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    Summary
    EudraCT Number:2020-004301-31
    Sponsor's Protocol Code Number:MLN0002-3025
    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:2021-09-01
    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 number2020-004301-31
    A.3Full title of the trial
    A Randomized, Double-Blind, Phase 3 Study to Evaluate the Efficacy and Safety of Vedolizumab Intravenous as Maintenance Therapy in Pediatric Subjects with Moderately to Severely Active Crohn’s Disease Who Achieved Clinical Response Following Open–Label Vedolizumab Intravenous Therapy
    Estudio de fase III, aleatorizado y doble ciego para evaluar la eficacia y la seguridad de vedolizumab intravenoso como tratamiento de mantenimiento en pacientes pediátricos con enfermedad de Crohn activa moderada a grave que lograron una respuesta clínica después del tratamiento intravenoso abierto con vedolizumab
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate efficacy and safety of Vedolizumab in pediatric subjects with moderately to severely active Crohn's disease
    Estudio para evaluar la eficacia y la seguridad de vedolizumab en pacientes
    pediátricos con enfermedad de Crohn moderada a grave
    A.4.1Sponsor's protocol code numberMLN0002-3025
    A.5.4Other Identifiers
    Name:INDNumber:009125
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/361/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorTakeda Development Center Americas, Inc.
    B.1.3.4CountryUnited States
    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 supportTakeda Development Center Americas, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Development Center Americas, Inc.
    B.5.2Functional name of contact pointDirector, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address40 Landsdowne Street
    B.5.3.2Town/ cityCambridge, Massachusetts
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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 ENTYVIO
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharma A/S
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameVedolizumab
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation Yes
    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 INNVedolizumab
    D.3.9.1CAS number 943609-66-3
    D.3.9.3Other descriptive nameMLN0002
    D.3.9.4EV Substance CodeSUB30452
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number100 to 300
    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
    Moderately to severely active Crohn’s disease (CD)
    Enfermedad de Crohn activa de moderada a grave (EC)
    E.1.1.1Medical condition in easily understood language
    Moderately to severely active Crohn’s disease. Crohn’s disease is a disease that causes inflammation of the digestive system.
    Enfermedad de Crohn activa de moderada a grave. La enfermedad de Crohn es una enfermedad que causa inflamación del sistema digestivo.
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10058815
    E.1.2Term Crohn's disease acute episode
    E.1.2System Organ Class 100000004856
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of 2 different dose regimens of vedolizumab IV in pediatric subjects with moderately to severely active CD during maintenance therapy, based on clinical and endoscopic assessments at Week 54.
    Evaluar la eficacia de 2 pautas posológicas diferentes de vedolizumab IV en pacientes pediátricos con EC activa de moderada a grave durante el tratamiento de mantenimiento, basándose en las valoraciones clínicas y endoscópicas de la semana 54.
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of high and low doses of vedolizumab IV as measured by clinical and endoscopic remission at Weeks 14 and 54.
    • To evaluate the effect of high and low doses of vedolizumab IV in each weight group on achieving corticosteroid-free remission at Week 54.
    • To evaluate the effect of vedolizumab IV in each weight group on clinical response and clinical remission over time up to Week 54.
    • To characterize the exposure of 2 different dose regimens of vedolizumab in pediatric subjects with moderately to severely active CD after IV administration.
    • To evaluate safety in pediatric subjects on maintenance therapy up to Week 54.
    • To evaluate the immunogenicity of vedolizumab in pediatric subjects with moderately to severely active CD treated with vedolizumab IV.
    • To evaluate the effect of vedolizumab on patterns of growth and pubertal development in pediatric subjects with moderately to severely active CD during their participation in the study.
    • Evaluar la eficacia de dosis altas y bajas de vedolizumab IV, determinada por la remisión clínica y endoscópica en las semanas 14 y 54
    • Evaluar el efecto de dosis altas y bajas de vedolizumab IV en cada grupo de peso sobre la consecución de remisión sin corticosteroides en la semana 54
    • Evaluar el efecto de vedolizumab IV en cada grupo de peso sobre la respuesta clínica y la remisión clínica a lo largo del tiempo hasta la semana 54
    • Caracterizar la exposición a 2 pautas diferentes de vedolizumab en pacientes pediátricos con EC activa de moderada a grave tras la administración IV
    • Evaluar la seguridad en pacientes pediátricos con tratamiento de mantenimiento hasta la semana 54
    • Evaluar la inmunogenicidad de vedolizumab en pacientes pediátricos con EC activa de moderada a grave tratados con vedolizumab IV
    • Evaluar el efecto de vedolizumab en los patrones de crecimiento y desarrollo puberal de pacientes pediátricos con EC activa de moderada a grave durante el estudio
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects male or female aged 2 to 17 years, inclusive, who weigh ≥10 kg at the time of screening and enrollment into the study.
