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    Summary
    EudraCT Number:2015-002530-50
    Sponsor's Protocol Code Number:EspeRare_RIM_001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-08-25
    Trial results View 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 number2015-002530-50
    A.3Full title of the trial
    A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy.
    Estudio abierto de fase Ib para evaluar la seguridad, tolerabilidad,
    farmacocinética y farmacodinámica de múltiples dosis orales ascendentes
    de rimeporida en pacientes con distrofia muscular de Duchenne (DMD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Rimeporide in patients with Duchenne Muscular Dystrophy
    Rimeporida en pacientes con distrofia muscular de Duchenne
    A.3.2Name or abbreviated title of the trial where available
    Rim4DMD
    Rim4DMD
    A.4.1Sponsor's protocol code numberEspeRare_RIM_001
    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 SponsorEspeRare
    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 supportEuropean Commission
    B.4.2CountryBelgium
    B.4.1Name of organisation providing supportL?Association Française Contre les Myopathies (AFM)
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportMerck Serono
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationQED Clinical Services
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressSnowdon Drive / Winterhill
    B.5.3.2Town/ cityMilton Keynes
    B.5.3.3Post codeMK6 1BP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number441908251 480
    B.5.5Fax number441908251 499
    B.5.6E-mailnmaruf@qed-clinical.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/3/15/1478
    D.3 Description of the IMP
    D.3.1Product nameRimeporide
    D.3.2Product code EMD 87580
    D.3.4Pharmaceutical form Capsule, hard
    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 INNN(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate
    D.3.9.2Current sponsor codeEMD 87580
    D.3.9.3Other descriptive nameRimeporide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 (DMD)
    Distrofia Muscular de Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy (DMD)
    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 18.1
    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 determine the preliminary safety and tolerability profile of multiple oral administrations of rimeporide.
    Determinar el perfil preliminar de seguridad y tolerabilidad de múltiples
    administraciones orales de rimeporida
    E.2.2Secondary objectives of the trial
    Secondary objectives:
    •Pharmacokinetics: To evaluate the pharmacokinetic profile of rimeporide in pediatric patients with DMD.

    •Pharmacodynamics:
    -To measure inflammatory and muscular injury biomarkers;
    -To explore the PK/PD relationship of a 4-week rimeporide treatment on those surrogate biomarkers.

    Exploratory objectives:
    -To explore the relationship between safety endpoints and pharmacokinetic parameters
    -To explore using Nuclear Magnetic Resonance (NMR) Spectroscopy and imaging the effect of a 4-week treatment with rimeporide on biomarkers including intracellular pH and intracellular Na, and degree of inflammation, oedema, fat fraction, muscle composition).
    Objetivos secundarios:
    •Farmacocinética: Evaluar el perfil farmacocinético de rimeporida en
    pacientes pediátricos con DMD.

    •Farmacodinámica:
    -Determinar los biomarcadores inflamatorios y de lesiones musculares
    -Analizar la relación FC/FD de un tratamiento de 4 semanas con
    rimeporida respecto a estos biomarcadores indirectos.

