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    EudraCT Number:2017-000858-18
    Sponsor's Protocol Code Number:281102
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2017-09-18
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-000858-18
    A.3Full title of the trial
    A phase 3, prospective, randomized, controlled, open-label, multicenter, 2-period crossover study with a single arm continuation evaluating the safety and efficacy of BAX 930 (rADAMTS13) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic thrombocytopenic purpura [hTTP])
    “Estudio cruzado en fase III, abierto, prospectivo, aleatorizado, controlado, multicéntrico y de 2 periodos con la continuación de un solo grupo para evaluar la seguridad y la eficacia de BAX 930 (rADAMTS13) en el tratamiento profiláctico y a demanda de sujetos con púrpura trombocitopénica trombótica congénita grave (PTTc, síndrome de Upshaw-Schulman [SUS], púrpura trombocitopénica trombótica hereditaria [PTTh])”
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3, randomized, controlled study of severe congenital thrombotic thrombocytopenic purpura, with Bax 930.
    Estudio en fase III, aleatorizado, controlado de púrpura trombocitopénica trombótica congénita con BAX 930
    A.3.2Name or abbreviated title of the trial where available
    A phase 3, randomized, controlled study of prophylactic and on-demand treatment of cTTP with BAX 930
    Estudio en fase III, aleatorizado, controlado de tto. profiláctico y bajo demanda de PTTc
    A.4.1Sponsor's protocol code number281102
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/048/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxalta Innovations GmbH
    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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta Innovation GmbH
    B.5.2Functional name of contact pointFrank Stout
    B.5.3 Address:
    B.5.3.1Street Address650 East Kenadall Street,
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02191
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34665 850140
    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/3/08/588
    D.3 Description of the IMP
    D.3.1Product nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
    D.3.2Product code BAX930 or SHP655
    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 INNapadamtase alfa
    D.3.9.2Current sponsor codeSHP655 (BAX930)
    D.3.9.3Other descriptive nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs
    D.3.9.4EV Substance CodeSUB183727
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1500
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    severe congenital thrombotic thrombocytopenic purpura
    (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic
    thrombocytopenic purpura [hTTP])
    Púrpura trombocitopénica trombótica congénita grave (PTTc, Síndrome Upshaw-Schulman, púrpura trombocitopénica trombótica hereditaria [PTTh])
    E.1.1.1Medical condition in easily understood language
    severe congenital thrombotic thrombocytopenic purpura
    Púrpura trombocitopénica trombótica congénita grave
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10043562
    E.1.2Term Thrombocytopenic purpura, thrombotic
    E.1.2System Organ Class 100000013328
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To determine the incidence of acute TTP episodes in subjects with severe cTTP receiving either standard of care (SoC) or BAX 930 as a prophylactic treatment
    1. Determinar la incidencia de episodios agudos de PTT en sujetos con PTTc que reciben el tratamiento
    estándar de atención o BAX 930 como tratamiento profiláctico.
    E.2.2Secondary objectives of the trial
    1. To evaluate the efficacy of BAX 930 in the treatment of acute TTP episodes as measured by the 1) number of acute TTP events responding to treatment and 2) time to resolution in both the prophylactic and the on-demand cohorts
    2. To evaluate the safety and tolerability of BAX 930 in terms of related adverse events (AEs) and serious adverse events (SAEs) in both the prophylactic and the on-demand cohorts
    3. To evaluate the pharmacokinetics (PK) of ADAMTS13 for up to 288 hours post-infusion in each treatment arm (BAX 930 and SoC) in the prophylactic cohort
    4. To evaluate the incidence of isolated TTP manifestations including thrombocytopenia,microangiopathic hemolytic anemia, renal dysfunction, neurologic signs and symptoms, and
    abdominal pain in the prophylactic cohort
    5. To assess the immunogenicity of BAX 930 as measured by the incidence of binding and inhibitory antibodies to ADAMTS13 in both the prophylactic and the on-demand cohorts
    1.Evaluar la eficacia de BAX 930 en el tratamiento de episodios agudos de PTT medida por 1) el número de acontecimientos agudos de PTT que responden al tratamiento y 2) el tiempo hasta la resolución en las cohortes profiláctica y a demanda.
