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    Summary
    EudraCT Number:2019-003374-91
    Sponsor's Protocol Code Number:SRP-9001-301
    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:2021-10-20
    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 number2019-003374-91
    A.3Full title of the trial
    A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
    Estudio de fase III multinacional, aleatorizado, doble ciego, controlado con placebo, de liberación génica sistémica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos con distrofia muscular de Duchenne (EMBARK)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy
    Estudio para evaluar la seguridad y eficacia de SRP-9001 en sujetos con Distrofia Muscular de Duchenne
    A.4.1Sponsor's protocol code numberSRP-9001-301
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/419/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSarepta Therapeutics, 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 supportSarepta Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSarepta Therapeutics, Inc.
    B.5.2Functional name of contact pointSarepta Clinical Trial Inquiries
    B.5.3 Address:
    B.5.3.1Street Address215 First Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.6E-mailsareptally@sarepta.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2250
    D.3 Description of the IMP
    D.3.2Product code SRP-9001
    D.3.4Pharmaceutical form 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 INNNot available
    D.3.9.1CAS number 2305040-16-6
    D.3.9.2Current sponsor codeSRP-9001
    D.3.9.3Other descriptive nameADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE
    D.3.9.4EV Substance CodeSUB197789
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number13300000000000
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 No
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    Distrofia Muscular de Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy
    Distrofia Muscular de Duchenne
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10052655
    E.1.2Term Duchenne muscular dystrophy gene carrier
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of SRP-9001 on physical function as assessed by the North Star Ambulatory Assessment (NSAA) score
    Evaluar el efecto de SRP-9001 sobre la función física, valorado mediante la puntuación de la Prueba Ambulatoria North Star (North Star Ambulatory Assessment, NSAA)
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of SRP-9001 on physical function as assessed by:
    o Number of skills gained or improved on the NSAA
    • To evaluate micro-dystrophin expression from SRP-9001 at 12 weeks (Part 1) as measured by Western blot of biopsied muscle tissue
    • To evaluate the effect of SRP-9001 on timed function tests as assessed by measuring:
    o Time to rise from the floor
    o 100-meter walk/run (100MWR)
    o Time to ascend 4 steps
    o 10-meter walk/run (10MWR)
    • To evaluate the effect of SRP-9001 on stride velocity 95th centile (SV95C) as measured by a wearable device
    • To evaluate subject (parent/caregiver proxy) reported Mobility and Upper Extremity Function using the Patient Reported Outcomes Measurement Information System (PROMIS®) tool
    • To evaluate the safety of SRP-9001
    • Evaluar el efecto de SRP-9001 sobre la función física, valorado mediante:
    o Número de habilidades adquiridas o mejoradas en la NSAA
    • Evaluar la expresión de micro-distrofina como consecuencia de SRP-9001 a las 12 semanas (Parte 1), determinada mediante la prueba de western blot en tejido muscular biopsiado
    • Evaluar el efecto de SRP-9001 en las pruebas funcionales cronometradas, determinando:
    o Tiempo requerido para levantarse del suelo
    o Tiempo requerido para correr/andar 100 metros (100-meter walk/run, 100MWR)
    o Tiempo requerido para subir 4 escalones
    o Tiempo requerido para correr/andar 10 metros (10-meter walk/run, 10MWR)
    • Evaluar el efecto de SRP-9001 sobre el percentil 95 de la velocidad de la zancada (SV95C), medido con un dispositivo portable
    • Evaluar la movilidad y la función de las extremidades superiores comunicadas por el sujeto (o representante: progenitor/cuidador) con la herramienta PROMIS®
    • Evaluar la seguridad de SRP-9001.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Select sites will participate in the MRI sub-studies; all subjects at these sites will undergo cardiac and musculoskeletal imaging assessments at the time points indicated in Section 1.1. A cardiac MRI will be performed to evaluate cardiac function. A musculoskeletal MRI will be performed to evaluate the structure and function of skeletal muscles as well as lean body mass. Refer to the MRI Study Manual for further details.
    Los sitios seleccionados participaran en los sub-estudios MRI; todos los sujetos en estos sitios seran evaluados usando imagen musculoesquelética y cardíaca en los momentos indicados en la sección 1.1. Se realizará una MRI cardíaca para evaluar la función cardíaca. Se realizará una MRI musculoesquelética para evaluar la estructura y funcion de los músculos esqueléticos así como la masa magra corporal. Para más detalles, consulte el manual del studio MRI.