    Objetivos exploratorios:
    - Explorar la relación entre variables de seguridad y los parámetros farmacocinéticos.
    - Efectuar el análisis mediante espectroscopia por resonancia magnética nuclear (RMN) y pruebas de imagen del efecto de un tratamiento de 4 semanas con rimeporida sobre los biomarcadores, incluyendo pH intracelular y Na intracelular, y el grado de inflamación, edema, fracción grasa y composición muscular.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Duchenne muscular dystrophy genetically confirmed;
    2. Males between 6 and 14 years old;
    3. Able to walk independently at least 75 meters;
    4. Patients on a stable dose of corticosteroids at least 6 months prior to baseline;
    5. Willing and able to comply with all protocol requirements and procedures;
    6. Signed informed consents by the parent(s)/legal guardian(s);
    7. France only: Affiliated to or a beneficiary of a social security system
    1. Distrofia muscular de Duchenne confirmada genéticamente
    2. Varones entre 6 y 14 años de edad
    3. Capaces de andar de forma independiente al menos 75 metros
    4. Pacientes tratados con una dosis estable de corticosteroides al menos 6 meses antes de la situación basal
    5. Disposición y capacidad para cumplir con todos los requisitos y procedimientos del protocolo
    6. Firma de los consentimientos informados por parte de los padres/tutores legales
    7. Solo en Francia: Afiliados a un sistema de seguridad social o beneficiarios de este.
    E.4Principal exclusion criteria
    1. Patients with significant renal disease or impairment, with Glomerular Filtration Rate estimated using plasma cystatin C level using the Filler formula less than 90ml/min/ 1.73m2;
    2. Current or history of liver disease or impairment,
    3. History of any significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. inflammatory, coagulation disease, unstable cardiac or respiratory disease;
    4. Acute illness within 4 weeks of the first administration of study medication which may interfere with study assessments;
    5. Significant change of dosage and/or dosing regimens for corticosteroids planned for the duration of study medication;
    6. Use of beta blockers / and ACEI or ARB unless at stable dose for at least 3 months prior to baseline;
    7. Use of protocol pump inhibitors unless at a stable dose for at least 3 months prior to baseline
    8. Use of aldosterone antagonists (i.e. spironolactone, eplerenone) within 3 months prior to first administration of study medication;
    9. Use of anticoagulants, antithrombotics or antiplatelet agents,
    10. Previous treatment with idebenone or other forms of Coenzyme Q10 within 1 month of the first administration of study medication;
    11. Previous treatment with investigational drugs within 4 weeks (or 7 half-life if longer than 4 weeks) of the first administration of study medication including placebo;
    12. A baseline QTc>450msec,or history of risk factors for torsades de pointes (eg, heart failure, hypokalaemia, family history of long QT syndrome);
    13. LVEF ≤ 45% at screening or within the past 6 months and/or history of acute heart failure;
    14. Ventilator dependent;
    15. Known individual hypersensitivity to any of the ingredients/excipients of the study medication;
    16. For patients willing to undergo MRI/MRS assessments: Patients with specific contraindication to MRI (e.g.: metallic foreign body, claustrophobia, etc.).
    1. Pacientes con enfermedad o insuficiencia renal significativa, con determinación de la filtración glomerular a través del nivel de cistatina C empleando la fórmula de Filler por debajo de 90 ml/min/1,73 m2
    2. Antecedentes o presencia actual de enfermedad o insuficiencia hepática
    3. Antecedentes de cualquier trastorno médico significativo que pueda confundir la interpretación de los datos de eficacia o seguridad, p. ej. enfermedad inflamatoria, coagulopatía, enfermedad cardíaca o respiratoria inestable
    4. Enfermedad aguda en las 4 semanas siguientes a la primera administración del medicamento del estudio que podría interferir con las evaluaciones del estudio
    5. Cambio significativo en la posología y/o las pautas posológicas de corticosteroides previsto para toda la administración de la medicación del estudio
    6. Uso de betabloqueantes e IECA o ARA a menos que se haya mantenido una dosis estable como mínimo durante 3 meses antes de la situación basal
    7. Uso de inhibidores de la bomba de protones a menos que se haya mantenido una dosis estable como mínimo durante 3 meses antes de la situación basal 8. Uso de antagonistas de la aldosterona (es decir, espironolactona, eplerenona) en los 3 meses anteriores a la primera administración del medicamento del estudio
    9. Uso de anticoagulantes, antitrombóticos o antiagregantes plaquetarios
    10. Tratamiento previo con idebenona u otras formas de coenzima Q10 en el mes anterior a la primera administración del medicamento del estudio
    11. Tratamiento previo con fármacos experimentales en las 4 semanas anteriores (o 7 semividas si son más de 4 semanas) a la primera administración del medicamento del estudio incluyendo placebo
    12. Intervalo QTc basal > 450 ms, o antecedentes de factores de riesgo de torsades de pointes (p. ej., insuficiencia cardíaca, hipopotasiemia, antecedentes familiares de síndrome del QT largo)
    13. FEVI ≤ 45 % en la selección o en los 6 meses anteriores y/o antecedentes de insuficiencia cardíaca aguda
    14. Dependencia de respirador
    15. Hipersensibilidad individual conocida a cualquiera de los
    componentes/excipientes del medicamento del estudio
    16. Para pacientes dispuestos a someterse a evaluaciones por RMN/ERMPacientes con contraindicaciones específicas a RMN (p. ej.: cuerpos extraños metálicos, claustrofobia, etc.).
    E.5 End points
    E.5.1Primary end point(s)
    Each subject will participate in only 1 dose cohort and will receive rimeporide for a total of 4 weeks.

    Safety assessment will be done on data collected in each cohort and during the 4 weeks treatment and 2 weeks of follow-up. The decision to progress to the next higher dose will be made after safety and tolerability data are reviewed for the preceding dose for 5 patients by a data Safety Monitoring Committee and determined that it is safe to proceed to the next dose level.

