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    Summary
    EudraCT Number:2009-012037-30
    Sponsor's Protocol Code Number:SNT-III-003
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-04-26
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2009-012037-30
    A.3Full title of the trial
    A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy
    Studio randomizzato di fase III, in doppio cieco e controllato verso placebo, sull'efficacia, la sicurezza e la tollerabilita' dell'idebenone in pazienti di 10-18 anni d'eta' con distrofia muscolare di Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    (DELOS) DuchEnne MD Long-term IdebenOne Study
    (DELOS) Studio di lungo termine con Idebenone nella malattia muscolare di Duchenne
    A.3.2Name or abbreviated title of the trial where available
    DELOS
    DELOS
    A.4.1Sponsor's protocol code numberSNT-III-003
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01022788
    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) LTD
    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 Limited
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanthera Pharmaceuticals Limited
    B.5.2Functional name of contact pointUfficio Ricerca Clinica
    B.5.3 Address:
    B.5.3.1Street AddressHammerstrasse
    B.5.3.2Town/ cityLiestal
    B.5.3.3Post codeCH-4410
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41 61 906 89 45
    B.5.5Fax number0041 619068951
    B.5.6E-mailraffaele.rubino@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 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 numberEU70/07/437
    D.3 Description of the IMP
    D.3.1Product nameIdebenone
    D.3.2Product code NA
    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.3.9.2Current sponsor codeSNT-MC17
    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
    Distrofia muscolare di Duchenne
    E.1.1.1Medical condition in easily understood language
    Hereditary neuromuscular disease
    Malattia neuromuscolare ereditaria
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    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
    To assess the efficacy of idebenone, compared to placebo, in improving respiratory function or delaying the loss of respiratory function in patients with DMD
    Valutare l'efficacia dell'idebenone, rispetto al placebo, nel migliorare la funzione respiratoria o ritardare la perdita della medesima nei pazienti con DMD.
    E.2.2Secondary objectives of the trial
    • To assess the efficacy of idebenone, compared to placebo, in improving respiratory function or delaying the loss of respiratory function using measures other than those used for the primary endpoint • To assess the efficacy of idebenone, compared to placebo, in improving skeletal muscle strength/ motor function or delaying the loss of skeletal muscle strength/ motor function • To assess the efficacy of idebenone, compared to placebo, in improving quality of life or delaying the loss in quality of life • To assess the safety and tolerability of idebenone in patients with DMD
    •Valutare l'efficacia dell'idebenone,rispetto al placebo,nel migliorare la funzione respiratoria o ritardare la perdita della medesima,attraverso criteri di misura diversi da quelli utilizzati per l'endpoint primario.•Valutare l'efficacia dell'idebenone,rispetto al placebo,nel migliorare la forza/motricità dei muscoli scheletrici o ritardare la perdita delle medesime.•Valutare l'efficacia dell'idebenone,rispetto al placebo,nel migliorare la qualità della vita del paziente o ritardare la perdita della medesima.•Valutare sicurezza e tollerabilità dell'idebenone nei pazienti con DMD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients 10 – 18 years of age at Baseline. 2. Signed and dated informed consent. 3. Documented diagnosis of DMD or 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 immunostain. 4. Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening). 5. 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.
    1. Pazienti di età compresa fra i 10 e i 18 anni alla baseline. 2. Consenso informato debitamente datato e firmato. 3. Diagnosi documentata di DMD conclamata o grave distrofinopatia e quadro clinico coerente con quello tipico della DMD alla diagnosi (vale a dire, rallentamento documentato delle capacità motorie e astenia manifestatisi entro i 5 anni d'età). La DMD dovrebbe essere confermata dall'analisi mutazionale del gene distrofina o da una sostanziale riduzione dei livelli di proteina distrofina (vale a dire, assenza della proteina oppure livello &lt;5% del normale) al western blot o all'immunostain. 4. Capacità di fornire PEF ripetute, affidabili e riproducibili, entro il 15% della prima valutazione (vale dire, baseline vs. screening). 5. I pazienti giudicati dallo sperimentatore idonei e in grado di soddisfare i requisiti dello studio dispongono delle capacità cognitive necessarie e sono in grado di assumere (per via orale) il farmaco di studio.
