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    Summary
    EudraCT Number:2012-004434-42
    Sponsor's Protocol Code Number:BAY94-9027/15912
    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:2015-10-05
    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 number2012-004434-42
    A.3Full title of the trial
    A multi-center, phase III, non-controlled, open-label trial to evaluate the pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated children (age <12 years) with severe hemophilia A
    Estudio de fase III, multicéntrico, abierto y no controlado para evaluar la farmacocinética, la seguridad y la eficacia de BAY 94-9027 para la profilaxis y el tratamiento de hemorragias en niños (edad < 12 años) con hemofilia A grave tratados previamente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial investigating safety and efficacy of a long-acting factor VIII in children (age <12 years) with severe hemophila A
    Estudio de la farmacocinética, la seguridad y la eficacia pediátricas de BAY 94-9027 en niños con hemofilia A severa (<12 años)
    A.3.2Name or abbreviated title of the trial where available
    PROTECT KIDS
    A.4.1Sponsor's protocol code numberBAY94-9027/15912
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/216/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBayer HealthCare AG
    B.1.3.4CountryGermany
    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 supportBayer HealthCare AG
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBayer HealthCare AG
    B.5.2Functional name of contact pointBayer Clin. Trials Contact CTP Team
    B.5.3 Address:
    B.5.3.1Street AddressBayer Pharma AG, S102, Level 2, Room 156
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code13342
    B.5.3.4CountryGermany
    B.5.4Telephone number0034.900102372
    B.5.6E-mailclinical-trials-contact@bayerhealthcare.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/847
    D.3 Description of the IMP
    D.3.1Product nameBAY 94-9027 (500 IU/vial)
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    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 INNDamoctocog alfa pegol
    D.3.9.2Current sponsor codeBAY 94-9027
    D.3.9.3Other descriptive namePEGylated B-domain deleted recombinant human antihemophilic Factor VIII
    D.3.9.4EV Substance CodeSUB33061
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/847
    D.3 Description of the IMP
    D.3.1Product nameBAY 94-9027 (1000 IU/vial)
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    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 INNDamoctocog alfa pegol
    D.3.9.2Current sponsor codeBAY 94-9027
    D.3.9.3Other descriptive namePEGylated B-domain deleted recombinant human antihemophilic Factor VIII
    D.3.9.4EV Substance CodeSUB33061
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/847
    D.3 Description of the IMP
    D.3.1Product nameBAY 94-9027 (3000 IU/vial)
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    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 INNDamoctocog alfa pegol
    D.3.9.2Current sponsor codeBAY 94-9027
    D.3.9.3Other descriptive namePEGylated B-domain deleted recombinant human antihemophilic Factor VIII
    D.3.9.4EV Substance CodeSUB33061
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1200
    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: 4
    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 numberEU/3/10/847
    D.3 Description of the IMP
    D.3.1Product nameBAY 94-9027 (250 IU/vial)
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    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 INNDamoctocog alfa pegol
    D.3.9.2Current sponsor codeBAY 94-9027
    D.3.9.3Other descriptive namePEGylated B-domain deleted recombinant human antihemophilic Factor VIII
    D.3.9.4EV Substance CodeSUB33061
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    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 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
    severe hemophilia A (<1% FVIII:C)
    Hemofilia A severa (<1% FVIII:C)
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is an X-linked congenital bleeding disorder causing frequent bleedings and recurrent spontaneous bleeds into the soft tissue and joints, leading to joint damage and severe disability.
    La hemofilia A es un trastorno hemorrágico hereditario ligado al cromosoma X causando hemorragias espontáneas recurrentes en tejidos y articulaciones, provocando daño articular y discapacidad grave.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10060612
    E.1.2Term Hemophilia A
    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 pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated patients with hemophilia A

    Expansion group (Part 2)
    To further evaluate safety of BAY 94-9027 for prophylaxis and treatment of bleeding in PTPs with hemophilia A below 6 years of age.
    Evaluar la farmacocinética, la seguridad y la eficacia de BAY 94-9027 para la profilaxis y el tratamiento de hemorragias en pacientes con hemofilia A tratados previamente.

    Grupo de ampliación (Parte 2)
    Evaluar con más detalle la seguridad de BAY 94-9027 para la profilaxis y el tratamiento de hemorragias en pacientes con hemofilia A menores de 6 años tratados previamente.
    E.2.2Secondary objectives of the trial
    Primary endpoints:
    - Number of bleeding events during prophylactic treatment
    - Assessment of PK, including Cmax, incremental recovery, MRT, Apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, in at least 12 subjects in each age subgroup, with at least 4 pre-selected time points between pre-dose and 72 h post-infusion.
    - Response of acute bleeding events to treatment will be rated using a 4-point scale (poor, moderate, good, or excellent) by the subject/parent or by the treating physician if the subject is hospitalized

