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    Summary
    EudraCT Number:2013-001194-25
    Sponsor's Protocol Code Number:H6D-MC-LVJJ
    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:2013-06-26
    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 number2013-001194-25
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy
    Estudio de fase 3, aleatorizado, doble ciego, controlado con placebo, para evaluar tadalafilo en la distrofia muscular de Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.
    Ensayo clinico para evaluar tadalafilo en la distrofia muscular de Duchenne en niños
    A.4.1Sponsor's protocol code numberH6D-MC-LVJJ
    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 SponsorLilly S.A.
    B.1.3.4CountrySpain
    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 supportEli Lilly
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEli Lilly
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street AddressAvda de la Industria 30
    B.5.3.2Town/ cityAlcobendas Madrid
    B.5.3.3Post code28108
    B.5.3.4CountrySpain
    B.5.4Telephone number34916635354
    B.5.5Fax number34916633481
    B.5.6E-mailEU_Lilly_Clinical_Trials@lilly.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 Yes
    D.2.1.1.1Trade name Cialis 2.5 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderEli Lilly Nederland B.V.
    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 INNTADALAFIL
    D.3.9.1CAS number 171596-29-5
    D.3.9.2Current sponsor codeLY450190
    D.3.9.4EV Substance CodeSUB12602MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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.IMP: 2
    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 Cialis 5 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderEli Lilly Nederland B.V.
    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 INNTADALAFIL
    D.3.9.1CAS number 171596-29-5
    D.3.9.2Current sponsor codeLY450190
    D.3.9.4EV Substance CodeSUB12602MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.IMP: 3
    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 Cialis 10 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderEli Lilly Nederland B.V.
    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 INNTADALAFIL
    D.3.9.1CAS number 171596-29-5
    D.3.9.2Current sponsor codeLY450190
    D.3.9.4EV Substance CodeSUB12602MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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.IMP: 4
    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 Cialis 20 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderEli Lilly Nederland B.V.
    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 INNTADALAFIL
    D.3.9.1CAS number 171596-29-5
    D.3.9.2Current sponsor codeLY450190
    D.3.9.4EV Substance CodeSUB12602MIG
    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
    D.8 Placebo: 2
    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
    D.8 Placebo: 3
    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
    D.8 Placebo: 4
    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
    Fatal muscle wasting disease
    Atrofia muscular progresiva
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    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
    The primary objective is to test the hypothesis that once daily tadalafil administered orally for 48 weeks lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD).
    El objetivo principal es evaluar la hipótesis de si tadalafilo (administrado por vía oral, una vez al día, durante 48 semanas) reduce el deterioro de la capacidad ambulatoria en niños con distrofia muscular de Duchenne (DMD), de acuerdo con la distancia recorrida en la prueba de marcha de 6 minutos, y en comparación con los pacientes tratados con placebo. Se compararán con placebo dos dosis de tadalafilo (0,3 mg/kg y 0,6 mg/kg).
    E.2.2Secondary objectives of the trial
    Test the hypothesis that once daily tadalafil administered orally for 48 weeks compared with placebo in boys with DMD:
    o lessens the decline in North Star Ambulatory Assessment (NSAA) global score,
    o lessens the decline in performance on timed function tests: rise from floor from supine, 10 meter walk/run, stair climb and stair descend,
    o delays the time to persistent 10% worsening in the 6MWD,
    o delays the time to persistent 10% worsening in timed function tests: rise from floor from supine, 10 meter walk/run test, stair climb, and stair descend,
    o lessens the decline in Quality of Life (QoL), as measured by the Pediatric Outcomes Data Collection Instrument (PODCI) global functioning scale and the following core scales: Upper Extremity/Physical Functioning, Transfer/Basic Mobility and Sports/Physical Functioning.
    o Characterize the PK of tadalafil in pediatric DMD patients, and assess relationships between tadalafil exposure and efficacy and safety outcomes.