    E.4Principal exclusion criteria
    1. Patients dependent on assisted ventilation at Screening and/or Baseline (defined as non-invasive nocturnal ventilation, daytime noninvasive ventilation or continuous invasive ventilation). 2. Patients with documented DMD-related hypoventilation for which assisted ventilation is needed according to current standard of care guidelines (e.g. FVC< 30%) or is required in the opinion of the Investigator. 3. Patients with a percent predicted PEF > 80% at Baseline. 4. Patients unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressures. 5. Symptomatic heart failure (high probability of death within one year of Baseline) and/or symptomatic ventricular arrhythmias. 6. Participation in the previous Phase II or Phase II Extension study (SNT-II-001 or SNT-II-001-E) for idebenone. 7. Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Baseline. 8. Use of carnitine, creatine, glutamine, oxatomide, or any herbal medicines (unless written evidence (e.g. from the manufacturer or a physician) that the herbal medicine does not contain steroids is provided) within 30 days prior to Baseline. 9. Use of coenzyme Q10 or vitamin E (if taken at a dose of 5 times above the daily physiological requirement) within 30 days prior to Baseline.
    1. Pazienti che allo screening e/o alla baseline dipendono dalla ventilazione assistita (definita come ventilazione notturna non invasiva, ventilazione diurna non invasiva o ventilazione invasiva continua). 2. Pazienti con documentata ipoventilazione DMD-associata tale da richiedere la ventilazione assistita secondo le attuali direttive standard di trattamento sanitario (es. FVC &lt;30%), oppure a giudizio dello sperimentatore. 3. Pazienti con percentuale prevista di PEF alla baseline &gt;80%. 4. Pazienti incapaci di stringere le labbra in modo tale da consentire un'esatta misurazione del flusso respiratorio e della pressione orale. 5. Scompenso cardiaco sintomatico (elevata probabilità di decesso entro un anno dalla baseline) e/o aritmie ventricolari sintomatiche. 6. Partecipazione alla precedente Fase II dello studio o allo studio d'estensione di Fase II (SNT-II-001 o SNT-II-001-E) sull'idebenone. 7. Partecipazione a qualunque altro studio terapeutico e/o assunzione di qualsivoglia farmaco sperimentale a partire da 90 giorni prima della data di baseline. 8. Assunzione di carnitina, creatina, glutammina, oxatomide o farmaci erboristici (fatto salvo il caso di prova scritta, da parte del produttore o di un medico, che il prodotto in questione non contiene steroidi) a partire da 30 giorni prima della data di baseline. 9. Assunzione di coenzima Q10 o vitamina E (in dose 5 volte superiore al normale fabbisogno quotidiano) a partire da 30 giorni prima della data di baseline. 10. Precedente assunzione di idebenone.
    E.5 End points
    E.5.1Primary end point(s)
    The change from Baseline to Week 52 in percent predicted Peak Expiratory Flow (PEF)1
    Variazione percentuale prevista del picco di flusso espiratorio (PEF)1 dalla baseline alla 52a settimana.
