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    EudraCT Number:2011-001266-17
    Sponsor's Protocol Code Number:DMD114349
    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:2011-10-03
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2011-001266-17
    A.3Full title of the trial
    An open-label extension study of the long-term safety,
    tolerability and efficacy of GSK2402968 in subjects with
    Duchenne Muscular Dystrophy
    Estudio de extensión abierto para evaluar la seguridad, tolerabilidad y eficacia a largo plazo de GSK2402968 en sujetos con distrofia muscular de Duchenne.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open-label extension study of the long-term safety,
    tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy
    Estudio de extensión abierto para evaluar la seguridad, tolerabilidad y eficacia a largo plazo de GSK2402968 en sujetos con distrofia muscular de Duchenne.
    A.4.1Sponsor's protocol code numberDMD114349
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/54/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGlaxoSmithKline, S.A.
    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 supportGlaxoSmithKline
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlaxoSmithKline
    B.5.2Functional name of contact pointClinical Trials Helpdesk
    B.5.3 Address:
    B.5.3.1Street AddressIron Bridge Road
    B.5.3.2Town/ cityUxbridge
    B.5.3.3Post codeUB11 1BU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442089904466
    B.5.5Fax number+442089901234
    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/3/08/599
    D.3 Description of the IMP
    D.3.1Product nameGSK2402968
    D.3.2Product code GSK2402968
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNGSK2402968
    D.3.9.2Current sponsor codeGSK2402968
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    Distrofia muscular de Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy (DMD)
    Distrofia muscular de Duchenne (DMD)
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    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 evaluate the long term safety, tolerability and efficacy of subcutaneous 6mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044.
    Evaluar la seguridad, tolerabilidad y eficacia a largo plazo de GSK2402968 6 mg/kg/semana SC en sujetos con DMD que hayan participado en los estudios DMD114117 o DMD114044.
    E.2.2Secondary objectives of the trial
    - To evaluate the long-term PK of subcutaneous 6 mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044.
    - To evaluate the long-term impact on health-related quality of life (HRQoL) and functional outcomes of continued treatment with GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044.
    -To evaluate DMD disease progression and outcomes (clinical, HRQoL and functional) in subjects who discontinue active treatment during the conduct of study (natural history component).
    - To evaluate the long-term safety, efficacy and PK of an intermittent dosing option in those subjects unable to tolerate GSK2402968 6mg/kg/week dosing.
    - Evaluar la FC (farmacocinética) a largo plazo de GSK2402968 6 mg/kg/semana por vía SC en sujetos con DMD que hayan participado en los estudios DMD114117 o DMD114044.
    - Evaluar el impacto a largo plazo sobre la calidad de vida relacionada con la salud (CdVRS) y los resultados funcionales del tratamiento continuado con GSK2402968 en sujetos con DMD que hayan participado en los estudios DMD114117 o DMD114044.
    - Evaluar la progresión de la enfermedad y los resultados (clínicos, CdVRS y funcionales) en la DMD en los sujetos que interrumpan el tratamiento activo durante el estudio (componente de observación de la historia natural de la enfermedad).
    - Evaluar la seguridad, eficacia y FC a largo plazo de una dosis intermitente en los sujetos que no toleren la dosis de GSK2402968 6mg/kg/semana.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects eligible for enrolment in the study must meet all of the following criteria:
    1. Subjects who have successfully completed either the DMD114117 study or DMD114044 study, OR
    Subjects who withdrew due to safety/tolerability issues, which have since resolved and where in the opinion of the PI along with consultation of the Medical Monitor, it is considered that the benefit of further treatment with GSK2402968 outweighs the risk to the individual subject. OR
    Subjects who either complete or withdraw early from studies DMD114117 or DMD114044, who do not wish to continue treatment with GSK2402968 but who are willing to participate in the natural history observation arm.
    2. Continued use of glucocorticoids with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticosteroids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor.
    3. Willing and able to comply with all protocol requirements and procedures,
    4. Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
    5. French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
    Los sujetos deberán cumplir todos los criterios siguientes para poder ser incluidos en el estudio:
    1. Sujetos que hayan completado con éxito el estudio DMD114117 o el estudio DMD114044, O BIEN
    Sujetos que se hayan retirado por problemas de seguridad/tolerabilidad que ya se hayan resuelto y en los que, en opinión del IP en consulta con el monitor médico, se considere que el beneficio del tratamiento con GSK2402968 supera a los riesgos para el sujeto. O BIEN
    Sujetos que hayan completado o se hayan retirado prematuramente de los estudios DMD114117 o DMD114044 y no deseen continuar el tratamiento con GSK2402968 pero quieran participar en el grupo de observación de la historia natural de la enfermedad.