    Safety and tolerability of rimeporide will be assessed as follows:
    - Incidence, severity, causality and outcomes of AE and SAE
    - Vital signs, Supine and standing blood pressure, heart rate and respiratory rate
    - Evolution of laboratory parameters from blood and urine samples including gastrin levels: substantial changes in laboratory parameters and gastrin;
    - Monitoring of heart function (ECG evolution parameters including QTc);
    - Number of patients withdrawn for safety issues
    Cada paciente participará solo en una cohorte y recibirá rimeporide para
    un total de 4 semanas.
    Se evaluará la seguridad en datos recabados en cada cohorte y durante las 4 semanas de tratamiento y 2 semanas de seguimiento. La decisión de avanzar a la siguiente dosis superior se tomará después de la revisión de los datos de seguridad y tolerabilidad de la dosis precedente para 5 pacientes por parte de un Comité de vigilancia de la seguridad de los datos, y de determinar que es seguro pasar al siguiente nivel de dosis.
    La seguridad y tolerabilidad de rimeporide serán evaluadas de la siguiente manera:
    - La incidencia, la gravedad, la causalidad y los resultados de AE y SAE
    - Los signos vitales, presión arterial en posición supina y de pie, la frecuencia cardíaca y la frecuencia respiratoria
    - Evolución de los parámetros de laboratorio a partir de muestras de sangre y orina, incluyendo los niveles de gastrina: cambios sustanciales en los parámetros de laboratorio y gastrina;
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety endpoints will be assessed druning on site visit at Baseline, day 14, day 28 and via a phone call at day 7 and day 21 (fexibility window for each visit or call is +/-2 days) during treatment period and 2 weeks after the last rimeporide dose.

    Safety endpoints will be also assessed if unplanned visit(s) may occur should the patient need to be assessed or treated for any clinical condition that arises during the study.
    Criterios de valoración de seguridad se evaluarán durante visita al inicio, día 14, el día 28 y a través de una llamada telefónica en el día 7 y el día 21 (ventana de flexibilidad para cada visita o llamada es de +/- 2 días) durante el período de tratamiento y 2 semanas después la última dosis de rimeporida.

    Criterios de valoración de seguridad serán también evaluados si surge alguna condición clínica durante el estudio no planificado,que requiera la evaluación o tratamiento de un paciente.
    E.5.2Secondary end point(s)
    Pharmacokinetic profile of rimeporide in plasma derived from modelling using sparse sampling

    Pharmacodynamics: Changes of plasma/urine biomarkers:
    - Plasma levels of Troponin I, myomesin, creatine kinase isoenzyme (MM fraction); miRNAs;
    - Urine concentration of titin fragments ;
    - Plasma levels of the inflammation markers: C - reactive protein (CRP), Tumor Necrosis Factor alfa (TNFα), Transforming Growth Factor beta (TGFβ).
    - Plasma level of fibrosis makers: MMP-9.

    Exploratory endpoints:

    Pharmacodynamics: Changes in NMRI and NMRS indices in skeletal muscle will be explored through:
    - 3 NMRI indices (water T2, fat fraction);
    - Nine 31P NMRS indices;
    - 23Na NMRS indices may also be included.

    Participation in MRI/MRS requires travel to the Institute of Myology in Paris and will be optional
    Farmacocinética:
    Se propone un modelo farmacocinético basado fisiológicamente (PBPK).
    Las muestras de sangre se obtendrán para determinar el perfil farmacocinético de rimeporide administrado por vía oral (muestreo disperso).
    Farmacodinamia:
    Se realizará una evaluación exploratoria de la respuesta biológica precoz de un tratamiento de 4 semanas con rimeporida en los músculos esqueléticos, a través del cambio observado desde el inicio hasta la semana 4 de biomarcadores de plasma / orina:
    Los niveles plasmáticos de troponina I, myomesin, MMP-9, la creatina quinasa (CK) en total, la creatina quinasa isoenzima (fracción MM); miRNAs (miR-29a y miR-29c)
    - La concentración de fragmentos titina en orina (muestra de orina provisional adicional se recogerá a W2)
    - Los niveles plasmáticos de los marcadores de inflamación: la proteína C reactiva (PCR), factor de necrosis tumoral alfa (TNF), factor de crecimiento transformante beta (TGF)
    Criterios exploratorios (opcional):
    Cambios en NMRI e índices NMRS en el músculo esquelético se explorarán a través:
    - 3 índices NMRI (T2 agua, fracción grasa);
    - Los índices Nueve 31P NMRS: 8 cocientes metabólicos que combinan:
    trifosfato de adenosina (ATP), fosfocreatina (PCr), mono fosfato y diésteres (PME, PDE) y dos piscinas de fosfato inorgánico: citosólica (Pia) y la combinación alcalina notable presente en el músculo distrófico (Pib), y los correspondientes valores ponderados de pH para las dos combinaciones Pi.
    - 23Na NMRS también pueden ser incluidos.

