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    Summary
    EudraCT Number:2014-003556-31
    Sponsor's Protocol Code Number:SHP-607-201
    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:2016-03-09
    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 number2014-003556-31
    A.3Full title of the trial
    Long-term Outcome of
    Children Enrolled in Study ROPP-2008-01 Previously Treated with rhIGF-1/rhIGFBP-3 for the Prevention of Retinopathy of Prematurity
    (ROP) or Who Received Standard Neonatal Care
    Esito a lungo termine sui bambini arruolati nello studio ROPP-2008-01 precedentemente
    trattati con rhIGF-1/rhIGFBP-3 per la prevenzione della retinopatia del prematuro (ROP) o che hanno ricevuto cure neonatali standard
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term Outcome of Children Enrolled in Study ROPP-2008-01 Previously Treated with rhIGF-1/rhIGFBP-3 for the Prevention of Retinopathy of Prematurity (ROP) or Who Received Standard Neonatal Care
    Esito a lungo termine sui bambini arruolati
    nello studio ROPP-2008-01 precedentemente trattati con rhIGF-1/rhIGFBP-3 per la prevenzione della retinopatia del prematuro
    (ROP) o che hanno ricevuto cure neonatali standard
    A.4.1Sponsor's protocol code numberSHP-607-201
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U0000-0000-0000
    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 SponsorPREMACURE AB, A MEMBER OF SHIRE GROUP OF COMPANIES
    B.1.3.4CountrySweden
    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 support Azienda Farmaceutica: Premacure AB, A Member of the Shire Group of Compa
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire
    B.5.2Functional name of contact pointMatthew Arkin
    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 number0017814829568
    B.5.5Fax number00
    B.5.6E-mailmarkin@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/3/06/399
    D.3 Description of the IMP
    D.3.1Product namemecasermina rinfabato
    D.3.2Product code rhIGF-I/rhIGFBP-3
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMECASERMIN RINFABATE
    D.3.9.1CAS number 478166-15-3
    D.3.9.2Current sponsor codeSHP-607
    D.3.9.4EV Substance CodeSUB25205
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(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 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 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 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 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
    Retinopathy of Prematurity (ROP)
    Retinopatia del prematuro (ROP)
    E.1.1.1Medical condition in easily understood language
    ROP is a rare disorder of the developing retinal blood vessels and retinal neurons of the preterm infant and is one of the leading causes of preventable blindness in children
    ROP è un raro disturbo dello sviluppo dei vasi sanguigni della retina e dei neuroni della retina del neonato pretermine ed è una delle cause principali della cecità prevenibile nei bambini
    E.1.1.2Therapeutic area Body processes [G] - Ocular Physiological Phenomena [G14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10038923
    E.1.2Term Retinopathy
    E.1.2System Organ Class 10015919 - Eye 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 evaluate the long-term efficacy outcomes
    following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D) as assessed
    by ROP associated visual outcomes
    -To evaluate the long-term safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in
    Study ROPP-2008-01 (Section D)
    E.2.1.IT Obiettivo principale:
    -Valutare gli esiti di efficacia a lungo termine a seguito dell'esposizione per un breve periodo a rhIGF-1/rhIGFBP-3 rispetto alle cure
    neonatali standard di cui allo studio ROPP-2008-01 (sezione D) secondo la valutazione degli esiti a livello visivo associati a ROP
    -Valutare gli esiti di sicurezza a lungo termine a seguito dell'esposizione per un breve periodo a rhIGF-1/rhIGFBP-3 rispetto alle
    cure neonatali standard di cui allo studio ROPP-2008-01 (sezione D)
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are
    to evaluate the effect following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01
    (Section D) on:
    •Growth parameters
    •Cognitive development
    •Physical development
    •Child behavior
    •Pulmonary morbidity
    •Survival
    •Health-related quality of life (HRQoL)
    •Health utility
    •Health care resource use (HCRU)
    Gli obiettivi secondari di questo studio prevedono la valutazione dell'effetto dell'esposizione per un
    breve periodo a rhIGF-1/rhIGFBP-3 rispetto alle cure neonatali standard di cui allo studio ROPP-2008-01 (sezione D) relativamente
    a:
    •Parametri di crescita
    •Sviluppo cognitivo
    •Sviluppo fisico
    •Comportamento del bambino
    •Morbilità polmonare
    •Sopravvivenza
    •Qualità di vita associata alla salute (HRQoL)
    •Utilità per la salute
    •Utilizzo delle risorse sanitarie (HCRU)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each subject must meet the following criteria to be enrolled in this
    study.
