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    Summary
    EudraCT Number:2012-003450-92
    Sponsor's Protocol Code Number:IG1005
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2012-11-29
    Trial 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 number2012-003450-92
    A.3Full title of the trial
    EVALUATION OF THE PHARMACOKINETIC PROFILE, CLINICAL EFFICACY AND SAFETY OF THE VON WILLEBRAND FACTOR CONTAINED IN FANHDI® (DOUBLE-INACTIVATED HUMAN ANTI-HEMOPHILIC FACTOR) IN PEDIATRIC PATIENTS WITH VON WILLEBRAND DISEASE
    EVALUACIÓN DEL PERFIL FARMACOCINÉTICO, EFICACIA CLÍNICA Y SEGURIDAD DEL FACTOR VON WILLEBRAND CONTENIDO EN FANHDI® (FACTOR ANTI-HEMOFÍLICO HUMANO DOBLEMENTE INACTIVADO) EN PACIENTES PEDIÁTRICOS CON ENFERMEDAD DE VON WILLEBRAND
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of the dynamic behavior, efficacy and safety of Fanhdi®, a high-purity von Willebrand containing FVIII concentrate, in pediatric patients with von Willebrand disease.
    A.3.2Name or abbreviated title of the trial where available
    Study of Fanhdi® in pediatric patients with VWD
    Estudio de Fanhdi® en pacientes pediatricos con EVW
    A.4.1Sponsor's protocol code numberIG1005
    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 SponsorInstituto Grifols S.A.
    B.1.3.4CountrySpain
    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 supportInstituto Grifols S.A.
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInstituto Grifols S.A.
    B.5.2Functional name of contact pointFanhdi pediatric study VWD
    B.5.3 Address:
    B.5.3.1Street AddressCan Guasch 2 (Polígono Levante)
    B.5.3.2Town/ cityParets del Vallès
    B.5.3.3Post code08150
    B.5.3.4CountrySpain
    B.5.4Telephone number+34935710584
    B.5.5Fax number+34935710381
    B.5.6E-mailmwoodward@grifols.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
    D.2.1.1.2Name of the Marketing Authorisation holderInstituto Grifols S.A.
    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.3.4Pharmaceutical form Concentrate and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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 Yes
    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 von Willebrand disease including types I, II, and III with VWF:RCof <15-20%
    Enfermedad de von Willebrand severa incluyendo los tipos I, II y III con VWF:RCof <15-20%
    E.1.1.1Medical condition in easily understood language
    Severe von Willebrand disease
    Enfermedad de von Willebrand severa
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10068986
    E.1.2Term Von Willebrand's factor activity decreased
    E.1.2System Organ Class 10022891 - Investigations
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10055168
    E.1.2Term Von Willebrand's factor deficiency
    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
    1. Evaluate the in vivo recovery and the pharmacokinetic profile of Fanhdi® in a pediatric population (<6 years of age) with severe von Willebrand disease
    2. Evaluate the efficacy of the product as a treatment for bleeds.
    3. Evaluate the efficacy of the product as prophylaxis for surgeries.
    1. Evaluar la recuperación in vivo y el perfil farmacocinético de Fanhdi® en una población pediátrica (<6 años) con enfermedad de von Willebrand grave.
    2. Evaluar la eficacia del producto como tratamiento en hemorragias.
    3. Evaluar la eficacia del producto como profilaxis en cirugías.
    E.2.2Secondary objectives of the trial
    1. Determine the long term clinical efficacy of prophylactic and/or on-demand treatment.
    2. Evaluate the clinical safety, as well as the immunogenicity and thrombogenicity of the product.
    3. Evaluate the tolerance to the product administration
    1. Determinar la eficacia clínica a largo plazo de la profilaxis y/o el tratamiento a demanda.
    2. Evaluar la seguridad clínica, así como la inmunogenicidad y la trombogenicidad del producto.
    3. Evaluar la tolerancia a la administración del producto.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients diagnosed with severe hereditary von Willebrand's disease (VWF:RCof<20 IU/dl) type 1, 2 or 3, independent of prior treatment
    2. Patients less than 6 years of age
    3. Patients that do not adequately respond to treatment with desmopressin
    4. Positive anti-HBs and anti-HAV antibodies due to prior exposure or vaccination. If negative, vaccination to hepatitis A and B with be initiated before the first infusion of Fanhdi®
    5. Signed informed consent by the patient's legal representative (mother, father o tutor)
    1. Pacientes diagnosticados con EVW hereditaria grave (VWF:RCof < 15-20 UI/dl), de tipo 1, 2 y 3, independientemente del tratamiento previo.
