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    Summary
    EudraCT Number:2015-002069-52
    Sponsor's Protocol Code Number:4045-301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-02-07
    Trial 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 number2015-002069-52
    A.3Full title of the trial
    A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
    Studio in doppio cieco, controllato con placebo, multicentrico, con un'estensione in aperto per valutare l'efficacia e la sicurezza di SRP-4045 e SRP-4053 in pazienti affetti da distrofia muscolare di Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients
    Studio di ricerca su un nuovo medicinale in fase di sperimentazione per il trattamento di pazienti affetti da Distrofia Muscolare di Duchenne
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    A.4.1Sponsor's protocol code number4045-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02500381
    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 SponsorSAREPTA THERAPEUTICS, INC.
    B.1.3.4CountryUnited States
    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 supportSarepta Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVoisin Consulting
    B.5.2Functional name of contact pointClinical Trial Operations
    B.5.3 Address:
    B.5.3.1Street Address3 rue des Longs Prés
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.3.4CountryFrance
    B.5.4Telephone number0033 1 41 31 83 00
    B.5.5Fax number0033 1 41 31 83 09
    B.5.6E-mailclinicaltrialinformation@voisinconsulting.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSRP-4045
    D.3.2Product code SRP-4045
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1422958-19-7
    D.3.9.2Current sponsor codeSRP-4045
    D.3.9.3Other descriptive nameOligomero morfolino fosforodiamidato per skipping dell’esone 45
    D.3.9.4EV Substance CodeSUB183702
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSRP-4053
    D.3.2Product code SRP-4053
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1422959-91-8
    D.3.9.2Current sponsor codeSRP-4053
    D.3.9.3Other descriptive nameOligomero morfolino fosforodiamidato per skipping dell’esone 53
    D.3.9.4EV Substance CodeSUB170839
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
    Pazienti con Distrofia Muscolare di Duchenne trattabile con lo Skipping dell'esone 45 o 53
    E.1.1.1Medical condition in easily understood language
    Ducheenne muscular dystrophy
    Distrofia muscolare di 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 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
    The primary objective of this study is to evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo on ambulation, endurance, and muscle function at Week 96, as measured by the 6-minute walk test (6MWT)
    L’obiettivo primario di questo studio è valutare l’effetto di SRP-4045 e SRP-4053 (gruppo attivo combinato) rispetto a placebo su deambulazione, resistenza e funzione muscolare alla Settimana 96, misurate dal 6 minute walk test (6MWT).
    E.2.2Secondary objectives of the trial
    The secondary objectives are to evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) on:
    • Dystrophin protein expression in biopsied muscle tissue as measured by:
    - Western blot (quantification)
    - Immunohistochemistry (IHC) fiber intensity
    • Functional status as measured by:
    - Ability to rise independently from the floor (without external support)
    - Loss of ambulation (LOA)
    - North Star Ambulatory Assessment (NSAA)
    - Respiratory muscle function, as measured by forced vital capacity (FVC) % predicted
    - Frequency of falls
    - Cardiac function, as measured by left ventricular ejection fraction (LVEF)
    • Safety and tolerability of SRP-4045 and SRP-4053
    Gli obiettivi secondari sono la valutazione dell’effetto di SRP-4045 e SRP-4053 (gruppo attivo combinato) su:
    • Espressione della proteina distrofina nei tessuti muscolari sottoposti a biopsia, misurata da:
    - Western blot (quantificazione)
    - Intensità della fibra misurata con la tecnica immunoistochimica (IHC)
    • Stato funzionale, misurato da:
    - Capacità di alzarsi in modo indipendente dal pavimento (senza supporto esterno)
    - Perdita di deambulazione (LOA)
    - North Star Ambulatory Assessment (NSAA)
    - Funzione dei muscoli respiratori, misurata dalla % prevista di capacità vitale forzata (FVC)
    - Frequenza delle cadute
    - Funzione cardiaca, misurata dalla frazione di eiezione del ventricolo sinistro (LVEF)
    • Sicurezza e tollerabilità di SRP-4045 e SRP-4053
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Is a male with an established clinical diagnosis of DMD and an out-offrame deletion amenable to:
    ● Exon 45 skipping (including but not limited to deletions of exons such as 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53, or 46-55) OR
    ● Exon 53 skipping (including but not limited to deletions of exons such as 42-52, 45-52, 47-52, 48-52,49-52, 50-52, 52, or 54-58) as documented by a genetic report from an accredited laboratory confirming deletion endpoints by multiplex ligation-dependent probe amplification or sequencing.
