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    Summary
    EudraCT Number:2017-004554-42
    Sponsor's Protocol Code Number:TAMDMD
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-09-17
    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 number2017-004554-42
    A.3Full title of the trial
    Tamoxifen in Duchenne muscular dystrophy - TAMDMD
    A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial
    Tamoxifeno para la distrofia muscular de Duchenne - TAMDMD
    Estudio de fase III, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la seguridad y eficacia durante 48 semanas
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.
    El estudio investigará el tratamiento de niños con distrofia muscular de Duchenne. El objetivo de este estudio es determinar los efectos y la tolerabilidad del tamoxifeno para el tratamiento de esta enfermedad.
    A.4.1Sponsor's protocol code numberTAMDMD
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03354039
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUniversity of Basel Children's Hospital, Division of Neuropediatrics
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSwiss National Science Foundation (SNSF)
    B.4.2CountrySwitzerland
    B.4.1Name of organisation providing supportScientific and Technological Research Council of Turkey (TÜBÄ°TAK)
    B.4.2CountryTurkey
    B.4.1Name of organisation providing supportFrench National Research Agency (ANF)
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportDuchenne UK
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportDuchenne Parent Project Netherlands
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportAssociation Monegasque contre les myopathies
    B.4.2CountryMonaco
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationmulti-service-monitoring
    B.5.2Functional name of contact pointDr. Josef Reisinger
    B.5.3 Address:
    B.5.3.1Street AddressMaxhüttenstr. 11
    B.5.3.2Town/ cityRegensburg
    B.5.3.3Post code93055
    B.5.3.4CountryGermany
    B.5.4Telephone number+499413782498
    B.5.5Fax number+499416308648
    B.5.6E-mailjosef.reisinger@multi-service-monitoring.de
    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 TAMOX
    D.2.1.1.2Name of the Marketing Authorisation holderRowex Ltd. Bantry Country York
    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.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTamoxifen
    D.3.9.1CAS number 54965-24-1
    D.3.9.3Other descriptive nameTAMOXIFEN CITRATE
    D.3.9.4EV Substance CodeSUB04672MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    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 test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients by at least 50% (using the MFM D1 subscore as primary clinical endpoint in group A patients).
    To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 10-16 years old non-ambulant DMD patients not treated with glucocorticoids (using the MFM D2 subscore as primary endpoint.)
    Evaluar si el tratamiento con tamoxifeno, en comparación con el placebo, reduce la progresión de la enfermedad en pacientes ambulantes de 6,5-12 años con DMD en al menos un 50 % (utilizando la subescala D1 de la MFM como criterio clínico de valoración principal en pacientes del grupo A).
    Evaluar si el tratamiento con tamoxifeno, en comparación con el placebo, reduce la progresión de la enfermedad en pacientes no ambulantes de 10-16 años con DMD que no están tratados con glucocorticoides (utilizando la subescala D2 de la MFM como criterio de valoración principal).
    E.2.2Secondary objectives of the trial
    Secondary clinical outcomes to assess muscle function:

    1.MFM total score, the D2, and D3 MFM subscores, North Star Ambulatory Assessment, proximal upper limb function from baseline to week 48 under TAM treatment compared to placebo.

    2.Timed function tests (6 minute walking distance in meter, 10 meter walking time in seconds, time to rise from lying on the floor / supine up in seconds,) from baseline to week 48 under TAM treatment compared to placebo.

    Secondary clinical outcomes to assess muscle force:

    3.Quantitative muscle testing (using Grip force) from baseline to week 48 under TAM treatment compared to placebo.

