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    Summary
    EudraCT Number:2014-000742-30
    Sponsor's Protocol Code Number:261202
    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:2014-06-09
    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 number2014-000742-30
    A.3Full title of the trial
    A phase 3 prospective, uncontrolled, multicenter study evaluating pharmacokinetics, efficacy, safety, and immunogenicity of BAX 855 (PEGylated full-length Recombinant FVIII) in previously treated pediatric patients with severe hemophilia A
    ESTUDIO DE FASE 3, PROSPECTIVO, NO CONTROLADO Y MULTICÉNTRICO QUE EVALÚA LA FARMACOCINÉTICA, LA EFICACIA, LA SEGURIDAD Y LA INMUNOGENICIDAD DE BAX 855 (FVIII RECOMBINANTE DE LONGITUD COMPLETA PEGILADO) EN PACIENTES PEDIÁTRICOS CON HEMOFILIA A GRAVE TRATADOS PREVIAMENTE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the pharmacokinetics, efficacy, safety, and immunogenicity of BAX-855 Administered for Prevention of Bleeding in Previously
    Treated Pediatric Patients with Severe Hemophilia A (a blood clotting disorder).
    Estudio de farmacocinetica, eficacia, seguridad e inmunogenicidad de BAX-855 administrado para la prevención de hemorragias en pacientes con Hemofilia A grave (un trastorno de la coagulacion de la sangre) tratados previamente.
    A.3.2Name or abbreviated title of the trial where available
    BAX 855 Pediatric
    BAX 855 pediatrico
    A.4.1Sponsor's protocol code number261202
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/72/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxter Innovations GmbH
    B.1.3.4CountryAustria
    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 supportBaxter Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxter Innovations GmbH
    B.5.2Functional name of contact pointEli Taube
    B.5.3 Address:
    B.5.3.1Street AddressIndustriestrasse 67
    B.5.3.2Town/ cityVienna
    B.5.3.3Post codeA-1221
    B.5.3.4CountryAustria
    B.5.4Telephone number+43 (1) 20100 247 1240
    B.5.6E-maileli_taube@baxter.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 namePEGylated rFVIII
    D.3.2Product code BAX855 250IU/vial
    D.3.4Pharmaceutical form Lyophilisate and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Intravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX-855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant product with covalently bound polyethyleneglycol.
    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.2.1.1.1Trade name Advate 1000 IU powder and solvent for solution for injection
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter AG
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameAdvate
    D.3.2Product code rAHF-PFM
    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 INNOCTOCOG ALFA
    D.3.9.1CAS number 139076-62-3
    D.3.9.4EV Substance CodeSUB16449MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 namePEGylated rFVIII
    D.3.2Product code BAX855 500IU/vial
    D.3.4Pharmaceutical form Lyophilisate and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Intravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX-855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant product with covalently bound polyethyleneglycol.
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePEGylated rFVIII
    D.3.2Product code BAX855 1000IU/vial
    D.3.4Pharmaceutical form Lyophilisate and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Intravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX-855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant product with covalently bound polyethyleneglycol.
    D.IMP: 5
    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 namePEGylated rFVIII
    D.3.2Product code BAX855 2000IU/vial
    D.3.4Pharmaceutical form Lyophilisate and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Intravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX-855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant product with covalently bound polyethyleneglycol.
    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 (FVIII<1%)
    Hemofilia A grave (FVIII<1%)
    E.1.1.1Medical condition in easily understood language
    Hemophilia A
    Hemofilia A
    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 17.0
    E.1.2Level LLT
    E.1.2Classification code 10060612
    E.1.2Term Hemophilia A
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate FVIII inhibitory antibodies (?0.6 BU using the Nijmegen modification of the Bethesda assay)
    Evaluar la incidencia de anticuerpos inhibidores del FVIII (? 0,6
    unidades Bethesda [UB] mediante la modificación de Nijmegen del
    ensayo Bethesda).
    E.2.2Secondary objectives of the trial
    1. To evaluate the PK parameters of BAX 855 in pediatric PTPs <12 years of age.
    2. To monitor incremental recovery (IR) of BAX 855 over time.
    3. To evaluate hemostatic efficacy of BAX 855 in the management of acute bleeding episodes and for prophylaxis over a period of 6 months.
    4. To assess all AEs possibly or probably related to BAX 855.
    5. To evaluate immunogenicity (binding antibodies to FVIII, BAX 855, PEG, and Chinese hamster ovary [CHO]) and clinically significant changes in routine laboratory parameters (hematology, clinical chemistry, and lipids) and vital signs.
    6. To evaluate changes in HRQoL and health resource use.
    1.Evaluar los parámetros farmacocinéticos de BAX 855 en PTP
    pediátricos < 12 años de edad.
    2.Supervisar la recuperación incremental (RI) de BAX 855 con el tiempo.
