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    Summary
    EudraCT Number:2015-002136-40
    Sponsor's Protocol Code Number:261203
    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:2015-10-15
    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 number2015-002136-40
    A.3Full title of the trial
    Phase 3, prospective, multi-center, open label study to investigate
    safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII
    (BAX 855) in previously untreated patients (PUPs) and minimally treated patients
    (MTPs) < 6 years with severe hemophilia A (FVIII < 1%)
    Estudio de fase III prospectivo, multicéntrico y abierto para investigar la seguridad,la inmunogenicidad y la eficacia hemostática del factor VIII PEGilado (BAX 855) en pacientes no tratados previamente (PNTP) y pacientes tratados mínimamente (PTM) < 6 años con hemofilia A grave (FVIII < 1 %)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of safety, effect on the immune system and blood clotting of BAX855 (factor VIII of blood clotting) in pediatric patients who have not received or received minimal treatment for their severe hemophilia A.
    Estudio de seguridad, efecto sobre el sistema inmune y la coagulación de la sangre
    de BAX855 (factor VIII de coagulación de la sangre) en pacientes pediátricos que
    no han recibido o recibido un tratamiento mínimo para su hemofilia A grave.
    A.3.2Name or abbreviated title of the trial where available
    BAX855 PUP study
    Estudio BAX855 PUP
    A.4.1Sponsor's protocol code number261203
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/072/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxalta Innovations GmbH
    B.1.3.4CountryAustria
    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 supportBaxalta Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMaria Teresa Alvarez Roman
    B.5.2Functional name of contact pointMaria Teresa Alvarez Roman
    B.5.3 Address:
    B.5.3.1Street AddressHospital Universitario La Paz, Paseo de la Castellana 261
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28046
    B.5.3.4CountrySpain
    B.5.4Telephone number+34912071945
    B.5.6E-mailtalvarezroman@gmail.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 namePEGylated rFVIII
    D.3.2Product code BAX855
    D.3.4Pharmaceutical form Powder and solution 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 INNNot applicable
    D.3.9.2Current sponsor codeBAX855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX 855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    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.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX 855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    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.3.8INN - Proposed INNNot applicable
    D.3.9.2Current sponsor codeBAX 855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant product with covalently bound polyethyleneglycol
    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 hemophilia A (FVIII <1%)
    Hemofilia A Grave (FVIII <1%)
    E.1.1.1Medical condition in easily understood language
    Hemophilia A
    Hemofilia A
    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 18.1
    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
    To determine safety including immunogenicity of BAX 855 based on the
    incidence of inhibitor development to FVIII (≥ 0.6 BU/mL using the Nijmegen modification of the Bethesda assay)
    El objetivo principal es evaluar la seguridad, incluida la inmunogenicidad, de BAX
    855 a partir de la incidencia del desarrollo de inhibidores del FVIII (≥0,6 unidades
    de Bethesda [UB]/ml mediante la modificación de Nijmegen del ensayo Bethesda).
