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    Summary
    EudraCT Number:2013-005512-10
    Sponsor's Protocol Code Number:997HA306
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-05-26
    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 ConcernedItaly - Italian Medicines Agency
    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
    Valutazione in aperto, multicentrica della sicurezza e dell’efficacia della proteina di fusione ricombinante costituita dal fattore VIII di coagulazione connesso al dominio Fc (rFVIIIFc; BIIB031) nella prevenzione e nel trattamento del sanguinamento in pazienti affetti da emofilia A severa non trattati in precedenza
    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.
    Studio clinico su pazienti con emofilia A non trattati in precedenza per comprendere se l’assunzione di una proteina di sostituzione del fattore VIII (nota come rFVIIIFc) sperimentale sia sicura e se questa funzioni nel prevenire e fermare i sanguinamenti
    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 pointN/A
    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
    Emofilia 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.
    L’emofilia A è un disturbo genetico che compromette la capacità del corpo di controllare la coagulazione del sangue, necessaria ad arrestare i sanguinamenti in caso di rottura di un vaso sanguigno.
    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.
    L’obiettivo primario dello studio è quello di valutare la sicurezza di rFVIIIFc in soggetti affetti da emofilia di tipo 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
    - valutare l'efficacia di rFVIIIFc nella prevenzione e nel trattamento degli episodi emorragici nei pazienti non trattati in precedenza (PUPs);
    - valutare il consumo di rFVIIIFc nella prevenzione e nel trattamento di episodi emorragici nei pazienti non trattati in precedenza;
    - descrivere l'esperienza con l'uso di rFVIIIFc per l'induzione di immunotolleranza (immune intolerance induction, ITI) in soggetti con inibitori.
    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. Capacità del soggetto o del rispettivo genitore o tutore legale di comprendere
    l’obiettivo e i rischi associati allo studio e di fornire un consenso informato
    firmato e datato e l’autorizzazione all’utilizzo delle informazioni sanitarie
    protette (protected health information, PHI) o equivalenti e/o a fornire il
    proprio assenso in accordo alle norme nazionali e locali.
    2. Soggetti di sesso maschile di età <18 anni al momento del consenso informato.
    3. Peso ≥3,5 kg al momento del consenso informato.
    4. Emofilia A severa, definita come valore <1 UI/dl (<1%) di FVIII endogeno
    documentato nella cartella clinica o testato durante il Periodo di
    screening. Qualsiasi soggetto arruolato in base ai risultati del laboratorio locale
    deve essere ritirato qualora i risultati dello screening eseguito dal laboratorio
    centrale indichino un livello di attività di FVIII al basale ≥1% della norma.
    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. Precedente anamnesi di inibitori secondo quanto definito dal laboratorio che redige il referto. La positività storica al test degli inibitori è definita in base al valore Bethesda per la positività del test degli inibitori del laboratorio locale (ossia uguale o maggiore al livello di rilevamento inferiore).
    2. Attività misurabile degli inibitori alla
    visita di screening, misurata mediante il saggio Bethesda modificato di Nijmegen
    eseguito presso il laboratorio centrale. Un risultato negativo al test degli inibitori presso
    il laboratorio locale potrà essere inizialmente utilizzato per determinare l’idoneità dei soggetti, tuttavia qualsiasi soggetto arruolato in base ai risultati del laboratorio locale
    deve essere ritirato qualora i risultati dello screening eseguito dal laboratorio
    centrale indichino un inibitore positivo.
    3. Anamnesi di reazioni di ipersensibilità associate alla somministrazione EV di immunoglobuline.
    4. Iniezione di qualsiasi prodotto sostitutivo di FVIII o di qualsiasi componente del sangue prima della conferma dell’idoneità.
    5. Iniezione di r FVIII prima della conferma dell’idoneità.
    6. Altro/i disturbo/i della coagulazione oltre all’emofilia A.
    7. Qualsiasi malattia grave clinicamente significativa concomitante che, nel parere
    dello sperimentatore, renderebbe il soggetto inadatto all’arruolamento.