    2.Evaluar la seguridad y tolerabilidad de BAX 930 en términos de acontecimientos adversos y acontecimientos adversos graves relacionados en las cohortes profilácticas y a demanda.
    3.Evaluar la farmacocinética de ADAMTS13 durante un máximo de 288 horas después de la infusión en cada grupo de tratamiento (BAX 930 y tratamiento estándar de atención) en la cohorte profiláctica.
    4.Evaluar la incidencia de manifestaciones aisladas de PTT, incluidas trombocitopenia, anemia hemolítica microangiopática, disfunción renal, signos y síntomas neurológicos y dolor abdominal en la cohorte profiláctica.
    5.Valorar la inmunogenia de BAX 930 medida por la incidencia de anticuerpos de unión e inhibidores para ADAMTS13 en las cohortes profiláctica y a demanda.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (signed by legal representative if subject is <18 years of age).
    2. Subject is 0 to 70 years of age, inclusive, at the time of screening. (Subjects <18 years of age will be enrolled only after at least 10 exposures with BAX 930 have been completed in at least 5 subjects over 18 years of age and reviewed by the study medical director).
    3. Subject has a documented diagnosis of severe hereditary ADAMTS13 deficiency, defined as:
    · Confirmed by molecular genetic testing, documented in subject history or at screening, and
    · ADAMTS13 activity <10% as measured by the FRETS-VWF73 assay, documented in subject history or at screening (subjects currently receiving SoC prophylactic therapy may exceed 10% ADAMTS13 activity at screening).
    Note: Subjects currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion
    4. Subject does not display any severe TTP symptoms signs (platelet count <100,000/µL and elevation of LDH >2×ULN) at screening. Subjects presenting with minor, but stable laboratory abnormalities at the time of screening may be enrolled (prophylactic cohort only).
    5. Subject is currently on a prophylactic dosing regimen or has a documented history of at least 1 TTP event and an ability to tolerate SoC prophylactic dosing (prophylactic cohort only).
    6. Subjects ≥16 years of age must have a Karnofsky score ≥70% and subjects <16 years of age must have a Lansky score ≥80%.
    7. Subject is hepatitis C virus (HCV)-negative as confirmed by antibody or polymerase chain reaction testing OR HCV-positive if their disease is chronic but stable.
    8. If female of childbearing potential, subject presents with a negative blood pregnancy test and agrees to employ adequate birth control measures for the duration of the study and to undergo quarterly pregnancy testing.
    9. Subject is willing and able to comply with the requirements of the protocol.
    1.Sujetos o representantes legalmente autorizados que han proporcionado un formulario de consentimiento informado firmado (≥18 años de edad) o un formulario de asentimiento (firmado por el representante legal si el sujeto tiene <18 años de edad).
    2.Sujetos que tienen entre 0 y 70 años de edad, inclusive, en el momento de la selección. (Los sujetos de <18 años de edad se inscribirán solo después de que se hayan completado 10 exposiciones con BAX 930 en al menos 5 sujetos mayores de 18 años de edad y cuyos resultados hayan sido revisados por el CVD).
    3.Sujetos que presentan un diagnóstico documentado de una deficiencia hereditaria grave de ADAMTS13, definida como:
    •confirmación por una prueba genética molecular, documentada en los antecedentes médicos o en la selección del sujeto, y
    •actividad de ADAMTS13 <10 % medida por la prueba FRET-FVW73, documentada en los antecedentes médicos o en la selección del sujeto (los sujetos que reciban en ese momento un tratamiento profiláctico del tratamiento estándar de atenciónpueden sobrepasar la actividad de ADAMTS13 en un 10 % durante la selección).
    Nota: Los sujetos que reciban en ese momento un tratamiento profiláctico se seleccionarán inmediatamente antes de su infusión profiláctica habitual.