    E.2.2Secondary objectives of the trial
    Safety
    1. To determine the immunogenicity of BAX 855 in terms of binding IgG and IGM antibodies to FVIII, PEG-FVIII and PEG
    2. To determine the safety of BAX 855 based on adverse events (AEs) and serious adverse events (SAEs)
    Hemostatic Efficacy
    3. To assess the efficacy of prophylactic treatment with BAX 855
    4. To characterize the efficacy of BAX 855 in the control of bleeding episodes
    5. To evaluate the efficacy of BAX 855 for perioperative management, if surgery is required
    Pharmacokinetics
    6. To determine the incremental recovery (IR) of BAX 855 at baseline and over time
    7. To determine half-life of BAX 855 at baseline (optional)
    Seguridad 1.-Determinar la inmunogenicidad de BAX 855 en cuanto a anticuerpos
    de unión IgG e IgM al FVIII, al PEG-FVIII y al PEG. 2.-Determinar la seguridad de
    BAX 855 a partir de los acontecimientos adversos (AA) y de los acontecimientos
    adversos graves (AAG). 3.-Evaluar la eficacia del tratamiento profiláctico con BAX
    855. 4.- Caracterizar la eficacia de BAX 855 en el control de los episodios
    hemorrágicos. 5.- Evaluar la eficacia de BAX 855 para tratamientos perioperatorios
    si es necesaria una intervención quirúrgica. 6.- Determinar la recuperación
    incremental (RI) de BAX 855 al inicio y a lo largo del tiempo. 7.- Determinar la
    semivida de BAX 855 al inicio (opcional).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is < 6 years old at the time of screening
    2. Subject is previously untreated or minimally treated with ≤ 3 EDs to ADVATE or BAX 855 at any time prior to screening
    3. Subject has severe hemophilia A (FVIII < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory and/or a FVIII gene mutation consistent with severe hemophilia A
    4. Subject is immune competent with a CD4+ count > 200 cells/mm3, as confirmed by central laboratory at screening
    5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocol
    Additional inclusion criteria for Part B (ITI):
    6. Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion
    7. Subject has a confirmed inhibitor titer (≥ 0.6 BU) as determined by the central laboratory based on a second repeat blood sample and requires ITI therapy
    1.-Sujeto < seis años de edad en el momento de la selección. 2.-Sujeto no tratado
    previamente o mínimamente tratado con ? 3 DE de ADVATE o BAX 855 en algún
    momento antes de la selección. 3.-Sujeto con hemofilia A grave (FVIII < 1 %),
    según lo determinado por el laboratorio central, unas concentraciones históricas de
    FVIII < 1 % según lo determinado por cualquier laboratorio local, o una mutación en
    el gen FVIII compatible con la hemofilia A grave. 4.-Sujeto inmunocompetente con
    una cifra de CD4+ > 200 células/mm3, según lo confirmado por el laboratorio
    central en la selección. 5.-Progenitor o representante legal dispuestos y capaces de
    cumplir los requisitos del protocolo Criterios de inclusión adicionales para la parte B
    (ITI): 6.-Progenitor o representante legal que otorguen su consentimiento informado
    firmado de forma voluntaria para la parte de ITI. 7.-Sujeto con un valor confirmado
    de inhibidores (? 0,6 UB) según lo determinado por el laboratorio central a partir de
    una segunda muestra de sangre repetida que requiera tratamiento de ITI.
    E.4Principal exclusion criteria
    1. Subject has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
    2. Subject has a history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening
    3. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease)
    4. Subject has been previously treated with FFP, cryoprecipitate, any type of FVIII concentrate other than ADVATE or BAX 855v or was administered ADVATE or BAX 855 for > 3 EDs at any time prior to screening
    5. Subject has received any kind of blood-transfusion such as PRBC, platelets or plasma prior to screening at any time prior to screening
    6. The subject’s weight is < 5 kg
    7. Subject’s platelet count is < 100,000/mL
    8. Subject has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80
    9. Subject has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented INR > 1.5] in his medical history or at the time of screening
    10. Subject has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)
    11. Subject has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation
    12. Subject is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy
    13. Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study
    14. Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
    15. Parent, legally authorized representative or subject are a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
    Additional exclusion criteria for Part B (ITI)
    16. Spontaneous disappearance of the inhibitor prior to ITI
    17. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory
    18. Inability or unwillingness to comply with the protocol
    1.-Sujeto con anticuerpos inhibidores del FVIII detectables (? 0,6 UB mediante la modificación de Nijmegen del ensayo Bethesda), según lo confirmado por el laboratorio central en la selección. 2.-Sujeto con antecedentes de anticuerpos inhibidores del FVIII (? 0,6 UB mediante la modificación de Nijmegen del ensayo Bethesda o el ensayo de Bethesda) en cualquier momento antes de la selección. 3.-Al sujeto se le ha diagnosticado un defecto hemostático heredado o adquirido diferente a la hemofilia A (como un defecto cualitativo de las plaquetas o la enfermedad de von Willebrand). 4.-Sujeto tratado previamente con plasma fresco congelado (PFC), crioprecipitado, cualquier tipo de concentrado de FVIII distinto de ADVATE o BAX 855 o al que se haya administrado ADVATE o BAX 855 durante >3 DE en algún momento antes de la selección. 5.-Sujeto que haya recibido cualquier tipo de transfusión de sangre, como concentrado de eritrocitos, trombocitos o plasma en cualquier momento antes de la selección. 6.-Peso del sujeto < 5 kg. 7.-Cifra de plaquetas del sujeto < 100 000/ml. 8.-El sujeto tiene una hipersensibilidad conocida a las proteínas de ratón o hámster, PEG o Tween 80.
