Focal Onset SeizureCENORNCT05747001
This is a Retrospective Study on the Use of CENOBAMATE as Adjunctive Treatment in Patients Suffering From Epilepsy in Early Access Program in Germany, France and UK
An observational study of Focal Onset Seizure and Epilepsy, run by Aziende Chimiche Riunite Angelini Francesco S.p.A at 23 sites in 3 countries.
Trial summary
Cenobamate is a newly-FDA and EMA approved drug used to treat -focal-onset seizures in adult patients. The aim of the current study is to analyse retrospectively the overall effectiveness and tolerability of cenobamate from real-world data collected in patients who partecipated in the Early Access Program (EAP) and… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Focal Onset Seizure, Epilepsy
- How many people
- 319 participants
- Where
- 23 sitesin 3 countries
- Study dates
- 27 Jan 2023to 30 Sep 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study population includes adult patients suffering from epilepsy with FOS who have been treated with cenobamate as adjunctive Anti-seizure medication (ASM) during the EAP in Germany, France and UK.
Who may be able to join
- Data from adult patients diagnosed with epilepsy with FOS participating in the EAP with cenobamate as adjunctive treatment, according to the authorization received from the local regulatory or ethic authorities will be collected and analyzed.
- Available data will be collected after obtaining consent from patient/legal representative to the processing of personal data according to the General Data Protection Regulation (GDPR) and applicable local regulation
Who cannot join
- Patient enrolled in other clinical trial during the EAP.
- Patient aged less than 18 years old.
- Patient with specific syndrome (e.g. LGS and Dravet)
Trial locations and contacts
23 sites in 3 countries.
23 sites in 3 countries
Germany12 sites
Epilepsieklinik Tabor
Bernau bei Berlin, 16321
Epilepsy Center Bethel hospital Mara
Bielefeld, 33617
Klinik und Poliklinik für Epileptologie, Universitätsklinikum Bonn
Bonn, 53127
Neurologische Klinik
Erlangen, 91054
Epilepsy Center Frankfurt Rhine-Main Neurocenter
Frankfurt am Main, 60528
Klinik für Neurochirurgie Uniklinik Freiburg -
Freiburg im Breisgau, 79106
Evangelische Krankenhaus Alsterdorf
Hamburg, 22297
Diakonie Kork Epilepsiezentrum
Kehl, 77694
Epilepsiezentrum Hessen, Klinik für Neurologie, Philipps Universität Marburg - Standort Marburg
Marburg, 35043
Epilepsiezentrum Kleinwachau gGmbH
Radeberg, 01454
Neurologie - Stroke Unit - Zentrum für Epilepsie
Reinickendorf, 13509
Universitätsklinikum Tubingen
Tübingen, 72016
France8 sites
Hôpital Pierre Wertheimer - Hopsices Civils de Lyon
Bron, 69677
CHRU de Lille - Hôpital Roger Salengro (LILLE)
Lille, 59037
Centre Hospitalier Universitaire (CHU) de Marseille - Hopital de la Timone
Marseille, 13005
CHRU de Nancy -Hopital Central, Service de Neurologie
Nancy, 54000
Hôpital de la Pitié-Salpêtrière
Paris, 75013
CHU Rennes - Pontchaillou Hospital
Rennes, 35000
CHU de Rouen Hôpital Charles-NicolleService de Neurophysiologie
Rouen, 76000
CHU de Strasbourg - Hôpital de Hautepierre
Strasbourg, 67098
United Kingdom3 sites
King's College Hospital NHS Foundation Trust
London, SE5 9RS
UCLH NHS Trust Epilepsy Department
London, WC1N 3BG
The Newcastle upon Tyne Hospitals
Newcastle, NE1 4LP
About this study
Official title: Retrospective Study on the Use of CENOBAMATE as Adjunctive Treatment in a Cohort of Patients Suffering From Epilepsy With Focal Onset Seizure (FOS) and Enrolled Into the Early Access Program (EAP) in Germany, France and UK
Cenobamate is a newly-FDA and EMA approved drug used to treat -focal-onset seizures in adult patients.
The aim of the current study is to analyse retrospectively the overall effectiveness and tolerability of cenobamate from real-world data collected in patients who partecipated in the Early Access Program (EAP) and were treated with cenobamate as adjunctive ASM.
Read the detailed description
Cenobamate is a new approved drug used to treat -focal-onset seizures in adult patients. This novel tetrazole-derived carbamate seems to act primarily by two mechanisms that are commonly associated with epilepsy: cenobamate acts as a positive allosteric modulator of the GABAA ion channels and is effective in reducing repetitive neuronal firing by inhibition of voltage-gated sodium channels, although the complete mechanism of action is currently unknown.
