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    Summary
    EudraCT Number:2012-004527-20
    Sponsor's Protocol Code Number:PTC124-GD-020-DMD
    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:2013-07-12
    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 number2012-004527-20
    A.3Full title of the trial
    A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
    Estudio de fase 3 sobre la eficacia y la seguridad del Atalureno (PTC124) en pacientes con distrofinopatía por mutación terminadora
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy
    Estudio del Atalureno en pacientes con distrofia muscular de Duchenne y de Becker por mutación terminadora
    A.3.2Name or abbreviated title of the trial where available
    N/A
    A.4.1Sponsor's protocol code numberPTC124-GD-020-DMD
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01826487
    A.5.4Other Identifiers
    Name:IND NumberNumber:68,431
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/202/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPTC 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 supportPTC Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVoisin Consulting
    B.5.2Functional name of contact pointClinical Trial Unit
    B.5.3 Address:
    B.5.3.1Street Address3 rue des Longs Prés
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.3.4CountryFrance
    B.5.6E-mailclinicaltrialinformation@voisinconsulting.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/3/05/278
    D.3 Description of the IMP
    D.3.1Product nameataluren
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Granules for oral suspension
    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 INNataluren
    D.3.9.1CAS number 775304-57-9
    D.3.9.2Current sponsor codePTC124
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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 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/3/05/278
    D.3 Description of the IMP
    D.3.1Product nameataluren
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Granules for oral suspension
    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 INNataluren
    D.3.9.1CAS number 775304-57-9
    D.3.9.2Current sponsor codePTC124
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 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/3/05/278
    D.3 Description of the IMP
    D.3.1Product nameataluren
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Granules for oral suspension
    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 INNataluren
    D.3.9.1CAS number 775304-57-9
    D.3.9.2Current sponsor codePTC124
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 placeboGranules for oral suspension
    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
    Nonsense mutation dystrophinopathy
    Distrofinopatía por mutación terminadora
    E.1.1.1Medical condition in easily understood language
    Duchenne and Becker muscular dystrophy
    Distrofia muscular de Duchenne y de Becker
    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 10059117
    E.1.2Term Becker's muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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
    To determine the ability of ataluren to slow disease progression as assessed by ambulatory decline (decrease in 6MWD) in patients with nonsense mutation dystrophinopathy.
    Determinar la capacidad del atalureno para ralentizar la progresión de la enfermedad según la evaluación del declive de la capacidad de marcha (reducción de la 6MWD) en pacientes con distrofinopatía por mutación terminadora.
    E.2.2Secondary objectives of the trial
    To determine the effect of ataluren on:
    - Ambulation
    - Proximal muscle function
    - Physical function
    - Patient and/or parent reported activities of daily living and disease symptoms
    - Parent-reported HRQL
    - Safety parameters
    - Compliance
    - Exposure of ataluren in plasma
    Determinar el efecto del atalureno sobre:
    -Ambulación
    -Función del músculo proximal
    -Función física
    -Síntomas de la enfermedad y actividades de la vida cotidiana comunicadas por parte del paciente y/o sus progenitores
    -CdVRS comunicada por los progenitores
    -Parámetros de seguridad
    -Cumplimiento
    -Exposición de atalureno en plasma
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial
    2. Male sex
    3. Age greater than or equal to 7 and less than or equal to 16 years.
    4. Phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical symptoms or signs (eg, proximal muscle weakness, waddling gait, and Gowers' maneuver) by 6 years of age, an elevated serum CK, and ongoing difficulty with ambulation.
    5. Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by College of American Pathologists (CAP), Clinical Laboratory Improvement Act/Amendment (CLIA), or an equivalent organization.
    6. Documentation that a blood sample has been drawn for confirmation of the presence of a nonsense mutation in the dystrophin gene.