    2. Subjects with moderately to severely active CD diagnosed at least 1 month before screening, defined by a PCDAI >30 and an SES-CD >6 (or an SES-CD ≥4 if disease is confined to terminal ileum).
    3. Subjects who have failed, lost response to, or been intolerant to treatment with at least 1 of the following agents: corticosteroids, immunomodulators (eg, AZA, 6-mercaptopurine, methotrexate), and/or TNF-α antagonist therapy (eg, infliximab, adalimumab). This includes subjects who are dependent on corticosteroids or exclusive or partial enteral nutrition to control symptoms and who are experiencing worsening of disease in the moderate-to-severe range when attempting to wean off corticosteroids or discontinue exclusive enteral nutrition.
    4. Subjects with extensive colitis or pancolitis of >8 years’ duration or left-sided colitis of >12 years’ duration must have documented evidence of a negative surveillance colonoscopy within 12 months before screening.
    5. Subjects with vaccinations that are up-to-date based on the countrywide accepted schedule of childhood vaccines.
    1. Pacientes de ambos sexos de 2 a 17 años de edad, ambos inclusive, con un peso ≥ 10 kg en el momento de la selección y la inclusión en el estudio.
    2. Pacientes con EC activa de moderada a grave diagnosticada al menos 1 mes antes de la selección, definida por un PCDAI > 30 y una SES-CD > 6 (o una SES-CD ≥ 4 si la enfermedad se limita al íleon terminal).
    3. Pacientes con fracaso, pérdida de respuesta o intolerancia al tratamiento con al menos 1 de los fármacos siguientes: corticosteroides, inmunomoduladores (p. ej., AZA, 6-mercaptopurina, metotrexato) y/o antagonistas del TNF-α (p. ej., infliximab, adalimumab). Esto incluye a los pacientes que dependen de corticosteroides o de la nutrición enteral exclusiva o parcial para controlar los síntomas y que experimentan empeoramiento de la enfermedad en el intervalo de moderado a grave cuando se intenta retirar los corticosteroides o interrumpir la nutrición enteral exclusiva.
    4. En los pacientes con colitis o pancolitis extensa de más de 8 años de duración o con colitis izquierda de más de 12 años de duración deberá haber pruebas documentadas de una colonoscopia de vigilancia negativa en los 12 meses previos a la selección.
    5. Pacientes con vacunación al día de acuerdo con el calendario nacional aceptado de vacunas infantiles.
    E.4Principal exclusion criteria
    1. Subjects who have had previous exposure to approved or investigational anti-integrins, including but not limited to natalizumab, efalizumab, etrolizumab, or AMG 181, or mucosal addressin cell adhesion molecule-1 (MAdCAM-1) antagonists or rituximab.
    2. Subjects who have had prior exposure to vedolizumab.
    3. Subjects with hypersensitivity or allergies to any of the vedolizumab excipients.
    4. Subjects who have received either (1) an investigational biologic (other than those listed in Exclusion Criterion #1) within 60 days or 5 half-lives before screening (whichever is longer); or (2) an approved biologic or biosimilar agent within 2 weeks before the first dose of study drug or at any time during the screening period.
    5. Subjects with active cerebral/meningeal disease, signs/symptoms or history of progressive multifocal leukoencephalopathy (PML) or any other major neurological disorders including stroke, multiple sclerosis, brain tumor or neurodegenerative disease.
    6. Subjects who currently require surgical intervention or are anticipated to require surgical intervention for CD during this study.
    7. Subjects who have had subtotal or total colectomy or have a jejunostomy, ileostomy, colostomy, ileo-anal pouch, known fixed stenosis of the intestine, short bowel syndrome, or >3 small intestine resections.
    8. Subjects with a current diagnosis of indeterminate colitis.
    9. Subjects with clinical features suggesting monogenic very early-onset inflammatory bowel disease.
    1. Pacientes con exposición previa a anti-integrinas aprobadas o en investigación, como natalizumab, efalizumab, etrolizumab o AMG 181, entre otras, o antagonistas de la molécula de citoadhesión adresina mucosal 1 (MAdCAM-1) o rituximab.
    2. Pacientes con exposición previa al vedolizumab.
    3. Pacientes con hipersensibilidad o alergia a cualquiera de los excipientes del vedolizumab.
    4. Pacientes que hayan recibido 1) un fármaco biológico en investigación (distinto de los citados en el criterio de exclusión nº 1) en los 60 días o 5 semividas previos a la selección (el plazo más largo), o 2) un fármaco biológico o biosimilar aprobado en las 2 semanas previas a la primera dosis de fármaco del estudio o en cualquier momento durante el período de selección.
