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    Summary
    EudraCT Number:2016-000602-10
    Sponsor's Protocol Code Number:SNT-III-012
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-01-12
    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 ConcernedFrance - ANSM
    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
    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)
    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
    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
     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
    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
    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.
    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).
    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
    E.5.1.1Timepoint(s) of evaluation of this end point
    78 weeks
    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
    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
    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 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.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 state20
    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 Decision2016-10-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-11-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-10-05
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