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    Summary
    EudraCT Number:2014-001703-41
    Sponsor's Protocol Code Number:WTX101-201
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-01-29
    Trial results View 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 number2014-001703-41
    A.3Full title of the trial
    A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 12 Months
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks with an Extension Phase of 12 Months
    A.3.2Name or abbreviated title of the trial where available
    Wilson Disease Patients to evaluate efficacy and safety of WTX101
    A.4.1Sponsor's protocol code numberWTX101-201
    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 Sponsor Wilson Therapeutics AB
    B.1.3.4CountryUnited States
    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 supportWilson Therapeutics AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationWilson Therapeutics AB
    B.5.2Functional name of contact pointSusan Flint
    B.5.3 Address:
    B.5.3.1Street Address15 New England Executive Park
    B.5.3.2Town/ cityBurlington
    B.5.3.3Post codeMA 01803
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 617 513 3787
    B.5.5Fax number+1 978 692 6816
    B.5.6E-mailsusan.flint@wilsontx.eu
    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 Yes
    D.2.5.1Orphan drug designation numberEU3/12/1089
    D.3 Description of the IMP
    D.3.2Product code WTX101
    D.3.4Pharmaceutical form Tablet
    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 INNnone
    D.3.9.1CAS number 64-9749-10-0
    D.3.9.2Current sponsor codeWTX101
    D.3.9.3Other descriptive namebis-choline TETRATHIOMOLYBDATE
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Wilson Disease
    E.1.1.1Medical condition in easily understood language
    Wilson's disease is an autosomal recessive genetic disorder in which copper accumulates in tissues; this manifests as neurological or psychiatric symptoms and liver disease.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10047988
    E.1.2Term Wilson's disease
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the efficacy of WTX101 for 24 weeks on non-ceruloplasmin-bound copper (NCC) levels adjusted for molybdenum (Mo) plasma concentration in newly diagnosed Wilson Disease (WD) patients aged 18 and older with NCC levels within or above the normal reference range at the time of enrolment.
    E.2.2Secondary objectives of the trial
    - Establish the safety and tolerability of individualised dosing of WTX101 over the course of the study period
    - Evaluate: change in and time to normalisation of NCC levels adjusted for Mo plasma concentration; the effects of WTX101 on neurological status using the UWDRS (neurological subscore; parts I, II and III); the effects of WTX101 on psychiatric status using M.I.N.I. Tracking; the global effects of WTX101 on clinical symptoms as assessed by the Investigators on the CGI scale items 1 (severity of illness) and 2 (global improvement); the effects of WTX101 on the following QoL / PRO endpoint measures: EQ5D, MAQ-8 and the TSQM, the effects of WTX101 on hepatic measures
    - Analyse copper endpoints (exchangeable copper, speciation profiling, and 24-hour urinary copper)
    - Collect PK data on WTX101 in this patient population based on the measurement of plasma total Mo
    - Evaluate the durability, and establish long-term safety and efficacy of WTX101 in a 12 month Extension Phase
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to be eligible for enrolment in this study:
    1. Willing and able to give informed consent for participation in the study.
    2. Male or female patients, aged 18 years or older as of signing the ICF.
    3. Able to understand and willing to comply with study procedures, restrictions and requirements, as judged by the Investigator.
    4. Newly established diagnosis of Wilson Disease by Leipzig-Score  4 (Ferenci et al 2003) documented by testing as outlined in 2012 EASL WD Clinical Practice
    Guidelines.
    5. NCC levels above the normal reference range (0.8-2.3 μM).
    6. Willing to undergo 48 hour washout from current Wilson Disease treatment.
    7. Adequate venous access to allow collection of a number of blood samples.
    8. Willing to avoid intake of foods and water with high concentrations of copper
    throughout the duration of the study.
    9. Females of childbearing potential will be included if they are either sexually inactive (abstinent) for 14 days prior to the first WTX101 dose continuing through 28 days after the last WTX101 dose, or using one of the following highly effective birth control methods:
    a. intrauterine device (IUD) (without Cu);
    b. surgical sterilization of the partner (vasectomy for 6 months minimum);
    c. combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
    d. progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
    e. intrauterine hormone releasing system (IUS);
    f. bilateral tubal occlusion.
    Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. In this trial abstinence is only acceptable if in line with the subjects preferred and usual lifestyle.
    Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method (LAM) are not acceptable methods of contraception. As well, female condom and male condom should not be used together.
    10. Females of childbearing potential agree to remain sexually inactive or to keep the same birth control method for at least 28 days following the last dose.
    11. A female of non-childbearing potential must have undergone one of the following sterilization procedures at least 6 months prior to the first WTX101 dose:
    a. hysteroscopic sterilization;
    b. bilateral tubal ligation or bilateral salpingectomy;
    c. hysterectomy;
    d. bilateral oophorectomy;
    or be postmenopausal with amenorrhea for at least 1 year prior to the first WTX101 dose and follicle stimulating hormone (FSH) serum levels consistent with
    postmenopausal status.
