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    Summary
    EudraCT Number:2017-004279-30
    Sponsor's Protocol Code Number:SNT-III-012-E
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-01-22
    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 number2017-004279-30
    A.3Full title of the trial
    A Phase III Open-Label Extension Study to Assess the Long-Term Safety
    and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy
    (DMD) who completed the SIDEROS study
    Estensione di studio di fase III in aperto per valutare la sicurezza a lungo termine e l’efficacia dell’idebenone in pazienti affetti da distrofia muscolare di Duchenne (DMD) che hanno completato lo studio SIDEROS.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to assess the long-term safety and efficacy of idebenone
    treatment in patients with Duchenne Muscular Dystrophy (DMD) who
    completed the SIDEROS study.
    studio clinico per valutare la sicurezza a lungo termine e l'efficacia del trattamento con Idebenone in pazienti con Distrofia Muscolare di Duchenne (DMD) che hanno completato lo studio SIDEROS.
    A.3.2Name or abbreviated title of the trial where available
    SIDEROS-E
    SIDEROS-E
    A.4.1Sponsor's protocol code numberSNT-III-012-E
    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
    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 pointQuentin Desvigne
    B.5.3 Address:
    B.5.3.1Street AddressHohenrainstrasse 24
    B.5.3.2Town/ cityPratteln
    B.5.3.3Post code4133
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number0041619068917
    B.5.5Fax number0041619068951
    B.5.6E-mailquentin.desvigne@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.3.2Product code [Idebenone]
    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 code58186-27-9
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 (DMD)
    Distrofia Muscolare di 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 è una malattia genetica caratterizzata da una rapida e progressiva debolezza e perdita di massa muscolare che porta a grave disabilità
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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 long-term safety of idebenone in DMD patients who completed the SIDEROS study.(up to Visit 4/Week 78)
    Valutare la sicurezza a lungo termine dell'idebenone nei pazienti con DMD che hanno completato lo studio SIDEROS (fino alla visita 4/settimana 78)
    E.2.2Secondary objectives of the trial
    To describe the long-term evolution of respiratory function in idebenonetreated
    DMD patients who completed the SIDEROS study, classified by
    background factors including, but not limited to age, DMD history (e.g.
    time of loss of ambulation, mutation type), type of steroid regimen and study treatment assignment in the SIDEROS study.(up to Visit 4/Week 78)
    Descrivere l’evoluzione a lungo termine della funzione respiratoria in pazienti affetti da DMD trattati con idebenone che hanno completato lo studio SIDEROS classificati per fattori di background come, in modo non esaustivo, età, storia della DMD (per es. perdita della deambulazione, tipo di mutazione), tipo di regime steroideo e assegnazione al trattamento dello studio durante lo studio SIDEROS. (fino alla visita 4/settimana 78)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Completion of the SIDEROS study at Visit 8/ Week 78.
    Signed and dated Informed Consent Form.
    inclusion criteria for the optionsal continued treatment with idebenone beyond Visit 4 of SIDEROS-E study:
    1. completion of Visit 4 / week 78 of Sideros-E study
    2. Signed and dated Informed Consent Form for continued treatment
    1. Completamento dello studio SIDEROS alla visita 8/settimana 78
    2. Datato e firmato il modulo di Consento informato per SIDEROS-E
    Criteri di inclusione per la continuazione del trattamento con idebenone facoltativa oltre la visita 4/settimana 78 dello studio SIDEROS:
    1. Completamento dello studio SIDEROS-E alla visita 4/settimana 78
    2. Datato e firmato il modulo di Consento informato per la continuazione del trattamento
    E.4Principal exclusion criteria
    1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8).
    2. Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the
    patient at significant risk or may interfere significantly with the patient's
    participation in the SIDEROS-E study.