    3.Evaluar la eficacia hemostática de BAX 855 en el tratamiento de los
    episodios hemorrágicos agudos y en la profilaxis durante un período de 6
    meses.
    4.Evaluar todos los AA posible o probablemente relacionados con BAX
    855.
    5.Evaluar la inmunogenicidad (anticuerpos de unión frente al FVIII, BAX
    855, PEG y las células de ovario de hámster chino [CHO]) y los cambios
    clínicamente significativos en los parámetros habituales de laboratorio (hematología, bioquímica clínica, y lípidos) y las constantes vitales.
    6.Evaluar los cambios en la CVRS y en la utilización de los recursos
    sanitarios
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The subject has severe hemophilia A (FVIII <1%) as determined by the central laboratory.
    2. Subject is <12 years old at the time of screening.
    3. Subjects aged ?6 to <12 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for a minimum of 150 EDs (based on the subjects? medical records).
    4. Subjects <6 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for at least 50 EDs (based on the subjects? medical records).
    5. Subject is human immunodeficiency virus (HIV) negative; or HIV positive with stable disease and CD4+ count of ?200 cells/mm3, as confirmed by central laboratory.
    6. The subject and/or legal representative accepts prophylactic treatment over a period of 6 months.
    7. The subject and/or the legal representative is willing and able to comply with the requirements of the protocol.
    1.El sujeto tiene hemofilia A grave (FVIII < 1 %), según lo determinado
    por el laboratorio central.
    2.El sujeto tiene < 12 años de edad en el momento de la selección.
    3.Los sujetos ? 6 a < 12 años de edad han sido tratados previamente
    con algún concentrado derivado de plasma y/o de FVIIIr durante un
    mínimo de 150 DE (según los informes médicos de los sujetos).
    4.Los sujetos < 6 años de edad han sido tratados previamente con algún
    concentrado derivado de plasma y/o de FVIIIr durante un mínimo de 50
    DE (según los informes médicos de los sujetos).
    5.El sujeto es negativo para el virus de la inmunodeficiencia humana
    (VIH); o es VIH positivo con enfermedad estable y un recuento de CD4+
    ? 200 células/mm3, según lo confirmado por el laboratorio central.
    6.El sujeto y/o el representante legal aceptan el tratamiento profiláctico
    durante un período de 6 meses.
    7.El sujeto y/o el representante legal están dispuestos y son capaces de
    cumplir los requisitos del protocolo.
    E.4Principal exclusion criteria
    1. Subject has detectable FVIII inhibitory antibodies (?0.4 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
    2. Subject has a history of FVIII inhibitory antibodies (?0.4 BU using the Nijmegen modification of the Bethesda assay or ?0.6 BU using the Bethesda assay) at any time prior to screening.
    3. Subject has known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80.
    4. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand?s disease).
    5. Subject?s platelet count is <100,000/?L.
    6. Subject has severe chronic hepatic dysfunction (eg, ?5 times upper limit of normal [ULN] alanine aminotransferase as confirmed by central laboratory at screening, or a documented international normalized ratio >1.5).
    7. Subject has severe renal impairment (serum creatinine >1.5 times ULN).
    8. Subject is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone >10 mg/day, or ?-interferon) other than anti-retroviral chemotherapy.
    9. Subject has current or recent (<30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation.
    10. Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
    11. Subject has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the Investigator, would affect subject safety or compliance.
    12. Subject?s legal representative is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
    1.-El sujeto tiene anticuerpos inhibidores del FVIII detectables (? 0,4 UB
    mediante la modificación de Nijmegen del ensayo Bethesda), según lo
    confirmado por el laboratorio central en la selección
    2.El sujeto tiene antecedentes de anticuerpos inhibidores del FVIII (?
    0,4 UB mediante la modificación de Nijmegen del ensayo Bethesda o ?
    0,6 UB mediante el ensayo de Bethesda) en cualquier momento antes de
    la selección.
    3.El sujeto tiene una hipersensibilidad conocida a las proteínas de ratón
    o hámster, PEG o Tween 80.
    4.Al sujeto se le ha diagnosticado un defecto hemostático heredado o
    adquirido diferente a la hemofilia A (como un defecto cualitativo de las
    plaquetas o la enfermedad de von Willebrand).
    5.El recuento plaquetario del sujeto es < 100.000/?l.
    6.El sujeto tiene una disfunción hepática crónica grave (p. ej., alanina
    aminotransferasa ? 5 veces el límite superior de la normalidad [LSN],
    según lo confirmado por el laboratorio central en la selección, o un índice
    internacional normalizado documentado > 1,5).
    7.El sujeto tiene insuficiencia renal grave (creatinina sérica > 1,5 veces
    el LSN).
    8.Se ha programado que el sujeto reciba durante el curso del estudio un
    fármaco inmunomodulador (como corticosteroides en una dosis
    equivalente a hidrocortisona > 10 mg/día o ?-interferón) distinto al de
    la quimioterapia antirretroviral.
