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    Summary
    EudraCT Number:2013-003450-24
    Sponsor's Protocol Code Number:HGT-SAN-093
    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:2014-05-09
    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 number2013-003450-24
    A.3Full title of the trial
    A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease
    Studio di Fase IIb randomizzato, controllato, in aperto, multicentrico circa la sicurezza e l'efficacia della somministrazione di HGT-1410 (Eparan N-solfatasi umana ricombinante) tramite dispositivo per la somministrazione intratecale di farmaci in pazienti pediatrici affetti da Mucopolisaccaridosi di Tipo III A allo stadio iniziale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A
    Studio relativo alla sicurezza e all’efficacia della somministrazione di HGT-1410 in pazienti pediatrici affetti da Sindrome di Sanfilippo di Tipo A allo stadio iniziale
    A.4.1Sponsor's protocol code numberHGT-SAN-093
    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 SponsorShire Human Genetic Therapies, 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 supportShire Human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Human Genetic Therapies Inc
    B.5.2Functional name of contact pointKenia Baez
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0017814829620
    B.5.5Fax number0017814821819
    B.5.6E-mailkbaez@shire.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/3/08/582
    D.3 Description of the IMP
    D.3.1Product nameRecombinant human heparan N-sulfatase (rhHNS)
    D.3.2Product code HGT-1410
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.2Current sponsor codeHGT-1410
    D.3.9.3Other descriptive nameRecombinant human heparan N-sulfatase
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeHuman protein heparan N-sulfatase produced in a human cell line by genetic engineering technology.
    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
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Sindrome di Sanfilippo di Tipo A o Mucopolisaccaridosi (MPS IIIA)
    E.1.1.1Medical condition in easily understood language
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Sindrome di Sanfilippo di Tipo A o Mucopolisaccaridosi (MPS IIIA)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10056918
    E.1.2Term Sanfilippo's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis III
    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 potential clinical efficacy of HGT-1410 administered via a surgically implanted IDDD in patients with Sanfilippo Syndrome Type A (MPS IIIA). Efficacy will be measured as a meaningful amelioration in the progression of cognitive decline, and will be measured using the Bayley Scales of Infant Development, 3rd Edition (BSID III).
    Valutare l’efficacia clinica potenziale di HGT-1410 somministrato tramite un IDDD impiantato chirurgicamente in pazienti affetti da Sindrome di Sanfilippo di Tipo A (MPS IIIA). L’efficacia sarà misurata come un miglioramento significativo nella progressione del declino cognitivo, e sarà misurata usando le Scale di Bayley per la valutazione dello sviluppo infantile, terza edizione (Bayley Scales of Infant Development, BSID-III).
    E.2.2Secondary objectives of the trial
    To determine the following: the safety and tolerability of HGT-1410 IT; the effect of HGT-1410 administration on BSID-III age-equivalent and DQ scores; the effect of HGT-1410 IT on adaptive behavioral function, assessed by Vineland Adaptive Behavior Scales, Second Edition (VABS-II); the effect of HGT-1410 IT treatment on the total cortical grey matter volume, as assessed by volumetric MRI of the brain; the effect of HGT-1410 IT treatment on the concentration of GAG in CSF and urine; and the pharmacokinetics of HGT-1410 in cerebrospinal fluid (CSF) and serum.
    Determinare quanto segue: la sicurezza e la tollerabilità di HGT-1410 intratecale (IT); l’effetto della somministrazione di HGT-1410 sui punteggi delle scale BSID-III equivalenti per l’età e sul quoziente di sviluppo (Development Quotient, DQ); l’effetto di HGT-1410 IT sulla funzione adattiva comportamentale, valutato tramite le Scale di comportamento adattivo secondo Vineland, seconda edizione (Vineland Adaptive Behavior Scales, VABSII); l’effetto del trattamento IT con HGT-1410 sul volume totale della materia grigia corticale, valutato tramite risonanza magnetica (RMI) volumetrica del cervello; l’effetto del trattamento IT con HGT-1410 sulla concentrazione di glicosamminoglicani (glycosaminoglicans, GAG) nel liquido cerebrospinale (Cerebrospinal Fluid, CSF) e nelle urine; e la farmacocinetica di HGT-1410 nel liquido cerebrospinale (CSF) e nel siero.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to be considered eligible for enrollment:
    1. Documented MPS IIIA diagnosis:
    a) All patients must show a documented deficiency in sulfamidase enzyme activity of ≤10% of the lower limit of the normal range as measured in fibroblasts or leukocytes
    AND
    b) Patients must show documented mutations in each SGSH allele OR there must be documentation of mutations in each SGSH allele in a sibling affected by MPSIIIA, provided parental consent is obtained to use this information.