    4.Sujetos que no presentan ningún signo grave de PTT (recuento de plaquetas <100 000 µl y aumento de la LDH >2 LSN) en la selección (solo en la cohorte profiláctica).
    5.Sujetos que reciben actualmente una pauta posológica profiláctica o cuentan con unos antecedentes médicos documentados de al menos 1 acontecimiento de PTT y la incapacidad de tolerar una dosis profiláctica del tratamiento estándar de atención (solo en la cohorte profiláctica).
    6.Sujetos de ≥16 años de edad que deben tener una puntuación de Karnofsky ≥70 % y sujetos de <16 años de edad que deben presentar una puntuación de Lansky ≥80 %.
    7.Sujetos no infectados por el virus de la hepatitis C (VHC) mediante determinación de anticuerpos o por reacción en cadena de la polimerasa O infectados por el VHC con hepatitis crónica estable.
    8.En el caso de mujeres en edad fértil, deben presentar una prueba de embarazo en sangre negativa y acceder a emplear medidas de control de la natalidad adecuadas durante todo el estudio, además de someterse a pruebas de embarazo trimestralmente.
    9.Sujetos que están dispuestos y son capaces de cumplir con los requisitos del protocolo.
    E.4Principal exclusion criteria
    1. Subject has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including acquired TTP.
    2. Subject has known hypersensitivity to hamster proteins.
    3. Subject has experienced an acute TTP episode less than 30 days prior to screening (prophylactic cohort only).
    4. Subject has a medical history or presence of a functional ADAMTS13 inhibitor at screening.
    5. Subject has a medical history of a genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including subjects who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count <200/mm3 or who are
    receiving chronic immunosuppressive drugs.
    6. Subject has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4).
    7. Subject with end stage renal disease requiring chronic dialysis.
    8. Subject has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following:
    a. Serum alanine aminotransferase (ALT) ≥2xULN
    b. International normalized ratio >1.5
    c. Severe hypoalbuminemia <24 g/L
    d. Portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices)
    9. In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
    10. Subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g., ointments, nasal sprays), within 30 days prior to enrollment. Use of corticosteroids in conjunction with administration of FFP to prevent allergic manifestations is permitted.
    11. Subject has an acute illness (e.g., influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylaxis cohort only).
    12. Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
    13. Subject has a history of drug and/or alcohol abuse within the last 2 years.
    14. Subject has a progressive fatal disease and/or life expectancy of less than 3 months.
    15. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
    16. Subject suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
    17. Subject is a family member or employee of the sponsor or investigator.
    18. If female, subject is pregnant or lactating at the time of enrolment.
    1.Sujetos a los que se les ha diagnosticado otro trastorno semejante a la PTT (anemia hemolítica microangiopática), incluida la PTT adquirida.
    2.Sujetos que tienen una hipersensibilidad conocida a las proteínas de hámster.
    3.Sujetos que han experimentado un episodio agudo de PTT con una diferencia de menos de 30 días antes de la selección (solo en la cohorte profiláctica).
    4.Sujetos que presentan antecedentes médicos o la presencia de un inhibidor funcional de ADAMTS13 en la selección.
    5.Sujetos que cuentan con antecedentes médicos de una deficiencia inmune genética o adquirida que podría interferir en la evaluación de la inmunogenia del producto, incluidos los sujetos con un resultado positivo para el virus de inmunodeficiencia humana (VIH) con un recuento de grupo de diferenciación absoluto 4 (CD4) <200/mm3 o que reciben inmunodepresores crónicos.
    6.Sujetos a los que se les ha diagnosticado una enfermedad cardiovascular grave (clases 3 a 4 según la clasificación de la Asociación de Cardiología de Nueva York [New York Heart Association]).
    7.Sujetos que presentan una enfermedad renal en estado terminal que requiere diálisis crónica.
