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    Summary
    EudraCT Number:2019-000601-77
    Sponsor's Protocol Code Number:5051-201
    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:2019-07-05
    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 number2019-000601-77
    A.3Full title of the trial
    A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with
    Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping
    Treatment
    Estudio de fase 2, de dos partes, de dosis múltiple ascendente, para la determinación de la dosis de SRP-5051, seguido de ampliación de la dosis, en pacientes con distrofia muscular de Duchenne susceptible al tratamiento de omisión del exón 51
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients
    Un estudio de investigación de un nuevo medicamento en investigación para el tratamiento de pacientes con Distrofia Muscular de Duchenne
    A.4.1Sponsor's protocol code number5051-201
    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 pointproject management
    B.5.3 Address:
    B.5.3.1Street Address4 The Fleming Building
    B.5.3.2Town/ cityEdinburgh Technopole
    B.5.3.3Post codeEH26 0BE
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44131448 1303
    B.5.6E-mailcarolyn.mills@syneoshealth.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSRP-5051
    D.3.2Product code SRP-5051
    D.3.4Pharmaceutical form Lyophilisate 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 INNSRP-5051
    D.3.9.1CAS number 2101569-97-3
    D.3.9.2Current sponsor codeSRP-5051
    D.3.9.3Other descriptive namepeptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
    D.3.9.4EV Substance CodeSUB197949
    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.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 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
    distrofia muscular de Duchenne (DMD)
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: Multiple-Ascending-Dose (MAD) for Dose Determination
    To evaluate the safety and tolerability of multiple ascending doses of SRP-5051 (4.0, 10.0, 16.0, and 20.0 mg/kg), administered intravenously (IV) every 4 weeks, and determine the maximum tolerated dose (MTD)