    Los objetivos secundarios del estudio son:
    ? Evaluar hipótesis niños con DMD y comparación con placebo, tadalafilo (oral, una vez al día, 48 semanas):
    o una menor disminución de la puntuación global en la Evaluación NSAA,
    o Reduce deterioro en desempeño del paciente en distintas pruebas funcionales cronometradas
    o Prolonga tiempo transcurrido hasta que se observe un empeoramiento persistente del 10% en la distancia recorrida prueba de marcha de 6 MWT
    o Prolonga tiempo transcurrido hasta que se observe un empeoramiento persistente del 10% en las pruebas funcionales cronometradas
    o Reduce deterioro en la calidad de vida del paciente, con la escala PODCI y escalas: extremidades superiores/función física, transferencia/movilidad básica y deportes/funcionalidad física
    ? Caracterizar farmacocinética de tadalafilo en niños con DMD, y evaluar la relación entre la exposición a tadalafilo y los resultados de eficacia y seguridad.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ? Males with proven DMD
    ? Ages 7-14 years inclusive
    ? Ambulant, defined as baseline 6MWD between 200 and 400 meters inclusive
    ? 6MWD measurements within 20% of each other at a minimum of 2 pre-randomization assessments
    ? Left ventricular ejection fraction (LVEF) ?50%
    ? Receiving corticosteroids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen (except those adjusting for weight changes) for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly (except for adjustments for weight) for the duration of the study. Corticosteroid treatment regimen can be daily, intermittent, high-dose weekend, or alternate days, but must be consistent with current clinical care recommendations.
    [1] Varones con diagnóstico confirmado de DMD, según se define en el apartado 8.1.1.
    [2] Edad entre 7 y 14 años, ambos inclusive.
    [3] Paciente ambulatorio, esto es, que en el momento basal recorra entre 200 y 400 metros (ambos valores incluidos) en la prueba de marcha de 6 minutos.
    [4] Presentar una desviación inferior al 20% en los resultados de la prueba de marcha de 6 minutos, que deberá haberse realizado al menos en 2 ocasiones antes de la aleatorización.
    [5] Presentar una fracción de eyección del ventrículo izquierdo (FEVI) ? 50%, de acuerdo con los resultados de una ecocardiografía realizada durante el proceso de selección o en el transcurso de los 30 días previos a la visita 1 (en caso de que pueda obtenerse el registro de dicha ecocardiografía como documentación original de la FEVI correspondiente a la selección).
    [6] Haber recibido corticosteroides al menos durante los 6 meses inmediatamente anteriores al proceso de selección, sin que se hayan realizado modificaciones significativas en la dosis diaria total o en la pauta posológica (excepto aquellas modificaciones que se realicen en función de las variaciones en el peso corporal) al menos en los 3 meses inmediatamente anteriores al proceso de selección, y con expectativas razonables de que la dosis diaria total y la pauta posológica no vayan a sufrir modificaciones significativas durante el estudio (excepto aquellas que se realicen en función de las variaciones en el peso corporal). El tratamiento con corticosteroides puede administrarse diariamente, de forma intermitente o en días alternos, o pueden administrarse dosis altas los fines de semana, pero debe ser coherente con las recomendaciones clínicas actuales (Bushby et al., 2010).
    [7] Los padres/representantes legales deberán proporcionar el consentimiento informado por escrito (y los pacientes deberán proporcionar su asentimiento por escrito), antes de que se lleve a cabo ningún procedimiento del estudio.
    E.4Principal exclusion criteria
    ? Symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV)
    ? Change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or reinitiating) in prophylactic treatment for heart failure within 3 months prior to start of study treatment
    ? Cardiac rhythm disorder defined as sinus rhythm with ectopic contractions or conductance disturbances, or any rhythm other than sinus, observed on screening ECG
    ? Use of continuous mechanical ventilator assistance. [Evening use of bi-level positive airway pressure (BPAP) or continuous positive airway pressure (CPAP) therapy is allowed]
    ? Previous treatment with investigational drugs or interventions (including shock training system) within 3 months of the first administration of study medication, or planned use during the study
    ? History of participation in gene or cell-based therapy
    ? History of antisense oligonucleotide (AON) or stop codon read-through therapy
    ? Unable to take orally administered tablets (without chewing, crushing or breaking), as assessed by the investigator
    ? Use of L-arginine supplements within 4 weeks (+/- 1 day) of the first administration of study medication
    ? Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength within 3 months prior to the start of study treatment (e.g., growth hormone, anabolic steroids including testosterone). Vitamin D, calcium, and combinations thereof will be allowed.
    ? New or changed treatment with herbal or dietary supplements being taken with an expectation of an effect on muscle strength or function during 1 month prior to first dose of study drug. Patients taking herbal or dietary supplements as defined above with no change in type or dose for 1 month prior to first dose of study drug with no expectation of adding or changing supplements for the 48 week double-blind period may be enrolled.
