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    Summary
    EudraCT Number:2015-002069-52
    Sponsor's Protocol Code Number:4045-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-03-10
    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 number2015-002069-52
    A.3Full title of the trial
    A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
    Estudio doble ciego, controlado con placebo y multicéntrico con una extensión abierta para evaluar la eficacia y la seguridad de SRP-4045 y SRP-4053 en pacientes con distrofia muscular de Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients
    Un estudio de investigación de un nuevo medicamento en investigación para el tratamiento de pacientes con distrofia muscular de Duchenne
    A.3.2Name or abbreviated title of the trial where available
    N/A
    A.4.1Sponsor's protocol code number4045-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02500381
    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 SponsorSarepta 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 supportSarepta 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 Operations
    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.5.4Telephone number+34900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSRP-4045
    D.3.2Product code SRP-4045
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet available
    D.3.9.1CAS number 1422958-19-7
    D.3.9.2Current sponsor codeSRP-4045
    D.3.9.3Other descriptive namePhosphorodiamidate morpholino oligomer for exon 45 skipping
    D.3.9.4EV Substance CodeSUB183702
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSRP-4053
    D.3.2Product code SRP-4053
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet available
    D.3.9.1CAS number 1422959-91-8
    D.3.9.2Current sponsor codeSRP-4053
    D.3.9.3Other descriptive namePhosphorodiamidate morpholino oligomer for exon 53 skipping
    D.3.9.4EV Substance CodeSUB170839
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
    Pacientes con distrofia muscular de Duchenne con mutaciones de deleción susceptibles de corrección mediante la omisión de los exones 45 y 53
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy
    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 19.1
    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
    The primary objective of this study is to evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo on ambulation, endurance, and muscle function at Week 96, as measured by the 6-minute walk test (6MWT)
    El objetivo principal de este estudio es evaluar el efecto de SRP-4045 y SRP-4053 (grupo de tratamiento activo combinado) en comparación con un placebo sobre la deambulación, la resistencia y la función muscular en la semana 96, determinadas mediante la prueba de marcha de 6 minutos (PM6M).
    E.2.2Secondary objectives of the trial
    The secondary objectives are to evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) on:
    - Dystrophin protein expression in biopsied muscle tissue as measured by:
    Western blot (quantification)
    Immunohistochemistry (IHC) fiber intensity
    - Functional status as measured by:
    Ability to rise independently from the floor (without external support)
    Loss of ambulation (LOA)
    North Star Ambulatory Assessment (NSAA)
    Respiratory muscle function, as measured by forced vital capacity (FVC) % predicted
    Frequency of falls
    Cardiac function, as measured by left ventricular ejection fraction (LVEF)
    - Safety and tolerability of SRP-4045 and SRP-4053
    Los objetivos secundarios consisten en la evaluación del efecto de SRP-4045 y SRP-4053 (grupo de tratamiento activo combinado) sobre:
    - La expresión de la proteína distrofina en tejido muscular biopsiado, determinada mediante:
    •Inmunoelectrotransferencia (cuantificación)
    •Intensidad de las fibras mediante inmunohistoquímica (IHQ)
    - El estado funcional determinado mediante los siguientes factores:
    •Capacidad para levantarse del suelo sin ayuda (sin apoyo externo)
    •Pérdida de la deambulación
    •Evaluación ambulatoria North Star (NSAA)
    •Función muscular respiratoria, medida por el porcentaje teórico de capacidad vital forzada (FVC)
    •Frecuencia de caídas
    •Función cardiaca, determinada por la fracción de eyección del ventrículo izquierdo (FEVI)
    - La seguridad y la tolerabilidad de SRP-4045 y SRP-4053
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Is a male with an established clinical diagnosis of DMD and an out-of-frame deletion amenable to:
    ● Exon 45 skipping (including but not limited to deletions of exons such as 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53, or 46-55) OR
    ● Exon 53 skipping (including but not limited to deletions of exons such as 42-52, 45-52, 47-52, 48-52,49-52, 50-52, 52, or 54-58) as documented by a genetic report from an accredited laboratory confirming deletion endpoints by multiplex ligation-dependent probe amplification or sequencing.