    2. Pacientes menores de 6 años de edad.
    3. Pacientes que no respondan adecuadamente al tratamiento con desmopresina
    4. Anticuerpos anti-HBs y anti-HVA positivos por exposición previa o vacunación. Si son negativos se iniciará la vacunación para las hepatitis A y B antes de la primera infusión de Fanhdi®
    5. Firma del consentimiento informado por parte del representante legal del paciente (madre, padre o tutor).
    E.4Principal exclusion criteria
    1. The subject has been diagnosed of acquired VWD2. Patients bleeding at the time of the first infusion or the 10 days prior to the infusion
    3. Subjects treated with desmopressin or another VWF containing FVIII concentrate during the 5 days prior to the infusion of the investigational product
    4. The subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF
    5. The subject is known to have history of intolerance to any Fanhdi® containing substance
    6. The subject is known to have history of anaphylactic reaction(s) to blood or blood components
    7. Subjects presenting severe platelet dysfunctions due to drugs (aspirin, other NSAIDs, etc.) or other pathologies (uremic thrombopathy, hematological diseases)
    8. Immunocompromised subjects or HIV positive that have less than 400 CD4+/?l, a viral load >400 copies/ml, platelet count lower than 100x10·9/l, present any additional factor that elevates the risk of bleeding or that have a life expectancy less than 1 year
    9. Subjects presenting anemia (hemoglobulin <11 g/dl)
    10. Subjects diagnosed with metabolic diseases that are not clinically controlled, such as diabetes mellitus, that could potentially interfere with the interpretations of the study
    11. The subject is participating in another clinical study involving an investigational treatment, or participated within the past month
    12. If it is anticipated that the subject will be treated with other products containing FVIII or VWF different from Fanhdi® during a period of one year
    13. The subject is unlikely to adhere to the protocol requirements of the study
    14. Any subject that does not dispose of a frozen plasma sample prior to the first infusion of Fanhdi®
    1.Pacientes que presenten enfermedad de von Willebrand adquirida.
    2.Pacientes que presenten un episodio hemorrágico en el momento de la primera infusión del fármaco en los 10 días previos a la infusión.
    3.Pacientes que hayan sido tratados con desmopresina o con otro concentrado de FVIII conteniendo FVW en los 5 días previos a la infusión del fármaco en estudio.
    4.Pacientes que en algún momento de la historia de su enfermedad hayan presentado o presenten anticuerpos anti-FVW o anti-FVIII (>0.5 UB).
    5.Pacientes con alergias conocidas a alguno de los componentes de Fanhdi®
    6.Pacientes con historia conocida de reacciones graves o frecuentes a productos derivados del plasma.
    7.Pacientes que presenten alteraciones de la función plaquetaria de forma relevante, debido a fármacos (aspirina, otros AINEs, etc.) o a otras patologías (trombopatía urémica, enfermedades hematológicas).
    8.Pacientes inmunocomprometidos o HIV positivos que tengan menos de 400 CD4+/?l, carga viral mayor a 400 copias/ml, recuento plaquetario menor a 100x10·9/l, presenten algún factor adicional que aumente el riesgo hemorrágico o tengan un pronóstico de vida inferior a 1 año.
    9.Paciente que presente anemia (hemoglobina < 11 g/dl).
    10.Paciente que padezca enfermedades metabólicas no controladas clínicamente, como diabetes mellitus, que pudiera interferir con las interpretaciones del estudio.
    11.Haber participado en otros ensayos clínicos o haber recibido cualquier otro fármaco en investigación durante el mes previo al inicio del estudio.
    12.Previsión de que pueda ser tratado con otros productos conteniendo FVIII o FVW diferentes a Fanhdi® en un período de un año.
    13.Sospecha de condiciones que pudieran afectar al cumplimiento del protocolo por parte del paciente.
    14.Cualquier paciente del que no se disponga de una muestra de plasma congelada previa a la primera infusión de Fanhdi®
    E.5 End points
    E.5.1Primary end point(s)
    1. Determine the half-life and in vivo recovery of VWF:RCof and VWF:Ag, the half-life and the apparent in vivo recovery of FVIII:C, and other pharmacokinetic parameters after a single dose of Fanhdi® in pediatric patients with severe VWD.
