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    Summary
    EudraCT Number:2012-004786-40
    Sponsor's Protocol Code Number:PB-102-F01
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-09-05
    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 number2012-004786-40
    A.3Full title of the trial
    A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients
    Estudio de fase 1/2, abierto, de determinación de la dosis para evaluar la seguridad, la tolerancia, la farmacocinética y los parámetros de eficacia exploratoria de PRX-102 administrado mediante infusión intravenosa cada 2 semanas durante 12 semanas a pacientes adultos con enfermedad de Fabry
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion to Adult Fabry Patients
    Estudio para evaluar la seguridad, la tolerancia, la farmacocinética y los parámetros de eficacia exploratoria de PRX-102 administrado mediante infusión intravenosa a pacientes adultos con enfermedad de Fabry
    A.4.1Sponsor's protocol code numberPB-102-F01
    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 SponsorProtalix Ltd.
    B.1.3.4CountryIsrael
    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 supportProtalix Ltd.
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCato Europe GmbH
    B.5.2Functional name of contact pointRainer Schuckelt
    B.5.3 Address:
    B.5.3.1Street AddressHertzstr. 7
    B.5.3.2Town/ cityKöln
    B.5.3.3Post code50859
    B.5.3.4CountryGermany
    B.5.4Telephone number004922343794411
    B.5.5Fax number004922343794425
    B.5.6E-mailr.schuckelt@catoeurope.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 namePRX102
    D.3.2Product code PRX102
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPRX102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX102
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 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 namePRX102
    D.3.2Product code PRX102
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPRX102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX102
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 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 namePRX102
    D.3.2Product code PRX102
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPRX102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX102
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    Fabry disease
    Enfermedad de Fabry
    E.1.1.1Medical condition in easily understood language
    Fabry disease is an inherited condition caused by a deficiency of an enzyme, leading to a range of systemic symptoms.
    La enfermedad de Fabry es un trastorno genético heredado provocado por la deficiencia de una enzima que conduce a una amplia gama de síntomas.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level PT
    E.1.2Classification code 10016016
    E.1.2Term Fabry's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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, tolerability, pharmacokinetics and exploratory efficacy parameters of PRX-102 in adult Fabry patients.
    Evaluar la seguridad, tolerabilidad, farmacocinética y parámetros de eficacia exploratoria de PRX-102 en pacientes adultos con enfermedad de Fabry.
    E.2.2Secondary objectives of the trial
    -Gb3 concentrations assessed by Gb3 concentrations in plasma and urine sediment. (Gb3 is a component in the cell membrane and its accumulation in body tissues results in Fabry Disease).
    -Globotriaosylsphingosine(LysoGb3) concentrations assessed by LysoGb3
    concentrations in plasma
    -Kidney function measurement of glomerular filtration and proteinuria
    -Pain assessed by a short term brief pain inventory
    -Assessment of gastrointestinal symptoms
    -Las concentraciones basales de Gb3 en plasma y sedimento urinario (Gb3 es un componente de la membrana celular y se acumula en los tejidos del cuerpo como resultado de la enfermedad de Fabry)
    -La concentración basal de globotriaosilsfingosina (Liso-Gb3) en plasma, y en cada infusión durante el ensayo
    -Evaluación de síntomas gastrointestinales en a nivel basal y en la última infusión
    -Funciones renales (eGFR y proteinuria) en nivel basal y en la última infusión
    -Cuestionario breve del dolor, formulario abreviado (Brief Pain Inventory-Short Form, BPI-SF) en nivel basal y en la última infusión