    9.El sujeto utiliza o ha utilizado recientemente (< 30 días) otros
    fármacos PEGilados antes de la participación en el estudio o se ha
    programado que utilice estos fármacos durante la participación en el
    estudio.
    10.El sujeto ha participado en otro estudio clínico que utilizó un PEI o un
    dispositivo en investigación durante los 30 días anteriores a la
    inscripción o está programado que participe en otro estudio clínico con
    un PEI o un dispositivo en investigación durante el transcurso de este
    estudio.
    11.El sujeto tiene una enfermedad médica, psiquiátrica o cognitiva o
    consume drogas/alcohol, lo que, en opinión del investigador, podría
    afectar a la seguridad o al cumplimiento del sujeto.
    12.El representante legal del sujeto es un miembro del equipo que
    realiza este estudio o tiene una relación de dependencia con uno de los
    miembros del equipo del estudio. Las relaciones dependientes incluyen a
    parientes cercanos (es decir, hijos, pareja/cónyuge, hermanos, padres),
    así como los empleados del investigador o el personal del centro que
    realizan el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measure is the incidence of FVIII inhibitory antibodies
    La medida prinicpal de los resultados es la incidencia de anticuerpos
    inhibidores de FVIII
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months (±2 weeks) or at least 50 EDs
    6 meses (± 2 semans) o al menos 50 DEs
    E.5.2Secondary end point(s)
    Pharmacokinetics
    1. Area under the plasma concentration versus time curve from 0 to ? hours post infusion (AUC0-?), AUC0-?/dose, MRT, clearance (CL), IR, T1/2, volume of distribution at steady state (Vss) following an initial dose of ADVATE followed by BAX 855.
    2. IR over time.
    Hemostatic Efficacy
    1. Annualized bleeding rate (ABR).
    2. Consumption of BAX 855: number of infusions and weight-adjusted consumption per month and per year.
    3. Number of infusions per bleeding episode, overall hemostatic efficacy rating at resolution of bleed.
    4. Weight-adjusted consumption per bleeding episode.
    Safety
    1. All AEs and SAEs possibly or probably related to BAX 855.
    2. Clinical significant changes in vital signs (pulse, respiration, supine blood pressure, and temperature) and clinical laboratory parameters (hematology, clinical chemistry, and lipids).
    3. Assessment of binding antibodies to FVIII, BAX 855, PEG, and CHO.
    Farmacocinética
    1.Área bajo la curva de concentración plasmática frente al tiempo de 0 a
    ? horas después de la infusión (ABC0-?), ABC0-?/dosis, tiempo medio
    de residencia, aclaramiento, RI, semivida de la fase de eliminación,
    volumen de distribución en estado de equilibrio después de una dosis
    inicial de ADVATE seguida de BAX 855.
    2.RI en el tiempo.
    Eficacia hemostática
    1.Tasa anualizada de hemorragias (TAH).
    2.Consumo de BAX 855: número de infusiones y consumo ajustado al
    peso por mes y por año.
    3.Número de infusiones por episodio hemorrágico, calificación de
    eficacia hemostática global cuando desparece la hemorragia.
    4.Consumo ajustado al peso por episodio hemorrágico.
    Seguridad
    1.Todos los AA y los AAG posible o probablemente relacionados con BAX
    855.
    2.Cambios clínicamente significativos en las constantes vitales (pulso, respiración, presión arterial en posición supina y temperatura) y los
    parámetros de laboratorio clínico (hematología, bioquímica clínica y
    lípidos).
    3.Evaluación de anticuerpos de unión al FVIII, BAX 855, PEG y las CHO.
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months (±2 weeks) or at least 50 EDs
    6 meses(±2 semanas) o al menos 50DEs
    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 Yes
    E.8.1.7.1Other trial design description
    Single-group
    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 EEA34
    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
    Australia
    Austria
    Belgium
    Bulgaria
    Czech Republic
    Germany
    Hong Kong
    Ireland
    Japan
    Korea, Republic of
    Lithuania
    Malaysia
    Netherlands
    Poland
    Romania
    Spain
    Taiwan
    Thailand
    Turkey
    Ukraine
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months22
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months22
    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: 50
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 50
    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 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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 50
    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)
    After study completion, subjects will have the option to transition into a continuation study (Baxter Clinical Study 261302)/ to continue receiving BAX 855 until he/she has accumulated a total of 100 EDs or BAX 855 is licensed in his/her country, whichever occurs last.
    Después de la finalización del estudio, los sujetos tendrán la opción de hacer la transición a un estudio de continuación (Estudio Clínico de Baxter 261302)/ seguir recibiendo BAX 855 hasta que el/ella hayan acumulado un total de 100 DE o BAX 855 esté autorizado en su país, lo que ocurra mas tarde.
    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 Decision2014-07-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-07-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-10-23
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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