    7. Use of systemic corticosteroids (prednisone, prednisolone, or deflazacort) for a minimum of 6 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen (not related to body weight change) for a minimum of 3 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
    8. Valid Screening 6MWD more than or equal to 150 meters. Valid Screening 6MWD must be less than or equal to 80% of predicted for age and height [Geiger 2007].
    9.Results of the 2 Baseline 6MWD results must be determined as valid and results of the Day 2 Baseline 6MWD must be within 20% of the Day 1 Baseline 6MWD.
    10. Baseline 6MWD (mean valid Day 1 and Day 2 values) must be no more than a 20% reduction from the valid Screening 6MWD.
    11. Confirmed screening laboratory values within the central laboratory ranges specified in in the protocol.
    12. In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 6-week follow-up period.
    13. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
    1. Constancia de los documentos de consentimiento informado/asentimiento firmados y fechados en los que se indique que se ha informado al sujeto (o a su progenitor/tutor legal) de todos los aspectos pertinentes del ensayo.
    2. Ser varón.
    3. Edad entre 7 y 16 años, ambos inclusive.
    4. Pruebas fenotípicas de distrofinopatía basadas en el inicio de los síntomas o signos clínicos característicos (por ejemplo, debilidad muscular proximal, marcha de pato y maniobra de Gowers) a los 6 años de edad, elevación de la CK sérica y dificultad en curso con la deambulación.
    5. Documentación de la presencia de una mutación puntual terminadora en el gen de la distrofina, según se determine mediante la secuenciación génica realizada por un laboratorio certificado por el Colegio Estadounidense de Patólogos (College of American Pathologists, CAP), la Ley/Enmienda de Mejora de los Laboratorios Clínicos (Clinical Laboratory Improvement Act/Amendment, CLIA) o una organización equivalente.
    6. Documentación de que se ha extraído una muestra de sangre para confirmar la presencia de una mutación terminadora en el gen de la distrofina.
    7. El uso de corticosteroides sistémicos (prednisona, prednisolona o deflazacort) durante un mínimo de 6 meses inmediatamente antes del inicio del estudio, sin cambio importante en la dosis o pauta posológica (que no esté relacionado con un cambio del peso corporal) durante al menos los 3 meses anteriores al inicio del tratamiento del estudio y expectativa razonable de que la dosis y la pauta posológica no cambiarán de forma significativa durante el transcurso del estudio.
    8. Resultado de 6MWD más de o igual a 150 metros válido en el momento de la selección. La 6MWD de selección válida debe ser menos de o igual a 80 % de lo previsto para su edad y altura [Geiger 2007].
    9. Los resultados de las 2 6MWD iniciales deben determinarse válidos y la diferencia entre los resultados de la 6MWD inicial del día 2 y la del día 1 debe ser inferior al 20 %.
    10. La 6MWD inicial (media de los valores válidos de los días 1 y 2) no puede ser inferior en más de un 20 % a la 6MWD de selección válida.
    11. Valores de selección del laboratorio confirmados comprendidos dentro de los intervalos del laboratorio central que se especifican en el protocolo.
    12. En el caso de los sujetos sexualmente activos, deben estar dispuestos a abstenerse de mantener relaciones sexuales o a utilizar un método anticonceptivo de barrera o médico durante la administración del fármaco del estudio y el período de seguimiento de 6 semanas.
    13. Voluntad y capacidad para cumplir con las visitas programadas, el plan de administración del fármaco, los procedimientos del estudio, las pruebas de laboratorio y las restricciones del estudio
    E.4Principal exclusion criteria
    1. Treatment with systemic aminoglycoside antibiotics within 3 months prior to start of study treatment.
    2. Initiation of systemic corticosteroid therapy within 6 months prior to start of study treatment.
    3. Change in systemic corticosteroid therapy (eg, change in type of drug, dose modification not related to body weight change, schedule modification, interruption, or reinitiation) within 3 months prior to start of study treatment.
    4. Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to start of study treatment.