    2. Age ≥12 months and ≤ 48 months
    3. The patient has a DQ score ≥60%, assessed by cognitive evaluation at screening assessment using the BSID-III
    4. The patient is medically stable, in the opinion of the Investigator, and able to accommodate the protocol requirements, including travel, assessments, and IDDD surgery, without placing an undue burden on the patient/patient's family
    5. The patient’s parent(s) or legal guardian must have voluntarily signed an Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient’s parent(s), or legal guardian. Consent of the patient’s parent(s) or legally authorized guardian(s) must be obtained prior to the start of any study procedures.
    I pazienti devono soddisfare tutti i seguenti criteri per essere considerati idonei per l’arruolamento:
    1. Diagnosi di MPS IIIA documentata:
    a) Tutti i pazienti devono presentare una deficienza documentata dell’attività dell’enzima sulfamidasi ≤10% al limite inferiore dell’intervallo normale misurata in fibroblasti o leucociti
    E
    b) I pazienti devono presentare mutazioni documentate in ogni allele sulfoglucosamina sulfoidrolasi (sulfoglucosamine sulfohydrolase, SGSH) O devono essere documentate le mutazioni in ogni allele SGSH in un fratello o una sorella affetti da MPSIIIA, fermo restando che sia stato ottenuto il consenso dei genitori per l’uso di queste informazioni.
    2. Età ≥12 mesi e ≤ 48 mesi
    3. Il paziente ha un punteggio DQ ≥60%, stabilito tramite valutazione cognitiva alla valutazione allo screening usando la scala BSID-III
    4. Secondo il parere dello Sperimentatore il paziente è clinicamente stabile e in grado di aderire ai requisiti del protocollo, inclusi viaggi, valutazioni e intervento chirurgico per l’IDDD, senza rappresentare un carico spropositato sul paziente/familiari del paziente
    5. Il/i genitore/i del paziente o rappresentante/i legalmente autorizzato/i deve/devono avere firmato volontariamente un modulo di consenso informato approvato da un Comitato etico indipendente dopo che tutti gli aspetti rilevanti dello studio sono stati illustrati e discussi con il/i genitore/i del paziente o il/i rappresentante/i legalmente autorizzato/i. Il consenso da parte del/i genitore/i del paziente o del/i rappresentante/i legalmente autorizzato/i deve essere ottenuto prima di iniziare qualsiasi procedura dello studio.
    E.4Principal exclusion criteria
    Patients will be excluded from the study if any of the following criteria are met at screening:
    1. The presence of significant non-MPS IIIA related central nervous system (CNS) impairment or behavioral disturbances that would confound the scientific integrity or interpretation of study assessments, as determined by the Investigator.
    2. The presence of at least one S298P mutation in SGSH, associated with attenuated disease OR there is documentation of the S298P mutation in a sibling affected by MPS IIIA, provided parental consent is obtained to use this information.
    3. The presence of relatively attenuated MPS IIIA disease in an older sibling, defined as preservation of any comprehensible speech beyond the age of 10 years
    4. Visual or hearing impairment, in the clinical judgement of the investigator, sufficient to preclude cooperation with neurodevelopmental testing.
    5. In the opinion of the Investigator, the patient is assessed as having an unacceptably high risk for anesthesia due to airway compromise, drug hypersensitivity, or other conditions (such as neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns).
    6. The patient has a history of poorly controlled seizure disorder.
    7. The patient is currently receiving psychotropic or other medications, which in the Investigator’s opinion would be likely to substantially confound test results.
    8. The patient has a history of bleeding disorder or is unable to abstain from medications that, in the opinion of the investigator, place them at risk of bleeding following surgery or lumbar puncture..