    2. Uso continuo de glucocorticoides con previsión razonable de que el sujeto siga tomándolos durante el estudio. Los cambios o la interrupción de los glucocorticoides quedarán a discreción del IP, en consulta con el sujeto/progenitor y el monitor médico.
    3. Disposición y capacidad para cumplir todos los requisitos y procedimientos del protocolo,
    4. Capacidad para dar el asentimiento o consentimiento informado por escrito, firmado por el sujeto o su progenitor o tutor legal (de acuerdo con las leyes locales).
    5. Sujetos franceses: En Francia sólo se podrá incluir en este estudio a los sujetos afiliados o beneficiarios de alguna categoría de la seguridad social.
    E.4Principal exclusion criteria
    Subjects meeting any of the following criteria must not be enrolled in the study:
    1. Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing at Visit 2. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor.
    2. Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for GSK2402968, within 1 month of the first administration of study medication.
    3. Current or anticipated participation in any other investigational clinical studies
    4. History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current or history of renal or liver disease/impairment, history of inflammatory illness.
    No se incluirá en el estudio a los sujetos que cumplan alguno de los criterios siguientes:
    1. Sujetos con un acontecimiento adverso grave o en los que se haya cumplido alguno de los criterios de interrupción de la administración del fármaco en los estudios DMD114117 o DMD114044 y continúe sin resolverse, siempre que, en opinión del investigador, pueda atribuirse a la medicación del estudio y que continúe en la visita 2. Una vez resuelto el acontecimiento o criterio, los sujetos podrán participar, previa consulta del IP con el monitor médico.
    2. Uso de anticoagulantes, antitrombóticos o antiplaquetarios, o tratamiento previo con fármacos en investigación, excepto GSK2402968, en el mes previo a la primera administración del fármaco del estudio.
    3. Participación actual o prevista en otros estudios clínicos de investigación.
    4. Antecedentes de trastorno médico importante que pueda confundir la interpretación de los datos de eficacia o de seguridad, como enfermedad/insuficiencia renal o hepática actual o previa, o antecedentes de enfermedad inflamatoria.
    E.5 End points
    E.5.1Primary end point(s)
    Primary efficacy endpoint:
    - Muscle function using 6 minute walking distance (6MWD) test.
    Variable principal de valoración de la eficacia:
    - Función muscular utilizando la prueba prueba TM6M (distancia caminada en 6 minutos)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weeks 12,24,48,72 and Week 104 or Early withdrawal
    Semanas 12, 24, 48, 72 y Semana 104 o Visita de retirada prematura.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints:
    -Timed function tests (times and grading):
    . Rise from floor
    . 4 stair climb
    . 10m walk/run
    - Muscle strength (total score): knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors (as determined by handheld myometery)
    - North Star Ambulatory Assessment
    - Creatine kinase serum concentrations
    - Pulmonary function (FEV1, FVC, PCF, PF, sniff pressure test)
    - Pediatric Quality of Life Neuromuscular module (PedsQL)
    - Clinician Global Impression of Improvement (CGI-I)
    - Health Utilities Index (HUI)
    - Time to major disease milestones (e.g. loss of ambulation, night time ventilation)
    - Frequency of accidental falls (during 6MWD)
    - Functional Outcomes assessment
    Safety endpoints:
    - Incidence and severity of adverse events.
    - Vital signs
    - ECG parameters
    - Safety haematology and biochemistry parameters including non-standard parameters such as coagulation parameters (in particular aPTT), serum cystatin C, Complement Factor C3, haptoglobin, fibrinogen, hsCRP, MCP-1
    - Urinalysis (including quantitative protein, creatinine and ratio, urine cystatin, KIM-1 and ?1-microglobulin)
    - Echocardiogram
    - AUC[0-24], AUC[0-7d],
    - Cmax
    - tmax
    - C24h,
    - C7d,
    DEXA Scan
    Lean body mass and bone density (exploratory endpoint)
    Assess changes in fat infiltration and oedematous inflammation in skeletal muscle in the thigh as determined by structural MRI measures over time in those subjects who had a baseline MRI in the DMD114044 study.