    E.3Principal inclusion criteria
    A subject must meet all of the following criteria to be eligible to participate in this study:
    1. Is male at birth, ambulatory, and ≥ 4 to < 8 years of age at the time of randomization.
    2. Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to absence of dystrophin protein.
    a. Mutations between or including exons 1-17 are not eligible.
    b. In-frame deletions, in-frame duplications, and variants of uncertain significance (“VUS”) are not eligible.
    c. Mutations fully contained within exon 45 (inclusive) are not eligible.
    3. Able to cooperate with motor assessment testing.
    4. Has a NSAA score > 16 and < 29 at the Screening visit.
    5. Has a time to rise from floor < 5 seconds at the Screening visit.
    6. Stable daily dose of oral corticosteroids for at least 12 weeks before Screening and the dose and regimen are expected to remain constant (except for modifications to accommodate changes in weight) throughout the study.
    7. Has rAAVrh74 antibody titers < 1:400 (ie, not elevated) as determined by an ELISA.
    8. Subjects who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive). Refer to Appendix 1 for guidance on highly effective contraceptive methods.
    9. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements.
    10. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide informed consent for the subject to participate in the study.
    Para ser elegibles para participar en el estudio, los sujetos deberán cumplir todos los criterios siguientes:
    1. Sujeto varón en el momento de su nacimiento, con ≥ 4 a < 8 años de edad en el momento de la aleatorización.
    2. Diagnóstico definitivo de DMD previamente a la selección, basado en hallazgos clínicos documentados y confirmación genética previa con una prueba genética de diagnóstico clínico. El informe genético deberá describir una deleción frameshift, duplicación frameshift, interrupción prematura ("nonsense"), mutación en el lugar del empalme (splice site mutation) clásica u otra variante patogénica del gen DMD contenida en su totalidad entre los exones 18 y 79 (ambos incluidos) que se espere que provoque una ausencia de la proteína distrofina. Son elegibles las duplicaciones «frameshift» entre los exones 1-17 (inclusive).
    a. No son elegibles las mutaciones entre o que incluyan los exones 1-17.
    b. Las deleciones y las duplicaciones «in-frame», así como las variantes de significado incierto (variants of uncertain significance, “VUS”), no son elegibles.
    c. Las mutaciones contenidas en su totalidad dentro del exón 45 (inclusive) no son elegibles.
    3. Sujeto capaz de cooperar en la prueba de evaluación motora.
    4. Puntuación NSAA > 16 y < 29 en la visita de selección.
    5. Tiempo requerido para levantarse del suelo <5 segundos en la visita de selección.
    6. Dosis diaria de corticoides estable durante las 12 semanas anteriores a la selección, como mínimo; deberá preverse que la dosis y la pauta se mantengan constantes (a excepción de las modificaciones necesarias para adaptarse a los cambios de peso) durante todo el estudio.
    7. Título de anticuerpos frente a rAAVrh74 <1:400 (es decir, no elevados) determinados mediante ELISA.
    8. Los sujetos sexualmente activos deberán estar de acuerdo en utilizar preservativos durante toda la duración del estudio, y la pareja sexual femenina deberá usar, adicionalmente, un método anticonceptivo médicamente aceptable (p. ej., anticonceptivos orales). Para obtener información sobre los métodos anticonceptivos de elevada eficacia, consúltese el Apéndice 1 .
    9. Los padres (o uno de los progenitores) o el tutor legal del sujeto tendrán que poder entender y cumplir el calendario de visitas del estudio y todo el resto de requisitos del protocolo.
    10. El sujeto desea proporcionar el asentimiento informado (si procede) y los padres (o uno de los progenitores) o el tutor legal del paciente están de acuerdo en otorgar su consentimiento informado para que el sujeto participe en el estudio.
    E.4Principal exclusion criteria
    A subject who meets any of the following criteria will be excluded from this study:
    1. Has left ventricular ejection fraction < 40% on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy.
    2. Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study for any time during this study.
    3. Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the subject’s ability to comply with the protocol required testing or procedures or compromise the subject’s wellbeing, safety, or clinical interpretability.
    4. Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
    5. Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
    6. Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the Investigator.
    7. Treatment with any of the following therapies according to the time frames specified:
    • Any time:
    − Gene therapy
    − Cell based therapy (eg, stem cell transplantation)
    − CRISPR/Cas9, or any other form of gene editing
    • Within 12 weeks of Day 1 and any time during the study:
    − Use of human growth factor or vamorolone
    • Within 6 months of Day 1 and any time during the study:
    − Any investigational medication
    − Any treatment designed to increase dystrophin expression (eg, Translarna™, EXONDYS 51™, VILTEPSO™)
    8. Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.