    9. The patient participated in a clinical trial of another investigational medicinal product, within the 30 days prior to the study (or within 5 elimination half lives of the investigational product), or is currently enrolled in another study that involves an investigational drug or device. NOTE: Nutritional supplements, including genistein are permitted if they are taken or administered outside the context of a formal investigation.
    10. The patient has received a hematopoietic stem cell or bone marrow transplant, or gene therapy.
    11. The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
    a) The patient has had an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
    b) The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
    c) The patient has a known or suspected local or general infection
    d) The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    e) The patient has a functioning CSF shunt device
    f) The patient has shown an intolerance to an implanted device
    12. The patient’s parent(s) or patient’s legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study, or do/does not agree to comply with the protocol defined schedule of assessments.
    13. The patient is unable to comply with the protocol (eg, has a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, or otherwise unlikely to complete the study) or is, in the opinion of the Investigator, otherwise unsuited for the study.
    14. The patient has any item (braces, tattoos, etc.) which would exclude the patient from being able to undergo MRI according to local Institutional Policy, or the patient has any other situation that would exclude the patient from undergoing any other procedure required in this study.
    I pazienti saranno esclusi dallo studio se uno qualsiasi dei seguenti criteri è rilevato allo screening:
    1. La presenza di deterioramento significativo del sistema nervoso centrale (SNC) non correlato alla MPS IIIA o disturbi comportamentali che confonderebbero l’integrità scientifica o l’interpretazione delle valutazioni dello studio, come determinato dallo Sperimentatore.
    2. La presenza di almeno una mutazione S298P in SGSH, associata alla malattia attenuata O mutazione documentata di S298P in un fratello o una sorella affetti da MPS IIIA, fermo restando che sia stato ottenuto il consenso dei genitori per l’uso di queste informazioni.
    3. La presenza di MPS IIIA relativamente attenuata in un fratello o una sorella di età maggiore, definita come preservazione di un eloquio comprensibile oltre i 10 anni di età
    4. Indebolimento visivo o uditivo sufficiente a precludere la cooperazione per i test di neurosviluppo, secondo il giudizio clinico dello sperimentatore.
    5. Secondo il parere dello Sperimentatore, l’anestesia potrebbe rappresentare per il paziente un rischio inaccettabile, dovuto a compromissione delle vie aeree, ipersensibilità al farmaco o altre condizioni (come sindrome maligna neurolettica, ipertermia maligna o altri problemi correlati all’anestesia).
    6. Il paziente presenta un’anamnesi di disturbo convulsivo scarsamente controllato.
    7. Il paziente sta attualmente ricevendo farmaci psicotropi o di altra natura, che secondo il parere dello Sperimentatore potrebbero confondere in misura sostanziale i risultati delle analisi.
    8. Il paziente ha un’anamnesi di disturbo emorragico o non è in grado di astenersi da farmaci che, secondo l’opinione dello sperimentatore, lo porrebbero a rischio di emorragia dopo un intervento chirurgico o la puntura lombare.
    9. Il paziente ha partecipato a una sperimentazione clinica di un altro medicinale sperimentale nei 30 giorni precedenti allo studio (o entro 5 emivite di eliminazione del prodotto sperimentale) o è attualmente arruolato in un altro studio che comporta l’assunzione di un farmaco o un dispositivo sperimentale. NOTA: gli integratori nutrizionali, inclusa la genisteina, sono permessi se presi o somministrati al di fuori del contesto di una investigazione formale.
    10. Il paziente ha ricevuto un trapianto di cellule staminali o di midollo osseo emopoietico o una terapia genica.
    11. Il paziente soffre di una condizione che è controindicata secondo le Istruzioni per l’uso dell’IDDD SOPH-A-PORT Mini S, tra cui:
    a) Il paziente ha avuto una reazione allergica ai materiali di costruzione del dispositivo SOPH-A-PORT Mini S.
    b) Il corpo del paziente è troppo piccolo per sostenere le dimensioni della porta di accesso SOPH-A-PORT Mini S, secondo il parere dello Sperimentatore.
    c) Il paziente ha un’infezione nota o sospetta a livello locale o generale.
    d) Il paziente ha una o più anomalie spinali che potrebbero complicare l’impianto o la fissazione sicura.
    e) Il paziente ha un dispositivo shunt cerebrospinale (CSF) in funzione.
    f) Il paziente ha mostrato intolleranza a un dispositivo impiantato 12. Il/i genitore/i del paziente o il/i tutore/i legale/i del paziente non è/sono in grado di comprendere la natura, l’ambito e le possibili conseguenze dello studio, o non acconsente/ono di aderire al programma di valutazioni previsto dal protocollo.