    2. Is 7 to 13 years of age, inclusive.
    3. Has stable pulmonary function (forced vital capacity % of predicted [FVC%] ≥50% and no requirement for nocturnal ventilation) that, in the Investigator’s opinion, is unlikely to decompensate over the duration of the study.
    4. Has intact right and left biceps muscles (the preferred biopsy site) or 2 alternative upper arm muscle groups.
    5. Has been on a stable dose or dose equivalent of oral corticosteroids for at least 24 weeks prior to Week 1 and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study. Note: patients are allowed to take other medications (excluding other ribonucleic
    acid [RNA] antisense or gene therapy agents) including angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), β adrenergic blockers, potassium, and coenzyme Q,
    provided they have been on a stable dose for 12 weeks prior to Week 1 and the dose is expected to remain constant throughout the study.
    6. Achieved a mean 6MWT distance of ≥300 to ≤450 meters (without assistance) at both the Screening and Baseline visits (prior to Week 1). The mean 6MWT distance at the Screening and Baseline visits is the average of 2 separate assessments on 2 consecutive days at each visit. The Baseline mean (average of Baseline Days 1 and 2) must be within 15% of the Screening mean distance (average of Screening Days 1 and 2).
    7. Sexually active subjects must agree to use contraceptives for the entire duration of the study and for 90 days following the last dose. Both a male condom and a medically acceptable form of female
    contraceptive (e.g., oral contraceptives) must be used.
    8. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
    9. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
    Los pacientes deberán cumplir todos los criterios siguientes para poder participar en este estudio.
    1.Paciente varón con diagnóstico clínico confirmado de DMD y una deleción fuera del marco susceptible de:
    •Omisión del exón 45 (por ejemplo, deleciones de exones como 12 44, 18 44, 44, 46 47, 46 48, 46 49, 46 51, 46 53 o 46 55) O BIEN
    •Omisión del exón 53 (por ejemplo, deleciones de exones como 42 52, 45 52, 47 52, 48 52, 49 52, 50 52, 52 o 54 58)
    que esté documentada en un informe genético de un laboratorio acreditado que confirme los resultados de deleción mediante secuenciación o amplificación de sondas dependiente de ligados múltiples.
    2.Edad de entre 7 y 13 años, ambos inclusive.
    3.Presentar una función pulmonar estable (porcentaje de capacidad vital forzada [%FVC] ≥ 50 % del valor teórico, sin necesidad de ventilación nocturna) que, a criterio del investigador, no sea probable que se descompense durante el transcurso del estudio.
    4.Tener intactos los bíceps derecho e izquierdo (el lugar preferido para la biopsia) u otros 2 grupos musculares del brazo.
    5.Haber recibido una dosis estable o una dosis equivalente de corticosteroides orales durante un mínimo de 24 semanas antes de la semana 1 y previsión de que la dosis se mantenga constante (excepto si debe modificarse por un cambio de peso) durante todo el estudio. Nota: se permite que los pacientes tomen otros medicamentos (a excepción de otros fármacos de genoterapia o ácido ribonucleico [ARN] antisentido) como inhibidores de la enzima convertidora de la angiotensina (IECA), antagonistas de los receptores de la angiotensina (ARA), bloqueantes adrenérgicos β, potasio y coenzima Q, siempre que hayan recibido una dosis estable durante las 12 semanas anteriores a la semana 1 y que se prevea que esa dosis se mantendrá constante durante todo el estudio.
    6.Haber alcanzado una distancia media de ≥ 300 a ≤ 450 meteros (sin ayuda) en la PM6M tanto en las visitas de selección como basal (antes de la semana 1). La distancia media de la PM6M en las visitas de selección y basal es el promedio de 2 evaluaciones independientes en 2 días consecutivos en cada visita. La media basal (promedio de los días 1 y 2 del período basal) debe encontrarse dentro del 15 % de la distancia media del período de selección (promedio de los días 1 y 2 del período de selección).
