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    Summary
    EudraCT Number:2015-002530-50
    Sponsor's Protocol Code Number:EspeRare_RIM_001
    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:2018-08-22
    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 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 (DMD)
    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 (DMD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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 (DMD)
    Studio di fase Ib in aperto volto a valutare la sicurezza, la tollerabilità e il profilo farmacocinetico e farmacodinamico di dosi multiple ascendenti
    di rimeporide somministrate per via orale a pazienti affetti da distrofia muscolare di Duchenne (DMD)
    A.3.2Name or abbreviated title of the trial where available
    RIM4DMD
    RIM4DMD
    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 FOUNDATION
    B.1.3.4CountryUnited Kingdom
    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 number441908251480
    B.5.5Fax number441908251499
    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.3.11 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
    Distrofia Muscolare di Duchenne (DMD)
    Distrofia Muscolare di Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    Distrofia Muscolare di Duchenne (DMD)
    Distrofia Muscolare di Duchenne (DMD)
    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 determine the preliminary safety and tolerability profile of multiple oral administrations of rimeporide.
    Determinare la sicurezza preliminare e il profilo di tollerabilità di dosi multiple di rimeporide somministrate per via orale.
    E.2.2Secondary objectives of the trial
    •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).
    • Farmacocinetica: Valutare il profilo farmacocinetico di rimeporide in pazienti pediatrici affetti da DMD.

    • Farmacodinamica:
    - Misurare i biomarcatori infiammatori e delle lesioni muscolari;
    - Esplorare il rapporto farmacocinetica/farmacodinamica del trattamento di 4 settimane con rimeporide su tali biomarcatori surrogati.
    Obiettivi esplorativi:
    • Esplorare il rapporto tra endpoint di sicurezza e parametri farmacocinetici;
    • Esplorare, mediante spettroscopia di risonanza magnetica nucleare (NMRS) e indagini di imaging, l'effetto del trattamento di 4 settimane con rimeporide sui biomarcatori, tra cui pH e Na intracellulare, e il grado di infiammazione, edema, frazione lipidica e composizione muscolare.
    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.Distrofia muscolare di Duchenne confermata mediante analisi genetica;
    2.Soggetti di sesso maschile di età compresa tra 6 e 14 anni;
    3.Capacità di deambulare autonomamente per almeno 75 metri;
    4.Pazienti in trattamento con dose stabile di corticosteroidi per almeno 6 mesi prima del basale;
    5.Disponibilità e capacità di rispettare tutti i requisiti e le procedure del protocollo;
    6.Consensi informati firmati dal/i genitore/i o dal/i tutore/i legale/i;
    7.Solo per la Francia: soggetti affiliati a o beneficiari di un sistema previdenziale.
    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.Pazienti affetti da malattia o insufficienza renale significativa, con tasso di filtrazione glomerulare stimato utilizzando la concentrazione plasmatica di cistatina C con formula di Filler inferiore a 90 ml/min/1,73 m2;
    2.Malattia o insufficienza epatica in corso o pregressa;
    3.Anamnesi positiva per qualsiasi patologia clinica significativa che potrebbe interferire con l'interpretazione dei dati di efficacia o di sicurezza, ad es. malattia infiammatoria, della coagulazione, respiratoria o cardiaca instabile;
    4.Malattia acuta nelle 4 settimane precedenti alla prima somministrazione del farmaco in studio che potrebbe interferire con le valutazioni della sperimentazione;
    5.Modifica significativa del dosaggio e/o dei regimi posologici dei corticosteroidi pianificata nel corso del trattamento con il farmaco in studio;
    6.Uso di betabloccanti e ACE-inibitori o antagonisti del recettore dell'angiotensina II (ARB), salvo qualora somministrati a dose stabile per almeno 3 mesi prima del basale;
    7.Uso di inibitori della pompa protonica definiti dal protocollo, salvo qualora somministrati a dose stabile per almeno 3 mesi prima del basale;
    8.Uso di antagonisti dell'aldosterone (ossia spironolattone, eplerenone) nei 3 mesi precedenti alla prima somministrazione del farmaco in studio;
    9.Uso di anticoagulanti, antitrombotici o antiaggreganti;
    10.Trattamento precedente con idebenone o altre forme del coenzima Q10 nel mese precedente alla prima somministrazione del farmaco in studio;
    11.Trattamento precedente con farmaci sperimentali nelle 4 settimane (o 7 emivite, se superiori a 4 settimane) precedenti alla prima somministrazione del farmaco in studio, compreso placebo;
    12.QTc >450 msec al basale o anamnesi positiva per fattori di rischio della torsione di punta (ad es. insufficienza cardiaca, ipopotassiemia, anamnesi familiare positiva per sindrome del QT lungo);
    13.Frazione di eiezione del ventricolo sinistro (LVEF) ≤45% allo screening o negli ultimi 6 mesi e/o anamnesi positiva per insufficienza cardiaca acuta;
    14.Dipendenza dal ventilatore;
    15.Ipersensibilità personale nota a uno qualsiasi dei componenti/degli eccipienti del farmaco in studio;
    16Per i pazienti disponibili a sottoporsi alle RM/spettroscopie di RM (MRS): pazienti che presentano controindicazioni specifiche alle RM (ad es. presenza di corpo estraneo metallico, claustrofobia, ecc.).
    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
    - Incidenza, gravità, causalità ed esiti degli eventi avversi (AE) e degli eventi avversi gravi (SAE);
    - Alterazioni sostanziali dei parametri di laboratorio e della gastrina;
    - Numero di pazienti che interrompono lo studio per problemi di sicurezza.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety endpoints will be assessed druning on site visit at Baseline, day 14, day 28 and via a phone call at day 7 and day 21 (fexibility window for each visit or call is +/-2 days) during treatment period and 2 weeks after the last rimeporide dose.