    1. Subject was randomized in Study ROPP-2008-01, Section D
    2. Subject's parent or legally authorized representative(s) must provide
    written informed consent prior to performing any study-related
    activities. Study-related activities are any procedures that would not
    have been performed during normal management of the subject.
    Per partecipare allo studio, ciascun soggetto deve soddisfare i criteri elencati di seguito:
    1. Il soggetto è stato randomizzato nello Studio ROPP-2008-01, Sezione D
    2. Prima che venga eseguita qualsiasi attività correlata allo studio, il genitore o il/i rappresentante/i legalmente autorizzato/i del
    soggetto devono fornire il consenso informato scritto. Per attività correlate allo studio si intendono tutte le procedure che non
    sarebbero altrimenti eseguite durante la normale gestione del soggetto.
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria will be excluded from the study.
    1. Any other condition or therapy that, in the Investigator's opinion, may pose a risk to the
    subject or interfere with the subject's ability to be compliant with this protocol or interfere with the interpretation of results
    2. The subject or subject's parent or legally authorized representative(s) is unable to comply
    with the protocol as determined by the Investigator
    Verranno esclusi dalla partecipazione allo studio i soggetti che soddisfino i criteri elencati di seguito:
    1. Qualsiasi altra condizione o terapia che, secondo il parere dello Sperimentatore, può mettere a rischio il soggetto o interferire con la capacità del soggetto di attenersi a questo protocollo o di interferire con l'interpretazione dei risultati
    2. Il soggetto o il genitore o il/i rappresentante/i legalmente autorizzato/i del soggetto non sono in grado di attenersi al protocollo, secondo il giudizio dello Sperimentatore
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoints of this study are: - Visual acuity as assessed by an age appropriate method - Ocular alignment and ocular motor examination in primary gaze and in as many of 9 positions of gaze as possible as assessed by corneal light reflex and by the cover test - Assessment of nystagmus by observation - Refraction as assessed by retinoscopy with cycloplegia - Stereoacuity as assessed with the Lang Stereotest
    Gli endpoint di efficacia primari di questo studio sono: •Acuità visiva valutata sulla base di un metodo appropriato per l’età •Esame dell'allineamento oculare e del movimento oculare in posizione primaria di sguardo e in altre 9 posizioni di sguardo, se possibile, tramite test del riflesso corneale alla luce e cover test •Valutazione del nistagmo sulla base dell'osservazione •Refrazione valutata tramite retinoscopia in cicloplegia •Stereoacuità valutata con lo stereotest di Lang
    E.5.1.1Timepoint(s) of evaluation of this end point
    will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA)
    varierà in base all'età al momento dell'arruolamento, ma i soggetti non saranno seguiti oltre i 5 anni e mezzo di età corretta (CA)
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints of this study are: •Growth parameters including body weight, body length (or height), and head circumference •Cognitive development as assessed by the following standardized, age-appropriate tools: Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV) •Physical development as assessed by standardized, age appropriate tools including physical exam, neurological examination for assessment of cerebral palsy, and hearing assessment •Child behavior as assessed by the following: Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Child Behavior Checklist (CBCL; 1 ½ to 5) Attention Deficit/Hyperactivity Disorder Rating Scale-fourth edition (ADHD RS-IV) for the assessment of symptoms of attention deficit/hyperactivity disorder (ADHD) Social Communication Questionnaire (SCQ) for screening of Autism Spectrum Disorder (ASD) •Pulmonary morbidity data (eg, hospitalizations, emergency room [ER] visits, pulmonary medications) •Survival as assessed by death during the study due to any cause The health economic outcome research endpoints of this study are: •Health-related quality of life (HRQoL) will be assessed by the Pediatric Quality of Life Inventory (PedsQL™) Scales appropriate for the child's age of development with the Total Scale Score and 5 domains within Physical Health (Physical Functioning and Physical Symptoms) and Psychosocial Health Scores (Emotional, Social, and Cognitive Functioning, respectively) •Health status (eg, health utility) will be measured by the Health Status Classification System-Preschool (HSCS-PS) •Resource use associated with inpatient visits, outpatient visits, medical utilization and pharmacy utilization will be assessed The safety endpoints of this study are: •Physical examination including tonsil examination •Adverse events (AEs), as follows: those considered related to rhIGF-1/rhIGFBP-3 (as administered in Study ROPP-2008-01, Section D) those considered related to procedures performed in this study (Study SHP-607-201) specified targeted medical events regardless of causality fatal SAEs regardless of causality •Cardiac size as assessed by echocardiogram •Kidney and spleen size and any other gross abnormalities as assessed by abdominal ultrasound Exploratory Endpoints: The exploratory endpoints of this study are related to the topography of the retinal layers and optic nerve as assessed by optical coherence tomography (OCT)