    8.Sujetos a los que se les ha diagnosticado disfunción hepática, que se ha podido demostrar, entre otros, con los siguientes:
    a.Alanina aminotransferasa sérica (ALT) ≥2 x LSN
    b.Índice internacional normalizado >1,5
    c.Hipoalbuminemia grave <24 g/l
    d.Hipertensión de la vena porta (p. ej., presencia de esplenomegalia sin otra explicación, antecedentes de varices esofágicas)
    9.A juicio del investigador, el sujeto tiene otra enfermedad concomitante clínicamente significativa que puede suponer riesgos adicionales para el sujeto.
    10.Sujetos que han recibido tratamiento con un fármaco inmunomodulador, excluidos los tratamientos tópicos (p. ej., pomadas o nebulizadores nasales) en los 30 días anteriores a la inscripción. Se permite el uso de corticoesteroides junto con la administración de PFC para prevenir las reacciones alérgicas.
    11.Sujetos que presentan una enfermedad aguda (p. ej., gripe, síndrome gripal, rinitis alérgica/conjuntivitis, asma bronquial...) en el momento de la selección (solo en la cohorte de profilaxis).
    12.Sujetos que reciben o prevén recibir otro fármaco en investigación o un fármaco intervencionista en los 30 días previos a la inscripción.
    13.Sujetos que presentan antecedentes de drogadicción o alcoholismo en los últimos 2 años.
    14.Sujetos que presentan una enfermedad mortal progresiva o esperanza de vida inferior a los 3 meses.
    15.Determinación por el investigador de que el sujeto no es capaz de colaborar con los procedimientos del estudio o no está dispuesto a hacerlo.
    16.Sujetos con una enfermedad mental que les incapacita para entender la naturaleza, el alcance y las posibles consecuencias del estudio, o bien con indicios de una actitud poco colaboradora.
    17.Sujetos que son familiares o empleados del promotor o el investigador.
    18.En el caso de los sujetos de sexo femenino, aquellas mujeres que estén embarazadas o en periodo de lactancia en el momento de la inscripción.
    E.5 End points
    E.5.1Primary end point(s)
    1.Incidence of acute TTP episodes among subjects receiving either BAX 930 or SoC prophylactically during the corresponding treatment periods
    1. Incidencia de episodios agudos de PTT entre sujetos que recibieron BAX 930 o SoC profilácticamente durante los períodos de tratamiento correspondientes
    E.5.1.1Timepoint(s) of evaluation of this end point
    After first 24 subjects completed the 2x2 crossover phase of the study (interim analysis) and at the end of the study
    Después de que los primeros 24 sujetos completaron la fase de cruce 2x2 del estudio (análisis intermedio) y al final del estudio
    E.5.2Secondary end point(s)
    1. Number and incidence of acute TTP episodes responding to BAX 930, defined as not requiring the use of another ADAMTS13 containing agent
    2. Time to resolution of clinical symptomatology, if present, and normalization of laboratory parameters (platelet count ≥150,000/µL; LDH ≤1.5×ULN) following initiation of treatment in acute TTP episodes with BAX 930 or SoC agent
    3. Incidence of thrombocytopenia defined as a drop in platelet count ≥25% of baseline or a platelet count <150,000/µL
    4. Incidence of microangiopathic hemolytic anemia defined as an elevation of LDH >1.5×ULN
    5. Incidence of neurological symptoms (e.g., confusion, dysphonia, dysarthria, focal or general motor symptoms including seizures)
    6. Incidence of renal dysfunction defined as an increase in serum creatinine >1.5×baseline
    7. Incidence of abdominal pain
    8. Incidence of supplemental doses prompted by subacute manifestations
    9. Incidence of dose modification not prompted by an acute event
    10. Incidence of acute TTP episodes while subjects are on their final dose and dosing regimen
    1. Incidence of product-related and unrelated AEs and SAEs during each treatment period
    2. Incidence of binding and inhibitory antibodies to ADAMTS13
    3. Clinically relevant changes in vital signs, clinical chemistry, and hematology
    4. Estimated total quantity of ADAMTS13 administered during the treatment of acute events
    1. Assessment of the PK parameters (incremental recovery [IR], area under the plasma curve [AUC], terminal half-life [t1/2], mean residence time [MRT], systemic clearance [CL], steady state volume of distribution [Vss], and maximum concentration following infusion [Cmax]) for both the SoC agent and BAX 930 at the beginning of the respective treatment periods with each agent and for BAX 930 at the conclusion of Treatment Period 2 in the prophylactic cohort
    2. Assessment of VWF:Ag, VWF:RCo, and VWF multimer pattern at baseline and following infusion of the SoC agent and BAX 930 treatment during the initial PK assessment
    3. Assessment of ADAMTS13 activity (trough levels) and VWF parameters prior to each infusion of SoC or BAX 930 and at the time of acute event presentation
    1.Número e incidencia de los episodios agudos de PTT como respuesta a BAX 930, definidos por no requerir el uso de otro fármaco que contenga ADAMTS13.
