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    Summary
    EudraCT Number:2016-000602-10
    Sponsor's Protocol Code Number:SNT-III-012
    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:2016-11-18
    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 number2016-000602-10
    A.3Full title of the trial
    A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids
    Estudio de fase III, doble ciego, aleatorizado y controlado con placebo para
    evaluar la eficacia, la seguridad y la tolerabilidad de idebenona en pacientes
    con distrofia muscular de Duchenne que reciben glucocorticoesteroides
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids
    A.3.2Name or abbreviated title of the trial where available
    SIDEROS
    A.4.1Sponsor's protocol code numberSNT-III-012
    A.5.4Other Identifiers
    Name:US INDNumber:103801
    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 SponsorSanthera Pharmaceuticals (Switzerland) Limited
    B.1.3.4CountrySwitzerland
    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 supportSanthera Pharmaceuticals (Switzerland) Limited
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanthera Pharmaceuticals (Switzerland) Limited
    B.5.2Functional name of contact pointIrina Coserea
    B.5.3 Address:
    B.5.3.1Street AddressHammerstrasse 49
    B.5.3.2Town/ cityLiestal
    B.5.3.3Post codeCH-4410
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+4161906 8938
    B.5.5Fax number+41619068951
    B.5.6E-mailIrina.Coserea@santhera.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 Raxone
    D.2.1.1.2Name of the Marketing Authorisation holderSanthera Pharmaceuticals (Deutschland) GmbH
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/07/437
    D.3 Description of the IMP
    D.3.1Product nameIdebenone
    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 INNIDEBENONE
    D.3.9.1CAS number 58186-27-9
    D.3.9.3Other descriptive nameIDEBENONE
    D.3.9.4EV Substance CodeSUB08114MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy (DMD)
    Distrofia muscular de Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    DMD is a genetic disease characterised by rapidly progressive muscle weakness and wasting which leads to severe disability
    La DMD es una enfermedad genética caracterizada por debilidad muscular y pérdida de peso rápidamente progresiva que conduce a una discapacidad severa
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    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 assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using hospital-based spirometry
    Evaluar la eficacia de idebenona en comparación con placebo, en la
    ralentización de la pérdida de la función respiratoria en pacientes con
    DMD que reciben glucocorticoesteroides medida por los cambios en
    el porcentaje previsto de la capacidad vital forzada (FVC %p) con
    espirometría realizada en el hospital.
    E.2.2Secondary objectives of the trial
     To assess the efficacy of idebenone compared to placebo in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by:
     Changes in Peak Expiratory Flow percent predicted (PEF %p) using hospital-based spirometry
     Time to loss of 10% of Baseline Forced Vital Capacity (FVC) using hospital-based spirometry
     To assess the efficacy of idebenone compared to placebo in delaying the loss of inspiratory muscle function as measured by changes in Inspiratory Flow Reserve (IFR) using hospital-based spirometry
    Evaluar la eficacia de idebenona en comparación con placebo en
    la ralentización de la pérdida de la función respiratoria en pacientes
    con DMD que reciben glucocorticoesteroides medida por:
     Los cambios en el porcentaje previsto en el flujo espiratorio
    máximo (PEF %p) con una espirometría realizada en el hospital.
     El tiempo transcurrido hasta la pérdida del 10 % del valor inicial
    de la capacidad vital forzada (FVC).
     Evaluar la eficacia de idebenona en comparación con placebo en
    la ralentización de la pérdida de la función del músculo
    inspiratorio medida por los cambios en la reserva de flujo
    inspiratorio (IFR) con una espirometría realizada en el hospital.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male patients with a 30% ≤ FVC ≤ 80% of predicted value at
    Screening and at Baseline.
    2. Minimum 10 years old at Screening.
    3. Signed and dated Informed Consent Form.
    4. Documented diagnosis of DMD (severe dystrophinopathy) and clinical
    features consistent of typical DMD at diagnosis (i.e. documented delayed
    motor skills and muscle weakness by age 5 years). DMD should be
    confirmed by mutation analysis in the dystrophin gene or by
    substantially reduced levels of dystrophin protein (i.e. absent or <5% of
    normal) on Western blot or immunostaining.
    5. Chronic use of systemic glucocorticoid steroids for DMD related conditions continuously for at least 12 months prior to Baseline without
    any dose adjustments on a mg/kg basis in the last 6 months (only dose
    adjustment determined by weight changes are allowed).