    7. Los sujetos sexualmente activos deberán acceder a utilizar anticonceptivos durante todo el estudio y durante los 90 días siguientes a la última dosis. Deberá utilizarse tanto un preservativo masculino como un tipo de anticonceptivo femenino aceptable (por ejemplo, anticonceptivos orales).
    8. Los padres o tutores son capaces de entender y cumplir todos los requisitos del estudio.
    9. Estar dispuesto a dar su asentimiento informado (si corresponde) y tener padres o tutores que estén dispuestos a proporcionar su consentimiento informado por escrito para que el paciente participe en el estudio.
    E.4Principal exclusion criteria
    1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to Week 1 that may have an effect on muscle strength or function. Growth hormone for short stature and testosterone for delayed puberty are permitted if an endocrinologist has documented the diagnosis and medical necessity of treatment, and the patient has been on a stable dose for at least 24 weeks prior to Week 1.
    2. Previous treatment with SMT C1100 (BMN-195) at any time.
    3. Previous treatment with PRO045 or PRO053 within 24 weeks prior to Week 1.
    4. Current or previous treatment with any other experimental treatment (other than deflazacort) within 12 weeks prior to Week 1.
    5. Major surgery within 3 months prior to Week 1 or planned surgery for any time during this study, except for protocol-specified surgery, as applicable.
    6. Presence of any other significant genetic disease other than DMD (e.g., dwarfism).
    7. Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or malignancy.
    8. Use of any aminoglycoside antibiotic or statin within 12 weeks prior to Week 1 or anticipated need for an aminoglycoside antibiotic or statin during the study.
    9. LVEF <50% on the Screening echocardiogram (ECHO) or QTcF ≥450 msec based on the Screening and Baseline ECG.
    10. Loss of ≥30 degrees of plantar flexion from the normal range of movement at the ankle joint due to contracture (i.e., fixed loss of more than 10 degrees of plantar flexion from plantigrade, assuming normal range of dorsiflexion of 20 degrees).
    11. Prior or ongoing medical condition that could, in the Investigator’s opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results. Additionally, patients who seem unable / unwilling to comply with the study procedures, in the Investigator’s opinion, are to be excluded.
    Se excluirá de este estudio a los pacientes que cumplan alguno de los criterios siguientes.
    1. Tratamiento farmacológico (excepto corticosteroides) en las 12 semanas anteriores a la semana 1 que pueda haber afectado a la función o la fuerza muscular. Se permiten hormonas del crecimiento para casos de estatura baja y testosterona para casos de retraso de la pubertad, si un endocrino ha documentado el diagnóstico y la necesidad médica del tratamiento, y si el paciente ha recibido una dosis estable durante al menos 24 semanas anteriores a la semana 1.
    2. Tratamiento previo con SMT C1100 (BMN-195) en cualquier momento.
    3. Tratamiento previo con PRO045 o PRO053 en las 24 semanas anteriores a la semana 1.
    4. Tratamiento previo o actual con otro fármaco en investigación (aparte de deflazacort) en las 12 semanas anteriores a la semana 1.
    5. Operación de cirugía mayor en los 3 meses anteriores a la semana 1 o cirugía programada durante el transcurso del estudio, a excepción de la cirugía especificada por el protocolo, si corresponde.
    6. Presencia de otra enfermedad genética importante diferente a la DMD (por ejemplo, enanismo).
    7. Presencia de otra enfermedad clínicamente significativa, como cardiopatía, neumopatía, hepatopatía, nefropatía, hemopatía, enfermedad inmunitaria o psiquiátrica significativas, o cáncer.
    8. Uso de antibióticos aminoglucósidos o estatinas en las 12 semanas anteriores a la semana 1 o previsión de la necesidad de estos fármacos durante el estudio.
    9. FEVI < 50 % en el ecocardiograma del período de selección o QTcF ≥ 450 ms conforme al ECG del período de selección o basal.
    10. Pérdida ≥ 30 grados de flexión plantar con respecto a la amplitud de movimiento normal en la articulación del tobillo debido a contractura (es decir, pérdida fija de más de 10 grados de flexión plantar con respecto a la posición plantígrada, presumiendo una amplitud normal de dorsiflexión de 20 grados).