    Secondary surrogate marker to assess muscle degeneration:

    4.Quantitative muscle MRI including muscle fat fraction (MFF) and T2 times of thigh muscles visualised by MRI from baseline to week 48 under TAM treatment compared to placebo.
    Objetivos clínicos secundarios para evaluar la función muscular:
    1. Puntuación total de MFM, subescalas D2 y D3, evaluación ambulatoria North Star, función proximal del miembro superior desde el inicio hasta la semana 48 con TAM en comparación con el placebo.
    2. Pruebas de función cronometrada (6 minutos a pié en metros, 10 metros a pié en segundos, tiempo para levantarse estirado desde el suelo / supino en segundos,) desde el inicio hasta la semana 48 con TAM en comparación con placebo.
    Objetivos clínicos secundarios para evaluar la fuerza muscular:
    3. Pruebas musculares cuantitativas (usando fuerza de agarre) desde el inicio hasta la semana 48 con TAM en comparación con el placebo.
    Marcador subrogado secundario para evaluar la degeneración muscular:
    4. RM muscular cuantitativa que incluye la fracción de grasa muscular (MFF) y tiempos T2 de los músculos del muslo visualizados mediante RM desde el inicio hasta la semana 48 con TAM en comparación con el placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Group A (ambulant patients)
    -Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
    -Stable treatment with glucocorticoids >6 months (no significant change in dosage (>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed
    -Male gender
    -6.5 to 12 years of age at time of screening
    -weight >15kg
    -ambulant patients
    -able to walk at least 350 meters in 6 minute walking distance test without assistance at screening
    -MFM D1 subdomain of the MFM scale >40% at screening
    -Ability to provide informed consent and to comply with study requirements

    Group B (non-ambulant patients)
    -Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
    -Not using glucocorticoids for >6 months
    -Male gender
    -Non-ambulant patients (walking distance less than 10 meters)
    -10 to 16 years of age at time of screening
    -Ability to provide informed consent and to comply with study requirements
    Grupo A (pacientes ambulantes)
    - Diagnóstico documentado de DMD mediante un análisis de la mutación del gen de la distrofina o por niveles sustancialmente reducidos de la proteína distrofina (es decir, ausencia o <5% de lo normal) en Western blot o inmunotinción.
    - Tratamiento estable con glucocorticoides >6 meses (sin cambios significativos de la dosis (>0.2mg/kg)) durante la selección; se permite adaptar la dosis de acuerdo a los cambios de peso.
    - Género masculino
    - 6,5 a 12 años de edad en el momento de la selección
    - Peso >15 kg
    - Pacientes ambulantes
    - Capaces de caminar al menos 350 metros en el test de la marcha de 6 minutos sin ayuda en la selección
    - Subdominio D1 de la escala de la MFM >40 % durante la selección
    - Capacidad para proporcionar un consentimiento informado y para cumplir los requisitos del estudio

    Grupo B (pacientes no ambulantes)
    - Diagnóstico documentado de DMD mediante un análisis de la mutación del gen de la distrofina o por niveles sustancialmente reducidos de la proteína distrofina (es decir, ausencia o <5% de lo normal) en Western blot o inmunotinción.
    - Que no hayan utilizado glucocorticoides durante >6 meses
    - Género masculino
    - Pacientes no ambulantes (recorrido de menos de 10 metros de distancia)
    - 10 a 16 años de edad en el momento de la selección
    - Capacidad para proporcionar un consentimiento informado y para cumplir los requisitos del estudio
    E.4Principal exclusion criteria
    -Known individual hypersensitivity or allergy to tamoxifen
    -Female gender
    -Use of tamoxifen or testosterone within the last 3 months
    -Known or suspected malignancy
    -Other chronic disease or clinically relevant limitation of renal, liver or heart function
    -Known or suspected non-compliance
    -Any injury which may impact functional testing, e.g. upper or lower limb fracture
    -Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapid progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 month prior to screening.
    -Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders of the participant/parents (as judged by the investigator)
    -Concomitant participation in any other interventional trial (and up to 3 months prior to screening)
    - Use of CYP2D6 inhibitors or of CYP3A4 inducers, platelet aggregation inhibitors and coumarin-type anti-coagulants.