    6. Ability to provide reliable and reproducible repeat FVC within 15% of
    the screening assessment at Baseline.
    7. Patients assessed by the Investigator as willing and able to comply
    with the requirements of the study, possess the required cognitive
    abilities and are able to swallow study medication.
    8. Patients who have been immunized with 23-valent pneumococcal
    polysaccharide vaccine or any other pneumococcal polysaccharide
    vaccine as per national recommendations, as well as annually immunized
    with inactivated influenza vaccine.
    1. Pacientes varones con un 30 % ≤ FVC ≤ 80 % del valor previsto en
    la vista de selección y en la visita inicial.
    2. Mínimo 10 años de edad en el momento de la selección
    3. Formulario de consentimiento informado firmado y fechado.
    4. Diagnóstico documentado de DMD (distrofinopatía grave) y
    características clínicas coherentes con el diagnóstico típico de la
    DMD, es decir, habilidades motrices ralentizadas y debilidad
    muscular a la edad de 5 años. Confirmación de la MDM mediante el
    análisis de mutaciones en el gen de la distrofina o mediante niveles
    sustancialmente reducidos de la proteína distrofina (es decir,
    ausente o < 5 % de lo normal) obtenidos con análisis de Western
    blot o por inmunotinción.
    5. Uso crónico de glucocorticoesteroides sistémicos para las
    afecciones relacionadas con la DMD durante al menos 12 meses
    antes de la visita inicial sin ajustes de la dosis basados en mg/kg en
    los últimos 6 meses (solo se permite el ajuste de la dosis
    determinado por los cambios en el peso).
    6. Habilidad para proporcionar una FVC de forma repetida, fiable y
    reproducible dentro del 15 % obtenido en la evaluación de
    selección en la visita inicial.
    7. Pacientes evaluados por el investigador y que se consideren
    con voluntad y capacidad para cumplir con los requisitos del
    estudio, que tengan las habilidades cognitivas necesarias y que
    puedan tragar la medicación del estudio.
    8. Pacientes que hayan recibido la vacuna antineumocócica
    polisacárida 23-valente o cualquier otra antineumococia
    polisacarida equivalente según recomendaciones nacionales y
    que además reciban la vacuna anual inactivada contra la gripe.
    E.4Principal exclusion criteria
    1. Symptomatic heart failure (defined as Stage C by ACCF/AHA guideline or NYHA III-IV) and/or symptomatic ventricular arrhythmias.
    2. Ongoing participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Baseline (only exception allowed is use of Deflazacort in US as part of the Expanded Access Program).
    3. Prior or ongoing exon-skipping or read-through gene therapy for DMD.
    4. Planned or expected spinal fixation surgery during the study period (as judged by the Investigator, i.e. due to rapidly progressing scoliosis), prior spinal fixation surgery is allowed if it took place more than 6 months prior to Screening.
    5. Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or presence of any other non-DMD respiratory illness that affects respiratory function.
    6. Chronic use of beta2-agonists or any use of other bronchodilating/bronchoconstricting medication (inhaled steroids, sympathomimetics, anti-cholinergics, antihistamines); chronic use is defined as a daily intake for more than 14 days.
    7. Any bronchopulmonary illness that required treatment with antibiotics within 3 months prior to Screening.
    8. Moderate or severe hepatic impairment (Child-Pugh class B [7 to 9 points] or Child-Pugh class C [10 to 15 points]) or severe renal impairment (eGFR <30 mL/min/1.73 m2).
    9. Prior or ongoing medical condition or laboratory abnormality which in the Investigator’s opinion may put the patient at significant risk, may confound the study results or may interfere significantly with the patient’s participation in the study .
    10. History of or current drug or alcohol abuse or use of any tobacco/marijuana products/smoking.
    11. Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the study medication.
    12. Daytime ventilator assistance (defined as use of any assisted ventilation while awake).
    1. Insuficiencia cardíaca sintomática (según la clasificación de la
    ACC/AHA estadio C o de grado III-IV según la NYHA) o arritmias
    ventriculares sintomáticas.
    2. Estar participando en otro ensayo terapéutico o estar tomando
    otro fármaco en fase de investigación durante los 90 días previos
    a la visita inicial (la única excepción es el uso de deflazacort en
    los EE. UU. como parte del programa de acceso ampliado).