    ? Surgery that might have an effect on muscle strength or function within 3 months before study entry or planned surgery at any time during the study
    ? Evidence of a lower limb injury that may in the judgment of the investigator affect performance on the 6MWD
    ? Severe behavioral problems, including severe autism or attention deficit disorders, that may in the judgment of the investigator interfere with completion of the 6MWD
    ? Any contraindication to tadalafil (use of any form of organic nitrate, either regularly and/or intermittently, or known serious hypersensitivity to tadalafil)
    ? History of significant renal insufficiency, defined as receiving renal dialysis or having a screening serum cystatin C level ? 2.35 mg/L
    ? Clinical evidence of cirrhosis
    ? Diagnosed with a retinal disorder (for example, hereditary retinal disorders, retinopathy of prematurity)
    ? Have severe hypotension or uncontrolled hypertension as determined by the investigator
    ? Current treatment with potent CYP3A4 inhibitors, such as antiretroviral therapy (protease inhibitor), systemic ketoconazole, or systemic itraconazole, or chronic use of potent CYP3A4 inducers, such as rifampicin
    ? Currently receiving treatment with doxazosin, nitrates, or cancer therapy
    ? Have known allergy to any of the excipients in tadalafil tablets, notably lactose
    ? Current PDE5 inhibitor therapy or treatment within the past 6 months
    ? Other medical condition deemed to place the patient at potential increased risk or reduced adherence to the study protocol
    ? History of loss of vision in 1 eye because of nonarteritic anterior ischemic optic neuropathy (NAION), regardless of whether this episode was in connection or not with previous phosphodiesterase type 5 (PDE5) inhibitor exposure.
    Se excluirá del estudio a cualquier paciente en que se constate alguno de los siguientes criterios:
    [8] Estar participando en un ensayo clínico, que se administre fármaco en fase de investigación, o se haga un uso no recogido en ficha técnica de un fármaco o dispositivo (salvo el producto en investigación que se administra en este estudio), o estar participando en cualquier otro tipo de investigación médica que se considere no es compatible con el estudio. Los pacientes que estén participando en un estudio observacional o no intervencionista podrán participar.
    [9] Miocardiopatía o insuficiencia cardiaca sintomáticas.
    [10] modificación en el tratamiento profiláctico para la insuficiencia cardiaca realizado en el transcurso de los 3 meses previos al inicio tratamiento del estudio.
    [11] Trastorno ritmo cardiaco, definido como ritmo sinusal con contracciones ectópicas o trastornos de conducción, o ritmo que no sea sinusal, que se observe en el ECG realizado durante el proceso de selección.
    [12] Uso de ventilación mecánica continua. [Se permite la administración nocturna de tratamiento con presión positiva en las vías respiratorias a dos niveles [PPVRDN] o de presión positiva continua en las vías respiratorias [PPCVR].
    [13] Haber recibido tratamiento con fármacos en fase de investigación o haberse sometido a intervenciones en fase de investigación (incluido el sistema de entrenamiento de choque en el transcurso de los 3 meses previos a la primera administración de la medicación, o tener previsto recibir dichos tratamientos o someterse a dichas intervenciones.
    [14] Antecedentes de terapia génica o terapia celular.
    [15] Antecedentes de terapia con un oligonucleótido antisentido o de terapia de lectura a través del codon de parada.
    [16] Ser incapaz de tomar comprimidos por vía oral.
    [17] Uso de complementos de L-arginina en el transcurso de las 4 semanas (+/- 1 día) previas a la primera administración de la medicación del estudio.
    [18] Uso de cualquier tratamiento farmacológico (excepto los tratamientos con corticosteroides) que pueda algún tipo efecto sobre la fuerza muscular, en el transcurso de los 3 meses previos al inicio del tratamiento . Se permite la administración de vitamina D, calcio y combinaciones de estos.
    [19] Nuevo tratamiento o modificaciones en un tratamiento con complementos dietéticos o fitoterapia, con el objetivo de ejercer algún tipo de efecto sobre la fuerza o la función muscular, en el transcurso del mes previo a la primera dosis del fármaco del estudio. Podrán ser reclutados los pacientes que estén recibiendo fitoterapia o complementos dietéticos cuyo tipo o dosis no haya sufrido ninguna modificación en el transcurso del mes previo a la primera dosis y que no tengan previsto añadir ningún otro complemento o realizar modificaciones en los tratamientos que ya estén tomando, durante el período doble ciego de 48 semanas.
    [20] Cirugía que pueda afectar a la fuerza o función muscular, que bien se haya realizado en el transcurso de los 3 meses previos a la inclusión o que esté previsto.
