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    EudraCT Number:2018-001762-42
    Sponsor's Protocol Code Number:4658-402
    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:2019-08-09
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2018-001762-42
    A.3Full title of the trial
    A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular
    Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
    Estudio comparativo de búsqueda de dosis,
    aleatorizado, doble ciego para evaluar la seguridad y la
    eficacia de una dosis alta de eteplirsen, precedido por un
    aumento de dosis abierto, en pacientes con distrofia
    muscular de Duchenne con supresión de mutaciones
    susceptible a la omisión del exón 51
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy
    Un estudio de investigación para comparar diferentes dosis de un nuevo medicamento en investigación para el tratamiento de ciertos pacientes con distrofia muscular de Duchenne
    A.4.1Sponsor's protocol code number4658-402
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of 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 organisationSyneos Health
    B.5.2Functional name of contact pointCarolyn Mills
    B.5.3 Address:
    B.5.3.1Street Address4 The Fleming Buildings, Edinburgh Technopole Milton Bridge
    B.5.3.2Town/ cityEdinburgh
    B.5.3.3Post codeEH26 0BE
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441314481303
    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. name Exondys 51
    D. of the Marketing Authorisation holderSarepta Therapeutics, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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/3/08/586
    D.3 Description of the IMP
    D.3.1Product nameEteplirsen
    D.3.2Product code AVI-4658
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNETEPLIRSEN
    D.3.9.1CAS number 1173755-55-9
    D.3.9.2Current sponsor codeAVI-4658
    D.3.9.4EV Substance CodeSUB129287
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    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) 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 No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    muscular de Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy is a rare, fatal degenerative neuromuscular disease with an X-linked recessive inheritance caused by mutations in the dystrophin gene
    La distrofia muscular de Duchenne es una enfermedad neuromuscular degenerativa rara y fatal con una herencia recesiva ligada al X causada por mutaciones en el gen de la distrofina
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Open-label Dose Escalation
    Evaluate the safety and tolerability of weekly IV doses of 100 and 200 mg/kg of eteplirsen.

    Double-blind Dose Finding:
    Evaluate the PK, PD (exon skipping
    quantitation), safety and tolerability of doses higher than 30 mg/kg of eteplirsen, to select a single higher dose of eteplirsen for Dose Comparison

    Double-blind Dose Comparison:
    Comparison of a selected high dose of eteplirsen with 30 mg/kg IV weekly
    To investigate the effect of a selected high dose of eteplirsen (100 mg/kg or 200 mg/kg) as compared with 30 mg/kg, administered weekly IV, on motor function in ambulant Duchenne muscular dystrophy (DMD) patients with confirmed deletion genotypes amenable to exon 51 skipping
    Escala de dosis abierta
    Evaluar la seguridad y tolerabilidad de las dosis IV semanales de 100 y 200 mg/kg de eteplirsen.
    Hallazgo de dosis doble ciego:
    Evaluar la FC, FD (cuantificación de omisión del exón), seguridad y tolerabilidad de dosis superiores a 30 mg/kg de eteplirsen, a fin de seleccionar una dosis única más alta de eteplirsen para la comparación de dosis.
    Comparación de dosis de doble ciego:
    Investigar el efecto de la dosis alta seleccionada de eteplirsen (100 mg/kg o 200 mg/kg) en comparación con 30 mg/kg, administrados IV semanalmente, en la función motora de pacientes con distrofia muscular de Duchenne (DMD) ambulatoria con genotipos de supresión confirmados susceptibles a la omisión del exón 51.
    E.2.2Secondary objectives of the trial
    Double-blind Dose Comparison:

