An Open-label Study of XEN1101 in Epilepsy

NCT ID: NCT05718817

Last Updated: 2025-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

880 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-25

Study Completion Date

2028-09-30

Brief Summary

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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 3 years.

Detailed Description

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This is an Open Label Extension study of the following Phase 3 clinical studies: XPF-010-301 (X-TOLE2), XPF-010-302 (X-TOLE3), and XPF-010-303 (X-ACKT). This study will evaluate the long-term safety, tolerability, PK, and efficacy of XEN1101 in subjects with FOS or PGTCS for the treatment of seizures for up to 3 years. Subjects who successfully completed and did not terminate early from one of the antecedent studies (X-TOLE2, X-TOLE3, or X-ACKT) are eligible to participate in X-TOLE4.

Following enrollment into X-TOLE4, subjects will undergo a treatment period of up to 3 years, during which there will be a visit at 2-, 4-, and 13-weeks post-entry, with subsequent visits occurring at 13-week intervals during the first year, and then at 26-week intervals (with a telephone call in between) until dosing is completed.

Subjects will be initially assigned to XEN1101 as follows:

* 25 mg QD for subjects aged ≥18 years
* For subjects aged ≥12 and \<18 years

* 15 mg QD for those

* who weigh \<45 kg at the start of the X-TOLE4 study
* who weighed \<45 kg during the X-ACKT study
* who weighed ≥45 kg at randomization in the X-ACKT study and had dose reduction(s) for intolerability
* 25 mg QD for all others

Subjects will be instructed to orally take XEN1101 once daily (QD) with an evening meal. Subjects will be expected to keep a daily seizure eDiary with a minimum of 80% compliance for the duration of the extension study (reporting on ≥80% of days between visits).

Upon completion of dosing at the end of the treatment period, there will be an 8-week follow up period.

Conditions

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Focal Epilepsy Tonic-Clonic Seizures

Keywords

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Epilepsy Seizures

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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XEN1101 15 or 25 mg/day

XEN1101 15 or 25 mg/day

Group Type EXPERIMENTAL

XEN1101

Intervention Type DRUG

XEN1101 capsules

Interventions

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XEN1101

XEN1101 capsules

Intervention Type DRUG

Other Intervention Names

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Azetukalner

Eligibility Criteria

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Inclusion Criteria

1. 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.
2. 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.
3. 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.
4. Subject is able to keep accurate seizure diaries.

Exclusion Criteria

1. Subject met any of the withdrawal criteria while in Study X-TOLE2, X-TOLE3, or X-ACKT.
2. 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.
3. 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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Worldwide Clinical Trials

OTHER

Sponsor Role collaborator

Xenon Pharmaceuticals Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Xenon Pharmaceuticals Inc.

Locations

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Xenoscience

Phoenix, Arizona, United States

Site Status

University of Arizona - Health Sciences

Tucson, Arizona, United States

Site Status

Clinical Trials, Inc

Little Rock, Arkansas, United States

Site Status

Brain Science Research Institute

Los Angeles, California, United States

Site Status

UC Irvine Health

Orange, California, United States

Site Status

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

Sacramento, California, United States

Site Status

University of Colorado Hospital Anschutz Outpatient Pavilion

Aurora, Colorado, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

Serenity Research Center, LLC

Miami, Florida, United States

Site Status

Research Institute of Orlando, LLC

Orlando, Florida, United States

Site Status

Panhandle Research & Medical Clinic

Pensacola, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Encore Medical Research of Weston, LLC

Weston, Florida, United States

Site Status

Hawaii Pacific Neuroscience

Honolulu, Hawaii, United States

Site Status

Consultants in Epilepsy and Neurology

Boise, Idaho, United States

Site Status

Indiana University School of Medicine

Indianapolis, Indiana, United States

Site Status

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Bluegrass Epilepsy Research, LLC

Lexington, Kentucky, United States

Site Status

Kentucky Clinic

Lexington, Kentucky, United States

Site Status

MMP Neurology

Scarborough, Maine, United States

Site Status

University of Maryland Medical Center

Baltimore, Maryland, United States

Site Status

Mid-Atlantic Epilepsy and Sleep Center

Bethesda, Maryland, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

UMass Chan Medical School

Worcester, Massachusetts, United States

Site Status

University of Michigan Hospitals

Ann Arbor, Michigan, United States

Site Status

Wayne State University

Detroit, Michigan, United States

Site Status

Michigan State University

East Lansing, Michigan, United States

Site Status

Cornwell Health (Spectrum Health Hospitals)