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Symptomatic adult Fabry patients (> or = to 18 yrs, males and females)
    2.Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein)
    3.Females: historical genetic test results consistent with Fabry mutations
    4.Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
    5.Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti PRX-102 antibody test
    6.Chronic kidney disease - stages 1 or 2 (CKD1 or 2) (Appendix 7) with proteinuria > 200 mg/g protein-to-creatinine ratio measured in a Spot urine sample or equivalent demonstrated in at least one of 2 separate samples (one sample in screening visit and the other from historical data)
    7.The patient signs informed consent
    8.Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method
    1.Pacientes adultos sintomáticos con enfermedad de Fabry (> ó = a 18 años, hombres y mujeres)
    2.Hombres: actividad de la galactosidasa alfa plasmática y/o leucocítica (mediante ensayo de actividad) menor que el límite inferior de la normalidad (LIN en el plasma=3,2 nmol/h/ml, LIN en los leucocitos=32 nmol/h/mg/proteína).
    3.Mujeres: Historial de las pruebas genéticas anteriores en concordancia con mutaciones de Fabry
    4.Concentración de globotriaosilceramida (Gb3) en orina >1,5 veces al límite superior de la normalidad
    5.Los pacientes que nunca hayan recibido terapia de reemplazo enzimático (TRE) con anterioridad, o los pacientes que no hayan recibido TRE en los últimos 6 meses y tengan resultados negativos en una prueba de anticuerpos anti PRX-102
    6.Estadios 1 o 2 (CKD1 o 2) de la enfermedad renal crónica (Apéndice 7) con proteinuria >200 mg/g respecto del cociente de proteína a creatinina, medida en una muestra de orina al azar o equivalente, demostrado por lo menos en una de las 2 muestras separadas (una muestra en la visita de selección y la otra de datos previos)
    7.El paciente firma el consentimiento informado
    8.Los pacientes de sexo femenino y los pacientes de sexo masculino cuyas parejas tengan capacidad para concebir aceptan usar un método anticonceptivo aceptable desde el punto de vista médico; se excluye el método del ritmo.
    E.4Principal exclusion criteria
    1.Participation in any trial of an investigational drug within 30 days prior to study screening
    2.Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
    3.History of dialysis or renal transplantation
    4.Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
    5.Severe myocardial fibrosis by MRI (> or = to 2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009)
    6.History of clinical stroke
    7.Pregnant or nursing
    8.Presence of HIV and/or HBsAg and/or Hepatitis C infections
    9.Known allergies to ERT
    10.Known allergy to Gadolinium based contrast agents
    11.Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patients compliance with the requirements of the study
    1.La participación en cualquier ensayo con fármacos en investigación durante los 30 días previos a la selección del ensayo
    2.Estadios 3-5 de enfermedad renal crónica (CKD 3-5) (Apéndice 7)
    3.Antecedentes de diálisis o trasplante renal
    4.Tratamiento iniciado con el inhibidor de la enzima convertidora de angiotensina (ECA) o con bloqueadores del receptor de angiotensina (ARB) o cambio de la dosis las 4 semanas previas a la selección
    5.Fibrosis miocárdica grave según RM (segmentos ventriculares positivos > o = a 2 segmentos ventriculares izquierdos con realce tardío [LE] positivo) (Weidemann et al. 2009)
    6.Antecedente clínico de accidente cerebrovascular
    7.Embarazo o lactancia
    8.Presencia de infecciones por VIH y/o HBsAg y/o Hepatitis C
    9.Alergias conocidas a la TRE
    10.Alergia conocida a los agentes de contraste de gadolinio
    11.Presencia de cualquier afección médica, emocional, conductual o psicológica que, según el criterio del Investigador y/o Director Médico, interfiera en el cumplimiento de los requisitos del ensayo por parte del paciente
    E.5 End points
    E.5.1Primary end point(s)
    The following PK parameters will be derived from the plasma concentration versus time profiles to determine the profile of the study drug: Cmax, t1/2, Tmax, AUC0-t, and AUC0-∞.
    Los siguientes parámetros de PK se derivarán de los perfiles de concentración plasmática frente a tiempo a fin de determinar el perfil del fármaco del ensayo: Cmáx, t1/2, Tmáx, AUC0-t y AUC0-∞.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Samples will be taken at first and last infusions at the following time points: pre-infusion (baseline); 1 hour after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 hours and 2 weeks ± 3 days post-infusion
    Las muestras se obtendrán en la primera y la última infusión y en los siguientes puntos cronológicos: antes de la infusión (basal); 1 h después del comienzo de la infusión; al final de la infusión; 1, 4, 8, 24, 48±3, 72±3, 96±3 h y 2 semanas±3 días después de la infusión
    E.5.2Secondary end point(s)
    Efficacy Variables:
    -Gb3 concentrations in plasma and urine sediment at baseline and at every infusion during the study
    -Globotriaosylsphingosine (Lyso-Gb3) concentration in plasma at baseline and at every infusion during the study
    -Assessment of gastrointestinal symptoms at baseline and at last infusion (Appendix 8)
    -Kidney functions (eGFR and proteinuria) at baseline and at last infusion
    -Short Form Brief Pain Inventory (BPI) at baseline and at last infusion (Appendix 9)
    The following additional procedures will be performed only at baseline in this protocol as a reference point for evaluation as an exploratory efficacy endpoint parameter in an extension protocol. These parameters are not expected to show significant response during the 12 weeks of dosing in this study and will not be repeated at the last infusion.
    -Kidney biopsy for Gb3 concentration (Appendix 5)
    -Skin punch biopsy for Gb3 concentration (Appendix 5)
    -MRI of the heart and brain
    -Mainz Severity Score Index (MSSI) (Appendix 6)
    -Cardiac function assessment (echocardiography and stress test)

