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    Summary
    EudraCT Number:2018-003221-29
    Sponsor's Protocol Code Number:XPF-008-201
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-12-27
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-003221-29
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of XEN1101 as Adjunctive Therapy in Focal-onset Epilepsy, with an Open-label Extension.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multicenter study to evaluate the safety, tolerability and efficacy of XEN1101 as additional therapy to standard treatment in focal epilepsy, with an Open-label Extension.
    A.3.2Name or abbreviated title of the trial where available
    X-TOLE
    A.4.1Sponsor's protocol code numberXPF-008-201
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorXenon Pharmaceuticals Inc
    B.1.3.4CountryCanada
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportXenon Pharmaceuticals Inc
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPivotal S.L.U.
    B.5.2Functional name of contact pointAlberto Viavattene
    B.5.3 Address:
    B.5.3.1Street AddressGobelas 19, La Florida
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28023
    B.5.3.4CountrySpain
    B.5.4Telephone number0034 91 7081250
    B.5.5Fax number0034 91 7081301
    B.5.6E-mailAlberto.Viavattene@pivotalcr.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameXEN1101
    D.3.2Product code XEN1101
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNXEN1101
    D.3.9.2Current sponsor codeXEN1101
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10 to 25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Adult focal (partial onset) epilepsy
    E.1.1.1Medical condition in easily understood language
    Adult focal epilepsy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10065337
    E.1.2Term Focal epilepsy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To assess the efficacy of XEN1101 compared to placebo on focal seizure frequency in adults with focal epilepsy taking 1-3 antiepileptic drugs (AEDs) in the DBP.
    - To assess the safety and tolerability of XEN1101 in adults with focal epilepsy taking 1-3 AEDs in the DBP.
    E.2.2Secondary objectives of the trial
    - To evaluate the 50% XEN1101 response rates in comparison to placebo in the DBP.
    - To evaluate trends in focal seizure frequency over time in the DBP.
    - To assess the effect of XEN1101 vs. placebo on seizure severity and impact in adults with focal epilepsy taking 1-3 AEDs in the DBP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Be properly informed of the nature and risks of the study and give informed consent in writing, prior to entering the study
    - BMI <40 kg/m2
    - Diagnosis (≥2 years) of focal epilepsy according to the International League Against Epilepsy [ILAE] Classification of Epilepsy (2017)
    - Treatment with a stable dose of 1 to 3 allowable current AEDs for at least one month prior to screening, during baseline, and throughout the DBP
    - Must be willing to comply with the contraception requirements
    - Able to keep accurate seizure diaries

    ADDITIONAL CRITERIA FOR THE DBP
    - During the 8-week Baseline period preceding the randomization visit (V3), patients must have a documented seizure frequency of ≥4 focal seizures per 28 days on average.
    - eDiary was completed a min of 80% of all days (i.e., ≥45 days) during the 8 week Baseline period as evidence of adequate compliance.
    - Patients should not be seizure-free for more than 21 consecutive days during the 8-week Baseline period.
    - Patient does not show retinal macular disease, or retinal pigment epithelium abnormality on the dilated ophthalmic exam prior to randomization.

    OPEN-LABEL EXTENSION:
    - Be properly informed of the nature and risks of the study and give informed consent in writing
    - Must have met all eligibility requirements and completed the DBP (to Visit 8* with a minimum of 80% compliance with eDiary entries and IMP), did not terminate early, patient had no important protocol deviations (e.g., that may impact patient safety or data integrity) that in the opinion of Sponsor should preclude participation in the OLE, and had no adverse events that, in the opinion of the Investigator, would preclude the patient’s entry into the OLE
    - Patient is expected to experience benefit from their participation, in the opinion of the Investigator
    - Must be willing to comply with the contraception requirements as defined in the protocol
    - Males must agree not to donate sperm until 6 months after the last dose of study drug. Females must agree not to donate ova until 6 months after the last dose of study drug
    E.4Principal exclusion criteria
    - History of pseudoseizures, psychogenic seizures, primary generalized seizure, or focal aware non-motor seizures only
    - Presence or previous history of Lennox-Gastaut syndrome
    - Seizures secondary to other diseases or conditions
    - History of repetitive seizures within the last 12 months where the individual seizures cannot be counted
    - History of neurosurgery for seizures <1 year prior to enrollment, or radiosurgery <2 years prior to enrollment
    - Schizophrenia and other psychotic disorders, or active suicidal plan/intent in the past 6 months, or a history of suicide attempt in the last 2 years, or more than 1 lifetime suicide attempt
    - History or presence of any significant medical or surgical condition or uncontrolled medical illness at screening, or history of cancer within the past 2 years, with the exception of appropriately treated basal cell or squamous cell carcinoma
    - Any clinically significant abnormalities on pre-study physical examination, vital signs, laboratory values or ECG indicating a medical problem that would preclude study participation including but not limited to:
    a. History of presence of long QT syndrome; QTcF > 450 msec at baseline; family history of sudden death of unknown cause
    b. History of skin or retinal pigment epithelium abnormalities caused by ezogabine
    - Use of vigabatrin in the last 5 years without stable visual fields tested twice over the 12 months after the last dose of vigabatrin (patients stopping vigabatrin more than 5 years prior to screening, must have no vigabatrin-related visual field abnormalities confirmed by examination within the past 6 months - concomitant use of vigabatrin is not allowed)
    - If felbamate is used as a concomitant AED, patients must be taking it for at least 2 years, with a stable dose for ≥49 days and acceptable hematology and LFT values (or discontinued felbamate no less than 49 days) prior to Screening
    - Have had multiple drug allergies or a severe drug reaction to an AED(s), including dermatological (e.g., Stevens-Johnson syndrome), hematological, or organ toxicity reactions
    - Current use of a ketogenic diet
    OPEN-LABEL EXTENSION
    - Patients who met any of the withdrawal criteria in the DBP
    - Any medical condition, personal circumstance, or ongoing adverse event that in the opinion of the Investigator exposes the patient to unacceptable risk by participating in the OLE, or prevents adherence to the protocol
    - Females who are pregnant, breastfeeding or planning to become pregnant until 6 months after the last dose of study drug
    - Patients planning to enter a clinical trial with a different investigational drug or plan to use any experimental device for treatment of epilepsy or any other medical condition
    E.5 End points
    E.5.1Primary end point(s)
    - Median percent change in monthly (28 days) focal seizure frequency from baseline to DBP for XEN1101 versus placebo
    - Severity and frequency of associated adverse events/serious adverse events (AEs/SAEs)
    - Clinically significant changes in clinical laboratory findings
    - Clinically significant changes in 12-lead ECG
    - Increase in suicide risk as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS) including increase in suicidal thoughts or an attempt
    - Clinically significant changes in vital signs including blood pressure, pulse or weight
    - Clinically significant changes in urological symptoms including retention as measured by the American Urological Association (AUA) Symptom Index in the DBP.
    E.5.1.1Timepoint(s) of evaluation of this end point
    When the 180th patient have completed the treatment period
    E.5.2Secondary end point(s)
    - Responders are defined as patients experiencing ≥50% reduction in monthly (28 days) focal seizure frequency from baseline to DBP.
    - Percent change from baseline in weekly focal seizure frequency for each week in the DBP.
    - Clinical and Patient Global Impression of Change scores during the DBP.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At the end of the study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    With an Open-label Extension phase
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA37
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Georgia
    Germany
    Moldova, Republic of
    Spain
    Ukraine
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days30
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 250
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 50
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state35
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 185
    F.4.2.2In the whole clinical trial 300
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-02-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-21
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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