    Part B – Dose Expansion
    • To evaluate exon-skipping and dystrophin protein production levels (derived from muscle) after additional treatment with SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    Parte A: Evaluar la seguridad y la tolerabilidad de dosis múltiples ascendentes de SRP-5051 (4, 10, 16 y 20 mg/kg), administradas por vía intravenosa (IV) cada 4 semanas, y determinar la dosis máxima tolerada (DMT)
    Parte B: Evaluar los niveles de omisión del exón y de producción de proteína distrofina (derivados del músculo) después del tratamiento adicional con SRP-5051, administrado por vía intravenosa cada 4 semanas a la DMT determinada en la parte A, en pacientes que han finalizado la parte A
    E.2.2Secondary objectives of the trial
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    To determine the pharmacokinetics (PK) of each dose level of the aforementioned multiple ascending doses of SRP-5051, administered IV every 4 weeks
    Part B – Dose Expansion
    • To evaluate the ongoing safety and tolerability of additional treatment with SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    • To evaluate the clinical effect of additional treatment with SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    • To determine the PK of SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    Parte A - Dosis de dosis ascendente (MAD) para la determinación de la dosis. Determinar la farmacocinética (FC) de cada nivel de dosis de las dosis múltiples ascendentes de SRP-5051 mencionadas, administradas por vía intravenosa cada 4 semanas
    Parte B - Expansión de la dosis
    • Evaluar la seguridad y la tolerabilidad continuadas del tratamiento adicional con SRP-5051, administrado por vía intravenosa cada 4 semanas a la DMT determinada en la parte A, en pacientes que han finalizado la parte A
    • Evaluar el efecto clínico del tratamiento adicional con SRP-5051, administrado por vía intravenosa cada 4 semanas a la DMT determinada en la parte A, en pacientes que han finalizado la parte A
    • Determinar la FC de SRP-5051, administrado por vía intravenosa cada 4 semanas a la DMT determinada en la parte A, en pacientes que han finalizado la parte A
    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. Is male.
    2. Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
    3. Is 7 to 21 years of age, inclusive.
    4. Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.
    5. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a highly effective form of contraceptive during this timeframe.
    6. Is willing to provide informed consent or 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 varón.
    I 2. Tener un diagnóstico genético de DMD y una mutación por deleción con pérdida del marco de lectura del gen DMD que sea susceptible al tratamiento por omisión del exón 51.
    I 3. Tener una edad comprendida entre los 7 y los 21 años, ambos incluidos.
    I 4. Haber estado recibiendo una dosis estable de corticoesteroides orales durante al menos 12 semanas antes de la administración del fármaco del estudio, o no haber recibido corticoesteroides durante al menos 12 semanas antes de la administración del fármaco del estudio.
    I 5. Si es sexualmente activo, aceptar el uso de un preservativo masculino para dicha actividad mientras dure el estudio y durante 90 días después de la última dosis del fármaco del estudio. La pareja sexual también deberá usar una forma altamente eficaz de anticonceptivo durante este período de tiempo
    E.4Principal exclusion criteria
    A patient who meets any of the following criteria will be excluded from this study:
    1. Has a LVEF < 40.0% based on an ECHO performed within 12 weeks prior to Screening or at the Screening visit.
    2. Has a QT interval corrected with Fridericia’s formula ≥ 450 msec on the Screening ECG.
    3. Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
    4. Requires anti-arrhythmic and/or diuretic therapy for heart failure.
    5. Has a FVC < 40.0% of predicted value (according to the American Thoracic Society/European Respiratory Society criteria) within 12 weeks prior to Screening or at Screening.
    6. Has a current infection, or history of an infection within 12 weeks prior to Screening requiring treatment with an antibiotic.
    7. Has a known kidney disease or had an acute kidney injury within 24 weeks prior to Screening.
    8. Use of any herbal medication/supplement containing aristolochic acid.
    9. Treatment with any exon 51-skipping therapy within 24 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.
    10. Participation in an interventional clinical trial within 12 weeks prior to Screening.
    11. Use of any aminoglycoside antibiotic or statin within 12 weeks prior to Screening.
    12. Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
    13. Major surgery within 12 weeks prior to Screening, or planned surgery or procedures that would interfere with the conduct of the study.
    14. Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy.
    15. Any other condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the trial.
    16. Inability to comply with the study protocol.
    17. Is an employee of the Investigator or study center, with direct involvement in the proposed study or other studies under the direction of that Investigator or study center, as well as family members of the employees or the Investigator.
    18. Any patient who is taking medications that increase the risk of bleeding, in the Investigator's opinion (eg, anticoagulants, antiplatelet agents, novel oral anticoagulants, selective norepinephrine reuptake inhibitors, etc.)
    19. Platelet count < 150 × 103/μL.
    Se excluirá de este estudio a todo paciente que cumpla cualquiera de los criterios siguientes:
    E 1. Tener una FEVI < 40 % según un ECO realizado dentro de las 12 semanas previas a la selección o en la visita de selección.
    E 2. Tener un intervalo QT corregido con la fórmula de corrección de Fridericia ≥ 450 ms en el ECG de selección.
    E 3. Haber iniciado o modificado la dosificación (excepto las modificaciones de ajuste a cambios de peso), dentro de las 12 semanas previas a la selección, de cualquiera de los siguientes tipos de fármacos: inhibidores de la enzima convertidora de la angiotensina, antagonistas de los receptores de la angiotensina, betabloqueantes o potasio.
    E 4. Requerir tratamiento antiarrítmico o diurético para la insuficiencia cardíaca.
    E 5. Tener una CVF < 40 % del valor previsto (de acuerdo con los criterios de la Sociedad Torácica Americana / Sociedad Respiratoria Europea) dentro de las 12 semanas previas a la selección o en el período de selección.
    E 6. Tener una infección actual o antecedentes de una infección dentro de las 12 semanas previas a la selección que requiera tratamiento con un antibiótico.
    E 7. Tener una enfermedad renal conocida o haber tenido una lesión renal aguda dentro de las 24 semanas previas a la selección.
    E 8. Tomar cualquier medicamento o suplemento herbario que contenga ácido aristolóquico.
    E 9. Haber recibido un tratamiento de omisión del exón 51 dentro de las 24 semanas previas a la selección o una genoterapia experimental para el tratamiento de la DMD en algún momento.
    E 10. Haber participado en un ensayo clínico de intervención dentro de las 12 semanas previas a la selección.
    E 11. Haber tomado un antibiótico aminoglucosídico o una estatina dentro de las 12 semanas previas a la selección.
    E 12. Haber iniciado o modificado la dosificación, dentro de las 12 semanas previas a la selección, de medicamentos de venta sin receta, como suplementos herbarios o no herbarios, vitaminas, minerales y preparaciones homeopáticas.
    E 13. Haberse sometido a una cirugía mayor dentro de las 12 semanas previas a la selección, o tener programados una cirugía o un procedimiento que pudieran interferir en la realización del estudio.
    E 14. Presentar otra patología clínicamente significativa, como afecciones cardíacas, pulmonares, hepáticas, renales, hematológicas, inmunológicas o conductuales, una infección o un cáncer.
    E 15. Presentar cualquier otra afección que, a juicio del investigador, pudiera interferir en la participación del paciente en el ensayo.
    E 16. Tener algún impedimento para cumplir con el protocolo del estudio.
    E 17. Ser un empleado del investigador o del centro del estudio con participación directa en el presente o en otros estudios dirigidos por el investigador o llevados a cabo en el centro del estudio, así como ser familiar del investigador o de alguno de los empleados.
    E 18. Estar tomando medicamentos que incrementen el riesgo de hemorragia, a juicio del investigador (por ejemplo, anticoagulantes, antiagregantes plaquetarios, nuevos anticoagulantes orales, inhibidores selectivos de la recaptación de noradrenalina, etc.).
    E 19. Presentar un recuento de plaquetas < 150 × 103/μL.
    E.5 End points
    E.5.1Primary end point(s)
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    • Incidence of AEs
    • Clinically significant laboratory abnormalities (eg, hematology, chemistry, coagulation, urinalysis)