    11. Enfermedad previa o actual que pueda, a criterio del investigador, afectar de forma adversa a la seguridad del paciente, reducir las probabilidades de completar el tratamiento o afectar a la evaluación de los resultados del estudio. Además, se excluirá del estudio a los pacientes que, según el criterio del investigador, parezcan no ser capaces o no estar dispuestos a cumplir los procedimientos del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Change from Baseline at Week 96 on the 6MWT for the combined-active group compared to placebo
    Variación de la PM6M, desde el momento basal hasta la semana 96, en el grupo de tratamiento activo combinado en comparación con el grupo del placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Baseline, Week 12, 24, 36, 48, 60, 72, 84, 96
    Screening, Basal, semana 12, 24, 36, 48, 60, 72, 84, 96
    E.5.2Secondary end point(s)
    ●Change from Baseline at Week 48 in the quantity of dystrophin protein expression as measured by Western blot of biopsied muscle tissue
    ●Change from Baseline at Week 48 in the intensity of dystrophin expression in biopsied muscle tissue, as measured by IHC
    ● Ability to rise independently from the floor (without external support) at Week 96, as indicated by an NSAA subscore of "2" (without modification) or "1" (Gower's maneuver)
    ● LOA status at Week 96, defined as an inability to perform the 6MWT, or result of "0" meters on 6MWT
    ● Change from Baseline at Week 96
    - NSAA total score
    - FVC% predicted
    - Frequency of falls
    - LVEF
    •Variación desde el momento basal hasta la semana 48 de la cantidad de expresión de proteína distrofina, determinada mediante electroinmunotransferencia del tejido muscular biopsiado.
    •Variación desde el momento basal hasta la semana 48 de la intensidad de la expresión de distrofina en tejido muscular biopsiado, determinada mediante IHQ.
    •Capacidad para levantarse del suelo sin ayuda (sin apoyo externo) en la semana 96, conforme a una subpuntuación de la NSAA de “2” (sin modificación) o “1” (maniobra de Gower).
    •Pérdida de deambulación en la semana 96, definida como la imposibilidad de realizar la PM6M o un resultado de “0” metros en la PM6M.
    •Variación desde el momento basal hasta la semana 96 en:
    - Puntuación total de la NSAA
    - %FVC del valor teórico
    - Frecuencia de caídas
    - FEVI
    E.5.2.1Timepoint(s) of evaluation of this end point
    ● Quantity of dystrophin protein expression: Baseline, week 48
    ● Intensity of dystrophin expression: Baseline, week 48
    ● Ability to rise independently from the floor: Screening, Baseline, Week 12, 24, 36, 48, 60, 72, 84, 96
    ● LOA status: Week 96
    ● Cantidad de expresión de proteína distrofina : basal, semana 48
    ● La intensidad de la expresión de distrofina : basal, semana 48
    ● Capacidad para levantarse del suelo sin ayuda : screening, basal, semana 12, 24, 36, 48, 60, 72, 84, 96
    ● Pérdida de deambulación : semana 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 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) 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 Yes
    E.8.1.7.1Other trial design description
    Los pacientes pueden participar en un período abierto de extensión del tratamiento de hasta 96 seman
    Patients may participate in an open-label treatment extension period of up to 96 weeks
    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 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 EEA24
    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
    Canada
    Israel
    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
    Last patient last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    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: 99
    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: 84
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    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
    This study is intended to be conducted with minors and will include males aged from 7 to 13 years old.
    Este estudio está dirigido a menores de edad e incluirá a varones entre 7 y 13 años.
    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 50
    F.4.2.2In the whole clinical trial 99
    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)
    Pending the outcome of this clinical trial, the open label extension would be lengthened or a registry would be opened so study patients could remain on treatment.
    Pendiente del resultado de este ensayo clínico, la extensión abierta se prolongaría o se abriría un registro de forma que los pacientes del estudio podrían seguir en tratamiento.
    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 Decision2017-05-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-02
    P. End of Trial
    P.End of Trial StatusOngoing
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