    Gli endpoint di efficacia secondari di questo studio sono: •Parametri di crescita compresi peso corporeo, lunghezza del corpo (o altezza) e circonferenza cranica •Sviluppo cognitivo valutato sulla base dei seguenti strumenti standardizzati e appropriati per l'età: Scala Bayley di sviluppo infantile (Bayley Scales of Infant and Toddler Development), terza edizione (BSID-III) Scala primaria di intelligenza per i bambini in età prescolare di Wechsler (Wechsler Preschool and Primary Scale of Intelligence), quarta edizione (WPPSI-IV) •Sviluppo fisico valutato sulla base di strumenti standardizzati e appropriati per l’età, tra cui esame obiettivo, esame neurologico per la valutazione di paralisi cerebrale e valutazione dell'udito •Comportamento del bambino valutato in base a quanto segue: Scala Vineland per il comportamento adattivo (Vineland Adaptive Behavior Scales), seconda edizione (VABS-II) Questionario sul comportamento del bambino (Child Behavior Checklist, CBCL 1 ½ - 5) Scala di classificazione del disturbo da deficit di attenzione e iperattività (Attention Deficit/Hyperactivity Disorder Rating Scale), quarta edizione (ADHD RS-IV), per la valutazione dei sintomi del disturbo da deficit di attenzione e iperattività (ADHD) Questionario di comunicazione sociale (Social Communication Questionnaire, SCQ) per lo screening dei disturbi dello spettro autistico (Autism Spectrum Disorders, ASD) •Dati sulla morbilità polmonare (ad es. ricoveri ospedalieri, visite al pronto soccorso, farmaci polmonari) •Sopravvivenza sulla base di un eventuale decesso durante lo studio per una causa qualsiasi Gli endpoint di ricerca degli esiti economici in relazione alla salute (health economic outcome research, HEOR) di questo studio sono: •La qualità della vita legata alla salute (HRQoL), valutata tramite le scale del questionario sulla qualità della vita in età pediatrica (Quality of Life Inventory, PedsQL™) appropriate per l'età di sviluppo del bambino con punteggio totale per ogni scala e 5 domini nell'ambito dei punteggi per salute fisica (funzioni fisiche e sintomi fisici) e salute psicosociale (rispettivamente, funzioni emotive, sociali e cognitive) •Lo stato di salute (ad es. utilità per la salute), misurato tramite sistema di classificazione dello stato di salute in età prescolare (Health Status Classification System-Preschool, HSCS-PS) •Valutazione dell'impiego delle risorse associato a visite ospedaliere, visite ambulatoriali, utilizzo di strutture sanitarie e utilizzo di farmaci Gli endpoint di sicurezza di questo studio sono: •Esame obiettivo, compreso esame delle tonsille •Eventi avversi (AE), quali: quelli ritenuti correlati a rhIGF-1/rhIGFBP-3 (secondo la somministrazione nello studio ROPP-2008-01, sezione D) quelli ritenuti correlati a procedure eseguite in questo studio (studio SHP-607-201) eventi medici mirati specifici, indipendentemente dalla causalità eventi avversi gravi (SAE) fatali indipendentemente dalla causalità
    E.5.2.1Timepoint(s) of evaluation of this end point
    will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA)
    varierà in base all'età al momento dell'arruolamento, ma i soggetti non saranno seguiti oltre i 5 anni e mezzo di età corretta (CA)
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    long-term developmental outcome study
    studio sugli esiti di sviluppo a lungo termine
    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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    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
    studio sugli esiti di sviluppo a lungo termine
    long-term developmental outcome study
    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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    Canada
    Italy
    Netherlands
    Poland
    Sweden
    United Kingdom
    United States
    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
    Duration for an individual subject's participation in the study will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA)
    La durata della partecipazione allo studio di un singolo soggetto varierà in base all'età al momento dell'arruolamento, ma i
    soggetti non saranno seguiti oltre i 5 anni e mezzo di età corretta (CA)
    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 months6
    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 months6
    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: 120
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 120
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 120
    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)
    NA
    NA
    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 Decision2015-04-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-24
    P. End of Trial
    P.End of Trial StatusOngoing
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