    13. Il paziente non è in grado di aderire al protocollo (ad es., è affetto da una condizione medica clinicamente rilevante che rende l’implementazione del protocollo difficile, ha una situazione sociale instabile o il completamento dello studio è improbabile) o è, secondo il parere dello Sperimentatore, inadatto per lo studio.
    14. Il paziente ha qualunque cosa (tutori, tatuaggi, ecc.) che escluderebbe il paziente dalla possibilità di sottoporsi a una RMI secondo la Politica istituzionale locale, o il paziente ha una qualsiasi situazione che lo escluderebbe dal sottoporsi a qualsiasi altra procedura richiesta in questo studio.
    E.5 End points
    E.5.1Primary end point(s)
    Achievement of response, defined as a maximum decline in cognitive DQ of 10 points over 48 weeks, as assessed by the Bayley Scales of Infant Development, 3rd Edition.
    Ottenimento della risposta, definita come un declino massimo del DQ cognitivo di 10 punti nell’arco di 48 settimane, valutato dalle Bayley Scales of Infant Development, terza edizione.
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 settimane
    E.5.2Secondary end point(s)
    • Safety endpoints including the assessment of adverse events (AEs), IDDD related issues, laboratory values, anti-rhHNS antibody development, vital signs, physical examination findings, and ECG results
    • The change from baseline to Week 48 in adaptive behavioral function, assessed by VABS II, using raw scores and age equivalent score
    • The change from baseline to Week 48 in the DQ assessed by neurocognitive testing, using the BSID-III age equivalent scores
    • The change from Baseline to Week 48 in total cortical grey matter volume, as assessed by MRI
    • The change from Baseline to Week 48 in concentrations of GAG in the CSF and urine
    • The concentration of HGT-1410 in CSF and serum
    • Endpoint di sicurezza, quali la valutazione di eventi avversi (EA), problemi correlati all’IDDD, valori degli esami di laboratorio, sviluppo di anticorpi anti-rhHNS, segni vitali, rilievi all’esame obiettivo e risultati dell’ECG.
    • La variazione dal basale alla Settimana 48 della funzione adattiva comportamentale, valutata tramite le scale VABS II, usando i punteggi grezzi e il punteggio equivalente per l’età.
    • La variazione dal basale alla Settimana 48 del DQ valutata tramite test neurocognitivi, usando i punteggi equivalenti per l’età secondo la scala BSID-III.
    • La variazione dal basale alla Settimana 48 del volume totale della materia grigia corticale, valutata tramite RMI.
    • La variazione dal basale alla Settimana 48 delle concentrazioni di GAG nel CSF e nelle urine.
    • La concentrazione di HGT-1410 nel CSF e nel siero.
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 settimane
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy 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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    assenza di gruppo di controllo del trattamento
    no treatment control group
    E.8.2.4Number of treatment arms in the trial3
    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 EEA6
    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
    Germany
    Netherlands
    Spain
    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 visit of the last patient in the clinical study
    L'ultima visita dell'ultimo paziente nello studio clinico
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years2
    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: 18
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 8
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    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
    Children between 12 and 48 months
    bambini di età compresa tra i 12 e i 48 mesi
    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 8
    F.4.2.2In the whole clinical trial 18
    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)
    It is anticipated that patients participating in this trial will be offered, upon completion of the study, enrollment in an extension trial, in which patients originally randomized to active treatment will continue to receive the same treatment regimen, while those originally randomized to the no-treatment control group will be re-randomized to one of the 2 active treatment regimens.
    A completamento dello studio, ai pazienti che partecipano a questa sperimentazione verrà offerta la possibilità di partecipare a una sperimentazione di estensione, nella quale i pazienti originariamente randomizzati al trattamento attivo continueranno a ricevere lo stesso regime posologico, mentre quelli originariamente randomizzati al gruppo di controllo senza trattamento saranno randomizzati di nuovo a uno dei 2 regimi di trattamento attivi.
    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 Decision2014-06-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-05-13
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