    Group A:
    -Glucocorticoid naïve patients
    -Start of glucocorticoid treatment or change in dosage <6 month prior to screening (dosing adaptations according to weight change are allowed)

    Group B:
    -Glucocorticoid treated patients or patients that stopped steroid treatment <6 month prior to screening
    -Assisted ventilation of any kind necessary
    - Hipersensibilidad o alergia individual conocida al tamoxifeno
    - Género femenino
    - Uso del tamoxifeno o testosterona en los últimos 3 meses
    - Conocimiento o sospecha de la existencia de un tumor maligno
    - Otra enfermedad crónica o una limitación clínica relevante de tipo renal, del hígado o de la función cardíaca
    - Conocimiento o sospecha de incumplimiento
    - Una lesión que pueda repercutir en las pruebas funcionales, p.ej:, una fractura del miembro superior o inferior
    - Cirugía de fusión espinal planificada o esperada durante el período de estudio (según lo juzgue el Investigador, p.ej:, debido a una escoliosis que progresa rápidamente), se permite una cirugía de fusión espinal previa si se realizó más de 6 meses antes de las pruebas.
    - Incapacidad de seguir los procedimientos del estudio, p.ej: debido a problemas del lenguaje, trastornos psicológicos del participante/padres (según lo juzgue el investigador)
    - Participación concomitante en otro ensayo de intervención (y hasta 3 meses antes de la selección)
    - Uso de inhibidores de CYP2D6 o de inductores de CYP3A4, inhibidores de agregación de plaquetas y anticoagulantes tipo cumarina.

    Grupo A:
    - Pacientes que no han recibido glucocorticoides
    - Comienzo del tratamiento con glucocorticoides o cambio de la dosis <6 meses antes de las pruebas (se permite adaptar la dosis según el cambio de peso)

    Grupo B:
    -Pacientes tratados con glucocorticoides o pacientes que suspendieron el tratamiento con glucocorticoides <6 meses antes de la selección.
    - Ventilación asistida necesaria de cualquier tipo
    E.5 End points
    E.5.1Primary end point(s)
    Group A: The primary efficacy outcome will be the change of motor function under TAM treatment compared to placebo. This will be assessed by the motor function measure (MFM) D1 subscore (standing and transfers) in ambulant patients.

    For the MFM D1 subscore several exercises that will be scored have to be done by the patients such as:
    -to sit up on a mat (without support of upper limbs),
    -to stand up from sitting on a mat,
    -to sit down on the chair from standing,
    -to walk forward 10 steps on both heels,
    -to walk forward 10 steps on a straight line,
    -to run 10 m,
    -and to hop 10 times on one foot,

    Group B: The primary efficacy outcome in this group will be the motor function from baseline to week 48 under TAM treatment compared to placebo. This will be assessed by the motor function measure (MFM) D2 subscore in non-ambulant patients allowing a partial extrapolation and comparison of MFM D2 values between group A and group B. Note that the MFM D1 subscore in non-ambulant patients is usually 0%; therefore the MFM D1 subscore is not suitable for this population.
    For the MFM D2 subscore several exercises that will be scored have to be done by the patients such as:
    -from supine on a mat to raise the head and maintain the raised position
    -from supine on a mat to raise one hand and move it to the opposite shoulder
    -from seated on the chair to raise the head from fully flexed position and maintain the raised position
    -from seated on the chair to place the two forearms and/or the hands on the table at the same time without moving the trunk.
    Grupo A: el objetivo primario de eficacia será el cambio de la función motora en el tratamiento TAM en comparación con el placebo. Esto se evaluará mediante la puntuación de la función motora (MFM) D1 subescala (de pie y transferencia) en pacientes ambulantes.

    Para la MFM D1 subescala, los pacientes deben realizar varios ejercicios que serán puntuados, tales como:
    - levantarse de una colchoneta (sin apoyo de las extremidades superiores),
    - levantarse desde posición sentada en una colchoneta,
    - sentarse en una silla desde posición levantada
    - avanzar 10 pasos en ambos talones,
    - avanzar 10 pasos en línea recta,
    - correr 10 m,
    - y saltar 10 veces en un pie,

