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    Summary
    EudraCT Number:2013-005512-10
    Sponsor's Protocol Code Number:997HA306
    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:2014-07-22
    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 number2013-005512-10
    A.3Full title of the trial
    An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
    Evaluación en abierto y multicéntrica de la seguridad y la eficacia de una proteína de fusión del factor VIII Fc recombinante de la coagulación (rFVIIIFc; BIIB031) en la prevención y el tratamiento de episodios de sangrado en pacientes con hemofilia A grave no tratados previamente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study involving previously untreated patients with haemophilia A to look at how safe an experimental replacement factor VIII protein (known as rFVIIIFc) is to take and how well it works to prevent and stop bleeds.
    Estudio clínico dirigido a pacientes con hemofilia A no tratados previamente paraevaluar como de seguro es tomar la proteina experimental de remplazo Factor VIII (conocida como rFVIIIFc) y como funciona para prevenir y detener hemorragias.
    A.4.1Sponsor's protocol code number997HA306
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/077/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBiogen Idec Research Ltd
    B.1.3.4CountryUnited Kingdom
    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 supportBiogen Idec
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiogen Idec Research Ltd
    B.5.2Functional name of contact pointBelén García
    B.5.3 Address:
    B.5.3.1Street AddressInnovation House, 70 Norden Road
    B.5.3.2Town/ cityMaidenhead, Berkshire
    B.5.3.3Post codeSL6 4AY
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailclinicaltrials@biogenidec.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/3/10/783
    D.3 Description of the IMP
    D.3.1Product namerecombinant Factor VIII-Fc
    D.3.2Product code rFVIIIFc
    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 INNTo be confirmed
    D.3.9.2Current sponsor codeBIIB031
    D.3.9.3Other descriptive namerFVIIIFc, recombinant Factor FVIII Fc
    D.3.9.4EV Substance CodeSUB13815MIG
    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 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/3/10/783
    D.3 Description of the IMP
    D.3.1Product namerecombinant Factor VIII-Fc
    D.3.2Product code rFVIIIFc
    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 INNTo be confirmed
    D.3.9.2Current sponsor codeBIIB031
    D.3.9.3Other descriptive namerFVIIIFc, recombinant Factor FVIII Fc
    D.3.9.4EV Substance CodeSUB13815MIG
    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 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/3/10/783
    D.3 Description of the IMP
    D.3.1Product namerecombinant Factor VIII-Fc
    D.3.2Product code rFVIIIFc
    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 INNTo be confirmed
    D.3.9.2Current sponsor codeBIIB031
    D.3.9.3Other descriptive namerFVIIIFc, recombinant Factor FVIII Fc
    D.3.9.4EV Substance CodeSUB13815MIG
    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: 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/3/10/783
    D.3 Description of the IMP
    D.3.1Product namerecombinant Factor VIII-Fc
    D.3.2Product code rFVIIIFc
    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 INNTo be confirmed
    D.3.9.2Current sponsor codeBIIB031
    D.3.9.3Other descriptive namerFVIIIFc, recombinant Factor FVIII Fc
    D.3.9.4EV Substance CodeSUB13815MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000
    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
    Hemophilia A
    Hemofilia A
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is a genetic disorder that impair the body's ability to control blood clotting or coagulation, which is used to stop bleeding when a blood vessel is broken.
    la hemofilia A es un desorden genético que afecta a la abilidad del cuerpo para controlar la coagulación, que es utilizada para detener una hemorragia cuando un vaso sanguineo se rompe.
    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 10053753
    E.1.2Term Hemophilia A without inhibitors
    E.1.2System Organ Class 100000004850
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the safety of rFVIIIFc in previously untreated subjects with severe hemophilia A.
    El objetivo principal del ensayo es evaluar la seguridad de rFVIIIFc en pacientes no tratados previamente con hemofilia A severa
    E.2.2Secondary objectives of the trial
    -To evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in PUPs
    -To evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs
    -To describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in subjects with inhibitors
    Evaluar la eficacia de rFVIIIFc en la prevención y tratamiento de episodios de sangrado en pacientes pediatricos no tratados previamente.. Evaluar el consumo de rFVIIIFc para la prevención y tratamiento de episodios de sangrado en pacientes pediatricos no tratados previamente. Describir la experiencia con el uso de rFVIIIFc para indución de tolerancia inmune (ITI) en sujetos con inhibidores.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Ability of the subject or his parent or legal guardian to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations. Subjects may provide assent in addition to the parental/guardian consent, if appropriate.
    2. Male, age <18 years at the time of informed consent.
    3. Weight ?3.5 kg at the time of informed consent.
    4. Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period. Any subject who is enrolled based on results of the local laboratory must be withdrawn if the central laboratory screening results indicate a baseline FVIII activity level ?1% of normal.
