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    Summary
    EudraCT Number:2015-002530-50
    Sponsor's Protocol Code Number:EspeRare_RIM_001
    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:2015-09-11
    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 number2015-002530-50
    A.3Full title of the trial
    A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy.
    Etude Clinique ouverte de phase Ib pour l'évaluation de la sécurité, de la tolérance, de la pharmacocinétique et de la pharmacodynamie de doses orales multiples et croissantes de Rimeporide chez les patients présentant une myopathie de Duchenne.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Rimeporide in patients with Duchenne Muscular Dystrophy
    Rimeporide chez les patients présentant une myopathie de Duchenne
    A.4.1Sponsor's protocol code numberEspeRare_RIM_001
    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 SponsorEspeRare
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportEuropean Commission
    B.4.2CountryBelgium
    B.4.1Name of organisation providing supportL’Association Française Contre les Myopathies (AFM)
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportMerck Serono
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation QED Clinical Services
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressSnowdon Drive / Winterhill
    B.5.3.2Town/ cityMilton Keynes
    B.5.3.3Post codeMK6 1BP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number441908251 480
    B.5.5Fax number441908251 499
    B.5.6E-mailnmaruf@qed-clinical.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 numberEU/3/15/1478
    D.3 Description of the IMP
    D.3.1Product nameRimeporide
    D.3.2Product code EMD 87580
    D.3.4Pharmaceutical form Capsule, hard
    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 INNN-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate
    D.3.9.2Current sponsor codeEMD 87580
    D.3.9.3Other descriptive nameRimeporide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate
    D.3.9.2Current sponsor codeEMD 87580
    D.3.9.3Other descriptive nameRimeporide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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
    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)
    Myopathie de Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy (DMD)
    Myopathie 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 18.0
    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 determine the preliminary safety and tolerability profile of multiple oral administrations of rimeporide.
    Déterminer un profil préliminaire de sécurité et de tolérance de l'administration de doses orales multiples de rimeporide.
    E.2.2Secondary objectives of the trial
    Secondary objectives:
    •Pharmacokinetics: To evaluate the pharmacokinetic profile of rimeporide in pediatric patients with DMD.

    •Pharmacodynamics:
    -To measure inflammatory and muscular injury biomarkers;
    -To explore the PK/PD relationship of a 4-week rimeporide treatment on those surrogate biomarkers.

    Exploratory objectives:
    • To explore the relationship between safety endpoints and pharmacokinetic parameters
    • To explore using Nuclear Magnetic Resonance (NMR) Spectroscopy and imaging the effect of a 4-week treatment with rimeporide on biomarkers including intracellular pH and intracellular Na, and degree of inflammation, oedema, fat fraction, muscle composition.
    Objectifs secondaires :
    •Pharmacocinétique : Évaluer le profil pharmacocinétique du rimeporide chez les patients pédiatriques atteints de DMD.

    •Pharmacodynamie :
    -Mesurer les biomarqueurs d'atteintes inflammatoires et musculaires ;
    -Explorer la relation PK/PD d'un traitement de 4 semaines par rimeporide sur ces biomarqueurs.