    5. Pacientes con enfermedad cerebral/meníngea activa, signos/síntomas o antecedentes de leucoencefalopatía multifocal progresiva (LMP) o cualquier otro trastorno neurológico importante como ictus, esclerosis múltiple, tumor cerebral o enfermedad neurodegenerativa.
    6. Pacientes que precisen en ese momento intervención quirúrgica o que se espera que la necesiten para la EC durante este estudio.
    7. Pacientes sometidos a colectomía subtotal o total o que tengan una yeyunostomía, ileostomía, colostomía, reservorio ileoanal, estenosis fija conocida del intestino, síndrome del intestino corto o > 3 resecciones del intestino delgado.
    8. Pacientes con diagnóstico actual de colitis indeterminada.
    9. Pacientes con rasgos clínicos que sugieran enfermedad inflamatoria intestinal monogénica de comienzo muy temprano.
    E.5 End points
    E.5.1Primary end point(s)
    • Coprimary 1 (based on PCDAI): Clinical remission defined as a PCDAI ≤10 at Week 54.
    • Coprimary 2 (based on SES-CD): Endoscopic response at Week 54, where a subject achieves endoscopic response if he or she has a 50% reduction in SES-CD score from baseline.
    • Coprincipal 1 (basado en el PCDAI): Remisión clínica definida como un PCDAI ≤ 10 en la semana 54.
    • Coprincipal 2 (basado en la puntuación SES-CD): Respuesta endoscópica en la semana 54, cuando un paciente consigue respuesta endoscópica si muestra una reducción del 50% de la puntuación SES-CD respecto a la basal.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The end of study is defined as the Week 54 study visit.
    El final del estudio se define como la visita de la semana 54.
    E.5.2Secondary end point(s)
    • Clinical and endoscopic remission at Week 14, where a subject achieves both clinical and endoscopic remission if he or she meets the following definition: PCDAI ≤10 and SES-CD ≤4 with at least a 2-point reduction from baseline and no subscore >1.
    • Clinical and endoscopic remission at Week 54, where a subject achieves clinical and endoscopic remission if he or she meets the following definition: PCDAI ≤10 and SES-CD ≤4 with at least a 2-point reduction from baseline and no subscore >1.
    • Sustained clinical and endoscopic remission at Week 54, where a subject achieves sustained clinical and endoscopic remission if he or she achieved clinical and endoscopic remission (based on PCDAI and SES-CD) at Week 14 and at Week 54.
    • Corticosteroid-free remission at Week 54, where a subject achieves corticosteroid-free clinical remission based on PCDAI at Week 54 and he or she has been off corticosteroids at least 12 weeks prior to Week 54.
    • Sustained endoscopic remission, where a subject achieves sustained endoscopic remission if he or she meets the following definition: SES-CD ≤4 with at least a 2 point reduction from baseline and no subscore >1, achieved at both Weeks 14 and 54.
    • Sustained clinical remission at Week 54, where a subject achieves sustained clinical remission if he or she achieved clinical remission (based on PCDAI) at Week 14 and at Week 54.
    • Serum trough concentrations of vedolizumab over time.
    • Positive antivedolizumab antibodies and positive neutralizing antivedolizumab antibodies during the study.
    • Sustained clinical response of subjects at Weeks 14 and 54, where a subject meets clinical response of if he or she has a PCDAI score <30 and reduction of the PCDAI by ≥15 points from baseline.
    • Clinical remission at Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54 where a subject achieves clinical remission if he or she meets the following definition: PCDAI ≤10.
    • Clinical response at Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54 where a subject achieves clinical response if he or she meets the following definition: PCDAI <30 with reduction in the PCDAI of ≥15 points from baseline.
    • Safety assessments: Descriptions of adverse events (AEs); serious adverse events (SAEs) and AEs of special interest (AESIs), including evaluation of opportunistic infection, such as PML, and liver injury, malignancies, infusion-related reactions, and hypersensitivity.
    • Change from baseline in weight gain and linear growth z-score during the course of dosing with vedolizumab.
    • Tanner Stage V at Week 54, where a subject achieves Tanner Stage V at Week 54 based on progression of pubertal changes from baseline.
    • Remisión clínica y endoscópica en la semana 14, cuando un paciente consigue remisión clínica y endoscópica si cumple la definición siguiente: PCDAI ≤10 y SES-CD ≤4 con una reducción de al menos 2 puntos respecto al valor basal y ninguna subpuntuación >1.
