Cenobamate in Adults With Focal-Onset Seizures

NCT05859854 | Active Not Recruiting

• 1 other identifier: 153(A)IM21348
• Study Type: observational
• Target: 936
• Locations: 1 country
• Sites: 10

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.

Conditions

Epilepsy; Focal-Onset Seizure; Neurological Disorder

Keywords

cenobamate; Real-word; epilepsy; focal-onset seizure; observational; focal epilepsy uncontrolled

Outcome Measures

Primary Outcomes (6)

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

  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").

  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)

  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").

  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.

  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).

  12, 24 and 52 weeks of cenobamate treatment initiation

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

  12, 24 and 52 weeks of cenobamate treatment initiation

• Intra-patient percent change in the seizure frequency (stratified)

  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).

  12, 24 and 52 weeks of cenobamate treatment initiation

Secondary Outcomes (17)

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

  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

  24 and 52 weeks of cenobamate treatment initiation.

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

  12, 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

Study Arms (1)

Adult patients diagnosed treated with adjunctive cenobamate in Italy.

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

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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-

You may not qualify if:

• 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.

Sponsors & Collaborators

• Aziende Chimiche Riunite Angelini Francesco S.p.A: lead
• Iqvia Pty Ltd: collaborator

Study Sites (10)

Policlinico di Bari

Bari, Bari, 70124, Italy

Location

Università degli Studi di Catanzaro "Magna Graecia"

Catanzaro, Catanzaro, 88100, Italy

Location

IRCCS Neuromed

Pozzilli, Isernia, 86077, Italy

Location

Fondazione Istituto Neurologico Casimiro Mondino

Pavia, Pavia, 27100, Italy

Location

Campus Bio-Medico

Roma, Roma, 00128, Italy

Location

Policlinico Umberto I

Roma, Roma, 00161, Italy

Location

Humanitas Gradenigo

Torino, Torino, 10153, Italy

Location

Associazione La Nostra Famiglia - IRCCS Eugenio Medea

Conegliano, Treviso, 31015, Italy

Location

Azienda Sanitaria Universitaria (A.O.U.) Integrata

Udine, Udine, 33100, Italy

Location

Ospedale San Bortolo

Vicenza, Vicenza, 36100, Italy

Location

Related Publications (1)

• Lattanzi S, Ranzato F, Di Bonaventura C, Bonanni P, Gambardella A, Tartara E, Assenza G, Procaccini M, Falsetto N, Villano V, Camattari G, Ori A, Di Gennaro G; BLESS Study Group. Effectiveness and Safety of Adjunctive Cenobamate in People with Focal-Onset Epilepsy: Evidence from the First Interim Analysis of the BLESS Study. Neurol Ther. 2024 Aug;13(4):1203-1217. doi: 10.1007/s40120-024-00634-5. Epub 2024 Jun 8.

  PMID: 38850402 DERIVED

MeSH Terms

Conditions

Epilepsy; Seizures; Nervous System Diseases

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2023
• First Posted: May 16, 2023
• Study Start: January 24, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: September 8, 2025

Record last verified: 2025-05

Locations

IT (10)