    Objectifs exploratoires :
    • Explorer la relation entre les critères de sécurité et les paramètres pharmacocinétiques.
    • Explorer par SRMN et par IRM, l'effet d'un traitement de 4 semaines par rimeporide sur des biomarqueurs incluant le pH intracellulaire , le Sodium intracellulaire, le degré de l'inflammation, l'œdème, le pourcentage de masse grasse et la composition/structure musculaire.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Duchenne muscular dystrophy genetically confirmed;
    2. Males between 6 and 14 years old;
    3. Able to walk independently at least 75 meters;
    4. Patients on a stable dose of corticosteroids at least 6 months prior to baseline;
    5. Willing and able to comply with all protocol requirements and procedures;
    6. Signed informed consents by the parent(s)/legal guardian(s);
    7. France only: Affiliated to or a beneficiary of a social security system
    1. Myopathie de Duchenne confirmée génétiquement.
    2. Patients de sexe masculin âgés de 6 à 14 ans.
    3. Patients ayant une autonomie de marche sur une distance minimum de 75 mètres.
    4. Patients sous une dose stable de corticoïdes pendant un minimum de 6 mois avant la détermination de l'état basal.
    5. Patients ayant la volonté et la capacité de respecter toutes les exigences et les procédures du protocole.
    6. Consentement éclairé signé du (des) parent(s)/représentant(s) légal(aux).
    7. Pour la FRANCE uniquement : Patient affilié ou bénéficiaire d'un système de sécurité sociale.
    E.4Principal exclusion criteria
    1. Patients with significant renal disease or impairment, with Glomerular Filtration Rate estimated using plasma cystatin C level using the Filler formula less than 90ml/min/1.73m2;
    2. Current or history of liver disease or impairment,
    3. History of any significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. inflammatory, coagulation disease, unstable cardiac or respiratory disease;
    4. Acute illness within 4 weeks of the first administration of study medication which may interfere with study assessments;
    5. Significant change of dosage and/or dosing regimens for corticosteroids planned for the duration of study medication;
    6. Use of beta blockers / and ACEI or ARB unless at stable dose for at least 3 months prior to baseline;
    7. Use of protocol pump inhibitors unless at a stable dose for at least 3 months prior to baseline
    8. Use of aldosterone antagonists (i.e. spironolactone, eplerenone) within 3 months prior to first administration of study medication;
    9. Use of anticoagulants, antithrombotics or antiplatelet agents,
    10. Previous treatment with idebenone or other forms of Coenzyme Q10 within 1 month of the first administration of study medication;
    11. Previous treatment with investigational drugs within 4 weeks (or 7 half-life if longer than 4 weeks) of the first administration of study medication including placebo;
    12. A baseline QTc>450msec,or history of risk factors for torsades de pointes (eg, heart failure, hypokalaemia, family history of long QT syndrome);
    13. LVEF≤ 45% at screening or within the past 6 months and/or history of acute heart failure;
    14. Ventilator dependent;
    15. Known individual hypersensitivity to any of the ingredients/excipients of the study medication;
    16. For patients willing to undergo MRI/MRS assessments: Patients with specific contraindication to MRI (e.g.: metallic foreign body, claustrophobia, etc.).
    1. Patients ayant une maladie ou une atteinte rénale significative, avec un débit de filtration glomérulaire estimé par le taux plasmatique de cystatine C selon la formule de Filler inférieur à 90 ml/min/1,73 m2.
    2. Maladie ou atteinte hépatique passée ou actuelle.
    3. Antécédent de toute affection médicale significative susceptible de fausser l'interprétation des données d'efficacité ou de tolérance, notamment un trouble de la coagulation, une maladie inflammatoire, une maladie cardiaque ou respiratoire instable.
    4. Maladie aiguë dans les 4 semaines précédant la première administration du médicament de l'étude, susceptible d'interférer avec les évaluations de l'étude.
    5. Changement significatif de la posologie ou des schémas d'administration des corticosteroids prévus pendant la durée du traitement de l'étude.
    6.Utilisation de bêtabloquants et d’Inhibiteurs de l’enzyme de conversion de l’angiotensine (IECA) ou d’ Inhibiteurs des récepteurs de l’angiotensine II (ARA), sauf en cas de dose stable depuis un minimum de 3 mois avant la détermination de l’état basal.
    7. Utilisation d'inhibiteurs de la pompe à protons, sauf en cas de dose stable depuis un minimum de 3 mois avant la détermination de l’état basal.
    8. Utilisation d'antagonistes de l'aldostérone (p. ex. spironolactone, éplérénone) dans les 3 mois précédant la première administration du médicament de l'étude.
    9. Prise d'anticoagulants, d'antithrombotiques ou d'agents antiplaquettaires.
    10. Traitement précédent par l’idébénone ou d'autres formes de coenzyme Q10 dans le mois précédant la première administration du médicament de l'étude.
    11. Traitement précédent par des médicaments expérimentaux dans les 4 semaines (ou les 7 demi-vies si elles sont supérieures à 4 semaines) précédant la première administration du médicament de l'étude, incluant le placebo.
    12. QTc initial > 450 msec, ou antécédents de facteurs de risques de torsades de pointes (p. ex. insuffisance cardiaque, hypokaliémie, antécédent familial de syndrome du QT long).
    13. FEVG ≤ 45 % à la sélection ou au cours des 6 derniers mois ou antécédent d'insuffisance cardiaque aiguë.
    14. Patients ventilo-dépendant.
    15. Hypersensibilité connue à l'une des substances/l’un des excipients du médicament de l'étude.
    16. Pour les patients acceptant de participer aux évaluations d’Imagerie par Résonance Magnétique (IRM) et Spectroscopie par Résonance Magnétique Nucléaire (SRMN) : Patients ayant des contre-indications spécifiques à l'IRM (p. ex. : corps étranger métallique, claustrophobie, etc.).
    E.5 End points
    E.5.1Primary end point(s)
    Each subject will participate in only 1 dose cohort and will receive rimeporide for a total of 4 weeks.