    1. Capacidad del sujeto o de su progenitor o tutor legal de comprender el objetivo y los riesgos del estudio y de dar el consentimiento informado firmado y fechado, y la autorización para usar información de salud protegida (PHI), de acuerdo con la
    normativa nacional y local sobre la privacidad de los sujetos. Los sujetos podrán otorgar su asentimiento además del consentimiento del progenitor/tutor, si es pertinente. 2. Hombre, edad < 18 años en el momento del consentimiento informado. 3. Peso ? 3,5 kg en el momento del consentimiento informado. 4.
    Hemofilia A grave definida como ? 1 UI/dl (? 1 %) de FVIII endógeno documentado en la historia clínica o como se haya analizado durante el período de selección.
    Cualquier sujeto que sea inscrito basándose en los resultados del laboratorio local debe ser retirado si los resultados de selección del laboratorio central indican un nivel de actividad del FVIII al inicio > 1 % de lo normal.
    E.4Principal exclusion criteria
    1. Prior history of inhibitor as defined by the reporting laboratory. The historical positive inhibitor test is defined as per local laboratory Bethesda value for a positive inhibitor test (i.e., equal to or above lower level of detection).
    2. Measurable inhibitor activity at the Screening Visit, measured using the Nijmegen-modified Bethesda assay performed at the central laboratory. A negative inhibitor test result at the local laboratory may be used initially to determine subject eligibility; however, any subject who is enrolled based on results of the local laboratory must be withdrawn if the central laboratory screening results indicate a positive inhibitor. Subjects actively bleeding and requiring emergent treatment may be enrolled and receive study drug after samples for inhibitor testing at the central laboratory have been obtained, with results pending. However, any such subject must be withdrawn if the central laboratory screening results indicate a positive inhibitor.
    3. History of hypersensitivity reactions associated with any IV immunoglobulin administration.
    4. Injection with any FVIII replacement product or any blood component prior to confirmation of eligibility.
    5. Injection with rFVIIIFc prior to confirmation of eligibility.
    6. Other coagulation disorder(s) in addition to hemophilia A.
    7. Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the subject unsuitable for enrollment.
    8. Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of corticosteroids for the treatment of asthma or management of acute allergic episodes is allowed with the exception of systemic corticosteroid treatment given to children daily or on alternate days at ?2 mg/kg per day of prednisone or its equivalent or ?20 mg/day if they weigh more than 10 kg with a duration of longer than 14 days.
    9. Participation within the past 30 days in any other clinical study involving investigational treatment.
    10. Current enrollment in any other clinical study involving investigational treatment.
    11. Inability to comply with study requirements.
    12. Other unspecified reasons that, in the opinion of the Investigator or Biogen Idec, make the subject unsuitable for enrollment.
    1. Antecedentes previos de inhibidor, tal como lo define el laboratorio que informa. La prueba histórica de inhibidor positiva se define como un valor de Bethesda del laboratorio local para una prueba positiva de inhibidor (es decir, igual o por encima del nivel inferior de detección). 2. Actividad inhibidora medible en la visita de selección, medida usando el ensayo de Bethesda modificado por Nijmegen realizado en el laboratorio central. Un resultado negativo de inhibidor en el laboratorio local puede utilizarse inicialmente para determinar la idoneidad del sujeto; sin embargo, todo sujeto que sea inscrito basándose en los resultados del laboratorio local debe ser retirado si los resultados de la selección del laboratorio
    central indican un inhibidor positivo. Los sujetos con sangrado activo y que requieran un tratamiento de emergencia pueden inscribirse y recibir el fármaco del estudio después de que se hayan obtenido las muestras para la prueba del inhibidor en el laboratorio central, con resultados pendientes. Sin embargo, si los resultados de detección del laboratorio central indican un inhibidor positivo, los sujetos deben ser retirados. 3. Antecedentes de reacciones de hipersensibilidad
    asociadas con alguna administración i.v. de inmunoglobulina. 4. Inyección con cualquier producto de reemplazo de FVIII o cualquier componente de la sangre antes de la confirmación de la idoneidad. 5. Inyección con rFVIIIFc antes de la confirmación de la idoneidad. 6. Otros trastornos de la coagulación además de la hemofilia A. 7. Cualquier enfermedad importante y clínicamente significativa
    concurrente que, en opinión del investigador, podría hacer que el sujeto no fuera apto para la inscripción. 8. Tratamiento sistémico actual con quimioterapia u otros fármacos inmunosupresores. Se permite el uso de corticosteroides para el tratamiento del asma o el control de episodios alérgicos agudos con la excepción del tratamiento con corticosteroides sistémicos que se da los niños diariamente o
    en días alternos de ? 2 mg/kg por día de prednisona o su equivalente, o ? 20 mg/día si pesan más de 10 kg, con una duración de más de 14 días. 9. Participación en los últimos 30 días en cualquier otro estudio clínico con algún tratamiento en investigación. 10. Inscripción actual en algún otro estudio clínico con un tratamiento en investigación. 11. Incapacidad para cumplir con los requisitos del estudio. 12. Otra razón no especificada que, en opinión del investigador o de Biogen Idec, haga que el sujeto no sea apto para la inscripción.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the occurrence of inhibitor development.
    l criterio de valoración principal del estudio es la aparición de desarrollo de inhibidores.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Subjects will be tested for inhibitor formation:
    - at each clinic visit including screening and baseline.