In clinical trials, cenobamate showed also low toxicity and adverse drug reaction profile.
In European Union (EU), cenobamate received the marketing authorisation, valid throughout the EU, in March 2021. Starting from September 2020 an EAP was initiated with cenobamate as adjunctive ASM in several EU Countries such as Germany, France, and UK.
Real-world data are of importance to understand and confirm the efficacy and safety profile of drugs outside of the clinical trial setting. The aim of the current study is to analyse the overall effectiveness and tolerability of cenobamate from real-world data in a large series of patients treated with cenobamate as adjunctive ASM.
As a consequence, a retrospective collection and analysis of the data of the patients who participated in the EAP, according to the authorization received from the local regulatory or ethic authorities, was conducted.
- Conditions
- Focal Onset Seizure, Epilepsy
- Keywords
- epilepsy, FOS, cenobamate, retrospective, Early Access Program, Europe
- Medical subject headings
- Seizures, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Cohort of patients suffering from epilepsy with Focal Onset Seizure (FOS) and enrolled into the Early Access Program (EAP) in Germany, France and UK |
What is being measured
The primary outcome is the main question the study is designed to answer.
Responder rate (%) at 3 months from the start of maintenance
Percentage of responder rate (defined as a ≥50% reduction from screening/baseline in focal onset seizure frequency) after 3 months of maintenance phase.
Measured: 3 months from the start of maintenance
Secondary outcomes (11)
Portion of responders
Portion of responders (defined as a ≥50% and <100% reduction from screening/baseline in focal onset seizure frequency) at 1 and 3 months after start of cenobamate therapy
Measured: 1 and 3 months after start of cenobamate therapy
Portion of responders
Portion of responders (defined as a ≥50% and <100% reduction from screening/baseline in focal onset seizure frequency) at 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Measured: 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Portion of seizure free
Portion of seizure free (100% reduction from screening/baseline) 1 and 3 months after start of cenobamate therapy
Measured: 1 and 3 months after start of cenobamate therapy
Portion of seizure free
Portion of seizure free (100% reduction from screening/baseline)3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Measured: 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Retention rate
Retention rate measured as percentage of patients remaining in the study and on adjunctive therapy at: 1 and 3 months after start of cenobamate therapy
Measured: 1 and 3 months after start of cenobamate therapy
Retention rate
Retention rate measured as percentage of patients remaining in the study and on adjunctive therapy at 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Measured: 3, 6 and 12 months after the completion of the titration
No. of Adverse Reactions (ADRs),
Adverse Reactions (ADRs), including DRESS, rash/hypersensitivity occurred during the EAP.
Measured: Through study completion, an average of 2 years
Change in Seizures Frequency
Change in Seizures Frequency at 1 and 3 months after start of cenobamate therapy,
Measured: 1 and 3 months after start of cenobamate therapy
Change in Seizures Frequency
Change in Seizures Frequency at 3, 6 and 12 months after the completion of the titration
Measured: 3, 6 and 12 months after the completion of the titration
Assessment of quality of life
The quality of life was assessed through the Questionnaire Quality of Life in Epilepsy Inventory - 31 items
Measured: 1 and 3 months after start of cenobamate therapy
Assessment of quality of life
The quality of life was assessed through the Questionnaire Quality of Life in Epilepsy Inventory - 31 items
Measured: 3, 6 and 12 months after the completion of the titration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2023
- Study start27 Jan 2023
- Primary completion30 Sep 2023
- Study completion30 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Focal Onset SeizureStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 319 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aziende Chimiche Riunite Angelini Francesco S.p.A
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hippocrates Research
- Responsible party
- Sponsor
- Investigators
- Sylvain Rheims, Principal investigator, Hôpital Neurologique Pierre Wertheimer - Hospices Civils de LyonRhys Thoma, Principal investigator, The Newcastle upon Tyne Hospitals NHS Trust Victoria Road, Newcastle, NE1 4LPFelix Rosenow, Principal investigator, Epilepsy Center Frankfurt Rhine-Main Neurocenter Schleusenweg 2 - 16 (Haus 95) 60528 Frankfurt am Main
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05747001
- Sponsor's protocol ID
- 169(A)MD21350
- Acronym
- CENOR
- Status last verified
- Mar 2023
- First submitted
- 30 Jan 2023
- Last updated
- 9 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Study on the Use of CENOBAMATE as Adjunctive Treatment in a Cohort of Patients Suffering From Epilepsy With Focal Onset Seizure (FOS) and Enrolled Into the Early Access Program (EAP) in Germany, France and UK
- ClinicalTrials.gov ID
- NCT05747001
- Acronym / sponsor ID
- CENOR / 169(A)MD21350
- Conditions
- Focal Onset Seizure, Epilepsy
- Keywords
- epilepsy, FOS, cenobamate, retrospective, Early Access Program, Europe
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 319 participants (actual)
- Lead sponsor
- Aziende Chimiche Riunite Angelini Francesco S.p.AIndustry (pharma, biotech, devices)
- Collaborators
- Hippocrates Research
- Study start
- 27 Jan 2023 (actual)
- Primary completion
- 30 Sep 2023 (actual)
- Study completion
- 30 Sep 2023 (actual)
- First posted
- 28 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Cenobamate is a newly-FDA and EMA approved drug used to treat -focal-onset seizures in adult patients.