    9.-Sujeto con disfunción hepática crónica grave (p. ej., alanina aminotransferasa [ALT] o aspartato aminotransferasa [AST] > cinco veces el límite superior de la normalidad o INR documentado > 1,5) en sus antecedentes médicos o en el momento de la selección. 10.-Sujeto con insuficiencia renal grave (creatinina sérica > 1,5 veces el límite superior de la normalidad).11.-Sujeto que utilice o haya
    utilizado recientemente (< 30 días) otros fármacos PEGilados antes de participar en el estudio o que tenga previsto utilizar estos fármacos durante su participación en el estudio. 12.-Sujeto que tenga previsto recibir durante el transcurso del estudio un fármaco inmunomodulador sistémico (como corticoesteroides en una dosis equivalente a hidrocortisona superior a 10 mg/día o ?-interferón) que no sea quimioterapia antirretrovírica . 13.-El sujeto ha participado en otro estudio clínico
    que utilizó un PEI o un dispositivo en investigación durante los 30 días anteriores a la inscripción o está programado que participe en otro estudio clínico con un PEI o un dispositivo en investigación durante el transcurso de este estudio. 14.-Progenitor o representante legal que presente una enfermedad médica, psiquiátrica o cognitiva, o que consuma drogas o alcohol, de manera que, en opinión del investigador, pueda afectar a la seguridad o al cumplimiento del sujeto. 15.-Progenitor, representante legal o sujeto que sean miembros del equipo que realiza este estudio o que tengan una relación de dependencia con alguno de los miembros del equipo del estudio. Las relaciones de dependencia incluyen a parientes cercanos (es decir, hijos, pareja/cónyuge, hermanos, padres), así como los empleados del investigador o el personal del centro que realizan el estudio. Criterios de exclusión adicionales para la parte B (ITI): 16.-Desaparición espontánea del inhibidor antes de la ITI. 17.-Valor de inhibidores del FVIII ? 0,6 UB sin confirmar mediante una segunda muestra nueva de sangre según lo determinado en el laboratorio central. 18.-Incapacidad o falta de disposición para cumplir el protocolo.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of FVIII inhibitor development
    The success rate of ITI therapy with BAX 855
    Incidencia del desarrollo de inhibidores del FVIII. Tasa de éxito del tratamiento de
    ITI con BAX 855 .
    E.5.1.1Timepoint(s) of evaluation of this end point
    The set of subjects to be analyzed includes all subjects who developed an inhibitor (at any time) and all subjects who did not develop an inhibitor and had ≥ 50 EDs. An interim analysis is planned after 50 subjects have completed 50 EDs. The final CSR will also analyze subjects who develop an inhibitor and subjects who did not, but completed at least 100 EDs.
    El conjunto de sujetos que se van a analizar incluye a todos los sujetos que hayan desarrollado un inhibidor (en cualquier momento) y a todos los sujetos que no hayan desarrollado un inhibidor y hayan tenido ? 50 DE. Está previsto efectuar un análisis intermedio cuando 50 sujetos hayan cumplido 50 DE. En el informe del estudio clínico (IEC) final también se analizarán los sujetos que hayan desarrollado un inhibidor y los que no, pero que hayan cumplido al menos 100 DE.