    9. Has abnormal laboratory values considered clinically significant including but not limited to:
    • Gamma-glutamyl transferase > 2× upper limit of normal (ULN)
    • Glutamate dehydrogenase (GLDH) > 15 U/L
    • Total bilirubin > ULN. Note; elevations in total bilirubin confirmed to be due to Gilbert’s syndrome are not exclusionary.
    • White blood cell count > 18,500 per µl
    • Platelets ≤ 150,000 per µL
    10. Family does not want to disclose subject’s study participation with general practitioner/primary care physician and other medical providers.
    11. In the opinion of the Investigator, the subject is not likely to be compliant with the study protocol.
    El sujeto quedará excluido del estudio si cumple cualquiera de los siguientes criterios:
    1. Fracción de eyección del ventrículo izquierdo <40% en el ECHO de la selección, o bien signos clínicos y/o síntomas de miocardiopatía.
    2. Cirugía mayor practicada dentro de los 3 meses previos al Día 1 o cirugía o procedimientos previstos que interferirían con la realización del estudio durante cualquier período de tiempo del mismo.
    3. Presencia de cualquier otra enfermedad clínicamente relevante, como patologías cardiacas, pulmonares, hepáticas, renales, hematológicas o inmunológicas, trastornos del comportamiento, infecciones, neoplasias malignas, enfermedades concomitantes, necesidad de tratamiento farmacológico crónico que, en opinión del investigador, suponga riesgos innecesarios para la transferencia génica, o bien enfermedades o circunstancias debilitantes que, según el criterio del investigador, puedan comprometer la capacidad del sujeto para cumplir con las pruebas o procedimientos requeridos por el protocolo o que afecten al bienestar o la seguridad del sujeto o a la interpretación clínica.
    4. Sujetos con pruebas serológicas que indiquen una infección actual, crónica o activa por el virus de la inmunodeficiencia humana, la hepatitis C o la hepatitis B.
    5. Infección sintomática (p. ej., infección de las vías respiratorias altas, neumonía, pielonefritis, meningitis) dentro de las 4 semanas previas al Día 1.
    6. Sujeto que muestra un retraso cognitivo o una afectación que, en opinión del investigador, podría provocar confusión respecto al desarrollo motor.
    7. Tratamiento con cualquiera de lo siguiente, de acuerdo con los marcos temporales que se especifican:
    • Cualquier período de tiempo:
    - Terapia génica
    - Terapia basada en células (p. ej.,, trasplante de progenitores hematopoyéticos)
    - CRISPR/Cas9, o cualquier otra forma de edición génica
    • Dentro de las 12 semanas previas al Día 1 y en cualquier momento durante el estudio:
    - Uso de factores de crecimiento humanos o de vamorolona
    • Dentro de los 6 meses previos al Día 1 y en cualquier momento durante el estudio:
    - Cualquier medicamento en investigación
    - Cualquier tratamiento diseñado para incrementar la expresión de distrofina (p. ej., Translarna™, EXONDYS 51™, VILTEPSO™)
    8. Sujetos que hayan recibido una vacuna con virus atenuados dentro de las 4 semanas previas, o bien vacunas con microorganismos inactivados dentro de las 2 semanas previas a la visita del Día 1, o que se prevea su vacunación dentro de los primeros 3 meses después del Día 1.
    9. Pacientes con anomalías de laboratorio que se consideren clínicamente relevantes, entre otras:
    • Gamma-glutamil transferasa >2 veces el límite superior de la normalidad (upper limit of normal, ULNULN)
    • Glutamato deshidrogenasa (GLDH) > 15 U/l
    • Bilirrubina total >ULN. Nota: no serán motivo de exclusión las elevaciones de la bilirrubina total que se confirmen debidas al síndrome de Gilbert .
    • Recuento de leucocitos >18.500 por µl
    • Recuento de plaquetas ≤150.000 por µl
    10. La familia no desea comunicar al médico de familia/de cabecera la participación del sujeto en el estudio, o a otros profesionales médicos.