Grand Rapids, Michigan, United States

Site Status

Minneapolis Clinic of Neurology

Burnsville, Minnesota, United States

Site Status

Northeast Epilepsy Group

Hackensack, New Jersey, United States

Site Status

Dent Neurosciences Research Center

Buffalo, New York, United States

Site Status

New York University Comprehensive Epilepsy Center

New York, New York, United States

Site Status

Mount Sinai Health

New York, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Montefiiore Medical Center

The Bronx, New York, United States

Site Status

Onsite Clinical Solutions

Charlotte, North Carolina, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Meridian Clinical Research, LLC

Raleigh, North Carolina, United States

Site Status

Summa Health

Akron, Ohio, United States

Site Status

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Providence Neurological Specialities

Portland, Oregon, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Austin Epilepsy Care Center

Austin, Texas, United States

Site Status

ANESC Research

El Paso, Texas, United States

Site Status

UT Health San Antonio

San Antonio, Texas, United States

Site Status

University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, United States

Site Status

Carilion Clinic

Roanoke, Virginia, United States

Site Status

Sentara Neurology Specialists

Virginia Beach, Virginia, United States

Site Status

University of Washington Main Hospital

Seattle, Washington, United States

Site Status

Aurora St. Luke's Medical Center

Milwaukee, Wisconsin, United States

Site Status

Hospital General de Agudos "José M. Ramos Mejía"

Buenos Aires, , Argentina

Site Status

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

Buenos Aires, , Argentina

Site Status

STAT Research S.A.

Buenos Aires, , Argentina

Site Status

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

Buenos Aires, , Argentina

Site Status

Hospital Italiano de Buenos Aires

Buenos Aires, , Argentina

Site Status

CENyR, Centro de Especialidades Neurologicas u Rehabilitacion

Buenos Aires, , Argentina

Site Status

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

Buenos Aires, , Argentina

Site Status

Hospital Córdoba

Córdoba, , Argentina

Site Status

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

San Juan Bautista, , Argentina

Site Status

Sanatorio del SUR S.A.

San Miguel de Tucumán, , Argentina

Site Status

Prince of Wales Hospital

Randwick, New South Wales, Australia

Site Status

Royal Prince Alfred Hospital

Camperdown, , Australia

Site Status

St Vincent's Hospital Melbourne

Fitzroy, , Australia

Site Status

Austin Health Pharmacy Clinical Trials

Heidelberg, , Australia

Site Status

Southern Neurology

Kogarah, , Australia

Site Status

Alfred Hospital Neurology CT Dept of Neuroscience

Melbourne, , Australia

Site Status

The Royal Melbourne Hospital

Parkville, , Australia

Site Status

Mater Misericordiae Ltd South Brisbane

South Brisbane, , Australia

Site Status

Westmead Hospital Dept. of Neurology Epilepsy Unit

Westmead, , Australia

Site Status

University Hospital Innsbruck

Innsbruck, , Austria

Site Status

Universitatsklinik fur Neurologie, Medizinische Universitat Wien

Vienna, , Austria

Site Status

Cliniques Universitaires Saint-Luc (UCL)

Brussels, , Belgium

Site Status

Universitair Ziekenhuis Gent

Ghent, , Belgium

Site Status

Hospital for active treatmnt Puls AD Neurology Dpt

Blagoevgrad, , Bulgaria

Site Status

MHATNP St. Naum

Sofia, , Bulgaria

Site Status

Center for Neurologic Research

Lethbridge, Alberta, Canada

Site Status

London Health Sciences Center

London, Ontario, Canada

Site Status

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

Montreal, , Canada

Site Status

Centro de investigación Clinica UC

Santiago, , Chile

Site Status

Hospital Clinico Viña del Mar

Viña del Mar, , Chile

Site Status

Altavita Centar j.d.o.o.

Zagreb, , Croatia

Site Status

Poliklinika Bonifarm

Zagreb, , Croatia

Site Status

University Hospital Centre Zagreb

Zagreb, , Croatia

Site Status

Motol University Hospital

Prague, , Czechia

Site Status

Forbeli s.r.o.