    [21] Indicios de una lesión en las extremidades inferiores que pueda afectar al desempeño en la prueba 6MW.
    [22] Problemas conductuales graves, que puedan dificultar la realización de la prueba 6MW.
    [23] Cualquier situación en que esté contraindicado administrar tadalafilo.
    [24] Antecedentes insuficiencia renal significativa, estar recibiendo diálisis renal o presentar una concentración sérica de cistatina C ? 2,35 mg/l.
    [25] Indicios clínicos de cirrosis.
    [26] Diagnóstico trastorno retiniano.
    [27] Hipotensión intensa o hipertensión sin controlar,
    [28] Estar recibiendo tratamiento con potentes inhibidores de la CYP3A4, como antirretrovíricos, administración sistémica de ketoconazol o itraconazol, o administración prolongada de inductores potentes de la CYP3A4, como rifampicina.
    [29] Estar recibiendo tratamiento con doxazosina o nitratos, o antineoplásicos.
    [30] Alergia a cualquier de los excipientes de los comprimidos de tadalafilo (en particular a la lactosa).
    [31] Estar recibiendo tratamiento con inhibidores de la PDE5, o haberlo recibido en de los últimos 6 meses.
    [32] Otras enfermedades que suponen un mayor riesgo o puedan implicar menor cumplimiento protocolo.
    [33] Ser personal del centro de investigación, directamente relacionado con el estudio, y/o familiares cercanos.
    [34] Ser empleados de Lilly o de una organización externa (TPO) que estén implicados en el estudio y que no puedan participar de acuerdo con sus requerimientos internos.
    [35] Haber interrumpido previamente su participación en este estudio o en cualquier otro estudio en el que se investigue tadalafilo. Este criterio de exclusión no es aplicable a los pacientes que se hayan vuelto a someter al proceso de selección previamente al reclutamiento.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy measure is the 6MWD, and the primary endpoint will be the comparison of the change in the 6MWD after 48 weeks in tadalafil and placebo patients.
    El criterio principal de valoración de la eficacia es la distancia recorrida en la prueba de marcha de 6 minutos, y el criterio principal de valoración será la comparación de la variación en la distancia recorrida en la prueba 6MW tras 48 semanas de tratamiento, entre los pacientes que reciban tadalafilo y los pacientes que reciban placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Week 0, Week 12, Week 24, Week 36, Week 48, Week 60, Week 96.
    Cribado, y a las 0, 12, 24, 36, 48, 60 y 96 semanas
    E.5.2Secondary end point(s)
    The North Star Ambulatory Assessment (NSAA)

    Timed function tests (rise from floor from supine, 10 meter walk/run, 4-stair climb/descend)

    Decline in Quality of Life (QoL), as measured by the Pediatric Outcomes Data Collection Instrument (PODCI)
    - Prueba ambulatoria North Star
    - Tiempos de los test de funcionalidad (prueba de levantarse desde el suelo (de una posición decúbito supino) y prueba de marcha/carrera de 10 metros, una prueba estándar de subir/bajar 4 peldaños)
    - Instrumento para la Obtención de Resultados Pediátricos? (PODCI), para evaluar la calidad de vida en niños con DMD
    E.5.2.1Timepoint(s) of evaluation of this end point
    NSAA and 6MWD:
    Screening, Week 0, Week 12, Week 24, Week 36, Week 48, Week 60, Week 96.

    PODCI:
    Week 0, Week 12, Week 24, Week 48,Week 96
    NSAA y 6MWD
    Cribado, y a las 0, 12, 24, 36, 48, 60 y 96 semanas
    PODCI:
    semanas 0, 12, 24, 48, 96
    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 No
    E.6.10Pharmacogenetic Yes
    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 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 EEA23
    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
    Argentina
    Belgium
    Canada
    Denmark
    France
    Germany
    Italy
    Korea, Republic of
    Netherlands
    Russian Federation
    Spain
    Taiwan
    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
    LVLS
    Ultima visita ultimo paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 306
    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: 245
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 61
    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
    Children below age of 18.
    Niños menores de 18 años
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 133
    F.4.2.2In the whole clinical trial 306
    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)
    Study drug will not be made available at the conclusion of the study. Patients will be referred to their local treatment centers for appropriate therapy.
    La medicacion no estara disponible a la finalizacion del estudio. Los pacientes seran tratados con el tratamiento habitual para su enfermedad
    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 Decision2013-08-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-26
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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