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    Summary
    EudraCT Number:2006-001383-23
    Sponsor's Protocol Code Number:PROWILL
    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:2012-03-01
    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 number2006-001383-23
    A.3Full title of the trial
    Efficacy, safety and pharmaco-economic assessment of secondary long term prophylaxis with highly purified, standardized, doubly virus inactivated FVIII/VWF concentrates in patients with severe, inherited VWD and frequent bleedings.
    Eficacia, seguridad y evaluación fármaco-económica de la profilaxis secundaria a largo plazo con concentrados de FVIII/FVW altamente purificados y doblemente inactivados en pacientes con enfermedad de Von Willebrand (EVW) severa heredada y con frecuentes sangrados
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Estudio de eficacia, seguridad e implicación económica de la administración continua del concentrado de factor VIII/VW en pacientes con enfermedad Von Willebrand severa heredada y con sangrados frecuentes comparado con la administración puntual cuando se produzca el sangrado
    A.4.1Sponsor's protocol code numberPROWILL
    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 SponsorFondazione IRCCS Cà Granda-Ospedale Maggiore Policlinico
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGrifols Biologicals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationANAGRAM-ESIC
    B.5.2Functional name of contact pointResponsable Médico
    B.5.3 Address:
    B.5.3.1Street AddressComte Urgell, 143
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08036
    B.5.3.4CountrySpain
    B.5.4Telephone number34934515250
    B.5.5Fax number34934516631
    B.5.6E-mailm.cardona@anagram-esic.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 Yes
    D.2.1.1.1Trade name Fanhdi 25 UI FVIII-30 UI FVW
    D.2.1.1.2Name of the Marketing Authorisation holderInstituto Grifols
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameFanhdi 25 UI
    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.3.9.3Other descriptive namecoagulación factor VIII
    D.3.9.4EV Substance CodeSUB13813MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive nameHUMAN VON WILLEBRAND FACTOR
    D.3.9.4EV Substance CodeSUB22288
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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 No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP 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 Yes
    D.2.1.1.1Trade name Fanhdi 50 UI FVIII-60 UI FVW
    D.2.1.1.2Name of the Marketing Authorisation holderInstituto Grifols
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameFanhdi 50 UI
    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.3.9.3Other descriptive namecoagulación factor VIII
    D.3.9.4EV Substance CodeSUB13813MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive nameHUMAN VON WILLEBRAND FACTOR
    D.3.9.4EV Substance CodeSUB22288
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number600
    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 No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP 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 Yes
    D.2.1.1.1Trade name Fanhdi 100 UI FVIII-120 UI FVW
    D.2.1.1.2Name of the Marketing Authorisation holderInstituto Grifols
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameFanhdi 100 UI
    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.3.9.3Other descriptive namecoagulación factor VIII
    D.3.9.4EV Substance CodeSUB13813MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive nameHUMAN VON WILLEBRAND FACTOR
    D.3.9.4EV Substance CodeSUB22288
    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 No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Severe, inherited von Willebrand disease (VWD) and frequent bleedings
    pacientes con enfermedad Von Willebrand (EVW) severa heredada y con frecuentes sangrados
    E.1.1.1Medical condition in easily understood language
    pacientes con enfermedad Von Willebrand (EVW) severa heredada y con frecuentes sangrados
    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 14.1
    E.1.2Level PT
    E.1.2Classification code 10047715
    E.1.2Term Von Willebrand's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 if long term, secondary prophylaxis with highly purified FVIII/VWF concentrates, with respect to on demand treatment with the same pharmacological agent, prevents spontaneous bleedings onset in patients with severe inherited VWD unresponsive to DDAVP and with frequent bleedings.