    2. Is 7 to 13 years of age, inclusive.
    3. Has stable pulmonary function (forced vital capacity % of predicted [FVC%] ≥50% and no requirement for nocturnal ventilation) that, in the
    Investigator's opinion, is unlikely to decompensate over the duration of the study.
    4. Has intact right and left biceps muscles (the preferred biopsy site) or 2 alternative upper arm muscle groups.
    5. Has been on a stable dose or dose equivalent of oral corticosteroids for at least 24 weeks prior to Week 1 and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study. Note: patients are allowed to take other medications (excluding other ribonucleic acid [RNA] antisense or gene therapy agents) including angiotensinconverting enzyme (ACE) inhibitors, angiotensin receptor blocking
    agents (ARBs), β adrenergic blockers, potassium, and coenzyme Q, provided they have been on a stable dose for 12 weeks prior to Week 1 and the dose is expected to remain constant throughout the study.
    6. Achieved a mean 6MWT distance of ≥300 to ≤450 meters (without assistance) at both the Screening and Baseline visits (prior to Week 1).
    The mean 6MWT distance at the Screening and Baseline visits is the average of 2 separate assessments on 2 consecutive days at each visit.
    The Baseline mean (average of Baseline Days 1 and 2) must be within 15% of the Screening mean distance (average of Screening Days 1 and
    2).
    7. Sexually active subjects must agree to use contraceptives for the entire duration of the study and for 90 days following the last dose. Both a male condom and a medically acceptable form of female
    contraceptive (e.g., oral contraceptives) must be used.
    8. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
    9. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
    Per risultare idoneo per la partecipazione a questo studio, ogni paziente deve soddisfare tutti i seguenti criteri.
    1. Deve essere di sesso maschile, con una diagnosi clinica definitiva di DMD e una delezione out-of-frame trattabile con:
    • Skipping dell’esone 45 (comprese, fra le altre, delezioni di esoni come 12 44, 18 44, 44, 46 47, 46 48, 46 49, 46 51, 46 53 o 46 55) OPPURE
    • Skipping dell’esone 53 (comprese, fra le altre, delezioni di esoni come 42 52, 45 52, 47 52, 48 52, 49 52, 50 52, 52 o 54 58) come documentato da un rapporto genetico da parte di un laboratorio accreditato che conferma gli endpoint di delezione per mezzo della tecnica MLPA (multiplex ligation-dependent probe amplification or sequencing).
    2. Deve avere dai 7 ai 13 anni di età, inclusi.
    3. Deve avere una funzionalità polmonare stabile (% prevista della capacità vitale forzata di [FVC%] ≥50% e assenza di necessità di ventilazione notturna) che, secondo l’opinione dello Sperimentatore, è improbabile che si scompensi nel corso della durata dello studio.
    4. Deve avere muscoli bicipiti sinistro e destro intatti (sede di preferenza per la biopsia) o 2 gruppi muscolari alternativi nel braccio.
    5. Sta assumendo una dose stabile o equivalente di dose di corticosteroidi orali da almeno 24 settimane prima della Settimana 1 e la dose deve rimanere costante (ad eccezione di modifiche dovute alla regolazione della dose per adeguarsi ai cambiamenti di peso) per tutta la durata dello studio. Nota: i pazienti possono assumere altri medicinali (esclusi altri agenti terapeutici genetici o acido ribonucleico [RNA] antisenso) inclusi inibitori dell'enzima convertitore dell'angiotensina (ACE), agenti bloccanti del ricettore dell’angiotensina (ARB), bloccanti dei β-adrenergici, potassio e coenzima Q, a condizione che ne assumano una dose stabile per 12 settimane prima della Settimana 1 e che si preveda che la dose rimarrà costante per tutta la durata dello studio.