Prague, , Czechia

Site Status

Hopital Neurologique Pierre Wertheimer, Hospices Civils de Lyon

Lyon, , France

Site Status

Hôpital Fondation Adolphe de Rothschild

Paris, , France

Site Status

CHU De Rennes - Hôpital Pontchaillou

Rennes, , France

Site Status

Hôpital de Hautepierre

Strasbourg, , France

Site Status

American Hospital Network LLC

Tbilisi, , Georgia

Site Status

Institute of Neurology and Neuropsychology

Tbilisi, , Georgia

Site Status

Krankenhaus Mara gGmbH

Bielefeld, , Germany

Site Status

Universitätsklinikum, ZNN - Epilepsiezentrum Frankfurt Rhein-Main

Frankfurt, , Germany

Site Status

University Hospital Freiburg

Freiburg im Breisgau, , Germany

Site Status

Philipps-Universität Marburg

Marburg, , Germany

Site Status

Klinikum der Universität München

München, , Germany

Site Status

Universitaets-Und Rehabilitations Ulm (RKU)

Ulm, , Germany

Site Status

Semmelweis Egyetem, Idegsebeszeti es Neurointervencios Klinika

Budapest, , Hungary

Site Status

Beaumont Hospital

Dublin, , Ireland

Site Status

Hadassah Medical Center (HMC)

Jerusalem, , Israel

Site Status

Kaplan Medical Center (KMC)

Rehovot, , Israel

Site Status

The Tel Aviv Sourasky Medical Center

Tel Aviv, , Israel

Site Status

IRCCS Istituto delle Scienze Neurologiche Bologna

Bologna, , Italy

Site Status

Universita' Degli Studi Gabriele d'Annunzio Di Chieti

Chieti, , Italy

Site Status

Azienda Ospedaliero Universitaria Pisana

Pisa, , Italy

Site Status

Azienda Ospedaliero Universitaria Policlinico Umberto I

Roma, , Italy

Site Status

Human Science Research Trials

Mexico City, , Mexico

Site Status

Grupo Medico Camino SC

Mexico City, , Mexico

Site Status

Neurociencias Estudios Clínicos SC

Sinaloa, , Mexico

Site Status

University of Auckland

Auckland, , New Zealand

Site Status

Waikato Hospital

Hamilton, , New Zealand

Site Status

Centrum Medyczne Neuromed

Bydgoszcz, , Poland

Site Status

COPERNICUS Podmiot Leczniczy Sp. z o.o.

Gdansk, , Poland

Site Status

NZOZ Neuromed M. i M.

Lublin, , Poland

Site Status

Twoja Przychodnia Nowosolskie Centrum Medyczne

Nowa Sól, , Poland

Site Status

MTZ Clinical Research Powered by Pratia

Warsaw, , Poland

Site Status

Neurosphera Sp z o.o.

Warsaw, , Poland

Site Status

Centro Hospitalar Universitário de Coimbra (CHUC)

Coimbra, , Portugal

Site Status

Hospital Senhora da Oliviera de Guimaraes

Guimarães, , Portugal

Site Status

Centro Hospitalar Lisboa Ocidental, EPE - Hospital Egas Moniz

Lisbon, , Portugal

Site Status

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

Porto, , Portugal

Site Status

Unidade Local de Saúde de São João

Porto, , Portugal

Site Status

Unidade Local Saúde Matosinhos

Porto, , Portugal

Site Status

Centro Hospitalar de Entre o Douro e Vouga

Santa Maria da Feira, , Portugal

Site Status

Hospital Universitario Cruces

Barakaldo, , Spain

Site Status

Vall d'Hebron Hospital

Barcelona, , Spain

Site Status

Hospital Vithas La Milagrosa

Madrid, , Spain

Site Status

Hospital Ramón Y Cajal

Madrid, , Spain

Site Status

Hospital Ruber Internacional

Madrid, , Spain

Site Status

Hospital Clinico San Carlos

Madrid, , Spain

Site Status

Hospital Universitario Fundación Jiménez Díaz

Madrid, , Spain

Site Status

Hospital Regional De Malaga

Málaga, , Spain

Site Status

Hospital La Fe

Valencia, , Spain

Site Status

Hospital Clinico Universitario de Valladolid

Valladolid, , Spain

Site Status

Leeds Teaching Hospitals NHS Trust

Leeds, England, United Kingdom

Site Status

Cardiff and Vale UHB

Cardiff, Wales, United Kingdom

Site Status

Queen Elizabeth Hospital Birmingham

Birmingham, , United Kingdom

Site Status

St George's Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

University College London

London, , United Kingdom

Site Status

John Radcliffe Hospital

Oxford, , United Kingdom

Site Status

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

Salford, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Belgium Bulgaria Canada Chile Croatia Czechia France Georgia Germany Hungary Ireland Israel Italy Mexico New Zealand Poland Portugal Spain United Kingdom

Other Identifiers

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2022-502282-24-00

Identifier Type: CTIS

Identifier Source: secondary_id

XPF-010-304

Identifier Type: -

Identifier Source: org_study_id