    Grupo B: el objetivo primario de eficacia en este grupo será la función motora desde el inicio hasta la semana 48 bajo tratamiento con TAM en comparación con el placebo. Esto se evaluará mediante la puntuación de la función motora (MFM) D2 subescala en pacientes no ambulantes, lo que permite una extrapolación parcial y comparación de los valores MFM D2 entre el grupo A y el grupo B. Tengase en cuenta que la subescala MFM D1 en pacientes no ambulantes suele ser 0 %; por lo tanto, la subescala MFM D1 no es adecuada para esta población.
    Para el subescala MFM D2, los pacientes deben realizar varios ejercicios que serán puntuados, tales como:
    - desde supino sobre una colchoneta, levantar la cabeza y mantener la posición elevada
    - desde supino sobre una colchoneta, levantar una mano y moverla al hombro opuesto
    - sentado en la silla, levantar la cabeza desde la posición completamente flexionada y mantener la posición elevada
    - sentado en la silla, colocar los dos antebrazos y / o las manos sobre la mesa al mismo tiempo sin mover el tronco.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Baseline, week 12, week 24, week 36, week 48
    Selección, Basal, semana 12, semana 24, semana 36, semana 48
    E.5.2Secondary end point(s)
    Secondary clinical outcomes to assess muscle function:
    -MFM total score, the D2, and D3 MFM subscores, North Star Ambulatory Assessment from baseline to week 48 under tamoxifen treatment compared to placebo.
    -Timed function tests (6 minute walking distance in meter, 10 meter walking time in seconds, time to rise from lying on the floor / supine up in seconds,) from baseline to week 48 under tamoxifen treatment compared to placebo.
    Secondary clinical outcomes to assess muscle force:
    -Quantitative muscle testing (using Grip force) from baseline to week 48 under tamoxifen treatment compared to placebo.
    Secondary surrogate marker to assess muscle degeneration:
    -Quantitative muscle MRI including muscle fat fraction (MFF) and T2 times of thigh muscles visualised by MRI from baseline to week 48 under tamoxifen treatment compared to placebo.
    Los objetivos Clínicos secundarios para evaluar la función muscular:
    - MFM puntuación total y las subescalas MFM D2 y D3, la Evaluación Ambulatoria North Star (NSAA) desde el inicio hasta la semana 48 bajo tratamiento con tamoxifeno en comparación con placebo.

    - Pruebas cronometradas de funciones (6 minutos a pié en metros, 10 a pié en segundos, tiempo para levantarse estirado desde el suelo / supino en segundos,) desde el inicio hasta la semana 48 bajo tratamiento con tamoxifeno en comparación con placebo.

    Los objetivos clínicos secundarios para evaluar la fuerza muscular:
    - Pruebas musculares cuantitativas (usando fuerza de agarre) desde el inicio hasta la semana 48 bajo tratamiento con tamoxifeno en comparación con placebo.

    Marcador subrogado secundario para evaluar la degeneración muscular:
    - RM muscular cuantitativa que incluye la fracción de grasa muscular (MFF) y tiempos T2 de los músculos del muslo visualizados mediante RM desde el inicio hasta la semana 48 bajo tratamiento con tamoxifeno en comparación con placebo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    MRI: Baseline, week 24, week 48
    Timed Function test, MFM: Screening, Baseline, week 12, week 24, week 36, week 48
    RM: Basal, semana 24, semana 48
    Prueba cronometrada de función: Selección, Basal, semana 12, semana 24, semana 36 , semana 48
    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 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 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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    France
    Germany
    Netherlands
    Spain
    Switzerland
    Turkey
    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
    LPLV
    última visita último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    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: 100
    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: 80
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    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
    Boys between 6,5 years and 16 years
    Niños entre 6,5 años y 16 años
    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 70
    F.4.2.2In the whole clinical trial 100
    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)
    Depending on the safety and efficacy data obtained during the study, the sponsor plans an open-label extension study to provide tamoxifen access to all DMD patients enrolled in this study.
    Dependiendo de los datos de seguridad y eficacia obtenidos durante el estudio, el patrocinador planea un estudio de extensión abierto para proporcionar acceso a tamoxifeno a todos los pacientes con DMD inscritos en este 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 Decision2019-01-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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