    3. Use of any investigational drug other than the study medication.
    Exclusion criteria for the optional continued treatment with idebenone
    beyond Visit 4/ Week 78 of SIDEROS-E study:
    1. Premature withdrawal from SIDEROS-E study before Visit 4/ Week 78
    2. Any conditions, which, in the opinion of the Investigator might trigger
    a negative risk-benefit assessment for the patient
    3. Use of any Investigational drug other than the study medication
    1. Pazienti che hanno interrotto prematuramente lo studio Studio SIDEROS (cioè non hanno sostenuto le visite dalla V1 alla V8)
    2. Sicurezza, tollerabilità o altri problemi emersi durante lo studio SIDEROS che secondo l’opinione dello sperimentatore possano comportare un rischio significativo per il paziente in SIDEROS-E o che possano interferire in modo significativo alla partecipazione del paziente a SIDEROS-E
    3. Uso di qualunque farmaco sperimentale diverso da quello dello studio
    Criteri di esclusione per la continuazione del trattamento con idebenone facoltativa oltre la visita 4/settimana 78 dello studio SIDEROS-E:
    1. Ritiro anticipato dallo studio SIDEROS-E prima della visita 4/settimana 78
    2. Qualunque condizione che secondo l’opinione dello Sperimentatore possa risultare in una valutazione del beneficio/rischio negativa per il paziente
    3. Uso di qualunque farmaco sperimentale diverso da quello dello studio
    E.5 End points
    E.5.1Primary end point(s)
    Primary:
    Standard safety assessments, including number of premature
    discontinuations of study treatment due to adverse events, incidence
    and severity of adverse events, actual values and changes from baseline in safety laboratory parameters, vital signs and electrocardiogram (ECG).
    Principale:
    • Normali valutazioni sulla sicurezza quali numero delle interruzioni anticipate del trattamento previsto dallo studio a causa di eventi avversi, incidenza e gravità degli eventi avversi, valori effettivi e variazioni dalla baseline dei parametri di laboratorio sulla sicurezza, segni vitali ed elettrocardiogramma (ECG).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Electrocardiogram: baseline
    Adverse events, safety laboratory parameters, vital signs: 26 weeks , 52 weeks, 78 weeks, post-completion follow-up visit
    ECG: baseline
    Eventi Avversi, parametri di sucurezza di laboratorio, segni vitali: 26, 52, 78 settimane, dopo il completamento della visita di Follow-up
    E.5.2Secondary end point(s)
    Secondary:
    Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p).
    Feasibility of continued idebenone treatment, measured with number of
    discontinuations of study treatment due to adverse events, will be the
    only applicable endpoint for the optional continued treatment period
    (beyond Visit 4).
    Secondario:
    • Scostamento dalla baseline della Capacità vitale forzata (FVC) come percentuale della previsione (FVC%p), picco di flusso espiratorio (PEF) come percentuale della previsione (PEF%p) e Volume espirato forzato in 1 secondo (FEV1) come percentuale del valore previsto (FEV1%p).
    La fattibilità del trattamento con idebenone continuato, misurata con il numero di interruzioni del trattamento in studio a causa di eventi avversi, sarà l'unico endpoint applicabile per il periodo di recupero continuo opzionale (oltre la visita 4).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Forced Vital Capacity (FVC), Peak Expiratory Flow (PEF), Forced Expiratory Volume in 1 second (FEV1) : baseline, 26 weeks , 52 weeks, 78 weeks . FVC and PEF in optional continued period : 130 weeks, 182 weeks .
    Adverse events for the optional continued treatment period : 104 weeks,
    130 weeks, 156 weeks, 182 weeks
    FVC, PEF, FEV1: baseline, 26, 52, 78 settimane. FVC e PEF nel periodo continuato opzionale: 130 settimane, 182 settimane
    Eventi avversi per il periodo di trattamento continuato opzionale: 104 settimane, 130 settimane, 156 settimane, 182 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 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 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 Yes
    E.8.1.7.1Other trial design description
    in aperto
    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.8.2.4Number 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA36
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    United States
    Austria
    Belgium
    France
    Germany
    Italy
    Netherlands
    Spain
    Sweden
    Switzerland
    United Kingdom
    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
    LVLS
    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 months43
    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 months43
    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.1.1.1In 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) Yes
    F.1.3.1Number of subjects for this age range: 1
    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
    DMD è soprattutto diagnosticata nei primi 10 anni di vita pertanto i pazienti sotto l'età di consenso potranno essere arruolati nello studio
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state11
    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)
    Upon completion of the main treatment period in the study (Visit 4),
    participants will be offered the possibility to enter an optional
    continued treatment period within the trial for up to 24 months. No
    particular post-trial treatment scheme is defined beyond this optional
    period.
    Al completamento del periodo di trattamento principale nello studio (Visita 4),
    ai partecipanti verrà offerta la possibilità di inserire un facoltativo
    ha continuato il periodo di trattamento all'interno della sperimentazione fino a 24 mesi. Nessun particolare schema di trattamento post-trial è definito oltre questo facoltativo periodo.
    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 Decision2018-08-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-13
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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