    E.5.2Secondary end point(s)
    Binding IgG and IgM antibodies to FVIII, PEG-FVIII and PEG
    AEs and SAEs
    Clinically significant changes in vital signs and clinical laboratory parameters (hematology and clinical chemistry)
    Efficacy:
     Annualized bleeding rate (ABR) for prophylactic and on-demand treatment
     Number of BAX 855 infusions per bleeding episode
     Overall hemostatic efficacy rating at 24 h after initiation of treatment and at resolution of bleed
     Weight-adjusted consumption of BAX 855 per month, per year and per event (prophylaxis, treatment of bleeding episode, surgery) and the number of infusions per month and per year
     Assessment of intra-, post- and perioperative hemostatic efficacy in case of surgery
     Intra- and postoperative blood loss in case of surgery
    Pharmacokinetics:
     IR at baseline and over time
     Half-life at baseline (optional)
    Additional Outcome Measures for ITI:
    The rate of partial success and failure of ITI with BAX 855
    ABR during ITI
    Weight-adjusted consumption of BAX 855 per month and per year for each ITI regimen employed
    Anticuerpos de unión IgG e IgM al FVIII, al PEG-FVIII y al PEG. AA y AAG. Cambios clínicamente significativos en las constantes vitales y en los parámetros clínicos analíticos (hematología y bioquímica clínica). Eficacia: Tasa anualizada de hemorragias (TAH) con el tratamiento profiláctico y a demanda. Número de infusiones de BAX 855 por episodio hemorrágico. Calificación global de la eficacia hemostática a las 24 horas de iniciar el tratamiento y en la resolución de la hemorragia. Consumo de BAX 855 ajustado por peso al mes, al año y por acontecimiento (profilaxis, tratamiento de episodios hemorrágicos e intervención
    quirúrgica), y número de infusiones por mes y por año. Evaluación de la eficacia
    hemostática intraoperatoria, posoperatoria y perioperatoria en caso de intervención
    quirúrgica. Pérdida de sangre intraoperatoria y posoperatoria en caso de
    intervención quirúrgica. Farmacocinética RI al inicio y a lo largo del tiempo.
    Semivida al inicio (opcional). Esto se basa en un parámetro de FC abreviado
    empleando dos puntos temporales posteriores a la infusión: 15-30 minutos y 24-48
    horas. Criterios de valoración adicionales para ITI: Las tasas de éxito parcial y de
    fracaso de la ITI con BAX 855. TAH durante la ITI. Consumo ajustado por peso de
    BAX 855 por mes y por año de cada pauta de ITI utilizada.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Binding antibodies to FVIII, BAX 855 and PEG and all other secondary safety outcome measures will be analyzed descriptively.
    The ABR will be analyzed by point and interval estimates derived from a negative binomial model with treatment regimen (on-demand vs. prophylaxis) as a covariate and the duration of the observation period as an offset. Other secondary efficacy outcome measures as well as IR over time will be analyzed descriptively.
    Outcomes of ITI will be summarized descriptively.
    Se analizarán descriptivamente los anticuerpos de unión al FVIII, BAX 855 y PEG,
    así como todos los demás criterios de valoración secundarios de la seguridad. La TAH se analizará mediante estimaciones puntuales e interválicas derivadas de un modelo binomial negativo con la pauta de tratamiento (a demanda frente a profiláctico) como covariable y la duración del periodo de observación como variable de exposición. Se analizarán descriptivamente otros criterios de valoración de la eficacia secundarios, así como la RI a lo largo del tiempo. Los resultados de la ITI se resumirán de forma descriptiva.
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA55
    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
    Austria
    Belgium
    Bulgaria
    Czech Republic
    Denmark
    Finland
    France
    Germany
    Hungary
    Italy
    Korea, Republic of
    Lithuania
    Malaysia
    Netherlands
    New Zealand
    Norway
    Poland
    Romania
    Singapore
    Spain
    Sweden
    Switzerland
    Taiwan
    Turkey
    Ukraine
    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
    LVLS
    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 months0
    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 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: 110
    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: 35
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 75
    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 age of 6 years
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 110
    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
    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 Decision2015-11-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-05
    P. End of Trial
    P.End of Trial StatusOngoing
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