    Variables secundarias de la eficacia:
    - Pruebas funcionales cronometradas (tiempos y puntuación):
    . Levantarse del suelo
    . Subir 4 escalones
    . Marchar/correr 10 m
    - Fuerza muscular (puntuación total): flexores de la rodilla, extensores de la rodilla, flexores del codo, extensores del codo, abductores del hombro y flexores de la cadera (lo que se determine mediante miometría manual)
    - North Star Ambulatory Assessment (Evaluación Ambulatoria North Star)
    - Concentraciones de creatina quinasa en suero
    - Pruebas de la función pulmonar (FEV1, FVC, PCF, PF, Test de presión inspiratoria-Sniff pressure test)
    - Módulo neuromuscular de calidad de vida pediátrica (PedsQL)
    - Impresión clínica global de mejoría (CGI-I)
    - Índice de utilidades de salud (HUI)
    - Tiempo hasta los principales hitos de la enfermedad (por ejemplo, pérdida de ambulación, ventilación nocturna)
    - Frecuencia de caídas accidentales (durante el TM6M)
    - Evaluación de los resultados funcionales

    Variables de seguridad:
    - Incidencia y gravedad de los acontecimientos adversos
    - Constantes vitales
    - Parámetros del ECG
    - Parámetros hematológicos y bioquímicos de seguridad, incluidos parámetros no habituales como los de coagulación (en particular, el TTPa), cistatina C en suero, factor del complemento C3, haptoglobina, fibrinógeno, PCRas, MCP-1
    - Análisis de orina (con cuantificación de proteínas, creatinina y proporción proteína/creatinina, cistatina en orina, KIM-1 y alfa1-microglobulina)
    - Ecocardiograma
    - AUC[0-24], AUC[0-7d],
    - Cmáx
    - tmáx
    - C24h,
    - C7d,
    DEXA Scan
    Masa corporal magra y densidad ósea (variable exploratoria)
    RM (Resonancia Magnética)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Functional secondary endpoints and collection of questionnaires will be performed at the same intervals as the primary endpoint. Samples for urinalysis and safety labs will be collected Baseline and every two weeks thereafter till visit 104 or Early withdrawal. Predose PK samples will be collected monthly. Serial PK at either Week 12 or 48: prior to treatment and 1/2, 2, 4 hours and between 9-12 hours and 48-72 hours after treatment. Vital signs & ECG Weeks 4,8,16,24,32,40,48,60,72,84,96, and Week 104 or Early withdrawal. Echo & DEXA: Weeks 24, 48, 72, and Week 104 or Early withdrawal.
    Las variables secundarias funcionales y los cuestionarios se realizarán en los mismos puntos que la variable principal. Las muestras para análisis de orina y muestras de laboratorio de seguridad se recogerán en la visita basal y cada dos semanas a partir de entonces hasta la visita de la semana 104 o la visita de retirada prematura. Las muestras FC predosis se recogerán una vez al mes. La FC seriada en la semana 12 o 48: antes de la dosis y después de la dosis a las siguientes horas: 0.5, 2, 4 horas, entre 9-12 horas, 24 horas y entre 48 72 horas. Los signos vitales y ECG: semanas 4,8,16, 24,32, 40, 48, 60, 72, 84, 96 y semana 104 o retirada prematura. Ecocardiograma y DEXA: semanas 24, 48, 72 y semana 104 o retirada prematura.
    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. 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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 No
    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 No
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA27
    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
    Czech Republic
    Korea, Republic of
    Russian Federation
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    This study has a duration of a minimum of 2 years or until treatment is commercially available locally.
    Este estudio durará 2 años como mínimo o hasta que esté comercialmente disponible el tratamiento localmente.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 200
    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. of subjects for this age range: 180
    F.1.1.6Adolescents (12-17 years) Yes
    F. 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. of subjects incapable of giving consent
    Assent and written informed consent must be obtained from each subject and/or subject's parent/legal guardian, in accordance with applicable local regulatory guidelines, prior to participation in the study.
    Se obtendrá el asentimiento/consentimiento informado por escrito de cada sujeto y/o progenitor/tutor del sujeto, de acuerdo con los requisitos reglamentarios locales, antes de la participación en el estudio.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 91
    F.4.2.2In the whole clinical trial 200
    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)
    The investigator is responsible for ensuring that consideration has been given to the poststudy care of the subject?s medical condition whether or not GSK is providing specific post study treatment.
    El investigador es responsable de garantizar que se valora la asistencia posterior al estudio del estado médico del sujeto, con independencia de que GSK suministre un tratamiento específico después del mismo.
    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 No aplica
    G.4.3.4Network Country Spain
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-11-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-11-07
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2014-03-17
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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