    Expansion group (Part 2)
    Primary endpoint: Characterization of the potential immune response.
    ? Número de acontecimientos hemorrágicos durante el tratamiento profiláctico.
    ? Evaluación de la FC, incluyendo la concentración máxima (Cmáx), el tiempo medio de residencia (TMR), el Volumen aparente de distribución en estado estacionario (VSS), la semivida, el área bajo la curva (AUC) y el aclaramiento, en por lo menos 12 pacientes de cada subgrupo de edad, con por lo menos 4 momentos preseleccionados entre el momento previo a la administración y 72 h después de la infusión.
    ? La respuesta de los acontecimientos hemorrágicos agudos al tratamiento se valorará mediante una escala de 4 puntos (escasa, moderada, buena o excelente) que evaluará el paciente o sus padres, o el médico responsable del tratamiento si el paciente está hospitalizado.
    Grupo de ampliación (Parte 2)
    ? Caracterización de la respuesta inmunitaria potencial.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male, age <12 years to be enrolled in 2 subgroups:
    - age 6 to <12 years
    - age < 6 years

    Subjects in the expansion group are to be <6 years of age.

    2. Severe hemophilia A defined as < 1% factor VIII concentration (FVIII:C) by measurement at the time of screening or from reliable prior documentation (eg, measurement in other clinical trials, results from approved clinical laboratory, or diagnostic genetic testing)

    3. > 50 ED with any FVIII concentrate(s) (plasma derived or recombinant)

    4. Willingness and ability of subjects and/or parents to complete training in the use of the EPD and to document infusions during the study

    5. Written informed consent by parent/legal representative. Assent should be sought from subjects, if appropriate
    1. Pacientes varones <= 12 años de edad. Todos los pacientes del grupo de ampliación (Expansion Study) serán pacientes varones de edad < 6 años.
    2. Hemofilia A severa, definida como una concentración de factor VIII (FVIII:C) < 1 %, documentada a partir de un análisis previo o en las pruebas analíticas de selección
    3. >= 50 DE con cualquier concentrado de FVIII (plasmático o recombinante)
    4. Disposición y capacidad de los pacientes y/o sus progenitores para completar la formación sobre el uso del diario electrónico del paciente (DEP) y anotar las infusiones durante el estudio.
    5. Consentimiento informado por escrito firmado por uno de los progenitores/representante legal. Asentimiento de los pacientes, si procede.
    E.4Principal exclusion criteria
    1. Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Subjects should not have received FVIII within 72 h prior to the collection of screening samples and should have had FVIII administered within the prior 2-3 weeks.

    2. History or presence of Factor VIII inhibitors. Inhibitor to FVIII is defined as a titer >0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. (Subjects with a maximum historical titer of <1.0 BU on no more than 1 occasion with the classical Bethesda assay but at least 3 subsequent negative results [<0.6 BU] are eligible.)

    3. Any other inherited or acquired bleeding disorder in addition to hemophilia A

    4. Platelet count < 100,000/mm3

    5. Creatinine > 2x upper limit of normal

    6. Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) > 5x upper
    limit of normal

    7. Known hypersensitivity to the drug substance, or any of its components (eg, mouse or hamster protein)

    8. The subject is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of study entry. Subjects who are currently participating in an investigational study in which they are treated with a currently marketed FVIII concentrate are not excluded. Subjects currently treated with BAY 81-8973 may continue treatment with the product up to the start of Visit 1.

    9. Any individual who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months.

    10. The subject is identified by the investigator as being unable or unwilling to perform study procedures.

    11. Previous assignment to treatment during this study.
    1. Evidencia de anticuerpos inhibidores determinada mediante el ensayo de Bethesda modificado por Nijmegen [< 0,6 unidades Bethesda (UB)/ml] realizado en el momento del screening (laboratorio central). Los pacientes no deben haber recibido FVIII en las 72 h previas a la obtención de muestras para las pruebas de detección de inhibidores de la visita de selección y deben haberse administrado FVIII durante las 2 - 3 semanas previas.
    2. Antecedentes o presencia de inhibidores del FVIII (definidos como > 0,6 unidades Bethesda (UB)/ml o registro de posible inhibidor en la historia clínica que requiera la modificación del tratamiento. Puede incluirse también a los pacientes con un valor histórico <1,0 UB en el ensayo de Bethesda clásico obtenido como máximo en una única ocasión, pero por lo menos con 3 resultados negativos sucesivos (< 0,6 UB) posteriores]