    E.5.1.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    E.5.2Secondary end point(s)
    • Pulmonary function: o The change from Baseline to Week 52 in percent predicted Forced Vital Capacity (FVC) o The change from Baseline to Week 52 in percent predicted Peak Cough Flow (PCF) o The change from Baseline to Week 52 in percent predicted Maximal Expiratory Pressure (MEP) o The change from Baseline to Week 52 in percent predicted Maximal Inspiratory Pressure (MIP) o Number of patients in each treatment arm that begin assisted ventilation during the study period (due to documented development of DMD-related hypoventilation) • Muscle strength / motor function: o The change from Baseline to Week 52 in muscle strength as measured by hand-held myometry (HHM) o The change from Baseline to Week 52 in motor function as assessed using the Brooke and Vignos scales • Quality of life: o The change from Baseline to Week 52 in Quality of Life assessed by the PedsQL™ Quality of Life Inventory (child and teen report forms) o The change from Baseline to Week 52 in the PedsQL™ Multidimensional Fatigue Scale (child and teen report forms) o Differences in the Clinical Global Impression of efficacy and tolerability at Week 52 between the two study arms o Differences between patient responses to the “satisfaction with treatment” question at Week 52 between the two study arms o Number of patients in each study arm that during the study period require 􀂃 acute hospitalization for treatment of a respiratory or other complication associated with disease progression 􀂃 the initiation of the use of a brace or spinal jacket 􀂃 initiation or change in systemic glucocorticoid treatment 􀂃 need for assisted ventilation • Measures of safety and tolerability of idebenone: o Nature and frequency of AEs o Laboratory parameters (haematology, biochemistry, and urinalysis) o Physical examinations and vital signs o ECG o Transthoracic echocardiography
    • Funzione polmonare: o Variazione percentuale prevista della capacità vitale forzata (FVC) dalla baseline alla 52a settimana. o Variazione percentuale prevista del picco di flusso espiratorio della tosse (PCF) dalla baseline alla 52a settimana. o Variazione percentuale prevista della pressione espiratoria massima (MEP) dalla baseline alla 52a settimana. o Variazione percentuale prevista della pressione inspiratoria massima (MIP) dalla baseline alla 52a settimana. o Numero di pazienti in ciascun braccio di trattamento che hanno dovuto sottoporsi a ventilazione assistita nel corso dello studio (in seguito all'insorgere documentato di ipoventilazione DMD-associata). • Forza muscolare/funzione motoria: o Variazione della forza muscolare, misurata tramite miometria manuale, dalla baseline alla 52a settimana. o Variazione della funzione motoria, misurata in base alle scale di Brooke e Vignos, dalla baseline alla 52a settimana. • Qualità di vita: o Variazioni della qualità di vita misurate tramite PedsQL™ (Quality of Life Inventory), formulario per bambini e adolescenti, dalla baseline alla 52a settimana. o Variazione rispetto alla scala multidimensionale PedsQL per la valutazione della fatica (formulario per bambini e adolescenti) dalla baseline alla 52a settimana. o Differenze fra i due bracci di studio nell'impressione clinica globale (CGI) di efficacia e tollerabilità alla 52a settimana. o Differenze fra i due bracci di studio in termini di soddisfazione dei pazienti nei confronti della terapia alla 52a settimana. o Numero di pazienti in ciascun braccio di studio che nel corso dello studio medesimo hanno richiesto: ricovero d'urgenza per complicanze respiratorie o d'altra natura associate alla progressione della malattia; applicazione di supporto dorsale o corsetto; avvio o modifica di terapia sistemica a base di glucocorticoidi; ventilazione assistita. • Sicurezza e tollerabilità dell'idebenone: o Natura e frequenza degli eventi avversi o Parametri di laboratorio (ematologia, biochimica, analisi delle urine) o Visita medica e segni vitali o ECG o Ecocardiografia transtoracica
    E.5.2.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    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 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 Yes
    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
    Disegno a gruppi sequenziali
    Group sequential design
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    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
    The end of the DELOS trial is defined as the Last Patient Last visit date.
    La fine dello studio DELOS è definita come data dell'ultima visita dell'ultimo paziente.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months25
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months30
    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: 260
    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: 60
    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: 20
    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
    Minors not capable to give consent
    Minori incapaci di dare il consenso
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 170
    F.4.2.2In the whole clinical trial 280
    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 protocol only foresees procedures for Handling Discontinued Patients
    Il protocollo prevede solo le procedure da attuare per i pazienti che interrompono lo studio prematuramente.
    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 Decision2012-03-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-05-09
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