    2.Tiempo hasta la resolución de la sintomatología, si existe, y la normalización clínica de los parámetros analíticos (recuento de plaquetas ≥150.000/µl; LDH ≤1,5 × LSN) posteriores al inicio del tratamiento en episodios agudos de PTT con BAX 930 o el fármaco del tratamiento estándar de atención.
    3.Incidencia de trombocitopenia definida por un descenso en el recuento de plaquetas ≥25 % con respecto al inicio o un recuento de plaquetas <150 000/µl.
    4.Incidencia de anemia hemolítica microangiopática definida como un aumento de la LDH >1,5 × LSN.
    5.Incidencia de síntomas neurológicos (p. ej., confusión, disfonía, disartria, síntomas motores locales o generales incluidas las convulsiones).
    6.Incidencia de disfunción renal definida como un aumento en la creatinina sérica >1,5 con respecto al inicio.
    7.Incidencia de dolor abdominal.
    8.Incidencia de dosis complementarias causadas por manifestaciones subagudas.
    9.Incidencia de la modificación de dosis no causada por un acontecimiento agudo.
    10.Incidencia de episodios agudos de PTT mientras los sujetos se encuentran en su última dosis y pauta posológica.
    1.Aparición de AA y AAG relacionados y no relacionados con el producto durante cada periodo del tratamiento.
    2.Aparición de anticuerpos de unión e inhibidores para ADAMTS13.
    3.Cambios relevantes clínicamente en constantes vitales, bioquímica clínica y hematología.
    4.Cantidad total estimada de ADAMTS13 administrada durante el tratamiento de acontecimientos agudos.
    1.Evaluación de parámetros FC (recuperación incremental [RI], área bajo la curva plasmática [ABC], semivida terminal [t1/2], tiempo medio de permanencia [TMP], aclaramiento sistémico [CL], volumen de distribución en estado de equilibrio [Vee] y concentración máxima tras la infusión [Cmáx]) tanto para el fármaco del tratamiento estándar de atención y BAX 930 al inicio de los respectivos periodos de tratamiento con cada fármaco y para BAX 930 al término del periodo de tratamiento 2 en la cohorte profiláctica.
    2.Evaluación del FVW: Ag, FVW:CoR y el patrón de multímetros del FVW al inicio y después de la infusión del fármaco del tratamiento estándar de atención y el tratamiento con BAX 930 durante la evaluación FC inicial.
    3.Evaluación de la actividad de ADAMTS13 (niveles de ADAMTS13 antes de la infusión) y de los parámetros de FVW antes de cada infusión del tratamiento estándar de atención o BAX 930 y en el momento de aparición de un acontecimiento agudo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Eifficacy analysis as above; final analysis at study end when all efficacy, safety and PK data are available
    Análisis de eficacia como el anterior; Análisis final al final del estudio cuando todos los datos de eficacia, seguridad y farmacocinética están disponibles
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA18
    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
    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
    last subject last visit
    último sujeto última visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 9
    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. of subjects for this age range: 1
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F. of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation 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 36
    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)
    Standard treatment
    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 Decision2017-11-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-10-31
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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