An Open-label Study of XEN1101 in Epilepsy

NCT05718817 | Enrolling By Invitation Phase 3 DMC FDA Drug

• 2 other identifiers: XPF-010-3042022-502282-24-00
• Study Type: interventional
• Target: 880
• Locations: 22 countries
• Sites: 137

Brief Summary

This study will evaluate the long term safety, tolerability, pharmacokinetics (PK), and efficacy of XEN1101 in subjects with Focal Onset Seizures (FOS) or Primary Generalized Tonic-Clonic Seizures (PGTCS) for the treatment of seizures for up to 6 years.

Conditions

Focal Epilepsy; Tonic-Clonic Seizures

Keywords

Epilepsy; Seizures

Outcome Measures

Primary Outcomes (1)

• The adverse events

  To assess the safety and tolerability of XEN1101

  From the start of treatment in the open-label extension (OLE) study through 8 weeks after the last dose.

Secondary Outcomes (5)

• Change in monthly seizure rate

  From baseline through the active extension treatment (Week 312).

• Proportion of responders

  From baseline through the active extension treatment (Week 312).

• Change in Clinical Global Impression of Severity (CGI-S)

  From baseline through the active extension treatment (Week 312).

• Change in Patient Global Impression of Severity (PGI-S)

  From baseline through the active extension treatment (Week 312).

• Change in Quality of Life in Epilepsy Inventory (QOLIE-31)

  From baseline through the active extension treatment (Week 312).

Study Arms (1)

XEN1101 15 or 25 mg/day

EXPERIMENTAL

XEN1101 15 or 25 mg/day

Drug: XEN1101

Interventions

XEN1101 DRUG

XEN1101 capsules

Also known as: Azetukalner

XEN1101 15 or 25 mg/day

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject must be properly informed of the nature and risks of the study and give informed consent in writing prior to entering the study (for adult subjects) and for adolescent subject's parent/legal guardian and subject gives informed consent or assent in writing prior to entering the study.
• Subject must have successfully completed the double-blind treatment period (DBP) and have not terminated early from Study X-TOLE2, X-TOLE3, or X-ACKT, met all eligibility requirements, and had no important protocol deviations (in the opinion of the sponsor) or adverse events (AEs) (in the opinion of the investigator) that would preclude the subject's entry into the long-term extension study.
• In the opinion of the investigator, the subject is able to understand verbal and written instructions and will adhere to all study schedules and requirements.
• Subject is able to keep accurate seizure diaries.

You may not qualify if:

• Subject met any of the withdrawal criteria while in Study X-TOLE2, X-TOLE3, or X-ACKT.
• Subject has any medical condition, personal circumstance, or ongoing AE (from Study X-TOLE2, X-TOLE3, or X-ACKT) that, in the opinion of the investigator, exposes the subject to unacceptable risk by participating in the study, or prevents adherence to the protocol.
• Subject is planning to enter a clinical study with a different investigational drug or planning to use any experimental device for treatment of epilepsy or any other medical condition during the study and until 28 days after completion of this study.

Sponsors & Collaborators

• Xenon Pharmaceuticals Inc.: lead
• Worldwide Clinical Trials: collaborator

Study Sites (146)

Xenoscience

Phoenix, Arizona, 85004, United States

Location

University of Arizona - Health Sciences

Tucson, Arizona, 85724, United States

Location

Clinical Trials, Inc

Little Rock, Arkansas, 72205, United States

Location

Brain Science Research Institute

Los Angeles, California, 90025, United States

Location

UC Irvine Health

Orange, California, 92868, United States

Location

University of California, Davis Clinical & Translational Science Center Clinical Research (CCRC)