    Evaluar si la profilaxis secundaria a largo plazo con concentrados FVIII/FVW altamente purificados, en comparación con tratamiento a demanda con el mismo agente farmacológico, previene sangrados espontáneos en pacientes con EVW severa heredada que no responden a DDAVP y con sangrados frecuentes
    E.2.2Secondary objectives of the trial
    To evaluate the effect of prophylaxis versus on demand therapy in the following:
    - safety, with particular reference to the likelihood of thrombotic complications occurrence
    - patient compliance
    - possible impact on the global costs of care and on patients? quality of life, describing and evaluating:
    a) incremental cost per unit of effect
    b) the Health Related Quality of Life (HRQoL) of adult and paediatric patients and their caregivers
    Evaluar el efecto del tratamiento en profilaxis versus a demanda en cuanto a:
    -Seguridad, particularmente en referencia a la probabilidad de ocurrencia de complicaciones trombóticas
    - Cumplimiento del paciente
    - Posible impacto en el coste global del tratamiento y en la calidad de vida del paciente, describiendo y evaluando:
    ?Coste incrementado por unidad de efecto
    ?Calidad de vida relacionada con la salud (CVRS) de pacientes adultos y pediátricos y sus cuidadores
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented unresponsiveness to DDAVP. All the patients, excluding type 3 and type 2B, should have been exposed to a documented infusion trial with DDAVP. Responsiveness to DDAVP is defined as an increase in plasma FVIII:C and VWF:RCo levels of at least three fold over baseline and in absolute reaching at least 30 IU/dl for both values and a BT of 12 minutes or less after 2 hours from administration of 0,3 mcg/kg of DDAVP
    OR
    contraindication to DDAVP (namely type 2B VWD patients or other patients who display significant side effects to DDAVP)

    2. Frequent, spontaneous bleedings i.e. at least 5 episodes in whatever anatomical site in the last 12 months, severe enough to require treatment with FVIII/VWF concentrates

    Prolonged or excess bleeding during menses will be allowed only when:
    i. No other medication can control frequent bleeds
    ii. Bleeding exceeds 10 consecutive days
    OR
    Recurrent spontaneous bleedings, severe enough to require treatment with FVIII/VWF concentrates of the following types:
    - Epistaxes (at least 5 episodes in the last 12 months)
    - Haemarthroses (at least 3 episodes at the same joint in the last 12 months)
    - Gastrointestinal bleeding (at least 2 episodes in the last 12 months, with a drop ? 2 g/dl of haemoglobin in 24-48 hours) due to unexplained reason or in association with underlying gastrointestinal angiodysplasia

    3. Willingness to participate in the study, expressed by signed, written informed consent
    Edad ? 6 años
    ?Falta de respuesta a DDAVP documentada. Todos los pacientes, excluyendo el tipo 3 y 2B, tienen que haber estado expuestos a un ensayo documentado de infusión con DDAVP. Respuesta al tratamiento con DDAVP se define como un incremento de los niveles de FVIII:C y FVW:RCo en plasma de al menos 3 veces por encima del nivel básico y en valor absoluto, alcanzando al menos 30 UI/dl para ambos valorescontraindicaciones a DDAVP (básicamente pacientes de EVW de tipo 2B u otros que presentan efectos secundarios significativos a DDAVP)
    ?Sangrados espontáneos frecuentes, por ejemplo, al menos 5 episodios en cualquier lugar anatómico en los últimos 12 meses (a excepción de los pacientes en profilaxis, ver más abajo), suficientemente severos como para requerir tratamiento con concentrados de FVIII/FVW
    Sangrado prolongado o en exceso durante menstruaciones se permitirá solamente cuando:
    i.Ninguna otra medicación puede controlar los sangrados frecuentes
    ii.El sangrado excede 10 días consecutivos
    Ó
    Sangrados espontáneos recurrentes, suficientemente severos como para requerir tratamientos con concentrados de FVIII/FVW, de los siguientes tipos:
    ?Epistaxis (al menos cinco 5 episodios en los últimos 12 meses)
    ?Hemartrosis (al menos tres 3 episodios en la misma articulación en los últimos 12 meses)
    ?Sangrado gastrointestinal (al menos dos 2 episodios en los últimos 12 meses, con una bajada de ? 2 g/dl de hemoglobina en 24-48 horas) por motivos inexplicados o en asociación con anginodisplasia gastrointestinal subyacente
    Los pacientes que estén bajo tratamiento profiláctico con VWF/FVIII en el momento de la evaluación, podrán ser incluidos en el estudio (después del periodo de lavado); los criterios de selección serán los mismos y se referirán al periodo de 12 meses antes del inicio del actual tratamiento profiláctico.
    ?Deseo de participación en el Estudio, expresado mediante consentimiento informado por escrito firmado
    E.4Principal exclusion criteria
    1. a life expectancy < 1 year
    2. presence of allo-antibodies to VWF or FVIII
    3. acquired Von Willebrand?s syndrome (AVWS)
    4. co-morbidity with other haemorrhagic diathesis, excluded those linked to VWD as the complication of treatment (for instance thrombocytopenia in Type 2B VWD)
    5. advanced liver cirrhosis with:
    - cirrhosis related coagulation abnormalities (thrombocytopenia, defined as platelet count < 50.000/mm3, INR >1,7, prolonged prothrombin time > 4 seconds versus normal)
    - portal hypertension with history of variceal bleeding
    6. pregnancy and lactation
    7. any known need for invasive procedures or elective surgery scheduled in the following 3 months (recruitment in these cases should be postponed)
    8. proven co-morbidity for other causes of gastrointestinal bleeding not related to the studied disease (as drug induced haemorrhagic gastropathy, upper GI tract ulcers or cancer, or operable conditions, e.g. haemorrhoids) with the exception of concomitant angiodysplasia which meets the inclusion criteria
    9. gastrointestinal bleeding due to trauma, invasive diagnostic or surgical procedures
    10. concomitant autoimmune anaemia and/or autoimmune thrombocytopenia
    ?Esperanza de vida < 1 año
    ?Presencia de alo-anticuerpos a FVW o FVIII
    ?Enfermedad de Von Willebrand adquirida
    ?Co-morbilidad con otras diátesis hemorrágicas, excluyendo las relacionadas con EVW como complicación del tratamiento (por ejemplo trombocitopenia en EVW tipo 2B)
    ?Cirrosis hepática avanzada con:
    ?Anormalidades de coagulación relacionadas con cirrosis (trombocitopenia, definida como número de plaquetas < 50.000/mm3, INR > 1,7, tiempo prolongado de protrombina ? 4 segundos versus normal)
    ?Hipertensión portal con historia de sangrado variceal
    ?Embarazo y lactancia
    ?Cualquier necesidad conocida de procedimientos invasivos o cirugía electiva programada para los siguientes 3 meses (reclutamiento en estos casos se debe posponer)
    ?Co-morbilidad demostrada para otras causas de sangrado gastrointestinal no relacionado con la enfermedad estudiada (como gastropatía hemorrágica inducida por un fármaco, úlceras de tracto gastrointestinal superior o cáncer, o condiciones operables, por ejemplo hemorroides) con la excepción de angiodisplasia concomitante que cumpla los criterios de inclusión
    ?Sangrado gastrointestinal debido a trauma, diagnóstico invasivo o procedimientos quirúrgicos
    ?Anemia autoinmune concomitante y/o trombocitopenia autoinmune
    E.5 End points
    E.5.1Primary end point(s)
    Prevention of spontaneous bleeding onset, expressed as the proportion of patients who do not present any spontaneous bleeding episode during the study period.
    La variable principal de eficacia será la prevención del inicio de hemorragias espontáneas
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    12 meses
    E.5.2Secondary end point(s)
    - Incidence rate of spontaneous bleedings (episodes/patient*time)
    - Incidence rate of all bleedings (episodes/patient*time)
    - Total number of bleedings (either spontaneous and all bleedings)
    - Mean duration of spontaneous bleeding episode (days)
    - Mean number of infusions per spontaneous bleeding episode
    - Mean dose of concentrate per spontaneous bleeding episode (expressed in FVIII:C and in VWF:RCo)
    - Transfusional needs*, expressed either as:
    Proportion of patients who need a transfusion
    Incidence rate of transfusions (events/patient*time)
    - Mean level of VWF:Rco (IU/dl) and Factor VIII (IU/dl) attained before infusion (trough levels) for patients in prophylaxis
    - Petterssen score, for patients with recurrent joint bleedings
    - Disease related days of in-hospitalisation in the period of interest. Hospitalisation in these patients reflects the need to control bleeding, or to transfuse blood components
    - Reduction of disease related in-hospital admissions and disease-related days of inhospitalisation in the period of interest. Hospitalisation in these patients reflects the need to control bleeding, or to transfuse blood components
    - Time free from event, the interval between randomization and the first bleeding episode after randomization.
    Incidencia de hemorragias espontáneas (episodios/paciente/tiempo)
    -Incidencia de hemorragias totales (episodios/paciente/tiempo)
    -Número total de hemorragias (ya sean espontáneas o totales)
    -Duración media del episodio hemorrágico espontáneo (días)
    -Número medio de infusiones por episodio hemorrágico espontáneo
    -dosis media de concentrado por episodio hemorrágico espontáneo (expresado en FVIII:C y en FVW:CoR)
    -Necesidades transfusionales*, expresadas como:
    -Proporción de pacientes que necesitan una transfusión
    -Incidencia de transfusiones (episodios/paciente/tiempo)
    -Concentración media de FVW:CoR (UI/dl) y Factor VIII (UI/dl) alcanzadas antes de la infusión (concentraciones mínimas) para pacientes en profilaxis
    -Puntuación de Petterssen para pacientes con hemorragias articulares recurrentes
    -Días de hospitalización relacionada con la enfermedad en el periodo de interés La hospitalización en estos pacientes refleja la necesidad de controlar la hemorragia o de transfundir componentes sanguíneos
    -Reducción de las admisiones hospitalarias relacionadas con la enfermedad y días de hospitalización relacionada con la enfermedad en el periodo de interés. La hospitalización en estos pacientes refleja la necesidad de controlar la hemorragia o de transfundir componentes sanguíneos
    -Periodo libre de episodios, el intervalo entre la aleatorización y el primer episodio hemorrágico después de la aleatorización
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    12 meses
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3.1Comparator description
    on demand treatment with the same product
    E.8.2.4Number of treatment arms in the trial4
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA19
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    útlima visita último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years7
    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: 2
    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: 1
    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) Yes
    F.1.2.1Number of subjects for this age range: 6
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 0
    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 Yes
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 24
    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)
    El mismo que seguia el paciente antes antes de participar en el estudio. Referido a su hematólogo habitual
    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 Decision2012-05-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-04-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-08-24
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