    6. Deve aver ottenuto una distanza media al 6MWT compresa fra ≥300 e ≤450 metri (senza aiuto) sia alla visita di Screening che alla visita Baseline (prima della Settimana 1). La distanza media al 6MWT alle visite di Screening e Baseline è rappresentata dalla media di due valutazioni separate durante due giorni consecutivi durante ogni visita. La media Baseline (media dei Giorni 1 e 2 della visita Baseline) deve rientrare entro il 15% della distanza media alla visita di Screening (media dei Giorni 1 e 2 di Screening).
    7. I soggetti sessualmente attivi devono acconsentire ad utilizzare concezionali per l’intera durata dello studio e per 90 giorni in seguito all’ultima dose. Devono essere utilizzati sia un preservativo maschile che una forma contraccettiva femminile accettabile dal punto di vista medico (ad esempio, un contraccettivo per via orale).
    8. Deve avere uno o più genitore/i o custode/i legale/i in grado di comprendere e conformarsi con tutti i requisiti dello studio.
    9. Deve essere disponibile a fornire un consenso informato (se applicabile) e deve avere uno o più genitore/i o custode/i legale/i disponibile/i a fornire un consenso informato scritto per la partecipazione del paziente allo studio.
    E.4Principal exclusion criteria
    1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to Week 1 that may have an effect on muscle strength or function. Growth hormone for short stature and testosterone for delayed puberty are permitted if an endocrinologist has documented the diagnosis and medical necessity of treatment, and the patient has been on a stable dose for at least 24 weeks prior to Week 1.
    2. Previous treatment with SMT C1100 (BMN-195) at any time.
    3. Previous treatment with PRO045 or PRO053 within 24 weeks prior to Week 1.
    4. Current or previous treatment with any other experimental treatment (other than deflazacort) within 12 weeks prior to Week 1.
    5. Major surgery within 3 months prior to Week 1 or planned surgery for any time during this study, except for protocol-specified surgery, as applicable.
    6. Presence of any other significant genetic disease other than DMD (e.g., dwarfism).
    7. Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or malignancy.
    8. Use of any aminoglycoside antibiotic or statin within 12 weeks prior to Week 1 or anticipated need for an aminoglycoside antibiotic or statin
    during the study.
    9. LVEF <50% on the Screening echocardiogram (ECHO) or QTcF ≥450 msec based on the Screening and Baseline ECG.
    10. Loss of ≥30 degrees of plantar flexion from the normal range of movement at the ankle joint due to contracture (i.e., fixed loss of more than 10 degrees of plantar flexion from plantigrade, assuming normal range of dorsiflexion of 20 degrees).
    11. Prior or ongoing medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results. Additionally, patients who seem unable / unwilling to comply with the study procedures, in the Investigator's opinion, are to be excluded.
    Il paziente che soddisfi qualunque dei seguenti criteri sarà escluso dallo studio.
    1. Uso di qualsiasi trattamento farmacologico (diverso dai corticosteroidi) entro 12 settimane prima della Settimana 1 che possa avere un effetto sulla forza o sulla funzione muscolare. L’ormone della crescita per una statura ridotta e del testosterone per una pubertà tardiva sono consentiti se un endocrinologo ha documentato la diagnosi e la necessità medica del trattamento, e se il paziente assume una dose stabile da almeno 24 settimane prima della Settimana 1.
    2. Trattamento precedente con SMT C1100 (BMN-195) in qualsiasi momento.
    3. Trattamento precedente con PRO045 o PRO053 entro 24 settimane prima della Settimana 1.
    4. Trattamento attuale o precedente con qualsiasi altro trattamento sperimentale (ad eccezione di deflazacort) entro 12 settimane prima della Settimana 1.
    5. Intervento chirurgico importante entro 3 mesi prima della Settimana 1 o intervento chirurgico in programma per qualsiasi momento durante lo studio, ad eccezione degli interventi chirurgici specificati dal protocollo, come applicabile.