    3. Omisión de exón o terapia de lectura génica previa o en curso
    para la DMD.
    4. Cirugía de fijación de la columna vertebral prevista o planificada
    durante el periodo del estudio (según el criterio del investigador, es
    decir, debido a una escoliosis de progresión rápida), pero se permite
    la cirugía previa de fijación de la columna vertebral si se ha
    realizado más de 6 meses antes de la selección.
    5. Asma, bronquitis/EPOC, bronquiectasia, enfisema, neumonía
    o presencia de otras enfermedades respiratorias no
    relacionadas con la DMD que afecten a la función
    respiratoria.
    6. Uso crónico de agonistas beta 2 o el uso de otra medicación
    broncodilatadora/broncoconstrictora (esteroides inhalados,
    simpaticomiméticos, anticolinérgicos, antihistaminas). El uso crónico
    se define como la toma diaria durante más de 14 días.
    7. Enfermedades broncopulmonares que requieran tratamiento
    con antibióticos durante los 3 meses previos a la selección.
    8. Insuficiencia hepática moderada o grave (Child-Pugh clase B [de 7 a
    9 puntos] o Child-Pugh clase C [de 10 a 15 puntos]) o insuficiencia
    renal grave (eGFR <30 ml/min/1,73 m2).
    9. Afección médica previa o en curso o anomalía de laboratorio que,
    según la opinión del investigador, pueda poner en grave riesgo al
    paciente, confundir los resultados del estudio o interferir de modo
    significativo en la participación del paciente en el estudio1.
    10. Antecedentes o adicción actual a las drogas, al alcohol, al
    tabaco o al consumo de marihuana.
    11. Hipersensibilidad individual conocida a idebenona o a alguno de los
    componentes/excipientes de la medicación del estudio.
    12. Asistencia ventilatoria diurna (definida como cualquier tipo de
    ventilación
    E.5 End points
    E.5.1Primary end point(s)
    The change from Baseline to Week 78 in FVC %p assessed by hospital-based spirometry measurements
    El cambio desde la visita inicial hasta la semana 78 en el FVC %p
    evaluado con las mediciones de la espirometría realizada en el
    hospital.
    E.5.1.1Timepoint(s) of evaluation of this end point
    78 weeks
    78 semanas
    E.5.2Secondary end point(s)
    The secondary endpoints will be evaluated in the following order in a hierarchical manner:
    1. The change from Baseline to Week 78 in PEF %p assessed by hospital-based spirometry measurements
    2. The time to first 10% decline in FVC (L) during the 78-week treatment period, assessed by hospital-based spirometry measurements
    3. The change from Baseline to Week 78 in IFR assessed by hospital-based spirometry measurements
    Los criterios de valoración secundarios se evaluarán en el siguiente orden
    jerárquico:
     El cambio desde la visita inicial hasta la semana 78 en el PEF %p
    evaluado con las mediciones de la espirometría realizada en el
    hospital.
     El tiempo transcurrido hasta el primer deterioro del 10 % en la
    FVC (l) durante el periodo de tratamiento de 78 semanas,
    evaluado con las mediciones de la espirometría realizada en el
    hospital.
     El cambio desde la visita inicial hasta la semana 78 en la IFR
    evaluado con las mediciones de la espirometría realizada en el
    hospital.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoint 1: 78 weeks
    Secondary endpoint 2: 13, 26, 39, 52, 65 and 78 weeks
    Secondary endpoint 3: 78 weeks
    Criterio de valoración secundario 1: 78 semanas
    Criterio de valoración secundario 2: 13, 26, 39, 52, 65 y 78 semanas
    Criterio de valoración secundario 3: 78 semanas
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 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 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 concerned4
    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
    Austria
    Belgium
    France
    Germany
    Italy
    Netherlands
    Spain
    Sweden
    Switzerland
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months40
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months40
    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: 266
    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: 26
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 200
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 40
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    DMD is mainly diagnosed in the first decade of life and therefore patients below the age of consent are expected to be enrolled into the trial
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 130
    F.4.2.2In the whole clinical trial 266
    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)
    All patients completing Visit 8/Week 78, and considered eligible by the Investigator will be able to participate in an open-label extension study (SIDEROS-E) and will continue to receive idebenone until the SIDEROS-E is terminated or Marketing Authorization is obtained for idebenone in DMD, whichever occurs first.
    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 Decision2016-12-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-12-02
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-10-05
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