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    Summary
    EudraCT Number:2012-002773-64
    Sponsor's Protocol Code Number:RP103-07
    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:2013-11-21
    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 number2012-002773-64
    A.3Full title of the trial
    A Long-Term Open-Label, Safety and Superior Effectiveness Study of
    Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with
    Cystinosis
    Studio a lungo termine, in aperto, sulla sicurezza e l’efficacia superiore delle capsule a rilascio ritardato di cisteamina bitartrato (RP103) in
    pazienti affetti da cistinosi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Long-Term Open-Label, Safety and Superior Effectiveness Study of
    Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with
    Cystinosis
    Studio a lungo termine, in aperto, sulla sicurezza e l’efficacia superiore di cisteamina bitartrato (RP103) in
    pazienti affetti da cistinosi
    A.3.2Name or abbreviated title of the trial where available
    RP103-07
    A.4.1Sponsor's protocol code numberRP103-07
    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 SponsorRaptor 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 supportRaptor Pharmaceuticals Europe BV
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPSR Group BV
    B.5.2Functional name of contact pointPeter van Twuyver
    B.5.3 Address:
    B.5.3.1Street AddressPlanetenweg 5
    B.5.3.2Town/ cityHoofddorp
    B.5.3.3Post code2132 HN
    B.5.3.4CountryNetherlands
    B.5.4Telephone number003102355632203224
    B.5.5Fax number00310235563230
    B.5.6E-mailpeter.vantwuyver@psr-group.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 nameCysteamine bitartrate (INN: mercaptamine bitartrate )
    D.3.2Product code RP103
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNmercaptamine bitartrate
    D.3.9.1CAS number CAS 27761-19
    D.3.9.2Current sponsor codeRP103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.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 nameCysteamine bitartrate (INN: mercaptamine bitartrate )
    D.3.2Product code RP103
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNmercaptamine bitartrate
    D.3.9.1CAS number CAS 27761-19
    D.3.9.2Current sponsor codeRP103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name CYSTAGON
    D.2.1.1.2Name of the Marketing Authorisation holderORPHAN EUROPE Srl
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameCystagon
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCystagon
    D.3.9.1CAS number 27761-19-9
    D.3.9.3Other descriptive nameCysteamine bitartrate
    D.3.9.4EV Substance CodeSUB34918
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 4
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name CYSTAGON
    D.2.1.1.2Name of the Marketing Authorisation holderORPHAN EUROPE Srl
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameCystagon
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCystagon
    D.3.9.1CAS number 27761-19-9
    D.3.9.3Other descriptive nameCysteamine bitartrate
    D.3.9.4EV Substance CodeSUB34918
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystinosis
    Cistinosi
    E.1.1.1Medical condition in easily understood language
    Cystinosis
    Cistinosi
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level PT
    E.1.2Classification code 10011777
    E.1.2Term Cystinosis
    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
    Primary Objective:
    To demonstrate superiority of RP103 versus Cystagon® in controlling white blood cell cystine (WBC) levels over 24 hours in patients with cystinosis and to assess long-term safety and tolerability of RP103
    Obiettivo primario: Dimostrare la superiorità di RP103 rispetto a
    Cystagon nel controllo dei livelli di cistina leucocitaria nell’arco delle
    24 ore in pazienti affetti da cistinosi. Valutare la sicurezza e la
    tollerabilità di RP103 a lungo termine.
    E.2.2Secondary objectives of the trial
    Secondary Objectives:

    To compare WBC cystine measurements provided by a central
    laboratory versus sites’ local (regional, not associated with the
    clinical trial) laboratories.
    To assess long-term quality of life and sleep using instruments
    appropriate to the subjects’ age and region (US or Europe).
    To assess subject dosing compliance with two different
    cysteamine bitartrate formulations, Q6H immediate-release
    Cystagon® versus Q12H delayed-release RP103.