    Comparison of a selected high dose of eteplirsen with 30 mg/kg IV weekly on:
    -Ambulatory performance
    -Pulmonary function
    -Dystrophin expression
    To evaluate safety and tolerability of a selected high dose as compared with 30 mg/kg of eteplirsen, administered weekly IV
    Comparación de dosis de doble ciego:
    Evaluar el efecto de la dosis alta seleccionada en comparación con 30 mg/kg de eteplirsen, administrados IV semanalmente, sobre lo siguiente:
    o Desempeño ambulatorio
    o Función pulmonar
    o Expresión de distrofina
    Para evaluar la seguridad y la tolerabilidad de una dosis alta seleccionada en comparación con 30 mg / kg de eteplirsen, administrado semanalmente IV
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A patient must meet all of the following criteria to be eligible to participate in this study.
    1. Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping (for example, deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, and 52-63).
    2. Be aged 7 to 13 years, inclusive
    3. Have achieved a mean 6-minute walk test (6MWT) distance of ≥300 and ≤450 meters (without assistance) at both the screening and baseline visits for the double-blind.
    4. Have intact right and left biceps muscles (the preferred biopsy site) or an alternative upper arm muscle group that will allow for sufficiently sized (1 cm3) muscle biopsies to be obtained prior to and on treatment.
    5. Have been on a stable dose or dose equivalent of oral corticosteroids for at least 24 weeks prior to randomization, and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study.
    6. Have stable pulmonary function (forced vital capacity ≥50% of predicted and no requirement for nocturnal ventilation) that, in the Investigator’s opinion, is unlikely to decompensate significantly over the duration of the study.
    7. If sexually active, agree to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose. The sexual partner must also use a medically acceptable form of contraceptive (ie, female oral contraceptives) during this timeframe.
    8. Have (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
    9. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
    Un paciente debe cumplir todos los criterios siguientes para que se le considere apto para participar en el estudio.
    I 1. Ser un varón con un diagnóstico clínico de DMD establecido y una supresión de mutaciones fuera de marco del gen de DMD susceptible a la omisión del exón 51 (por ejemplo, supresión de los exones 45-50, 47-50, 48-50, 49-50, 50, 52 y 52-63).
    I 2. Tener entre 7 y 13 años, ambos incluidos.
    I 3. Haber alcanzado una distancia media en el test de marcha de 6 minutos (6MWT) ≥300 y ≤450 metros (sin asistencia) únicamente en la visita de selección para la parte de aumento de dosis abierto, y tanto en la visita de selección como en la de referencia para la parte doble ciego.
    I 4. Tener los músculos del bíceps derecho e izquierdo intactos (el sitio preferido para la biopsia) o un grupo de músculos del brazo superior alternativo que permita obtener biopsias musculares de tamaño suficiente (1 cm3) antes y durante el tratamiento.
    I 5. Haber estado recibiendo una dosis estable o equivalente de dosis de corticosteroides orales durante al menos 24 semanas antes de la asignación aleatoria, y con previsión de que la dosis permanezca constante (a excepción de las modificaciones para ajustar cambios de peso) a lo largo del estudio.
    I 6. Tener una función pulmonar estable (CVF ≥50 % de predicción y sin requisitos de ventilación nocturna) que, en opinión del investigador, es improbable que se descompense significativamente durante el estudio.
    I 7. Si es sexualmente activo, aceptar el uso de un condón masculino para dicha actividad mientras dure el estudio y durante 90 días después de la última dosis. La pareja sexual también debe usar una forma médicamente aceptable de anticonceptivo (es decir, anticonceptivos orales femeninos) durante este período de tiempo.
    I 8. Tener padres o tutores legales capaces de entender y cumplir todos los requisitos del estudio.
    I 9. Estar dispuesto a proporcionar el consentimiento informado (si corresponde) y tener padres o tutores legales dispuestos a proporcionar el consentimiento informado por escrito para que el paciente participe en el estudio.
    E.4Principal exclusion criteria
    A patient who meets any of the following criteria will be excluded from this study:
    1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization that may have an effect on muscle strength or function. Growth hormone for short stature and testosterone for delayed puberty are permitted if an endocrinologist has documented the diagnosis and medical necessity of treatment and if the patient has been on a stable dose for at least 24 weeks prior to randomization.
    2. Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; exceptions are listed in number 3 below.
    3. Previous treatment with drisapersen, ezutromid, or domagrozumab in the last 24 weeks prior to study enrollment.
    4. Major surgery within 3 months prior to randomization or planned surgery for any time during this study, except for allowed protocol-specified surgery, as applicable.
    5. Presence of any other significant neuromuscular or genetic disease other than DMD (eg, dwarfism).
    6. Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease or malignancy.
    7. Has evidence of cardiomyopathy, as defined by LVEF <50% on the screening ECHO or the Fridericia’s correction formula (QTcF) ≥450 milliseconds based on the screening ECGs.
    8. Prior or ongoing medical condition that could, in the Investigator’s opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
    9. Is, in the Investigator’s opinion, unable or unwilling to comply with the study procedures.
    Se excluirá de este estudio a todo paciente que cumpla cualquiera de los criterios siguientes:
    E 1. Haber usado cualquier tratamiento farmacológico (distinto de corticosteroides) en las 12 semanas previas a la asignación aleatoria que pueda afectar a la fuerza o la función muscular. Se permiten la hormona de crecimiento para la estatura corta y la testosterona para la pubertad retrasada si un endocrinólogo ha documentado el diagnóstico y la necesidad médica del tratamiento y si el paciente ha estado recibiendo una dosis estable por lo menos 24 semanas antes de la asignación aleatoria.
    E 2. Tratamiento actual o previo con cualquier otro tratamiento farmacológico experimental para la DMD o cualquier exposición previa a oligonucleótido antisentido, terapia génica o edición génica; las excepciones se indican en E3.
    E 3. Tratamiento previo con drisapersen, ezutromid, o domagrozumab en las últimas 24 semanas anteriores a la inscripción en el estudio.
    E 4. Cirugía mayor en un plazo de 3 meses antes de la asignación aleatoria o cirugía planificada durante cualquier momento de este estudio, excepto la cirugía especificada que permite el protocolo, según corresponda.
    E 5. Presencia de cualquier otra enfermedad neuromuscular o genética significativa que no sea DMD (por ejemplo, enanismo).
    E 6. Presencia de otra enfermedad clínicamente significativa, lo que incluye enfermedades cardíacas, pulmonares, hepáticas, renales, hematológicas, inmunológicas, conductuales o malignas.
    E 7. Tener evidencias de miocardiopatía, lo que se define como FEVI <50 % en el ECO de selección o una fórmula de corrección de Fridericia (QTcF) ≥450 milisegundos en el ECG de selección.
    E 8. Afección médica previa o en curso que podría, a juicio del investigador, afectar negativamente a la seguridad del paciente, hacer improbable la conclusión del tratamiento o desvirtuar la evaluación de los resultados del estudio.
    E 9. Ser incapaz, en opinión del investigador, o no estar dispuesto a cumplir los procedimientos del studio.
    E.5 End points
    E.5.1Primary end point(s)
    Open-label Dose Escalation
    Incidence of AEs, adverse events of special interest (AESIs) serious adverse events (SAEs), Safety laboratory assessments, ECGs and ECHO