    Part B – Dose Expansion
    • Change from Baseline biopsy in exon-skipping levels (as measured by PCR) in muscle.
    • Change from Baseline biopsy in dystrophin protein production levels (as measured by Western blot and/or other relevant methods) in muscle.
    Parte A: Dosis de dosis múltiple ascendente (MAD) para la determinación de la dosis
    • Incidencia de AA
    • Alteraciones clínicamente significativas en las pruebas analíticas (por ejemplo, hematología, química, coagulación, análisis de orina)
    Parte B- expansion de dosis
    • Cambio de la biopsia de referencia en los niveles de omisión de exón (medido por PCR) en el músculo.
    • Cambio de la biopsia de referencia en los niveles de producción de proteína distrofina (medido por Western blot y / u otros métodos relevantes) en el músculo
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination

    All AEs will be reported from the time of providing signed informed consent/assent through the last follow-up visit.
    Clinically significant laboratory abnormalities will be assessed from Baseline at week 12
    All the routine clinical laboratory tests will be performed during clinic visits at the time points specified in the Schedule of Events for Part A as stated in the Table 1 of the protocol

    Part B – Dose Expansion
    -biopsy in exon-skipping levels (as measured by PCR) in muscle collected from the Part B, Week 24 biopsy.
    -dystrophin protein production levels from the Part B, Week 24 biopsy.
    Parte A: Dosis de dosis múltiple ascendente (MAD) para la determinación de la dosis
    Todos los AA serán informados desde la firma del asentimiento / consentimiento informado firmado hasta la última visita de seguimiento.
    Las anomalías de lab clínicamente significativas se evaluarán a partir de la línea de base en la semana 12
    Todas las pruebas de laboratorio clínicas de rutina se realizarán durante las visitas clínicas en los puntos de tiempo especificados en el Programa de eventos para la Parte A como se indica en la Tabla 1 del protocolo.

    Parte B - Expansión de la D
    - Biopsia en los niveles de omisión de exón (medido por PCR) en el músculo recolectado de la Parte B, biopsia de la semana 24.
    -Los niveles de producción de proteína estrofina de la Parte B, biopsia de la semana 24.
    E.5.2Secondary end point(s)
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    • PK parameters

    Part B – Dose Expansion
    • Incidence of AEs
    • Clinically significant laboratory abnormalities (eg, hematology, chemistry, coagulation, urinalysis)
    • Change from Baseline assessment in the following, conducted at Part B, Week 24:
    o Forced vital capacity (FVC) (% predicted) calculation
    o North Star Ambulatory Assessment (NSAA) (in ambulatory patients)
    o Performance Upper Limb (PUL)
    o Brooke Upper Extremity Scale score (“Brooke Score”)
    • PK parameters at the MTD determined in Part A
    Parte A: Dosis de dosis múltiple ascendente (MAD) para la determinación de la dosis
    • parámetros PK

    Parte B - Expansión de la dosis
    • Incidencia de AA
    • Anomalías de laboratorio clínicamente significativas (p. Ej., Hematología, química, coagulación, análisis de orina)
    • Cambio de la evaluación de referencia en lo siguiente, realizado en la Parte B, semana 24:
    -Cálculo de la capacidad vital forzada (FVC) (% predicho)
    -North Star Ambulatory Assessment (NSAA) (en pacientes ambulatorios)
    -Rendimiento del miembro superior (PUL)
    -Puntuación de Brooke Upper Extremity Scale ("Puntuación de Brooke")
    • Parámetros de PK en el MTD determinado en la Parte A
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    • PK parameters at each dose level
    Part B – Dose Expansion
    •All AEs will be reported from Baseline through the last follow-up visit.
    •Clinically significant laboratory abnormalities will be conducted in part B Week 24
    • Change from baseline assessment in functional test conducted at part B, capture all timepoints
    • PK parameters at MTD
    Parte A: Dosis de dosis múltiple ascendente (MAD) para la determinación de la dosis
    • Parámetros de PK a cada nivel de dosis.
    Parte B - Expansión de la dosis
    • Todos los AA se informarán desde la línea de base hasta la última visita de seguimiento.
    • Anomalías de laboratorio clínicamente significativas se llevarán a cabo en la parte B Semana 24
    • Cambio de la evaluación de línea de base en la prueba funcional realizada en la parte B, captura todos los puntos de tiempo
    • PK parámetros en MTD
    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 Yes
    E.6.7Pharmacodynamic No
    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
    Multiple-Ascending-Dose Study for Dose Determination, then Dose Expansion
    Estudio de dosis múltiple ascendente para la determinación de la dosis y luego la expansión de la dosis
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Estudio de dosis múltiple ascendente para la determinación de la dosis y luego la expansión de la do
    Multiple-Ascending-Dose Study for Dose Determination, then Dose Expansion
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 EEA14
    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
    Belgium
    Canada
    Germany
    Ireland
    Italy
    Netherlands
    Spain
    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
    LPLV
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 24
    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: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 4
    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
    This study will be conducted in both ambulatory and nonambulatory patients, aged 7 to 21 years, inclusive, with DMD amenable to exon 51-skipping treatment.
    Este estudio se llevará a cabo en pacientes ambulatorios y no ambulatorios, de 7 a 21 años de edad, ambos inclusive, con DMD susceptible de tratamiento con exón 51 y omisión.
    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 16
    F.4.2.2In the whole clinical trial 24
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    none
    ninguna
    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 Decision2020-01-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-24
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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