    12. A non-vasectomized male subject agrees to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study medication and the female partner agrees to comply with inclusion 9 or 11. For a vasectomized male who has had his vasectomy 6 months or more prior to study start, it is required that they use a condom during sexual intercourse. A male who has been vasectomized less than 6 months prior to study start must follow the same restrictions as a non- vasectomized male.
    Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. In this trial abstinence is only acceptable if in line with the subjects preferred and usual lifestyle.
    Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method (LAM) are not acceptable methods of contraception. As well, female condom and male condom should not be used together.
    10. Females of childbearing potential agree to remain sexually inactive or to keep the same birth control method for at least 28 days following the last dose.
    13. If male, agrees not to donate sperm from the first WTX101 dose until 90 days after dosing.
    E.4Principal exclusion criteria
    1. Treatment for greater than 24 months for Wilson Disease with chelation therapy (i.e.penicillamine, trientine hydrochloride) or zinc therapy.
    2. Decompensated hepatic cirrhosis.
    3. Model for End-Stage Liver Disease (MELD) score > 11.
    4. Modified Nazer score > 6 (Dhawan et al. Liver Transplant 2005).
    5. GI bleed within past 6 months.
    6. ALT > 5x upper limit of normal (ULN).
    7. Marked neurological disease requiring either nasogastric (NG) feeding or intensive in-patient medical care.
    8. Severe anaemia with a haemoglobin < 9 mg/dL.
    9. Participation in a clinical trial of an experimental or unapproved/unlicensed therapy at the same time or within the 4 weeks prior to this study.
    10. History of seizure activity within 6 months of study start.
    11. Pregnant (or women who are planning to become pregnant) or lactating women.
    12. Known sensitivity to WTX101, WTX101 excipients (anhydrous di-calcium
    phosphate, anhydrous sodium carbonate), any of the ingredients contained in
    WTX101, PPIs or to related compounds.
    13. Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus (patients with positive hepatitis C antibody result would require confirmation of active disease with a positive hepatitis C polymerase chain reaction (PCR) test), seropositivity for human immunodeficiency virus (HIV).
    14. Any disability acquired from trauma or another illness that, in the opinion of the Investigator, could interfere with evaluation of disability due to WD.
    15. Previous treatment with tetrathiomolybdate.
    16. Major systemic disease or other illness that would, in the opinion of the Investigator, compromise patient safety or interfere with the collection or interpretation of study results.
    17. In the opinion of the Investigator, the patient is likely to be non-compliant or uncooperative during the study.
    18. Any deviation in laboratory values that are confirmed on re-examination to be
    clinically significant by the Investigator that would jeopardise the safety of the patient or impact the validity of the study results.
    E.5 End points
    E.5.1Primary end point(s)
    Non-ceruloplasmin-bound copper (NCC) levels adjusted for molybdenum (Mo) plasma concentration
    E.5.1.1Timepoint(s) of evaluation of this end point
    Evaluation is done over a period of 24 weeks
    E.5.2Secondary end point(s)
    - Change in NCC levels adjusted for Mo plasma concentration
    - Time to normalisation of NCC adjusted for Mo plasma concentration
    - Change in exchangeable copper during 24 weeks of treatment
    - Change in speciation profiling (Mo, Cu and protein complex profiling with SEC) during 24 weeks of treatment
    - Change in 24-hour urinary copper during 24 weeks of treatment
    - Changes in hepatic measures (ALT, AST, INR and bilirubin)
    - Changes in neurological status using the UWDRS (neurological subscore; parts I, II and III) and additionally:
    - Number of patients that deteriorate = increase ≥ 4 (part III of neurological sub-score) or increase ≥ 1 (part II of neurological sub- score).
    - Number of patients that improve = decrease ≥ 4 (part III of neurological sub-score) or decrease ≥ 1 (part II of neurological sub- score).
    - Changes in psychiatric status using M.I.N.I. Tracking
    - Changes in the global effects of WTX101 on clinical symptoms assessed by the Investigators on the CGI scale items 1 (severity of illness) and 2 (global improvement)
    - Absolute changes in QOL / PRO measures EQ5D, MAQ-8, TSQM
    - Pharmacokinetic (PK) data based on measurement of plasma total molybdenum (Mo)
    - Durability and long term efficacy of WTX101 in a 12 month Extension Phase.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Evaluation is done over a period of 24 weeks.
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Austria
    Germany
    Poland
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    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 years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days17
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days12
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 0
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    In case a patient is unable to give consent him/herself due to neurological disease, it will be possible for a caregiver to provide
    informed consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 30
    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 are offered the opportunity to participate in the 12 month
    extension phase to evaluate the durability, and establish long-term safety and efficacy of WTX101 or are assisted in their transition to standard of care for WD under the guidance of their local physician.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation NIHR CRN National Coordinating Centre reference: HEPA - 4066
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-03-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-11-07
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