    Double-blind Dose Finding
    Dystrophin expression in biopsied muscle tissue
    Plasma PK parameters of 30, 100, and 200 mg/kg of eteplirsen, administered IV weekly
    Incidence of AEs, AESIs and SAEs
    Abnormal changes from baseline or worsening of vitals or physical examination findings
    Safety laboratory assessments
    ECGs and ECHO
    Tissue concentration of eteplirsen from biopsied muscle tissue

    Dose Comparison
    Change from baseline at Week 144 in North Star Ambulatory Assessment
    (NSAA) total score
    Aumento de dosis abierto
    • Incidencia de acontecimientos adversos (AA)
    • Incidencia de acontecimientos adversos de interés especial (AAIE)
    • Incidencia de acontecimientos adversos graves (AAG)
    •Evaluaciones analíticas de seguridad
    • Electrocardiogramas (ECG)
    • Ecocardiogramas (ECO)
    Doble ciego, determinacion de dosis
    Expresión de distrofina en tejido muscular biopsiado
    Parámetros de PK en plasma de 30, 100 y 200 mg / kg de eteplirsen, administrados por vía intravenosa IV semanalmente
    • Incidencia de AA
    • Incidencia de AAIE
    • Cambios anormales desde los valores de referencia o empeoramiento de las constantes vitales y hallazgos de la exploración física
    • Incidencia de AAG
    • Evaluaciones analíticas de seguridad
    • ECG
    • ECO
    Concentración tisular de eteplirsen a partir de tejido muscular biopsiado
    Comparacion de Dosis
    Cambio desde los valores de referencia en la semana 144 en la evaluación ambulatoria de North Star (NSAA) puntaje total
    E.5.1.1Timepoint(s) of evaluation of this end point
    Open-label Dose Escalation
    At the completion of every 2-week treatment by 4 subjects at each
    dose level.

    Double-blind Dose Finding
    At baseline all patients undergo muscle biopsy pre-dose. One additional muscle biopsy at either 12, 24, or 48 weeks.
    Interim analyses will be performed on muscle biopsy data at Weeks 12 to assess dystrophin expression for high dose selection; additional interim analyses will be conducted at Week 24 and, if a high dose is not selected, at Week 48.

    AE assessment at baseline and continuous throughout the study. ECG
    at screening, week 1, 4, 8, and 12, and every 12 weeks thereafter.
    ECHO at screening, week 48, 96, and 144.