    La participación en la RM / MRS requiere viajar al Instituto de Miología en París y será opcional.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacokinetics:
    At day 1, for half of the patients, samples will be taken at screening or D1 and at (0.5 - 1h), (1 - 2h), (2.5 - 3.5h), 6h after the first dose. For the other half of the patients, samples will be taken before at screening or D1 and at (0.5 - 1h), (1 - 2h), (2.5 - 3.5h), 6h after the second dose. In this way, samples will be taken up to 12h after the first dose, corresponding to approximately 3 half-lives. At week 4, samples will be taken at (0.5 - 1h), 6h after the last dose.

    Pharmacodynetics: Blood samples will be collected at D1 before rimeporide administration and then at week 4 after the last rimeporide dose.

    Exploratory enpoints: Just before first dose of rimeporide and at the end of treatment, between day 21 and day 27, patient will undergo to a MRI/MRS.
    Farmacocinética:
    En el día 1, la mitad de los pacientes, las muestras se tomarán en el cribado o D1 y a (0,5-1h), (1-2h), (2,5-3,5 h), 6h después de la 1ª dosis.
    Para la otra mitad de los pacientes, las muestras se tomarán antes en el cribado o D1 y al (0,5-1 h), (1-2 h), (2,5-3,5 h), 6h después de la segunda dosis. Así,las muestras se tomarán hasta un máx. 12h tras la 1ª dosis, que corresponde aprox. a 3 medias vidas. En la S4, las muestras se tomarán a (0,5 - 1 h), 6h tras la última dosis.

    Farmacodinámica: Las muestras de sangre se recogerán el D1 antes de la admón rimeporida y en la S4 tras la última dosis de rimeporida.

    C. exploratorios: Justo antes de la primera dosis de rimeporide y al final del tratamiento, entre el día 21 y el día 27, los pacientes serán
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    first adminitration in Children with DMD (tested in phase 1 in healthy subject and CHF patients)
    primera administración en niños con DMD (probada en fase 1 en sujetos sanos y pacientes CHF
    E.7.2Therapeutic exploratory (Phase II) No
    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 grupo secuencial de dosis orales ascendentes de IMP
    sequential-group study of ascending oral doses of IMP
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Italy
    Spain
    Switzerland
    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
    LVLS
    Ultima visita del 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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 20
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    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
    DMD is characterized by a progressive loss of skeletal muscle and degeneration that lead to a loss of independent ambulation before 13y without disease modifying treatment. For this reason, to satisfy inclusion criteria, this age range is required.
    DMD se caracteriza por una pérdida progresiva del músculo esqult. que
    conduce a una pérdida de la deambulación independiente antes de los
    13a sin tto. modificador. Para satisfacer los criterios de inclusión, se
    requiere este rango de edad.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    In this pilot study, no further treatment beyond Week 4 is planned as no efficacy is claimed by this study. Subject who has ended the participation in the trial, will be treated according to the standard of care at the site as specific rescue therapy is foreseen for DMD. . However in case of positive data that will allow the sponsor to move forward with a pivotal study, participation of patients already enrolled in this study will be prioritized in the pivotal study, if the patient wish so.
    En este estudio, no se ha previsto ningún tto adicional más allá de la semana 4 ya que no se reclama ninguna eficacia. El paciente que haya puesto fin a la participación en el ensayo, se tratará de acuerdo con el estándar de cuidado en el sitio ya que está prevista terapia de rescate específica. Sin embargo, en el caso datos positivos que permitan al promotor seguir adelante con el estudio, se dará prioridad a la participación de los pacientes que ya están inscritos,si el paciente así lo desea.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Duchenne Parent Project Italy
    G.4.3.4Network Country Italy
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation Duchenne Parent Project Spain
    G.4.3.4Network Country Spain
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Duchenne Parent Project Netherlands
    G.4.3.4Network Country Netherlands
    G.4 Investigator Network to be involved in the Trial: 4
    G.4.1Name of Organisation AFM_ Téléthon
    G.4.3.4Network Country France
    G.4 Investigator Network to be involved in the Trial: 5
    G.4.1Name of Organisation Muscular Dystrophy Campaign
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 6
    G.4.1Name of Organisation Action Duchenne
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 7
    G.4.1Name of Organisation FSRMM
    G.4.3.4Network Country Switzerland
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-11-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-30
    P. End of Trial
    P.End of Trial StatusCompleted
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