    5. Ongoing use of coumarin-based anticoagulants (eg. warfarin), phenytoin, tolbutamide, or paclitaxel.
    6. Prior therapy with ataluren.
    7. Known hypersensitivity to any of the ingredients or excipients of the study drug [eg. refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate]
    8. Exposure to another investigational drug within 3 months prior to start of study treatment.
    9. History of major surgical procedure within 6 weeks prior to start of study treatment
    10. Ongoing immunosuppressive therapy (other than corticosteroids)
    11. Ongoing participation in any clinical trial (except for studies specifically approved by PTC Therapeutics)
    12. Expectation of major surgical procedure (eg, scoliosis surgery) during the 12-month treatment period of the study.
    13. Requirement for daytime ventilator assistance.
    14. Uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D) [Hunt 2001]
    15. Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings (eg. lower limb injury that may affect 6MWT performance), ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
    1. Tratamiento con antibióticos aminoglucósidos sistémicos en los 3 meses anteriores al inicio del tratamiento del estudio.
    2. Inicio del tratamiento con corticosteroides sistémicos en los 6 meses anteriores al inicio del tratamiento del estudio.
    3. Cambio en el tratamiento con corticosteroides sistémicos (por ejemplo, cambio en el tipo de fármaco, modificación de la dosis no relacionada con cambios en el peso corporal, modificación, interrupción o reinicio de la pauta posológica) en los 3 meses anteriores al inicio del tratamiento del estudio.
    4. Cualquier cambio (inicio, cambio de tipo de fármaco, modificación de la dosis, modificación del intervalo de administración, interrupción, suspensión o reinicio) en la profilaxis/el tratamiento crónico de la insuficiencia cardíaca congestiva (ICC) en los 3 meses antes del inicio del tratamiento del estudio.
    5. Uso en curso de anticoagulantes cumarínicos (como la warfarina), fenitoína, tolbutamida o paclitaxel.
    6. Tratamiento anterior con atalureno.
    7. Hipersensibilidad conocida a alguno de los ingredientes o excipientes del fármaco del estudio [polidextrosa refinada, polietilenglicol 3350, poloxámero 407, manitol 25C, crospovidona XL10, hidroxietil celulosa, sílice coloidal o estearato de magnesio].
    8. Exposición a otro fármaco en investigación en los 3 meses anteriores al inicio del tratamiento del estudio.
    9. Antecedentes de procedimiento quirúrgico mayor en las 6 semanas anteriores al inicio del tratamiento del estudio.
    10. Terapia inmunodepresora en curso (diferente de los corticosteroides).
    11. Participación en curso en algún ensayo clínico (excepto por estudios específicamente aprobados por PTC Therapeutics).
    12. Previsión de algún procedimiento quirúrgico mayor (por ejemplo, operación quirúrgica por escoliosis) durante el período de tratamiento de 12 meses del estudio.
    13. Necesidad de ayuda de respirador durante el día.
    14. Signos y síntomas clínicos no controlados de ICC (estadios C o D de la American College of Cardiology/American Heart Association) [Hunt 2001].