    Dose Comparison
    Change from baseline at Week 144
    Aumento de Dosis(D) diaria
    Al finalizar cada tto de 2 semanas por 4 sujetos en cada nivel de dosis.
    Hallazgo de D doble ciego
    Al inicio del estudio,los pacientes se someten a una D previa de biopsia muscular. Una biopsia muscular adicional en semana 12, 24 o 48.
    Se realizarán análisis interinos en los datos de biopsia muscular en semanas 12 para evaluar la expresión de distrofina para selección de D altas; Se realizarán análisis intermedios adicionales en la semana 24 y, si no se selecciona una D alta, en la semana 48.
    Evaluación de la EA en la línea de base y continua a lo largo del estudio. ECG
    en la selección, semana 1, 4, 8 y 12, y cada 12 semanas a partir de entonces.
    ECHO en la proyección, semana 48, 96 y 144.
    Comparacion de D
    Cambio desde la línea de base en la semana 144
    E.5.2Secondary end point(s)
    Change from baseline at Week 144 in
    o 6 MWT
    o 10 meter walk/run time
    o Timed 4 step stair ascend test
    o Time to rise from floor
    Annual decline rate in FVC % predicted Time to LOA
    Change from baseline at Week 48 (also at Week 12 if Dose Finding is not necessary, based on Dose Escalation, and Week 24 if dose selection is at Week 12) in skeletal muscle dystrophin expression by:
    o Western blot (quantitation)
    o IHC fiber intensity
    o Exon skipping quantitation by PCR
    IIncidence of AEs
    Incidence of AESIs
    Incidence of SAEs
    Safety laboratory assessments
    • Cambio desde los valores de referencia en la semana 144 de lo siguiente:
    o 6MWT
    o Tiempo para caminar/correr 10 metros
    • Tiempo hasta pérdida de ambulación (LOA)
    • Cambio desde los valores de referencia en la semana 48 (también en la semana 12 si no es necesaria la determinación de dosis, de acuerdo con el aumento de dosis, y en la semana 24 si la selección de la dosis se realiza en la semana 12) en la expresión de distrofina del músculo esquelético, determinada por lo siguiente:
    o Western Blot (cuantificación)
    o Intensidad de fibra por IHQ
    o Cuantificación de omisión del exón mediante PCR
    • Incidencia de AA
    • Incidencia de AAIE
    • Incidencia de AAG
    • Evaluaciones analíticas de seguridad
    • ECG
    • ECO
    o Prueba de subida de escalera de 4 peldaños
    o Tiempo para levantarse del suelo
    • Tasa de reducción anual prevista en % de la capacidad vital forzada (CVF)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change from baseline at Week 144
    Change from baseline at Week 48 (also at Week 12 if Dose Finding is not necessary, based on Dose Escalation, and Week 24 if dose selection is at Week 12)
    AE assessment at baseline and continuous throughout the study. ECG at
    screening, week 1, 4, 8, and 12, and every 12 weeks thereafter. ECHO at
    screening, week 48, 96, and 144.
    Cambio desde los valores de referencia en la semana 144.
    Cambio desde los valores de referencia en la semana 48 (también en la semana 12 si no es necesario encontrar la dosis, según la escala de dosis, y en la semana 24 si la selección de la dosis es en la semana 12)
    Evaluación de la EA en los valores de referencia y continua a lo largo del estudio. ECG en semana 1, 4, 8 y 12, y cada 12 semanas a partir de entonces. ECO en
    Proyección, semana 48, 96, y 144.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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
    The open-label period of the study evaluates the tolerability of the higher dose eteplirsen in DMD patients
    El período abierto del estudio evalúa la tolerabilidad de la dosis más alta de eteplirsen en pacientes con DMD
    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 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 Yes
    E. trial design description
    Etiqueta abierta Escalada de dosis seguida de una parte de Búsqueda de dosis doble ciego y una parte
    Open-label Dose Escalation followed by a Double-blind Dose Finding and Dose Comparison part
    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. description
    Dosis diferente del mismo producto
    different dosage of the same product
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    Korea, Republic of
    Russian Federation
    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
    ultima visita del ultimo paciente
    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 months7
    E.8.9.1In the Member State concerned days18
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days18
    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: 152
    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. of subjects for this age range: 125
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 27
    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. of subjects incapable of giving consent
    Male subjects 7-13 years old with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping
    Sujetos masculinos de 7 a 13 años de edad con un diagnóstico clínico establecido de DMD y una mutación de deleción fuera del marco del gen de la DMD susceptible de omisión del exón 51
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 152
    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 trial the patients will get back to their normal treatment of that condition under their physician
    Después de completar el ensayo, los pacientes volverán a su tratamiento normal con esa condición bajo control con su médico.
    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 Decision2019-10-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-09
    P. End of Trial
    P.End of Trial StatusOngoing
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