Real-world Clinical Response to Cenobamate Early add-on in France, Germany and Spain

Observational Cohort Study on Adult Patients With Not-adequately Controlled FOcal-onset Seizures Treated With Cenobamate as Early adjUnctive Therapy in a Real-world Setting - FOCUS

Aim of the study is to better characterize the clinical profile of adjunctive cenobamate by collecting data from the current standard clinical practice in France, Germany, and Spain, to describe the real-world clinical response among adult patients affected by focal epilepsy not adequately controlled despite a history of 2 or 3 ASMs before starting treatment with cenobamate (including previous and concomitant ASMs).

Study Overview

• Status: Recruiting
• Conditions: Focal Epilepsy With and Without Secondary Generalization
• Intervention / Treatment: Drug: Cenobamate

• Study Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

• Study Contact: Name: Enrica Salvatori; Phone Number: +39 3458063168; Email: Enrica.salvatori@angelinipharma.com
• Study Contact Backup: Name: Valeria Tellone; Phone Number: +393452493461; Email: valeria.tellone@angelinipharma.com

Study Locations

• France
  • France
    • Paris, France, France
      • Recruiting
      • Hôpital TARNIER COCHIN
      • Contact: Edouard HIRSCH

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients affected by epilepsy with focal-onset seizures with or without secondary generalization who have not been adequately controlled despite a history of treatment with 2 or 3 anti-seizure medications (ASMs).

Description

Inclusion Criteria:

1. Male and female patients ≥18 years old at the time of cenobamate treatment initiation.
2. Patients with a diagnosis of epilepsy with focal-onset seizures, with or without secondary generalization.
3. Patients under titration phase (i.e., maintenance dose not reached yet according to clinical judgement) with cenobamate as adjunctive therapy in third or fourth line with 1 to maximum 2 (for third line)/3 (for fourth line) concomitant anti-seizure medications (ASMs).
4. Patients who have not been adequately controlled despite treatment with 2 or 3 (maximum) ASMs before cenobamate treatment initiation (including concomitant ASMs started before initiating cenobamate).
5. Patients with available retrospective data in medical charts, seizure diaries or patient's notes, including reliable information about seizure frequency (intended as the number of seizures and the corresponding time period) in the last 3 months before cenobamate treatment initiation.
6. Written informed consent (including consent for the processing of personal data) signed by the patient, or by the legally designated representative in case of patient lacking capacity, prior to entering the study following local regulation.

Exclusion Criteria:

1. Patients who meet any of the contraindications to the administration of cenobamate according to its approved Summary of Product Characteristics (SmPC).
2. Patients with progressive neurodegenerative central nervous system (CNS) diseases or (benign or malignant) brain tumors.
3. Patients with unstable psychiatric diagnosis, including suicidal ideation and behavior within 6 months prior to enrolment, current psychotic disorder, or acute mania.
4. Patients with known substance abuse or dependence (except for caffeine and nicotine).
5. Patients participating in any interventional study from cenobamate treatment initiation until enrolment visit.
6. Patients with ongoing pregnancy or breast-feeding from cenobamate treatment initiation until enrolment visit.
7. Patients who are seizure-free in the last 3 months before cenobamate treatment initiation.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients affected by epilepsy with focal-onset seizures with or without secondary generalization; Adult patients affected by epilepsy with focal-onset seizures with or without secondary generalization who have not been adequately controlled despite a history of treatment with 2 or 3 anti-seizure medications (ASMs).	Drug: Cenobamate; Adult patients affected by epilepsy with focal-onset seizures with or without secondary generalization who have not been adequately controlled despite a history of treatment with 2 or 3 anti-seizure medications.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Responder rate	To describe the ≥50% responder rate during the 6 and 12 months of maintenance-dosing treatment with cenobamate with respect to the pretreatment baseline.	At 6 and at 12 months of maintenance treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Responder rate	To describe the ≥75%/≥90%/=100% responder rate during the 6 and 12 months of maintenance-dosing treatment with cenobamate with respect to the pre-treatment baseline.	At 6 and at 12 months of maintenance-dosing treatment
Safety evaluation	To describe real-world safety from treatment initiation until 12 months of maintenance-dosing treatment with cenobamate. Absolute and relative frequencies of patients treated with cenobamate who experienced at least one AE/Adverse Drug Reaction (ADR)/Serious Adverse Event (SAE)/Serious Adverse Drug Reaction (SADR)/AE of special interest during the observation period. Total number of AEs/ADRs/SAEs/SADRs/AEs of special interest occurred during the observation period.	0 -12 months of maintenance-dosing treatment
Health-Related Quality of Life evaluation (QOLIE-31-P)	QOLIE-31-P is a survey of health-related quality of life for adults with epilepsy. This questionnaire should be completed only by the person who has epilepsy (not a relative or friend). There are 39 questions about health and daily activities.The QOLIE-31-P consists of 38 self-reported items and is divided in eight parts which evaluate energy, mood, daily activities, cognition, medication effects, seizure worry, overall QoL , and health state. The first and the last items are scored with 0-10 and 0-100 scales, respectively, from the worst to the best situation. The remaining 36 items are scored using a Likert rating scale from 1 to 3,4,5, or 6. The raw scores are reorganized to have scores from 0 to 100. The final score is obtained from the subscale scores and is rescaled from 0 to 100 with higher scores associated with better QoL.	At 6 and at 12 months maintenance-dosing treatment
Health-Related Quality of Life evaluation (SF-6Dv2 )	The SF-6Dv2 Health Utility Survey provides keen insights into patient health and product performance by measuring six health domains. The SF-6Dv2™ Health Survey consists of six questions asking about different aspects of patient's health; for each question, patients should select the response (among 5 or 6 options per question) that best describe his/her health status.	At 6 and at 12 months maintenance-dosing treatment
Sleepiness status evaluation	To describe the sleepiness status of patients, by means of the Epworth Sleepiness Scale (ESS), at 6 and at 12 months of maintenance-dosing treatment with cenobamate. The ESS scale is designed to measure the patient's general level of daytime sleepiness. The ESS scale is designed to measure the patient's general level of daytime sleepiness and consists of 8 questions with a rating ranging from "0 = no chance of dozing" to "3 = high chance of dozing".	At 6 and at 12 months of maintenance-dosing treatment
Healthcare resource evaluation	Absolute and relative frequencies of patients according to the type of resources used (ED accesses; inpatient hospitalizations; specialist outpatient visits; diagnostic exams) during the observation period. Descriptive statistics (mean, SD, median, 25th and 75th percentiles, min. and max.) of total number of resources used (ED accesses; inpatient hospitalizations; specialist outpatient visits; diagnostic exams) during the observation period. Descriptive statistics (mean, SD, median, 25th and 75th percentiles, min. and max.) of total duration of inpatient hospitalizations (calculated for each patient by summing the duration of all his/her inpatient hospitalizations) during the observation period.	0-12 months of maintenance-dosing treatment

Collaborators and Investigators

• Sponsor: Aziende Chimiche Riunite Angelini Francesco S.p.A
• Collaborators: IQVIA Pty Ltd

Study record dates

Study Major Dates

• Study Start (Actual): December 19, 2024
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: November 22, 2024
• First Submitted That Met QC Criteria: November 29, 2024
• First Posted (Actual): December 4, 2024

Study Record Updates

• Last Update Posted (Estimated): September 19, 2025
• Last Update Submitted That Met QC Criteria: September 16, 2025
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Keywords: epilepsy; focal-onset seizures
• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Epilepsy; Seizures; Anticonvulsants; Cenobamate
• Other Study ID Numbers: 169(A)MD23324

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No