Sacramento, California, 95817, United States

Location

University of Colorado Hospital Anschutz Outpatient Pavilion

Aurora, Colorado, 80045, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

Serenity Research Center, LLC

Miami, Florida, 33176, United States

Location

Research Institute of Orlando, LLC

Orlando, Florida, 32806, United States

Location

Panhandle Research & Medical Clinic

Pensacola, Florida, 10021, United States

Location

University of South Florida

Tampa, Florida, 33606, United States

Location

Encore Medical Research of Weston, LLC

Weston, Florida, 33331, United States

Location

Hawaii Pacific Neuroscience

Honolulu, Hawaii, 96817, United States

Location

Consultants in Epilepsy and Neurology

Boise, Idaho, 83704, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

The University of Kansas Medical Center

Kansas City, Kansas, 66103, United States

Location

Bluegrass Epilepsy Research, LLC

Lexington, Kentucky, 40504, United States

Location

Kentucky Clinic

Lexington, Kentucky, 40536, United States

Location

MMP Neurology

Scarborough, Maine, 04074, United States

Location

University of Maryland Medical Center

Baltimore, Maryland, 21201, United States

Location

Mid-Atlantic Epilepsy and Sleep Center

Bethesda, Maryland, 20817, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

UMass Chan Medical School

Worcester, Massachusetts, 06155, United States

Location

University of Michigan Hospitals

Ann Arbor, Michigan, 48109-2435, United States

Location

Wayne State University

Detroit, Michigan, 48201, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Cornwell Health (Spectrum Health Hospitals)

Grand Rapids, Michigan, 49503, United States

Location

Minneapolis Clinic of Neurology

Burnsville, Minnesota, 55337, United States

Location

Saint Louis University Medical School

St Louis, Missouri, 63104, United States

Location

Northeast Epilepsy Group

Hackensack, New Jersey, 07601, United States

Location

Dent Neurosciences Research Center

Buffalo, New York, 14226, United States

Location

New York University Comprehensive Epilepsy Center

New York, New York, 10016, United States

Location

Mount Sinai Health

New York, New York, 10029, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Montefiiore Medical Center

The Bronx, New York, 10467, United States

Location

Onsite Clinical Solutions

Charlotte, North Carolina, 28211, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Meridian Clinical Research, LLC

Raleigh, North Carolina, 27607, United States

Location

Summa Health

Akron, Ohio, 44304, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

The Ohio State University Brain and Spine Hospital

Columbus, Ohio, 43210, United States

Location

Providence Neurological Specialities

Portland, Oregon, 97225, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Austin Epilepsy Care Center

Austin, Texas, 78758, United States

Location

ANESC Research

El Paso, Texas, 79912, United States

Location

UT Health San Antonio

San Antonio, Texas, 78229, United States

Location

University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, 84132, United States

Location

Carilion Clinic

Roanoke, Virginia, 24013, United States

Location

Sentara Neurology Specialists

Virginia Beach, Virginia, 23456, United States

Location

University of Washington Main Hospital

Seattle, Washington, 10012, United States

Location

Aurora St. Luke's Medical Center

Milwaukee, Wisconsin, 53215, United States

Location

Hospital General de Agudos "José M. Ramos Mejía"

Buenos Aires, 1221, Argentina

Location

Asociacion Medica de Lomas de Zamora S.A. - Policlinicio Lomas

Buenos Aires, B1832BQD, Argentina

Location

STAT Research S.A.

Buenos Aires, C1023AAB, Argentina

Location

Centro de Psicología Médica San Martín de Tours SRL (CITES-INECO)

Buenos Aires, C1060AAF, Argentina

Location

Hospital Italiano de Buenos Aires

Buenos Aires, C1199ABB, Argentina

Location

CENyR, Centro de Especialidades Neurologicas u Rehabilitacion

Buenos Aires, C1424BYD, Argentina

Location

FLENI (Fundación para la Lucha contra las Enfermedades Neurológicas de la Infancia)

Buenos Aires, C1428DGG, Argentina

Location

Hospital Córdoba

Córdoba, X5000, Argentina

Location

Hospital De Alta Complejidad en Red El Cruce - Dr Nester Carlos Kirchner Servicio de Atencion Medica Integral Para la Communidad

San Juan Bautista, 1888, Argentina

Location

Sanatorio del SUR S.A.