    Safety assessment will be done on data collected in each cohort and during the 4 weeks treatment and 2 weeks of follow-up. The decision to progress to the next higher dose will be made after safety and tolerability data are reviewed for the preceding dose for 5 patients by a data Safety Monitoring Committee and determined that it is safe to proceed to the next dose level.

    Safety and tolerability of rimeporide will be assessed as follows:
    - Incidence, severity, causality and outcomes of AE and SAE
    - Vital signs, Supine and standing blood pressure, heart rate and respiratory rate
    - Evolution of laboratory parameters from blood and urine samples including gastrin levels: substantial changes in laboratory parameters and gastrin;
    - Monitoring of heart function (ECG evolution parameters including QTc);
    - Number of patients withdrawn for safety issues
    Chaque patient participera dans une seule cohorte et recevra rimeporide pour une durée de 4 semaines au total.

    L'évaluation de la sécurité sera effectuée sur les données recueillies dans chaque cohorte, ainsi qu'au cours des quatre semaines de traitement et des deux semaines de suivi. La décision progressant à la dose supérieure sera prise après la revue des données de sécurité et de tolérance de 5 patients à la dose précédente par un comité de surveillance des données de sécurité, et après détermination de
    l'absence de risque lié au passage à la dose supérieure.

    La sécurité et la tolérabilité de rimeporide seront évaluées de la façon suivante:
    - Incidence, sévérité, causalité et évolution des EI et des EIG
    - Constantes vitales, la pression artérielle dans la position couchée et debout, le pouls et la fréquence respiratoire
    - Evolution de paramètres biologiques du sang et de l' urine: variations substantielles des paramètres biologiques et des taux de gastrine.
    - Evaluation de l'activité électrique du coeur (ECG paramètres incluant QTc)
    - Nombre de patients retirés pour des raisons de sécurité.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety endpoints will be assessed during the on site visit at Baseline (day 1) , day 14, day 28 and via a phone call at day 7 and day 21 (flexibility window for each visit or call is +/-2 days) during treatment period and 2 weeks after the last rimeporide dose.

    Safety endpoints will also be assessed if unplanned visit(s) occur and if the patient should need to be assessed or treated for any clinical condition that arises during the study.
    Les critères d'évaluation de la sécurité sont déterminées pendant les visites hôpitaux à la détermination de l' état basal (jour 1), au jour 14, au jour 28 et par téléphone au jour 7 et jour 21 (fenêtre de flexibilité à chaque visite ou appel de téléphone est +/- 2 jours) pendant la période de traitement et 2 semaines après la dernière dose de rimeporide.

    Les critères d' évaluation sont aussi déterminées si des visites non-programmées ont lieu et si le patient sera traité pour une condition clinique qui se présente durant l' étude.
    E.5.2Secondary end point(s)
    • Pharmacokinetic profile of rimeporide in plasma derived from modelling using sparse sampling

    • Pharmacodynamics: Changes of plasma/urine biomarkers:
    - Plasma levels of Troponin I, myomesin, creatine kinase isoenzyme (MM fraction); miRNAs;
    - Urine concentration of titin fragments ;
    - Plasma levels of the inflammation markers: C - reactive protein (CRP), Tumor Necrosis Factor alfa (TNFα), Transforming Growth Factor beta (TGFβ).
    - Plasma level of fibrosis makers: MMP-9.

    Exploratory endpoints:

    Pharmacodynamics: Changes in NMRI and NMRS indices in skeletal muscle will be explored through:
    - 3 NMRI indices (water T2, fat fraction);
    - Nine 31P NMRS indices;
    - 23Na NMRS indices may also be included.

    Participation in MRI/MRS requires travel to the Institute of Myology in Paris and will be optional
    • Profil pharmacocinétique du rimeporide dans le plasma obtenu par modélisation physiologique des parametres pharmacocinétiques (PBPK) sur d’échantillons épars.

    • Pharmacodynamie: Variations des biomarqueurs plasmatiques/urinaires :
    -Taux plasmatiques de troponine I, de myomésine, d'isoenzymes de la créatine kinase (fraction MM) ; micro-ARN.
    -Concentration urinaire des fragments de titine.
    -Taux plasmatiques de marqueurs de l'inflammation : Protéine C réactive (CRP), TNF-α (Tumor Necrosis Factor alfa), TGF-β (Transforming Growth Factor beta).
    -Taux plasmatique de marqueurs de la fibrose : MMP9.


