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    Summary
    EudraCT Number:2009-015619-42
    Sponsor's Protocol Code Number:XPF-001-202
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2009-10-01
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2009-015619-42
    A.3Full title of the trial
    Phase 2a, Single-Blind, Placebo-Controlled, 3-Period, 2-Treatment, Crossover Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Multiple Oral Doses of XPF-001 in Patients with Inherited Erythromelalgia
    A.4.1Sponsor's protocol code numberXPF-001-202
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 nameXPF-001
    D.3.2Product code XPF-001
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeXEN402
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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, hard
    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
    Inherited erythromelalgia
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.0
    E.1.2Level LLT
    E.1.2Classification code 10015284
    E.1.2Term Erythromelalgia
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To evaluate the safety and efficacy (onset, duration of relief and overall efficacy) of multiple doses of XPF-001 (400 mg bid) for relief of pain in patients with IEM.
    - To evaluate the efficacy of multiple doses of XPF-001 (400 mg bid) for relief of vasomotor signs in patient with IEM.
    - To evaluate the PK profile of XPF-001 and correlate plasma levels of drug to the pharmacodynamic/efficacy endpoints.
    E.2.2Secondary objectives of the trial
    · QST thresholds to electrical and thermal stimulations
    · QST central sensitization to electrical and thermal stimulations
    · TOTPAR-4 and SPID-4
    · SPID-6
    · TOTPAR-8 and SPID-8
    · Time to first perceptible relief (confirmed)
    · Time to meaningful relief
    · Mean PID and REL scores at each observation time
    · Time to rescue medication
    · Amount of rescue medication per day
    · Average total daily dose of rescue medication
    · Duration of relief
    · Cumulative percentage of subjects taking rescue medication by each time point
    · Percentage of subjects rescuing at 2, 4, 6, 8, and 12 hours
    · Global Evaluation (GE) at 6 hours post morning-dose
    Safety evaluations will include medical history, physical examination, vital signs, ECGs, laboratory tests, and AEs.
    It is planned that pharmacokinetic variables Cmax, Tmax, AUC0-t, and others parameters of interest, will be evaluated.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects will include males and females 18 – 75 years of age (inclusive), with a BMI between 19.5 and 34.0 kg/m2 (inclusive) who have a clinical and genetic diagnosis of Inherited Erythromelalgia (IEM);
    Subjects must (either spontaneously, or using one of the protocol-defined pain induction methods) achieve pain scores of
    NRS ³4, and/or moderate/severe on the CRS, or an intolerable level of pain irrespective of pain scores;
    Subjects must be willing to comply with all study procedures, including the stopping use of all medication, including those for pain management between Check-in and Discharge;
    Subjects must be in general good health and have no contraindications to the IMP, its excipients, or the permitted rescue medication.
    E.4Principal exclusion criteria
    Subjects with a coexistent source of pain from other conditions that may interfere with the study interpretation;
    History or evidence of any condition that, in the opinion of the PI, may pose undue risk to the subject;
    A positive test for HIV, Hepatitis C antibodies, or Hepatitis B surface antigen;
    Use of any prescription or over the counter (OTC) medication or supplement from Check-in until Discharge (except for rescue medication);
    Receiving professional psychological support specifically for coping with IEM;
    Treatment for significant depression within the 6 months prior to Screening;
    Females who are pregnant, lactating, or who test positive on a serum-based regnancy test at Screening or Check-in;
    Not currently undertaking adequate measures to prevent a pregnancy throughout the entire study;
    Clinically significant abnormal laboratory values, ECG, vital signs or physical examination findings at Screening or Check-in;
    History or presence of alcoholism or alcohol or substance abuse (not including nicotine or caffeine);
    Presence or history of major psychiatric disturbance and/or substance abuse, or a positive urine drug test at Check-in;
    Ingestion of any caffeine-containing food or beverages (including chocolate) in excess of the permitted 1 cup of caffeine containing beverage per day, between Day -1 until Discharge;
    Consumption of alcohol from Check-in until Discharge, or a positive alcohol breath test on Check-in;
    Consumption of grapefruit or grapefruit containing products within 7 days of Day 1 until Discharge;
    Smoking more than 3 cigarettes per day (or the equivalent in tobacco or nicotine substitutes) within the 1 week prior to ICheck-in, and the inability to refrain from all nicotine use between Check-in and Discharge;
    Has taken an investigational drug within the 60 days prior to Day 1;
    Donation or loss of whole blood or plasma (excluding the volume of blood that will be drawn during the Screening procedures of this study) prior to Day 1 as follows: 50 mL to 499 mL within 30 days, or more than 499 mL within 56 days prior to drug administration;
    Has previously been enrolled into this study;
    Study site or Sponsor employee or relative of an employee who is directly involved in the study;
    Any other reason that would make the subject, in the opinion of the PI or Sponsor, unsuitable for the study.
    E.5 End points
    E.5.1Primary end point(s)
    TOTPAR-6 (Total Pain Relief score of 6 on an 11-point Numerical Rating Scale (NRS), where 0 = no pain at all, 10 = worst pain imaginable).
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.8.1.2Open No
    E.8.1.3Single blind Yes
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    Last patient's last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 state10
    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)
    Patients will return to using their usual pain medication(s).
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2009-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-02-09
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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