    15. Afección médica anterior o en curso (p. ej.: enfermedad concomitante, trastorno psiquiátrico, trastorno conductual, alcoholismo, drogadicción), antecedentes médicos, datos obtenidos en la exploración física (como una lesión en las extremidades inferiores que pudiera afectar al resultado de la 6MWT) o en los ECG, o anomalías en los valores de laboratorio que, en opinión del investigador, podrían afectar de forma adversa a la seguridad del sujeto, que hacen improbable que se completen el curso de tratamiento o el seguimiento, o que podrían afectar a la evaluación de los resultados del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Change in 6MWD
    Cambio en la 6MWD
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening visit and every 8 weeks
    Visita de selección y cada 8 semanas
    E.5.2Secondary end point(s)
    1. Time to 10% persistent worsening in 6MWD
    2. Proportion of patients with ?more than or equal to 10% worsening in 6MWD at Week 48
    3. Change in %-predicted 6MWD
    4. Change in timed function tests (time to walk/run 10 meters and time to climb/descend 4 stairs)
    5. Change in physical function as measured by the NSAA
    6. Change in patient and/or parent/caregiver reports of activities of daily living as measured by a standardized survey administered by site personnel
    7. Change in PODCI Transfers/Basic Mobility and Sports/Physical Functioning scores
    8. Safety profile characterized by type, frequency, severity, timing, and relationship to study drug of any adverse events, or of abnormalities of laboratory tests, vital signs, physical examinations, or ECGs
    9. Study drug compliance as assessed by quantification of unused study drug
    10. Plasma concentration as assessed by a validated bioanalytical method
    1.Tiempo hasta el empeoramiento persistente del 10 % en 6MWD
    2. Proporción de pacientes con un empeoramiento de más de o igual a 10 % en 6MWD en la semana 48
    3. Cambio en el porcentaje predicho de 6MWD
    4. Cambio en las pruebas funcionales cronometradas (el tiempo necesario para caminar/correr 10 metros y tiempo necesario para subir/bajar 4 escalones)
    5. Cambio en la función física según la medición con el NSAA
    6. Cambios en las actividades de la vida cotidiana comunicados por el paciente y/o sus progenitores/cuidador según su medición mediante una encuesta normalizada administrada por personal del estudio
    7. Cambio en las puntuaciones de función deportiva/física y de movilidad básica/transferencia de PODCI
    8. Perfil de seguridad caracterizado por el tipo, la frecuencia, la gravedad, el momento y la relación con el fármaco del estudio de cualquier acontecimiento adverso o anomalía de laboratorio, constantes vitales, exploración física o ECG
    9.Cumplimiento con el fármaco del estudio, valorado según la cuantificación del fármaco del estudio sin usar
    10. Concentraciones plasmáticas valoradas mediante un método bioanalítico validado
    E.5.2.1Timepoint(s) of evaluation of this end point
    1, 3, 4, 5, 6, 7: Screening visit and every 8 weeks
    2. Week 48
    8. Adverse events: every 8 weeks until end of treatment, then 6 weeks post-treatment
    Laboratory tests and vital signs: Screening, every 8 weeks until end of treatment, then 6 weeks post-treatment
    Physical examinations: Screening, week 1, week 24, week 48 and 6 weeks post-treatment
    ECGs: Screening, week 48 and 6 weeks post-treatment
    9, 10: Every 8 weeks from week 8
    1, 3, 4, 5, 6, 7 : Visita de selección y cada 8 semanas
    2. Semana 48
    8. Efectos adversos : cada 8 semanas hasta el final del tratamiento, y 6 semanas postratamiento
    Parámetros de laboratorio y signos vitales : Visita de selección, cada 8 semanas hasta el final del tratamiento, y 6 semanas postratamiento
    Exámenes físicos : Visita de selección, semana 1, semana 24, semana 48 y 6 semanas postratamiento
    ECGs: Visita de selección, semana 48 y 6 semanas postratamiento
    9,10: Cada 8 semanas desde la semana 8
    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 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 No
    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 trial2
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Australia
    Brazil
    Canada
    Chile
    Israel
    Korea, Republic of
    Switzerland
    Turkey
    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
    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 months3
    E.8.9.1In the Member State concerned days17
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days17
    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: 220
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 110
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 110
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 and Adolescents
    Niños y adolescentes
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 220
    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)
    At the completion of blinded treatment, an open-label extension study will be available to patients (who successfully complete the double-blind study) in countries where ataluren is not commercially available.
    Al finalizar el tratamiento enmascarado, se pondrá a disposición de los pacientes (que hayan terminado con éxito el estudio doble ciego) un estudio de extensión abierto en los países en los que el atalureno no esté comercializado.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-07-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-08-20
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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