    6. Presenza di qualsiasi altra malattia genetica significativa diversa da DMD (ad esempio, nanismo).
    7. Presenza di altra malattia significativa dal punto di vista clinico, comprese malattia cardiaca, polmonare, epatica, renale, ematologica, immunologica o comportamentale significativa, o tumore maligno.
    8. Uso di qualsiasi antibiotico o statina aminoglicosidi entro 12 settimane prima della Settimana 1 o prevista necessità di antibiotico o statina aminoglicosidi durante la durata dello studio.
    9. LVEF <50% all’ecocardiogramma durante la visita di Screening (ECHO) o QTcF ≥450 msec sulla base dell’ECG alle visite di Screening e Baseline.
    10. Perdita di ≥30 gradi di flessione plantare dal normale range di movimento all’articolazione della caviglia a causa di una contrattura (ad esempio, perdita fissa di più di 10 gradi di flessione plantare del piede, presupponendo che il range normale di dorsiflessione sia di 20 gradi).
    11. Precedente o attuale problema medico che, secondo l’opinione dello Sperimentatore, potrebbe influenzare negativamente il paziente, rendere impossibile il completamento del trattamento o pregiudicare la valutazione dei risultati dello studio. Inoltre, sono esclusi i pazienti che, secondo lo Sperimentatore, non sembrano in grado / disposti a conformarsi con le procedure dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    Change from Baseline at Week 96 on the 6MWT for the combined-active group compared to placebo
    Cambiamento dalla Baseline alla Settimana 96 nel 6MWT per il gruppo attivo combinato rispetto al gruppo placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Baseline, Week 12, 24, 36, 48, 60, 72, 84, 96
    Screening, Baseline, Settimane 12, 24, 36, 48, 60, 72, 84, 96
    E.5.2Secondary end point(s)
    ●Change from Baseline at Week 48 in the quantity of dystrophin protein expression as measured by Western blot of biopsied muscle tissue ●Change from Baseline at Week 48 in the intensity of dystrophin expression in biopsied muscle tissue, as measured by IHC ● Ability to rise independently from the floor (without external support) at Week 96, as indicated by an NSAA subscore of "2" (without modification) or "1" (Gower's maneuver) ● LOA status at Week 96, defined as an inability to perform the 6MWT, or result of "0" meters on 6MWT ● Change from Baseline at Week 96 - NSAA total score - FVC% predicted - Frequency of falls – LVEF
    • Cambiamento dalla Baseline alla Settimana 48 nella quantità di espressione della proteina distrofina, misurata da Western blot del tessuto muscolare sottoposto a biopsia. • Cambiamento dalla Baseline alla Settimana 48 nell’intensità dell’espressione della distrofina nel tessuto muscolare sottoposto a biopsia, misurato da IHC. • Capacità di alzarsi in modo indipendente dal pavimento (senza supporto esterno) alla Settimana 96, come indicato da un sottopunteggio NSAA pari a “2” (senza modifiche) o “1” (segno di Gowers). • Stato LOA alla Settimana 96, definito quale incapacità di eseguire il 6MWT, o risultato di “0” metri nel 6MWT. • Cambiamento dalla Baseline alla Settimana 96 in: o Punteggio totale NSAA o FVC% previsto o Frequenza delle cadute o LVEF
    E.5.2.1Timepoint(s) of evaluation of this end point
    ● Quantity of dystrophin protein expression: Baseline, week 48 ● Intensity of dystrophin expression: Baseline, week 48 ● Ability to rise independently from the floor: Screening, Baseline, Week 12, 24, 36, 48, 60, 72, 84, 96 ● LOA status: Week 96
    ● Quantità di espressione della proteina distrofina: Baseline, Settimane 48 ● Intensità dell’espressione della distrofina : Baseline, Settimane 48 ● Possibilità di salire indipendentemente dal pavimento: Screening, Baseline, Settimane 12, 24, 36, 48, 60, 72, 84, 96 ● Stato LOA : Settimane 96
    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 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) 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 EEA20
    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
    Belgium
    France
    Germany
    Netherlands
    Sweden
    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
    Last patient last visit
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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: 84
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 99
    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)
    None
    Nessuno
    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 Decision2017-01-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-11-17
    P. End of Trial
    P.End of Trial StatusOngoing
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