The aim of the current study is to analyse retrospectively the overall effectiveness and tolerability of cenobamate from real-world data collected in patients who partecipated in the Early Access Program (EAP) and were treated with cenobamate as adjunctive ASM.
Detailed description
Cenobamate is a new approved drug used to treat -focal-onset seizures in adult patients. This novel tetrazole-derived carbamate seems to act primarily by two mechanisms that are commonly associated with epilepsy: cenobamate acts as a positive allosteric modulator of the GABAA ion channels and is effective in reducing repetitive neuronal firing by inhibition of voltage-gated sodium channels, although the complete mechanism of action is currently unknown.
In clinical trials, cenobamate showed also low toxicity and adverse drug reaction profile.
In European Union (EU), cenobamate received the marketing authorisation, valid throughout the EU, in March 2021. Starting from September 2020 an EAP was initiated with cenobamate as adjunctive ASM in several EU Countries such as Germany, France, and UK.
Real-world data are of importance to understand and confirm the efficacy and safety profile of drugs outside of the clinical trial setting. The aim of the current study is to analyse the overall effectiveness and tolerability of cenobamate from real-world data in a large series of patients treated with cenobamate as adjunctive ASM.
As a consequence, a retrospective collection and analysis of the data of the patients who participated in the EAP, according to the authorization received from the local regulatory or ethic authorities, was conducted.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | –Cohort of patients suffering from epilepsy with Focal Onset Seizure (FOS) and enrolled into the Early Access Program (EAP) in Germany, France and UK |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population includes adult patients suffering from epilepsy with FOS who have been treated with cenobamate as adjunctive Anti-seizure medication (ASM) during the EAP in Germany, France and UK.
Key inclusion criteria (2 listed)
- Data from adult patients diagnosed with epilepsy with FOS participating in the EAP with cenobamate as adjunctive treatment, according to the authorization received from the local regulatory or ethic authorities will be collected and analyzed.
- Available data will be collected after obtaining consent from patient/legal representative to the processing of personal data according to the General Data Protection Regulation (GDPR) and applicable local regulation
Key exclusion criteria (3 listed)
- Patient enrolled in other clinical trial during the EAP.
- Patient aged less than 18 years old.
- Patient with specific syndrome (e.g. LGS and Dravet)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Responder rate (%) at 3 months from the start of maintenance
Time frame: 3 months from the start of maintenance
Percentage of responder rate (defined as a ≥50% reduction from screening/baseline in focal onset seizure frequency) after 3 months of maintenance phase.
11 secondary outcomes
Portion of responders
Time frame: 1 and 3 months after start of cenobamate therapy
Portion of responders
Time frame: 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Portion of seizure free
Time frame: 1 and 3 months after start of cenobamate therapy
Portion of seizure free
Time frame: 3, 6 and 12 months after the completion of the titration and its relation with the dosage.
Retention rate
Time frame: 1 and 3 months after start of cenobamate therapy
Retention rate
Time frame: 3, 6 and 12 months after the completion of the titration
No. of Adverse Reactions (ADRs),
Time frame: Through study completion, an average of 2 years
Change in Seizures Frequency
Time frame: 1 and 3 months after start of cenobamate therapy
Change in Seizures Frequency
Time frame: 3, 6 and 12 months after the completion of the titration
Assessment of quality of life
Time frame: 1 and 3 months after start of cenobamate therapy
Assessment of quality of life
Time frame: 3, 6 and 12 months after the completion of the titration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sylvain Rheims principal investigator
Hôpital Neurologique Pierre Wertheimer - Hospices Civils de Lyon
Rhys Thoma principal investigator
The Newcastle upon Tyne Hospitals NHS Trust Victoria Road, Newcastle, NE1 4LP
Felix Rosenow principal investigator
Epilepsy Center Frankfurt Rhine-Main Neurocenter Schleusenweg 2 - 16 (Haus 95) 60528 Frankfurt am Main
23 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Focal Onset Seizure and Epilepsy, run by Aziende Chimiche Riunite Angelini Francesco S.p.A at 23 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in Germany, France, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Responder rate (%) at 3 months from the start of maintenance (time frame: 3 months from the start of maintenance). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2024.