    Safety endpoints will be also assessed if unplanned visit(s) may occur should the patient need to be assessed or treated for any clinical condition that arises during the study.
    Gli endpoints di sicurezza verranno stimati durante le visite al centro al Baseline, al giorno 14, al giorno 28 e tramite contatto telefonico al giorno 7 e giorno 21 (finestra di flessibilità per ogni visita o telefonata di +/- 2 giorni) durante il periodo di trattamento e 2 settimane dopo l'ultima dose di rimeporide.
    Gli endnpoint di sicurezza verranno stimati in caso di visite non pianificate se il paziente necessita di essere valutato o trattato per qualsiasi condizione medica si presentasse durante lo studio
    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
    • Profilo farmacocinetico di rimeporide nel plasma ottenuto mediante modellazione con tecnica di compressed sensing.
    • Alterazioni dei biomarcatori plasmatici/urinari:
    - Livelli plasmatici di troponina I, miomesina, isoenzima della creatinchinasi (frazione MM); miRNA;
    - Concentrazione di frammenti di titina nelle urine;
    - Livelli plasmatici dei marcatori infiammatori: proteina C-reattiva (PCR), fattore di necrosi tumorale alfa (TNFα), fattore di crescita trasformante beta (TGFβ);
    - Livello plasmatico dei marcatori della fibrosi: MMP9.

    Endpoint esplorativi:
    Farmacodinamica

    • Verranno esaminate approfonditamente le alterazioni degli indici delle NRM e delle NMRS nei muscoli scheletrici.
    - 3 indici NRM (acqua T2, frazione lipidica);
    - Nove indici NMRS 31P;
    - Potrebbero essere inclusi anche indici NMRS 23Na.

    La partecipazione alle RM/MRS richiede che i pazienti si rechino presso l'Istituto di miologia di Parigi e sarà facoltativa.
    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.

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

    Exploratory enpoints: 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.
    Farmacocinetica:
    al giorno 1, per metà dei pazienti, i campioni verranno raccolti allo screening o D1 e a (0.5-1h), (1 – 2h), (2.5 – 3.5h), 6h dopo la prima dose. Per l'altra metà dei pazienti, i campioni verranno raccolti allo screening o D1 e a (0.5 – 1h), (1 – 2h), (2.5 – 3.5h), 6h dopo la seconda dose. In questo modo, i campioni verranno raccolti fino a 12h dopo la prima dose, corrispondente approssimamente a 3 emi-vite. Alla settimana 4, i campioni verranno raccolti a (0.5 – 1h), 6h dopo l'ultima dose.

    Farmacodinamica: i campioni verranno raccolti a D1 prima della somministrazione di rimeporide e poi alla settimana 4 dopo l'ultima dose di rimeporide.

    Enpoints esplorativi: i pazienti saranno sottoposti a MRI/MRS appena prima la prima dose di rimeporide e alla fine del trattamento
    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 administration in Children with DMD (tested in phase 1 in healthy subject and CHF patients)
    Prima somministrazione in bambini con DMD (testato in fase 1 in volontari sani e pazienti CHF)
    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 Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    a dosi multiple ascendenti somministrate per via orale.
    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.8.4.1Number of sites anticipated in Member State concerned1
    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 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
    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
    LVLS
    LVLS
    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: 1
    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 and degeneration of the skeletal muscle which leads to a loss of ability to walk independently before 13 years without change the treatment of the disease, For this reason, it is required this age range .
    DMD causa perdita progressiva e degenerazione del muscolo scheletrico che porta a perdita di capacità di deambulazione indipendente prima del 13 anni senza modificare il trattamento della malattia, Per questa ragione, è richiesto questo range di età.
    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 15
    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, it is not planned further treatment beyond 4 weeks. The subjects who completed the trial will be treated according to the standard of care of the center as a specific rescue therapy is scheduled for DMD. However, in case of positive data that will enable the promoter to go to a pivotal study, will be prioritized in the pivotal study included patients already enrolled in this study, if patients so desire.
    In questo studio pilota, non è pianificato un ulteriore trattamento oltre le 4 settimane. I soggetti che hanno terminato il trial verranno trattati in accordo allo standard of care del centro poichè una specifica rescue terapia è prevista per DMD. Comunque in caso di dati positivi che consentano al Promotore di andare verso uno studio pivotal, verrà priorizzata nello studio pivotal la partecipazione di pazienti già arruolati in questo studio, se i pazienti lo desiderano.
    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 Netherlands
    G.4.3.4Network Country Netherlands
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation AFM_ Téléthon
    G.4.3.4Network Country France
    G.4 Investigator Network to be involved in the Trial: 3
    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: 4
    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: 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 Decision2016-01-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-19
    P. End of Trial
    P.End of Trial StatusCompleted
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