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    Summary
    EudraCT Number:2017-004135-36
    Sponsor's Protocol Code Number:WTX101-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-01-22
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2017-004135-36
    A.3Full title of the trial
    A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months
    Estudio de fase III, multicéntrico, randomizado y con enmascaramiento del evaluador, para evaluar la eficacia y la seguridad de WTX101 administrado durante 48 semanas frente al tratamiento de referencia en pacientes con enfermedad de Wilson de 18 años en adelante con una fase de extensión de hasta 60 meses
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease.
    Estudio de fase III para evaluar la eficacia y la seguridad de WTX101 en pacientes adultos que tienen enfrmedad de Wilson.
    A.4.1Sponsor's protocol code numberWTX101-301
    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 SponsorWilson Therapeutics AB
    B.1.3.4CountrySweden
    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 Address1500 District Ave
    B.5.3.2Town/ cityBurlington
    B.5.3.3Post codeMA 01801
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16175133787
    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.3.1Product nameWTX101
    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 INNNot applied
    D.3.9.1CAS number 649749-10-0
    D.3.9.2Current sponsor codeWTX101
    D.3.9.3Other descriptive nameBIS-CHOLINE TETRATHIOMOLYBDATE
    D.3.9.4EV Substance CodeSUB168578
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.2Name of the Marketing Authorisation holderLaboratorios Rubió S.A.
    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.3.4Pharmaceutical 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 INNPENICILLAMINE
    D.3.9.1CAS number 52-67-5
    D.3.9.4EV Substance CodeSUB09667MIG
    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.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.2Name of the Marketing Authorisation holderUnivar BV
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/03/172
    D.3 Description of the IMP
    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 INNTRIENTINE DIHYDROCHLORIDE
    D.3.9.1CAS number 38260-01-4
    D.3.9.4EV Substance CodeSUB04953MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1.2-2.4 to (4-8 capsules)
    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.IMP: 4
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.2Name of the Marketing Authorisation holderOrphan Europe SARL
    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.3.4Pharmaceutical 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 INNZINC ACETATE DIHYDRATE
    D.3.9.1CAS number 5970-45-6
    D.3.9.4EV Substance CodeSUB12413MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number150 to 3 times daily
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Wilson Disease
    Enfermedad de Wilson
    E.1.1.1Medical condition in easily understood language
    Wilson Disease
    Enfermedad de Wilson
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    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 administered for 48 weeks, compared to standard of care (SoC), on copper (Cu) control in WD subjects aged 18 and older. Copper control will be assessed in terms of the percentage change from baseline (Day 1) to 48 weeks in non-ceruloplasmin-bound copper (NCC) levels. For WTX101-treated subjects, the NCC level will be corrected for the amount of Cu bound to the WTX101 tripartite complex (TPC) (NCCcorrected).
    El objetivo principal es evaluar la eficacia de WTX101 administrado durante 48 semanas, en comparación con el tratamiento de referencia (TR), en el control del cobre (Cu) en pacientes con EW de 18 años en adelante. El control del cobre se evaluará en términos del cambio porcentual desde el periodo basal (día 1) hasta las 48 semanas en los niveles de cobre no unido a ceruloplasmina (NCC). Para los pacientes tratados con WTX101, el nivel de cobre no unido a ceruloplasmina se corregirá por la cantidad de Cu unido al complejo tripartito (CTP) con WTX101 (NCCcorregido).
    E.2.2Secondary objectives of the trial
    The secondary objectives are to:
    - Establish the safety and tolerability of individualised dosing of WTX101;
    - Evaluate the effects of WTX101 on hepatic status assessed by the Model for End-Stage Liver Disease (MELD)
    - Evaluate the effects of WTX101 on disability assessed by the Unified WD Rating Scale (UWDRS) Part II
    - Evaluate the effects of WTX101 on neurological status assessed by the UWDRS Part III
    please see study protocol for more details.
    Los objetivos secundarios son:
    -Establecer la seguridad y la tolerabilidad de la administración individualizada de WTX101;
    -Evaluar los efectos de WTX101 en el estado hepático evaluado por la puntuación del modelo para hepatopatía terminal MELD (Model for End-Stage Liver Disease);
    -Evaluar los efectos de WTX101 sobre la discapacidad evaluada por la Escala de valoración de la EW unificada (Unified WD Rating Scale, UWDRS), Parte II;
    -Evaluar los efectos de WTX101 sobre el estado neurológico evaluado por la escala UWDRS Parte III;
    Por favor consulte el protocolo del estudio para más detalles.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Willing and able to give informed consent for participation in the study;
    - Male or female subjects, aged 18 years or older as of signing the informed consent form (ICF);
    - Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator;
    - Established diagnosis of WD by Leipzig-Score ≥ 4 documented by testing as outlined in the 2012 European Association for the Study of Liver WD Clinical Practice Guidelines;
    - Treatment for >28 days for WD with chelation therapy (ie, penicillamine, trientine hydrochloride), Zn therapy, or a combination of a chelator and Zn; OR Treatment naïve or treatment for ≤ 28 days for WD with chelation therapy (ie, penicillamine, trientine hydrochloride), Zn therapy, or a combination of a chelator and Zn;
    - Willing to undergo ≥ 48-hour washout from current WD treatment;
    - Willing to avoid use of vitamins and/or minerals containing Cu, Zn, or Mo throughout the study duration
    - Willing to avoid intake of foods and drinks with high contents of Cu throughout the study duration;
    please see the study protocol for details
    -Estar dispuesto a dar el consentimiento informado para participar en el estudio y ser capaz de darlo
    -Varones o mujeres, de 18 años en adelante en el momento de la firma del documento de consentimiento informado (DCI)
    -Capaz de entender los procedimientos, restricciones y requisitos del estudio, y estar dispuesto a cumplirlos, a juicio del investigador
    -Diagnóstico confirmado de enfermedad de Wilson mediante una puntuación de Leipzig  4 documentada mediante pruebas como las indicadas en las guías de práctica clínica en la enfermedad de Wilson de 2012 de la Asociación Europea para el Estudio del Hígado
    -Haber recibido tratamiento durante > 28 días contra la enfermedad de Wilson con quelación (es decir, penicilamina, clorhidrato de trientina), Zn o una combinación de un quelante y Zn; O No haber recibido tratamiento anteriormente o haber recibido tratamiento durante 28 días contra la enfermedad de Wilson con quelación (es decir, penicilamina, clorhidrato de trientina), Zn o una combinación de un quelante y Zn
    -Estar dispuesto a someterse a un periodo de reposo farmacológico de  48 horas de los tratamientos contra la enfermedad de Wilson que se estén recibiendo
    -Estar dispuesto a evitar el uso de vitaminas y de minerales que contengan Cu, Zn o Mo durante todo el estudio
    -Estar dispuesto a evitar alimentos y bebidas con alto contenido de Cu durante todo el estudio
    Por favor ver el protocolo del estudio para más detalles
    E.4Principal exclusion criteria
    - Decompensated hepatic cirrhosis;
    - MELD score > 13;
    - Participation in a clinical study of an experimental or unapproved/unlicensed therapy at the same time or within the 4 weeks prior to this Screening Visit;
    - Severe anaemia with a haemoglobin <9 g/dL;
    - Pregnant (or women who are planning to become pregnant) or lactating women;
    - Major systemic disease or other illness that would, in the opinion of the Investigator, compromise subject safety or interfere with the collection or interpretation of study results;
    - Modified Nazer score >7
    - Alanine aminotransferase >2 × ULN for subjects treated for >28 days with WD therapy (Cohort 1);
    - Alanine aminotransferase Alanine aminotransferase > 5 × ULN for treatment naïve subjects or subjects who have been treated for ≤ 28 days (Cohort 2);
    please see study protocol for more details
    -Cirrosis hepática descompensada
    -Puntuación MELD > 13
    -Participación en un estudio clínico de un tratamiento experimental o no aprobado/autorizado simultáneamente o en las 4 semanas previas a la visita de screening
    -Anemia grave con unos niveles de hemoglobina< 9 g/dl
    -Mujeres embarazadas (o que tienen previsto quedarse embarazadas) o en periodo de lactancia
    -Enfermedad sistémica grave u otra afección que, en opinión del investigador, pondría en peligro la seguridad del sujeto o interferiría en la recogida o interpretación de los resultados del estudio
    -Puntuación de Nazer modificada >7
    -Alanina-transaminasa >2 × LSN para los sujetos tratados durante >28 días con el tratamiento de la enfermedad de Wilson (Cohorte 1)
    -Alanina-transaminasa >5 × LSN para los sujetos que no han recibido anteriormente tratamiento o para los sujetos que han recibido tratamiento durante <=28 días (Cohorte 2)
    Por favor ver el protocolo del estudio para más detalles
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy assessment will be control of free Cu, measured as the percent change from baseline (Day 1) to 48 weeks in NCC levels. For WTX101-treated subjects, the NCC level will be corrected for the amount of Cu bound to the WTX101 TPC.
    La evaluación de la variable principal de eficacia será el control de Cu libre, medido como el cambio porcentual desde el periodo basal (día 1) hasta las 48 semanas en los niveles de cobre no unido a ceruloplasmina. Para los pacientes tratados con WTX101, el nivel de cobre no unido a ceruloplasmina se corregirá por la cantidad de Cu unido al complejo tripartito con WTX101.
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    E.5.2Secondary end point(s)
    The secondary endpoints include the following:
    a. Change from baseline in hepatic status at 48 weeks assessed by MELD score;
    b. Change from baseline to 48 weeks in UWDRS Part II score;
    c. Change from baseline to 48 weeks in UWDRS Part III score;
    d. Change from baseline to 48 weeks in the CGI-I and CGI-S; and
    e. NCC responder rate at 48 weeks.
    Los criterios secundarios de valoración incluyen los siguientes:
    a.Cambio respecto al periodo basal en el estado hepático a las 48 semanas, evaluado mediante la puntuación MELD;
    b.Cambio desde el periodo basal hasta las 48 semanas en la escala UWDRS Parte II;
    c.Cambio desde el periodo basal hasta las 48 semanas en la escala UWDRS Parte III;
    d.Cambio desde el periodo basal hasta las 48 semanas en las escalas CGI-I y CGI-S; y
    e.Tasa de respuesta según los niveles de cobre no unido a ceruloplasmina en la semana 48.
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Rater Blinded
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Standard of care (Penicillamine, Trientine, Zinc acetate,or combinations)
    E.8.2.4Number of treatment arms in the trial2
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    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
    Czech Republic
    France
    Germany
    Hungary
    Israel
    Italy
    Poland
    Spain
    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
    Última visita ultimo sujeto(UVUS)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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.1.2.1Number of subjects for this age range: 102
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 72
    F.4.2.2In the whole clinical trial 102
    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)
    Subjects who complete the 48-week treatment period will be offered the opportunity to participate in an Extension Phase of the study to evaluate the long-term safety and durability of treatment effect of WTX101. If the subject does not choose to participate in this Extension Phase, he/she will be assisted in their transition to SoC for WD under the guidance of their local physician.
    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 Decision2018-04-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-27
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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