    Critères exploratoires :
    Pharmacodynamie

    Les variations des signaux IRM et SRMN dans le muscle squelettique seront explorées par le biais de
    - trois signaux IRM (eau, T2, graisse) ;
    - neuf signaux SRM du 31P ;
    - des signaux SRM du 23Na pourront également être inclus.

    La participation à la procédure IRM/SRMN nécessite un déplacement à l'Institut de myologie de Paris et sera facultative.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacokinetics:
    At day 1, for half of the patients, samples will be taken at screening or D1 and at (0.5 – 1h), (1 – 2h), (2.5 – 3.5h), 6h after the first dose. For the other half of the patients, samples will be taken before at screening or D1 and at (0.5 – 1h), (1 – 2h), (2.5 – 3.5h), 6h after the second dose. In this way, samples will be taken up to 12h after the first dose, corresponding to approximately 3 half-lives. At week 4, samples will be taken at (0.5 – 1h), 6h after the last dose.

    Pharmacodynamics: Blood samples will be collected at D1 before rimeporide administration and then at week 4 after the last rimeporide dose

    Exploratory end points: Just before first dose of rimeporide and at the end of treatment, between day 21 and day 27, patient will undergo to a MRI/MRS
    PK: Pour la moitié des patients, les échantillons sont pris pendant la visite de la détermination de l'état basal (EB) ou au Jour 1 (J1) et à (0.5-1h), (1 -2h), (2.5-3.5h) et 6h après la 1e dose. Pour l' autre moitié des patients, des échantillons sont pris avant la visite EB ou au J1 et à (0.5-1h), (1 -2h), (2.5-3.5h) et 6h après la deuxième dose. De cet te façon, les échantillons seront pris jusqu'à 12h après la 1e dose, correspondant à +/- 3 demi-vies. Durant la semaine 4, des échantillons seront pris à (0.5-1h) et 6h après la dernière dose.

    PD: Les échantillons de sang seront pris au J1 avant l'administration de rimeporide et à la semaine 4 après la dernière dose.

    Les critères exploratoires: avant la 1e dose et à la fin du traitement, entre J21-J27, le patient subira un IRM/SRMN
    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 No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    first adminitration in Children with DMD (tested in phase 1 in healthy subject and CHF patients)
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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
    étude de groupes séquentielles de doses orales croissantes de médicament de l' étude
    sequential-group study of ascending oral doses of IMP
    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.8.3 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 EEA4
    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
    France
    Italy
    Spain
    Switzerland
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    End of the study is defined as last patient last visit.
    La fin de l'étude est définie par la dernière visite du dernier patient.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 20
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    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
    DMD is characterized by a progressive loss of skeletal muscle and degeneration that lead to a loss of independent ambulation before 13y without disease modifying treatment. For this reason, to satisfy inclusion criteria, this age range is required.
    DMD est caractérisée par une perte progressive des muscles squelettiques et la dégénérescence qui conduit à une perte de déambulation indépendante avant 13 ans sans traitement modifiant la maladie.
    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 20
    F.4.2.2In the whole clinical trial 20
    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)
    In this pilot study, no further treatment beyond Week 4 is planned as no efficacy is claimed by this study. Subject who has ended the participation in the trial, will be treated according to the standard of care at the site. However in case of positive data that will allow the sponsor to move forward with a pivotal study, participation of patients already enrolled in this study will be prioritized in the pivotal study, if the patient wish so.

    Dans cette étude pilote, aucun traitement supplémentaire est prévu aprés semaine 4 parce que l' efficacité n'est pas le but de cette étude. Après participation, les patients seront traités selon le traitement standard de base. Toutefois, en cas de résultats positifs permettant le promoteur de développer une étude pivotale, la participation de patients inclus dans l' étude actuelle sera prioritisée dans l'étude pivotale, si le patient le souhaite.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Duchenne Parent Project Italy
    G.4.3.4Network Country Italy
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation Duchenne Parent Project Spain
    G.4.3.4Network Country Spain
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Duchenne Parent Project Netherlands
    G.4.3.4Network Country Netherlands
    G.4 Investigator Network to be involved in the Trial: 4
    G.4.1Name of Organisation AFM_ Téléthon
    G.4.3.4Network Country France
    G.4 Investigator Network to be involved in the Trial: 5
    G.4.1Name of Organisation Muscular Dystrophy Campaign
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 6
    G.4.1Name of Organisation Action Duchenne
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 7
    G.4.1Name of Organisation FSRMM
    G.4.3.4Network Country Switzerland
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-10-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-12-20
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