    Safety Variables
    -Safety will be assessed by the frequency, severity, and duration of treatment-emergent AEs (adverse events), including clinically significant laboratory abnormalities, ECG changes from baseline, physical examination findings and assessment of the injection site after administration of the study drug
    -Anti-PRX-102 antibodies will be assessed before dosing at: baseline, every month, at last infusion, and 1 and 3 months after last infusion
    Variables de eficacia:
    -Las concentraciones de Gb3 en plasma y sedimento urinario basales y en cada infusión durante el ensayo
    -La concentración basal de globotriaosilsfingosina (Liso-Gb3) en el plasma y en cada infusión durante el ensayo
    -Evaluación de síntomas gastrointestinales en el nivel basal y en la última infusión (Apéndice 8)
    -Funciones renales (eGFR y proteinuria) en el nivel basal y en la última infusión
    -Cuestionario breve del dolor, formulario abreviado (BPI-SF) en el nivel basal y en la última infusión (Apéndice 9)

    Los siguientes procedimientos adicionales de este protocolo se realizarán únicamente a nivel basal, como punto de referencia para la evaluación como un parámetro del criterio de valoración de la eficacia exploratoria en un protocolo de extensión. No se espera que estos parámetros presenten una respuesta significativa durante las 12 semanas de administración de la dosis en este ensayo y no se repetirán en la última infusión
    -Biopsia de riñón para determinar la concentración de Gb3 (Apéndice 5)
    -Biopsia de piel en sacabocados para determinar la concentración de Gb3 (Apéndice 5)
    -RM del corazón y del cerebro
    -Índice de isogravedad de Mainz (MSSI) (Apéndice 6)
    -Evaluación de la función cardíaca (ecocardiograma y prueba de esfuerzo)

    Variables de seguridad:

    -La seguridad se evaluará mediante la frecuencia, la gravedad y la duración de reacciones adversas debidas al tratamiento, incluidas las anomalías de laboratorio significativas desde el punto de vista clínico, los cambios en el ECG respecto del nivel basal, los hallazgos del examen físico y la exploración del lugar de aplicación de la inyección después de la administración del fármaco del ensayo.
    -Se evaluarán los anticuerpos anti- PRX-102 antes de la administración de la dosis en los siguientes momentos: en el nivel basal, cada mes, en la última infusión y 1 y 3 meses después de la última infusión

    E.5.2.1Timepoint(s) of evaluation of this end point
    12 weeks
    12 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 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) Yes
    E.7.1.1First administration to humans Yes
    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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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
    Argentina
    Australia
    Spain
    Israel
    Paraguay
    Serbia
    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
    LPLV
    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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    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: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 18
    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 tolerate the infusions well and complete the study are eligible to enroll in the extension study.
    Los pacientes que toleren bien las dosis y completen este estudio, se les dará la opción de participar en un ensayo abierto de extensión
    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 Decision2013-11-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-05-18
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