeFrame: 12, 24 and 52 weeks
measure: Retention with cenobamate
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Pozzilli
country: Italy
facility: IRCCS Neuromed
state: Isernia
city: Conegliano
country: Italy
facility: Associazione La Nostra Famiglia - IRCCS Eugenio Medea
state: Treviso
city: Bari
country: Italy
facility: Policlinico di Bari
city: Catanzaro
country: Italy
facility: Università degli Studi di Catanzaro "Magna Graecia"
city: Pavia
country: Italy
facility: Fondazione Istituto Neurologico Casimiro Mondino
city: Roma
country: Italy
facility: Campus Bio-Medico
city: Roma
country: Italy
facility: Policlinico Umberto I
city: Torino
country: Italy
facility: Humanitas Gradenigo
city: Udine
country: Italy
facility: Azienda Sanitaria Universitaria (A.O.U.) Integrata
city: Vicenza
country: Italy
facility: Ospedale San Bortolo
Study officials:
References: