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    Summary
    EudraCT Number:2011-002415-28
    Sponsor's Protocol Code Number:CSL654_3001
    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:2011-10-28
    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 number2011-002415-28
    A.3Full title of the trial
    A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B
    Estudio de Fase II/III, abierto y multicéntrico, de la seguridad y la eficacia de una proteína de fusión del factor IX recombinante de la coagulación con la albúmina (rIX-FP) en sujetos con hemofilia B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A safety and Efficacy Study of a recombinant Factor IX in patients with severe Hemophilia B
    EStudio de la eficacia y seguridad del factor recombinante IX en pacientes con hemofilia B severa
    A.4.1Sponsor's protocol code numberCSL654_3001
    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 SponsorCSL Behring GmbH
    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 supportCSL Behring GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring
    B.5.2Functional name of contact pointSr. Clinical Program Manager
    B.5.3 Address:
    B.5.3.1Street AddressEmil-von-Behring-Str. 76
    B.5.3.2Town/ cityMarburg
    B.5.3.3Post code35041
    B.5.3.4CountryGermany
    B.5.4Telephone number+496421392930
    B.5.5Fax number+496421393172
    B.5.6E-mailkerstin.jung@cslbehring.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 nameProteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
    D.3.2Product code CSL654
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCSL654 or rIX-FP
    D.3.9.3Other descriptive nameProteína de fusión recombinante que une el factor de coagulación IX con albúmina humana.
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameProteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
    D.3.2Product code CSL654
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCSL654 or rIX-FP
    D.3.9.3Other descriptive nameProteína recombinante de fusión que une el factor de coagulación IX con albúmina humana.
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameProteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
    D.3.2Product code CSL654
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCSL654 or rIX-FP
    D.3.9.3Other descriptive nameProteína de fusión recombinante que une el factor de coagulación IX con albúmina humana.
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameProteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
    D.3.2Product code CSL654
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCSL654 or rIX-FP
    D.3.9.3Other descriptive nameProteína de fusión recombinante que une el factor de coagulación IX con albúmina humana.
    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
    Prophylaxis and treatment of bleeding episodes in subjects with congenital Factor IX (FIX) deficiency (Hemophilia B).
    Profilaxis y tratamiento de los episodios hemorrágicos en sujetos con deficiencia congénita del factor IX (FIX) (Hemofilia B).
    E.1.1.1Medical condition in easily understood language
    A study on prophylaxis and treatment of a non-plasma source (recombinant) product that replaces the missing clotting factor IX in patients with sever Hemophilia B.
    Estudio sobre la profilaxis y el tratamiento de un producto de fuente no-plasmática (recombinante) que sustituye la falta del factor de coagulación IX en pacientes con hemofilia severa B.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level LLT
    E.1.2Classification code 10060614
    E.1.2Term Hemophilia B (Factor IX)
    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
    The primary objective of the study is to evaluate the efficacy of rIX-FP and the clinical response in preventing bleeding episodes (prophylaxis) in patients with severe hemophilia B.
    El objetivo principal del estudio es evaluar la eficacia de la rIX-FP y la respuesta clínica en la prevención de los episodios hemorrágicos (profilaxis) en pacientes con hemofilia B severa.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    ?To evaluate the safety of rIX-FP.
    ?To evaluate the clinical response to rIX-FP for the treatment of bleeding episodes in patients with severe hemophilia B.
    ?To evaluate the efficacy of rIX-FP in surgical prophylaxis.
    ?To evaluate the pharmacokinetics (PK) of a single dose of rIX-FP.
    Los objetivos secundarios del estudio son:
    ?Evaluar la seguridad de la rIX-FP.
    ?Evaluar la respuesta clínica a la rIX-FP en el tratamiento de los episodios hemorrágicos en pacientes con hemofilia B severa.
    ?Evaluar la eficacia de la rIX-FP en la profilaxis quirúrgica.
    ?Evaluar la farmacocinética (FC) de una dosis única de rIX-FP.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B?Surgical Substudy
    Estudio de Fase II/III, abierto y multicéntrico, de la seguridad y la eficacia de una proteína de fusión del factor IX recombinante de la coagulación con la albúmina (rIX-FP) en sujetos con hemofilia B-Subestudio quirúrgico.
    E.3Principal inclusion criteria
    - Male subjects, 12 to 65 years of age.
    - Documented severe hemophilia B (FIX activity of ? 2%), or confirmed at Screening by the central laboratory.
    - Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs),
    confirmed by their treating physician.
    - No confirmed prior history of FIX inhibitor formation (defined as two consecutive positive tests ?requiring a confirmatory test
    on a second separately drawn blood sample shortly after the previous positive test), no confirmed detectable inhibitors
    (defined as
    < 0.6 Bethesda Units [BU]) at Screening by the central laboratory, and no family history of inhibitor formation against FIX.
    - Written informed consent for study participation obtained before undergoing any study specific procedures.

    For on-demand subjects ONLY:
    - Subjects who have experienced a minimum average of 2 non-trauma induced bleeding episodes per month in the past 3 to 6
    months, which required FIX replacement therapy and are documented in their medical records.
    - Subjects who are willing to switch to a prophylaxis regimen.
    Podrán entrar en el estudio los sujetos que cumplan todos los criterios de inclusión siguientes:
    ?Varones de 12 a 65 años de edad.
    ?Hemofilia B severa documentada (actividad del FIX ? 2%), o confirmada en la Selección por el laboratorio central.
    ?Sujetos que han recibido productos de FIX (FIX derivado del plasma y/o recombinante) durante > 150 días de exposición (DE), confirmado por su médico.
    ?Ausencia de historia previa confirmada de formación de inhibidores del FIX (lo que se define como dos estudios positivos consecutivos ? que requieren un estudio confirmador en una segunda muestra extraída poco después de la positiva previa), ausencia confirmada de inhibidores detectables (lo que se define como < 0,6 unidades Bethesda [UB]) en la Selección por el laboratorio central, y ausencia de historia familiar de formación de inhibidores frente al FIX.
    ?Obtención del consentimiento informado por escrito para la participación en el estudio antes de la práctica de cualquier procedimiento específico del estudio.

    SOLAMENTE en los sujetos a demanda:
    ?Sujetos que han presentado un promedio mínimo de 2 episodios hemorrágicos de origen no traumático al mes en los 3 a 6 últimos meses, que requirieron tratamiento sustitutivo con FIX y que se encuentran documentados en su historia médica.
    ?Sujetos que están dispuestos a cambiar a una pauta de profilaxis.
    E.4Principal exclusion criteria
    - Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein.
    - Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
    - Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
    - Platelet count < 100,000/?L at Screening.
    - HIV positive subjects with a CD4 count < 200/mm3. An HIV-positive subject may participate in the study and receive antiviral
    therapy at the discretion of the Investigator.
    - Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 5 x ULN at Screening.
    - Serum creatinine concentration > 2 x ULN at Screening.
    - Evidence of thrombosis, including deep vein thrombosis, stroke, myocardial infarction or arterial embolus within 4 months
    prior to dosing on Day 1.
    - Experienced a life-threatening bleeding episode, including bleeding in the central nervous system, gastrointestinal tract,
    neck/throat or severe trauma-induced bleeding episode, or had major surgical intervention within 4 months prior to dosing on
    Day 1.
    - Use of any Investigational Medicine Product (IMP) other than rIX-FP within 4 weeks prior to the first rIX-FP administration on
    Day 1.
    - Concurrent non-hemophiliac inflammatory joint disease or other medical condition that, in the Investigator?s judgment, could
    confound study results.
    - Suspected inability (e.g., language problem or mental condition) or unwillingness to comply with study procedures or history
    of noncompliance.
    - Subjects who have active synovitis.
    - Subject routinely receives factor IX infusion prior to activity (ie, sports) as a preventative measure more than 2 times per
    month.
    No podrán entrar en el estudio los sujetos que cumplan cualquiera de los siguientes criterios de exclusión:
    ?Hipersensibilidad conocida (reacción alérgica o anafilaxia) a cualquier producto de FIX o a las proteínas del hámster.
    ?Trastorno conocido de la coagulación, congénito o adquirido, distinto de la deficiencia congénita de FIX.
    ?En tratamiento actualmente con agentes inmunomoduladores IV, como inmunoglobulina, o en tratamiento crónico con corticosteroides sistémicos.
    ?Recuento de plaquetas < 100.000/µl en la Selección.
    ?Sujetos positivos para el VIH con un recuento de CD4 < 200/mm3. A criterio del investigador, un sujeto VIH positivo podrá participar en el estudio si recibe tratamiento antiviral.
    ?Concentración sérica de aspartato aminotransferasa (AST) o de alanina aminotransferasa (ALT) > 5 x LSN en la Selección.
    ?Concentración sérica de creatinina > 2 x LSN en la Selección.
    ?Evidencia de trombosis, incluidos trombosis venosa profunda, ictus, infarto de miocardio o émbolo arterial, en el plazo de los 4 meses previos a la administración del Día 1.
    ?Episodio hemorrágico con riesgo para la vida, como hemorragia en el sistema nervioso central, tracto gastrointestinal, cuello/garganta o episodio hemorrágico severo de origen traumático, o intervención quirúrgica mayor, en el plazo de los 4 meses previos a la administración del Día 1.
    ?Tratamiento con cualquier producto en investigación (PEI) distinto de la rIX-FP en las 4 semanas previas a la primera administración de la rIX-FP el Día 1.
    ?Enfermedad articular inflamatoria no hemofílica concurrente u otro proceso médico que, en opinión del Investigador, pudiera actuar como factor de confusión de los resultados del estudio.
    ?Sospecha de incapacidad (por ejemplo, problemas de lenguaje o trastorno mental) o de falta de deseos de cumplir con los procedimientos del estudio, o historia de incumplimiento del tratamiento.

    SOLAMENTE en los sujetos a demanda:
    ?Sujetos con sinovitis activa.
    ?Sujetos que reciben habitualmente un infusión de factor IX antes de una actividad (por ejemplo, deporte) como medida preventiva más de 2 veces al mes.
    E.5 End points
    E.5.1Primary end point(s)
    Annualized spontaneous bleeding events during the on-demand treatment period compared to the annualized spontaneous bleeding events during the routine prophylaxis treatment period.
    Tasas anualizadas de hemorragia espontánea durante el tratamiento a demanda comparado con las tasas anualizadas espontáneas durante el tratamiento profiláctico.
    E.5.1.1Timepoint(s) of evaluation of this end point
    approximately 20 weeks
    Aproximadamente 20 semanas
    E.5.2Secondary end point(s)
    - The frequency of related Adverse Events (AEs) to rIX-FP over the course of the study.
    - The number of subjects with FIX inhibitors.
    - The number of subjects with antibodies against rIX-FP.
    - Proportion of bleeding episodes requiring one or ? two infusions of rIX-FP to achieve hemostasis.
    - Investigator?s overall clinical assessment of hemostatic efficacy for treatment of bleeding episodes, based on a four point
    ordinal scales (excellent, good, moderate, poor/ none).
    - rIX-FP consumed per month while maintaining assigned prophylactic treatment interval during routine prophylaxis.
    - Incremental recovery (IU/mL/IU/kg) at 30 minutes following infusion of 50 IU/kg rIX-FP.
    - Half-life (t1/2) of a single dose of 50 IU/kg rIX-FP.
    - AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of 50 IU/kg rIX-FP.
    - Clearance of a single dose of 50 IU/kg rIX-FP.
    - Investigator?s (or surgeon?s) overall clinical assessment of hemostatic efficacy for surgical prophylaxis, based on a four point
    ordinal scale (excellent, good, moderate, poor/ none)
    ?Número de episodios hemorrágicos espontáneos por sujeto después del comienzo del tratamiento.
    ?Porcentaje de episodios hemorrágicos que han precisado una o dos infusiones de rIX-FP para alcanzar la hemostasia.
    ?Evaluación clínica global por el Investigador de la eficacia hemostática en el tratamiento de los episodios hemorrágicos.
    ?Uso de rIX-FP en el tratamiento profiláctico.
    E.5.2.1Timepoint(s) of evaluation of this end point
    168 hours
    168 horas
    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) Yes
    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 No
    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.8.3 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 concerned3
    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
    Austria
    Bulgaria
    France
    Germany
    Israel
    Italy
    Japan
    Russian Federation
    Spain
    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 study participation for an individual subject occurs with the end of the follow-up period, which is defined as completion of the final study visit, after which no further study-related procedures will be performed. Prophylaxis subjects (Arm 1) and On-demand subjects (Arm 2) will complete the end-of-study visit on approximately Week 60. The end of the trial is defined as the last-patient-last-visit.
    El fin de la participación en el estudio de un sujeto individual se produce con el final del período de seguimiento, que se define como la finalización de la última visita del estudio, tras el cual no más estudios relacionados con los procedimientos se llevarán a cabo. Los sujetos en profilaxis (grupo 1) y en a demanda (grupo 2) completarán la visita de fin de estudio aproximadamente a la semana 60. El final del ensayo está definido como la última visita del último paciente.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months4
    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: 3
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 55
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    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)
    Patients who end their participation in the trial can return to standard therapy according to their health care scheme. There are several other recombinant Factor IX products available on the market.
    Pacientes que terminaron su participación en el ensayo pueden volver a la terapia estandar de acuerdo a su plan de tratamiento. Hay otros productos Factor recombinante IX disponibles en el mercado.
    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 Decision2011-12-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-12-01
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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