    2. Determine the clinical efficacy of Fanhdi® as replacement therapy in preventing excessive bleeding during spontaneous or traumatic induced bleeding episodes through the study of the duration and severity of the bleed, achievement of hemostasis, total number of infusions and doses administered for each bleeding episode.
    3. Determine the clinical efficacy of Fanhdi® as replacement therapy in preventing excessive bleeding in patients who undergo surgical procedures or invasive procedures through the study of the duration and seriousness of bleeding episodes and the achievement of hemostasis.
    1. Determinar la semivida y recuperación in vivo de FVW:RCof y FVW:Ag así como la semivida aparente y recuperación in vivo del FVIII:C, y otros parámetros farmacocinéticos después de una única infusión de concentrado de Fanhdi® en pacientes pediátricos con EVW grave.
    2. Determinar la eficacia clínica en el tratamiento de episodios hemorrágicos, espontáneos o traumáticos mediante el estudio de la duración y severidad del sangrado, consecución de hemostasia, número de infusiones y dosis total requerida
    para cada episodio hemorrágico.
    3. Determinar la eficacia clínica como profilaxis en pacientes sometidos a cirugías y procedimientos invasivos mediante el estudio de la duración y gravedad del sangrado y la consecución de hemostasia.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Pharmacokinetic parameters will be assessed within a maximum of 15 days after the recruitment visit. Subjects with type III VWD will undergo a second pharmacokinetic study 6 months after the first PK analysis.
    2. Whenever a bleeding treated in the hospital occurs throughout the 12 month follow-up period.
    3. Whenever a surgery or invasive procedure occurs throughout the 12 month follow-up period.
    1. Los parámetros farmacocinéticos serán evaluados en un máximo de 15 días después de la visita de reclutamiento. Los sujetos con EVW tipo III se someterán a un segundo estudio farmacocinético 6 meses después del primer análisis farmacocinético.
    2. Siempre que ocurra un sangrado tratado en el hospital a lo largo de los 12 meses de seguimiento.
    3. Siempre que ocurra una cirugía o un procedimiento invasivo a lo largo de los 12 meses de seguimiento.
    E.5.2Secondary end point(s)
    1. Evaluate long term clinical efficacy of profilaxis and/or long term demand studying concentrates requirements and the number of haemorragic epsiodes treated.
    2. Evaluate product's clinical security, inmunogenicity and trombogenicity detecting presence of FVIII and FVW inhibitors and evaluating clinical trombosis in patients undergoing quirurgic or invasive procedures.
    3. Evaluate the product's administration tolerance detecting adverese reactions, including clinically significant changes in vital signs or lab paramenters.
    1. Evaluar la eficacia clínica a largo plazo de la profilaxis y/o el tratamiento a demanda mediante el estudio de los requerimientos de concentrado y del número de episodios
    hemorrágicos tratados.
    2. Evaluar la seguridad clínica, así como la immunogenicidad y la trobogenicidad del producto mediante la detección de la presencia de inhibidores de FVIII y del FVW y la evaluación clínica de trombosis en aquellos pacientes sometidos a procedimientos quirúrgicos o invasivos.
    3. Evaluar la tolerancia a la administración del producto mediante la detección de reacciones adversas, incluyendo cambios clínicamente significativos en los signos vitales o parámetros de laboratorio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. The clinical safety and tolerance to Fanhdi will be assessed since recruitment and throughout the 12 month follow-up period.
    2. Immunogenicity will be measured before the first infusion of the investigational product, every three months throughout the follow-up period and when there exists a clinical suspicion of the formation of inhibitors.
    3. Thrombogenicity will be measured in all subjects undergoing surgery or an invasive procedure.
    1. La seguridad clínica y la tolerancia a Fanhdi se evaluará desdel reclutamiento a lo largo de los 12 meses de seguimiento.
    2. La inmunogenicidad se evaluará antes de la primera infusión del producto en investigación, cada tres meses a lo largo del período de seguimiento y cuando exista una sospecha clínica de formación de inhibidores.
    3. La trombogenicidad se evaluará en todos los sujetos que se sometan a cirugías o a procedimientos invasivos.
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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.8.3 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 concerned5
    E.8.5The trial involves multiple Member States No
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned 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: 8
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 4
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 4
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations 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
    Pediatric patients under 6 years of age.
    Pacientes pediatricos menores de 6 años de edad.
    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 8
    F.4.2.2In the whole clinical trial 8
    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)
    None
    Ninguno
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-11-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-10-31
    P. End of Trial
    P.End of Trial StatusOngoing
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