    11. En opinión del investigador, no es probable que el sujeto cumpla con el protocolo del estudio
    E.5 End points
    E.5.1Primary end point(s)
    • Change in NSAA total score from Baseline to Week 52 (Part 1)
    • Cambio en la puntuación total de la NSAA desde la situación basal hasta la semana 52 (Parte 1)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline to Week 52 (Part 1)
    Desde la situación basal hasta la semana 52 (Parte 1)
    E.5.2Secondary end point(s)
    • Number of skills gained or improved at Week 52 (Part 1) as measured by the NSAA
    • Quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by Western Blot
    • Change in time to rise from the floor from Baseline to Week 52 (Part 1)
    • Change in time of 100MWR from Baseline to Week 52 (Part 1)
    • Change in time to ascend 4 steps from Baseline to Week 52 (Part 1)
    • Change in time of 10MWR from Baseline to Week 52 (Part 1)
    • Change in SV95C from Baseline to Week 52 (Part 1)
    • Change in PROMIS score per domain from Baseline over 52 weeks (Part 1)
    • Incidence of treatment-emergent adverse events
    • Incidence of serious adverse events
    • Incidence of adverse events of special interest
    • Clinically significant changes in vital signs and physical examination findings
    • Clinically significant changes in safety laboratory assessments, electrocardiograms (ECGs), and echocardiograms (ECHOs)
    • Número de habilidades adquiridas o mejoradas en la semana 52 (Parte 1), valoradas por la NSAA
    • Cantidad de la proteína micro-distrofina expresada en la semana 12 (Parte 1), determinada mediante la prueba de western blot.
    • Cambio en el tiempo requerido para levantarse del suelo desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el tiempo requerido para correr/andar 100 metros desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el tiempo requerido para subir 4 escalones desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el tiempo requerido para correr/andar 10 metros desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el SV95C desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en la puntuación PROMIS, por dominios, desde la situación basal hasta la semana 52 (Parte 1).
    • Incidencia de acontecimientos adversos aparecidos durante el tratamiento.
    • Incidencia de acontecimientos adversos graves.
    • Incidencia de los acontecimientos adversos de especial interés
    • Cambios clínicamente relevantes en las constantes vitales y los hallazgos de la exploración física.
    • Cambios clínicamente relevantes en las determinaciones de laboratorio de seguridad, el electrocardiograma (ECG) y el ecocardiograma (echocardiograms, ECHOs).
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Number of skills gained or improved at Week 52 (Part 1) as measured by the NSAA
    • Quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by Western Blot
    • Change in time to rise from the floor from Baseline to Week 52 (Part 1)
    • Change in time of 100MWR from Baseline to Week 52 (Part 1)
    • Change in time to ascend 4 steps from Baseline to Week 52 (Part 1)
    • Change in time of 10MWR from Baseline to Week 52 (Part 1)
    • Change in SV95C from Baseline to Week 52 (Part 1)
    • Change in PROMIS score per domain from Baseline over 52 weeks (Part 1)
    • For other secondary endpoints, full duration of the study
    • Número de habilidades adquiridas o mejoradas en la semana 52 (Parte 1), valoradas por la NSAA
    • Cantidad de la proteína micro-distrofina expresada en la semana 12 (Parte 1), determinada por western blot.
    • Cambio en el tiempo requerido para levantarse del suelo de la situación basal a la semana 52 (Parte 1).
    • Cambio en el tiempo requerido para correr/andar 100 metros desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el tiempo requerido para subir 4 escalones desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el tiempo requerido para correr/andar 10 metros desde la situación basal hasta la semana 52 (Parte 1).
    • Cambio en el SV95C desde la situación basal hasta la semana 52 (Parte 1).
    • El protocolo contiene el resto de objetivos secundarios.
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 Yes
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Hong Kong
    Japan
    Taiwan
    United States
    Belgium
    France
    Germany
    Italy
    Spain
    United Kingdom
    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 Patient Last Visit
    Ultimo Paciente Ultima Visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days17
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months1
    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: 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: 120
    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 No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Patients ≥ 4 to < 8 years of age at the time of randomization
    Sujeto con ≥ 4 a < 8 años de edad en el momento de la aleatorización.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 53
    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)
    After completion of the Part 2 Week 52 assessments, subjects could be enrolled into an extension study to assess long-term safety and efficacy.
    Una vez completadas las evaluaciones de la Semana 52 de la Parte 2, los sujetos podrán ser elegibles para su inclusión en un estudio de extensión a fin de evaluar la seguridad y la eficacia a largo plazo.
    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 Decision2022-02-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-09
    P. End of Trial
    P.End of Trial StatusOngoing
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