San Miguel de Tucumán, 4000, Argentina

Location

Prince of Wales Hospital

Randwick, New South Wales, 2031, Australia

Location

Royal Prince Alfred Hospital

Camperdown, 2050, Australia

Location

St Vincent's Hospital Melbourne

Fitzroy, 3065, Australia

Location

Austin Health Pharmacy Clinical Trials

Heidelberg, 3084, Australia

Location

Southern Neurology

Kogarah, 2217, Australia

Location

Alfred Hospital Neurology CT Dept of Neuroscience

Melbourne, 30004, Australia

Location

The Royal Melbourne Hospital

Parkville, 3050, Australia

Location

Mater Misericordiae Ltd South Brisbane

South Brisbane, 4101, Australia

Location

Westmead Hospital Dept. of Neurology Epilepsy Unit

Westmead, 2145, Australia

Location

University Hospital Innsbruck

Innsbruck, 6020, Austria

Location

Universitatsklinik fur Neurologie, Medizinische Universitat Wien

Vienna, 1090, Austria

Location

Cliniques Universitaires Saint-Luc (UCL)

Brussels, 1200, Belgium

Location

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

Location

Hospital for active treatmnt Puls AD Neurology Dpt

Blagoevgrad, 2700, Bulgaria

Location

MHATNP St. Naum

Sofia, 1113, Bulgaria

Location

Center for Neurologic Research

Lethbridge, Alberta, T1J 0N9, Canada

Location

London Health Sciences Center

London, Ontario, N6A 5A5, Canada

Location

Le Centre Hospitalier de l'Universite' de Montreal (CHUM)

Montreal, H2X 0C1, Canada

Location

Centro de investigación Clinica UC

Santiago, 8330073, Chile

Location

Hospital Clinico Viña del Mar

Viña del Mar, 2520612, Chile

Location

Altavita Centar j.d.o.o.

Zagreb, 10000, Croatia

Location

Poliklinika Bonifarm

Zagreb, 10000, Croatia

Location

University Hospital Centre Zagreb

Zagreb, 10000, Croatia

Location

Motol University Hospital

Prague, 150 06, Czechia

Location

Forbeli s.r.o.

Prague, 160 00, Czechia

Location

Hopital Neurologique Pierre Wertheimer, Hospices Civils de Lyon

Lyon, 69003, France

Location

Hôpital Fondation Adolphe de Rothschild

Paris, 75010, France

Location

Hôpitaux Universitaires Pitié Salpêtrière

Paris, 75013, France

Location

CHU De Rennes - Hôpital Pontchaillou

Rennes, 35033, France

Location

Hôpital de Hautepierre

Strasbourg, 67200, France

Location

American Hospital Network LLC

Tbilisi, 0102, Georgia

Location

Institute of Neurology and Neuropsychology

Tbilisi, 0186, Georgia

Location

Universitätsklinikum Aachen

Aachen, 52074, Germany

Location

Krankenhaus Mara gGmbH

Bielefeld, 33617, Germany

Location

Universitätsklinikum, ZNN - Epilepsiezentrum Frankfurt Rhein-Main

Frankfurt, 60528, Germany

Location

University Hospital Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Philipps-Universität Marburg

Marburg, 35043, Germany

Location

Klinikum der Universität München

München, 81377, Germany

Location

Universitaets-Und Rehabilitations Ulm (RKU)

Ulm, 89081, Germany

Location

Semmelweis Egyetem, Idegsebeszeti es Neurointervencios Klinika

Budapest, 1145, Hungary

Location

Beaumont Hospital

Dublin, NO9 V2NO, Ireland

Location

Hadassah Medical Center (HMC)

Jerusalem, 9112001, Israel

Location

Kaplan Medical Center (KMC)