    3. Presencia de otro trastorno hemorrágico diferente de la hemofilia A (p. ej., enfermedad de von Willebrand, hemofilia B)
    4. Trombocitopenia (cifra de plaquetas < 100 000/mm3)
    5. Niveles de creatinina > 2 veces por encima del límite superior de la normalidad
    6. Niveles de aspartato-aminotransferasa (AST)/alanina-aminotransferasa (ALT) > 5 veces el límite superior de la normalidad
    7. Hipersensibilidad conocida al principio activo o a las proteínas de ratón o hámster.
    8. Tratamiento con otro fármaco experimental en los últimos 3 meses. No se excluye a pacientes en Tratamiento con otro producto de FVIII ya comercializado. Los pacientes tratados con BAY 81-8973 pueden continuar con el tratamiento hasta el inicio de la visita 1.
    9. Tratamiento actual con quimioterapia, fármacos inmunomoduladores (quimioterapia anti-retroviral, uso crónico de corticosteroides por vía oral o i.v. >14 días), o tratamiento con otro fármaco experimental en los últimos 3 meses
    10. No estar dispuesto a cumplir las visitas del estudio o los demás requisitos del protocolo o no ser apto para participar en este estudio por cualquier motivo, según el criterio del investigador
    11. Participación previa en este estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoints:
    - Number of bleeding events during prophylactic treatment
    - Assessment of PK, including Cmax, incremental recovery, MRT, Apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, in at least 12 subjects in each age subgroup, with at least 4 pre-selected time points between pre-dose and 72 h post-infusion.
    - Response of acute bleeding events to treatment will be rated using a 4-point scale (poor, moderate, good, or excellent) by the subject/parent or by the treating physician if the subject is hospitalized

    Expansion group (Part 2)
    Primary endpoint: Characterization of the potential immune response.
    ? Número de acontecimientos hemorrágicos durante el tratamiento profiláctico.
    ? Evaluación de la FC, incluyendo la concentración máxima (Cmáx), el tiempo medio de residencia (TMR), el Volumen aparente de distribución en estado estacionario (VSS), la semivida, el área bajo la curva (AUC) y el aclaramiento, en por lo menos 12 pacientes de cada subgrupo de edad, con por lo menos 4 momentos preseleccionados entre el momento previo a la administración y 72 h después de la infusión.
    ? La respuesta de los acontecimientos hemorrágicos agudos al tratamiento se valorará mediante una escala de 4 puntos (escasa, moderada, buena o excelente) que evaluará el paciente o sus padres, o el médico responsable del tratamiento si el paciente está hospitalizado.
    Grupo de ampliación (Parte 2)
    ? Caracterización de la respuesta inmunitaria potencial.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - Number of bleedings: at the end of treatment visit
    - Assessment of PK values: at the PK visit
    - Response to treatment of acute bleedings: end of treatment visit
    ? Número de acontecimientos hemorrágicos al final del tratamiento profiláctico.
    -Evaluación de la FC en las visitas donde se realice la FC
    -Respuesta al tratamiento de las hemorragias agudas: al final del tratamiento profiláctico
    E.5.2Secondary end point(s)
    - Inhibitor development after 10-15 and 50 ED
    - Assess incremental recovery in all subjects
    - Safety and tolerability assessments
    ? Se evaluará en todos los pacientes el desarrollo de inhibidores despues de los 10-15 y 50 Días de Exposición al fármaco
    ? Evaluar la recuperación incremental en todos los sujetos
    ? Evaluar la seguridad y la eficacia del producto en investigación
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Inhibitor development: after 10-15 and after 50 ED of each subject
    - Safety and tolerability assessments: at the end of treatment visit and FU visit
    Se evaluará en todos los pacientes el desarrollo de inhibidores despues de los 10-15 y 50 Días de Exposición al fármaco
    ? Evaluar la seguridad y la eficacia del producto en investigación al final del tratamiento y en la visita de seguimiento
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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) 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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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.8.2.4Number of treatment arms in the trial1
    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 EEA30
    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
    Canada
    Dominican Republic
    European Union
    Israel
    New Zealand
    Puerto Rico
    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
    End of the trial is the last visit (follow-up phone call 1-2 weeks after end of treatment) of the last subject undergoing study.
    El Final del estudio es la última visita (Llamada telefónica de seguimiento 1 - 2 semanas después de la finalización del tratamiento) del último paciente del estudio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years5
    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 Yes
    F.1.1Number of subjects for this age range: 60
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 55
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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
    Minors below 12 years of age.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 37
    F.4.2.2In the whole clinical trial 60
    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)
    At the end of the main study, all participants who have completed a minimum of 50 ED and at least 6 months in the study will be offered the option of participation in an extension study.
    Participation in the extension study will allow continuation of treatment for at least 50 additional ED (minimum total 100 ED) or until marketing authorization of the drug.
    Al final del estudio, se ofrecerá a todos los participantes que hayan completado un mínimo de 50 DE y al menos 6 meses en el estudio, la posibilidad de participar en un estudio de extensión. La participación en el estudio de extensión permitirá continuar el tratamiento hasta acumular al menos 100 DE, o hasta que se conceda la autorización de comercialización.
    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 Decision2015-11-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-10-15
    P. End of Trial
    P.End of Trial StatusCompleted
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