    • Remisión clínica y endoscópica en la semana 54, cuando un paciente consigue remisión clínica y endoscópica si cumple la definición siguiente: PCDAI ≤10 y SES-CD ≤4 con una reducción de al menos 2 puntos respecto al valor basal y ninguna subpuntuación >1.
    • Remisión clínica y endoscópica mantenida en la semana 54, cuando un paciente consigue remisión clínica y endoscópica mantenida si ha logrado remisión clínica y endoscópica (según el PCDAI y la SES-CD) en las semanas 14 y 54.
    • Remisión sin corticoides en la semana 54, cuando un paciente consigue remisión clínica sin corticoides según el PCDAI en la semana 54 y ha estado sin corticosteroides al menos 12 semanas antes de la semana 54.
    • Remisión endoscópica mantenida, cuando un paciente consigue remisión endoscópica mantenida si cumple la definición siguiente: SES-CD ≤4 con una reducción de al menos 2 puntos respecto al valor basal y ninguna subpuntuación >1, lograda en las semanas 14 y 54.
    • Remisión clínica mantenida en la semana 54, cuando un paciente consigue remisión clínica mantenida si ha logrado remisión clínica (según el PCDAI) en las semanas 14 y 54.
    • Concentraciones séricas mínimas de vedolizumab a lo largo del tiempo.
    • Positividad de anticuerpos antivedolizumab y de anticuerpos antivedolizumab neutralizantes durante el estudio.
    • Respuesta clínica mantenida de los pacientes en las semanas 14 y 54, cuando un paciente consigue respuesta clínica si tiene una puntuación PCDAI <30 y una reducción de la puntuación PCDAI respecto a la basal ≥15 puntos.
    • Remisión clínica en las semanas 2, 6, 10, 14, 22, 30, 38, 46 y 54, cuando un paciente consigue remisión clínica si cumple la definición siguiente: Un PCDAI ≤10.
    • Respuesta clínica en las semanas 2, 6, 10, 14, 22, 30, 38, 46 y 54, cuando un paciente consigue respuesta clínica si cumple la definición siguiente: Un PCDAI <30 con una reducción del PCDAI respecto al basal ≥15 puntos.
    • Valoraciones de la seguridad: Descripciones de acontecimientos adversos (AA); acontecimientos adversos graves (AAG), y AA de interés especial (AAIE), incluida la evaluación de infecciones oportunistas, como LMP, y de lesión hepática, neoplasias malignas, reacciones relacionadas con la infusión e hipersensibilidad.
    • Cambio respecto al momento basal de la ganancia de peso y la puntuación z del crecimiento lineal durante la administración del vedolizumab.
    • Estadio V de Tanner en la semana 54, cuando un paciente alcanza el estadio V de Tanner en la semana 54 basándose en la progresión de los cambios puberales respecto al momento basal.
    E.5.2.1Timepoint(s) of evaluation of this end point
    LVLS
    Última visita del último paciente
    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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    Inmunogenicidad
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Diferentes pautas posológicas de medicación en estudio en los grupos de tratamiento
    Different doses of IMP are used in treatment arms
    E.8.2.4Number of treatment arms in the trial3
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA100
    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
    Bosnia and Herzegovina
    Canada
    China
    Croatia
    Czechia
    France
    Germany
    Greece
    Hungary
    Israel
    Italy
    Japan
    Lithuania
    New Zealand
    Poland
    Romania
    Russian Federation
    Slovakia
    Spain
    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
    The end of study is defined as the Week 54 study visit.
    El final del estudio se define como la visita de la semana 54.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days15
    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: 120
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 60
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2021-09-01. Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children & adolescents (2-17)
    Niños y adolescentes (2-17 años)
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Pediatric patients
    Pacientes pediátricos
    F.4 Planned number of subjects to be included
    F.4.1In the member state26
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 120
    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)
    Follow-up safety visit 18 weeks after the last dose of study drug and a final LTFU safety survey phone call every 6 months for 2 years after the last dose of study drug for all subjects who do not continue into the extension study and for subjects who discontinue study drug at any time during the study.
    Visita de seguridad de seguimiento 18 semanas después de la última dosis del fármaco del estudio y una encuesta de seguridad de seguimiento a largo plazo por teléfono cada 6 meses durante 2 años después de la última dosis del fármaco del estudio en los pacientes que no continúen en el estudio de extensión y los que suspendan el fármaco del estudio en cualquier momento durante el estudio.
    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 Decision2021-06-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-28
    P. End of Trial
    P.End of Trial StatusOngoing
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