    8. Trattamento sistemico concomitante con chemioterapia e/o altri
    farmaci immunosoppressori. L’uso di corticosteroidi per il trattamento dell’asma o per la gestione di episodi allergici acuti è permesso con l’esclusione di trattamenti del trattamento sistemico con corticosteroidi somministrato a bambini giornalmente o
    a giorni alterni a ≥ 2 mg / kg al giorno di prednisone o equivalente o ≥ 20 mg / giorno se pesano più di 10 kg per non più di 14 giorni.
    9. Partecipazione, nei 30 giorni precedenti, ad un altro studio clinico
    che preveda un trattamento sperimentale.
    10. Arruolamento concomitante in un altro studio clinico che preveda
    un trattamento sperimentale.
    11. Incapacità di aderire ai requisiti dello studio.
    12. Altre ragioni non specificate che, nel parere dello Sperimentatore o
    di Biogen Idec, rendano il soggetto inadatto all’arruolamento.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the occurrence of inhibitor development.
    L’endpoint primario dello studio è l’incidenza dello sviluppo dell’inibitore.
    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
    I soggetti saranno testati per la formazione di inibitori: ad ogni visita in clinica incluso screening e baseline;a 5 (±1),10-15,20-25,50,75 e 100 ED;da 2 a 4 sett. prima di un intervento chirurgico maggiore elettivo,prima dell’intervento,dopo intervento e all’ultima visita dopo intervento (Sez 4.2.2 Protocollo).Se questo non coincide con una visita programmata,una visita aggiuntiva deve essere programmata.Per interventi minori,i test per inibitori saranno eseguiti se indicato dalla natura della procedura,secondo lo standard di cura locale;se si sospetta lo sviluppo di inibitori in qualunque momento dello studio,il soggetto sarà testato per inibitori dal laboratorio centrale.Ulteriori analisi non programmate per inibitori potranno essere effettuate se richiesto da standard di cura locali
    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)
    - numero di episodi emorragici annuali per soggetto
    - numero di episodi emorragici articolari spontanei annuali per soggetto
    - numero di iniezioni e dose per iniezione di rFVIIIFc necessari per risolvere un episodio emorragico
    - valutazioni della risposta al trattamento con rFVIIIFc per gli episodi
    emorragici utilizzando la scala di risposta dei sanguinamenti a 4 punti
    - numero totale di ED all’anno per soggetto
    - consumo annuale totale di rFVIIIFc per soggetto per la prevenzione e il trattamento di episodi emorragici
    - recupero incrementale di rFVIIIFc
    - Risposta a ITI con rFVIIIFc (successo, successo parziale, failure, recesso prematuro)
    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.
    L’incidenza degli episodi emorragici, la dose e il numero di iniezioni e
    la risposta al trattamento saranno costantemente monitorati dal medico
    e saranno ricavati dai diari elettronici dei pazienti (electronic patient diary, EPD), dalle schede elettroniche di raccolta dati (electronic case report form, eCRF) e dalle cartelle cliniche. Durante le visite in clinica in cui è prevista un’iniezione di rFVIIIFc, saranno effettuati dei prelievi di sangue per valutare il recupero incrementale.
    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 concerned4
    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.
    La fine dello studio avrà luogo quanto almeno 100 soggetti avranno raggiunto
    almeno 100 ED con rFVIIIFc. Una volta raggiunto questo traguardo,
    ogni altro soggetto dello studio che non abbia raggiunto i 100 ED dovrà
    presentarsi al centro dello studio per le valutazioni della Visita di interruzione
    anticipata/fine dello studio.
    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.
    Ai genitori/ tutori legali dei soggetti sarà fornita l’informativa e consenso informato prima della visita di screening per consentire un tempo adeguato per la revisione e l'opportunità di discutere lo studio con Sperimentatore.
    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 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.
    Standard di cura; non sono previste ulteriori disposizioni per l’accesso ai trattamenti in studio al di là della durata del trattamento specificata nel protocollo.
    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 Decision2014-07-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-16
    P. End of Trial
    P.End of Trial StatusCompleted
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