Rehovot, 76100, Israel

Location

The Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

IRCCS Istituto delle Scienze Neurologiche Bologna

Bologna, 40139, Italy

Location

Universita' Degli Studi Gabriele d'Annunzio Di Chieti

Chieti, 66100, Italy

Location

Azienda Ospedaliero Universitaria Pisana

Pisa, 56126, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Umberto I

Roma, 00185, Italy

Location

Human Science Research Trials

Mexico City, 14050, Mexico

Location

Grupo Medico Camino SC

Mexico City, 3310, Mexico

Location

Neurociencias Estudios Clínicos SC

Sinaloa, Mexico

Location

University of Auckland

Auckland, 1023, New Zealand

Location

Waikato Hospital

Hamilton, 3204, New Zealand

Location

Centrum Medyczne Neuromed

Bydgoszcz, 85-163, Poland

Location

COPERNICUS Podmiot Leczniczy Sp. z o.o.

Gdansk, 80-803, Poland

Location

NZOZ Neuromed M. i M.

Lublin, 20-064, Poland

Location

Twoja Przychodnia Nowosolskie Centrum Medyczne

Nowa Sól, 67-100, Poland

Location

MTZ Clinical Research Powered by Pratia

Warsaw, 02-172, Poland

Location

Neurosphera Sp z o.o.

Warsaw, 03-829, Poland

Location

Centro Hospitalar Universitário de Coimbra (CHUC)

Coimbra, 3000-075, Portugal

Location

Hospital Senhora da Oliviera de Guimaraes

Guimarães, 4835-044, Portugal

Location

Centro Hospitalar Lisboa Ocidental, EPE - Hospital Egas Moniz

Lisbon, 1349-019, Portugal

Location

Centro Hospitalar Universitario de Santo António, E.P.E

Porto, 4050-342, Portugal

Location

Unidade Local de Saúde de São João

Porto, 4200-319, Portugal

Location

Unidade Local Saúde Matosinhos

Porto, 4464-513, Portugal

Location

Centro Hospitalar de Entre o Douro e Vouga

Santa Maria da Feira, 520-211, Portugal

Location

Hospital Universitario Cruces

Barakaldo, 48903, Spain

Location

Vall d'Hebron Hospital

Barcelona, 08035, Spain

Location

Hospital Vithas La Milagrosa

Madrid, 28010, Spain

Location

Hospital Ramón Y Cajal

Madrid, 28034, Spain

Location

Hospital Ruber Internacional

Madrid, 28034, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Universitario Fundación Jiménez Díaz

Madrid, 28040, Spain

Location

Hospital Regional De Malaga

Málaga, 29010, Spain

Location

Hospital La Fe

Valencia, 46026, Spain

Location

Hospital Clinico Universitario de Valladolid

Valladolid, 47003, Spain

Location

Leeds Teaching Hospitals NHS Trust

Leeds, England, LS97TF, United Kingdom

Location

Cardiff and Vale UHB

Cardiff, Wales, CF14 4XW, United Kingdom

Location

Queen Elizabeth Hospital Birmingham

Birmingham, B15 2TH, United Kingdom

Location

St George's Hospital NHS Foundation Trust

London, SW17 0RE, United Kingdom

Location

University College London

London, WC1N 3BG, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX39DU, United Kingdom

Location

Salford Royal NHS Foundation Trust - Greater Manchester Neuroscience Centre (GMNC)

Salford, M6 8HD, United Kingdom

Location

MeSH Terms

Conditions

Epilepsies, Partial; Seizures; Epilepsy

Interventions

XEN1101

Condition Hierarchy (Ancestors)

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

Study Officials

• Medical Director

  Xenon Pharmaceuticals Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 13, 2023
• First Posted: February 8, 2023
• Study Start: April 25, 2023
• Primary Completion (Estimated): April 1, 2033
• Study Completion (Estimated): June 1, 2033
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

IL (3); AU (2); CA (3); FR (5); HU (1); PL (6); GB (7); NZ (2); CZ (2); CR (3); PT (7); IE (1); GE (2); US (53); ME (3); BE (2); BU (2); CL (2); IT (4); AR (10); ES (10); DE (7)