    - 5 (±1), 10-15, 20-25, 50, 75, and 100 EDs
    - 2 to 4 weeks prior to elective major surgery, preoperatively, postoperatively, and at the Last Postoperative Visit (see Section 4.2.2 of protocol).If this does not align with a scheduled visit, an additional visit must be scheduled. For minor surgeries, testing for inhibitors will only be performed if indicated by the nature of the procedure, according to local standard of care.
    - if inhibitor development is suspected at any time during the study the subject will be tested for inhibitors by the central lab

    Additional unscheduled testing for inhibitors may be performed, if required by local standards of care
    Se evaluará la formación de inhibidores en sujetos:
    - en cada visita clinica incluyendo basal y screening
    5(+/1), 10-15, 20-25,50,75, and 100 EDs
    -2 a 4 semanas antes de elección de cirugía mayor, preoperatorio y posteroperatorio, y en la última visita postoperatoria (ver seccion 4.2.2. del protocolo) Si no coincide con una visita programada una visita adicional deberá programarse. Para cirugías menores, la evaluación de inhibidores será realizada solo si está indicada por la naturaleza del procedimiento de acuerdo a los estandares habituales de cuidado. Si se sospecha un desearrollo de inhibidores en algún momento durante la duración del estudio el sujeto será sometido a una evaluación de inhibidores por el lab. central
    E.5.2Secondary end point(s)
    -The annualized number of bleeding episodes per subject
    - The annualized number of spontaneous joint bleeding episodes per subject
    - The number of injections and dose per injection of rFVIIIFc required to resolve a bleeding episode
    - Assessments of response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale
    - The total number of EDs per subject per year
    - Total annualized rFVIIIFc consumption per subject for the prevention and treatment of bleeding episodes
    - rFVIIIFc incremental recovery (IR)
    - Response to ITI with rFVIIIFc (success, partial success, failure, early withdrawal)
    ? El número de episodios de sangrado anualizados por sujeto ? El número de episodios de sangrado articular espontáneos anualizados por sujeto ? El número de inyecciones y dosis por inyección de rFVIIIFc necesario para resolver un episodio de sangrado ? Las evaluaciones de la respuesta al tratamiento con rFVIIIFc para los episodios de sangrado, utilizando la escala de respuesta de sangrado de 4 puntos ? El número total de DE por sujeto por año ? El consumo total anualizado de rFVIIIFc por sujeto para la prevención y el tratamiento de los episodios de sangrado ? La recuperación incremental (RI) de rFVIIIFc ? La respuesta a la ITI con rFVIIIFc (éxito, éxito parcial, fracaso, retirada anticipada)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Incidence of bleeding episodes, dose and number of injections, and response to treatment will be monitored by the physician on an ongoing basis and will be obtained from electronic patient diaries (EPDs), electronic case report forms (eCRFs), and medical records. During clinic visits when rFVIIIFc is injected, blood samples will be taken to assess incremental recovery.
    La incidencia de episodios de sangrado, dosis y número de inyecciones y respuesta al tratamiento serán monitorizadas por el médico de manera regular y se obtendrán del diario de paciente (DPs), el cuaderno de recogida de datos electronico (eCRF) yarchivos médicos.Durante las visitas clínicas al inyectar rFVIIIFc, se tomarán muestras para evaluar la tasa incremental de respuesta.
    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 EEA28
    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
    Belgium
    Canada
    Czech Republic
    Denmark
    France
    Germany
    Ireland
    Italy
    Netherlands
    New Zealand
    Poland
    South Africa
    Spain
    Sweden
    Switzerland
    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
    The end of the study will occur when at least 100 subjects have reached at least 100 EDs with rFVIIIFc. Once this milestone has been achieved, any additional study subjects who have not reached 100 EDs will be required to attend the study center for the Early Termination/End of Study Visit assessments.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years8
    E.8.9.2In all countries concerned by the trial months3
    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: 125
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 40
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 82
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    Parents/legal guardians of subjects must be provided with the informed consent information prior to the Screening Visit to allow adequate time for review and an opportunity to discuss the study with the Investigator/designee.
    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 60
    F.4.2.2In the whole clinical trial 125
    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)
    Standard of care; no further provisions are made for access to the study treatments beyond the protocol-specified treatment duration.
    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 Decision2014-09-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-05
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2019-09-23
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