    To assess halitosis during Cystagon® and RP103 treatment in a
    subgroup of subjects
    Obiettivi secondari: Confrontare le misurazioni di cistina leucocitaria
    fornite da un laboratorio centrale con quelle di laboratori locali
    (regionali, non associati alla sperimentazione clinica). Valutare la
    qualità della vita e del sonno a lungo termine utilizzando strumenti
    appropriati per l’età e la regione del soggetto (Stati Uniti o Europa).
    Valutare la compliance di assunzione del soggetto con due formulazioni
    differenti di cisteamina bitartrato, Cystagon a rilascio immediato Q6H
    rispetto a RP103 a rilascio ritardato Q12H. Valutare l’alitosi durante il
    trattamento con Cystagon e RP103 in un sottogruppo di soggetti
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male or female with a documented diagnosis of cystinosis.
    2. On a stable dose of Cystagon® for at least 21 days prior to
    Screening.
    3. WBC cystine level > 1 nmol ½ cystine/mg of protein on
    average over at least 2 measurements collected during the 2
    years prior to Screening.
    4. No clinically significant change in liver function tests, i.e. 1.5
    times ULN for ALT and AST, and/or 1.5 times ULN for total
    bilirubin, within 6 months prior to Screening.
    5. No clinically significant change in renal function, i.e.
    estimated GFR within 6 months prior to Screening.
    6. Must have an estimated GFR > 20 mL/minute/1.73 m2 (using
    the equation from Schwartz 2009 J Am Soc Nephrol 20:629-
    637) 1.
    7. Female subjects who are sexually active and of childbearing
    potential, i.e. not surgically sterile (tubal ligation, bilateral
    oopherectomy, or hysterectomy) or at least 2 years naturally
    postmenopausal must agree to use an acceptable form of
    contraception from Screening through completion of the
    study. Acceptable forms of contraception for this study
    include hormonal contraceptives (oral, implant, transdermal
    patch, or injection) at a stable dose for at least 3 months prior
    to Screening, barrier (spermicidal condom or diaphragm with
    spermicide), IUD, or a partner who has been vasectomized
    for at least 6 months.
    8. Subject or their parent or guardian must provide written
    informed consent and assent (where applicable) prior to
    participation in the study.
    1. Pazienti di sesso maschile o femminile con una diagnosi documentata
    di cistinosi. 2. A dose stabile di Cystagon per almeno 21 giorni prima
    dello screening. 3. Livello di cistina leucocitaria > 1 nmol ½ cistina/mg
    di proteina in una media di almeno 2 misurazioni raccolte durante i 2
    anni precedenti allo screening. 4. Nessuna alterazione clinicamente
    significativa dei test della funzionalità epatica, vale a dire 1,5 volte
    l’ULN per ALT e AST e/o 1,5 volte l’ULN per la bilirubina totale, nei 6 mesi precedenti lo screening. 5. Nessuna alterazione clinicamente
    significativa della funzione renale, vale a dire una VFG stimata nei 6
    mesi precedenti lo screening. 6. È necessario presentare una VFG
    stimata > 20 ml/minuto/1,73 m2 (utilizzando l’equazione di Schwartz
    2009 J Am Soc Nephrol 20:629-637) 1. 7. I soggetti di sesso femminile
    sessualmente attivi e in età fertile, vale a dire non chirurgicamente
    sterili (legamento delle tube, ovariectomia bilaterale o isterectomia) o
    naturalmente in postmenopausa da almeno 2 anni, devono acconsentire
    a utilizzare un metodo contraccettivo accettabile dal momento dello
    screening al completamento dello studio. I metodi contraccettivi
    accettabili per questo studio includono contraccettivi ormonali (orale,
    impianto, cerotto transdermico o iniezione) a dose stabile per almeno 3
    mesi precedenti allo screening, contraccettivi di barriera (preservativo
    con crema spermicida o diaframma con spermicida), IUD o partner
    vasectomizzato da almeno 6 mesi. 8. Il soggetto o il genitore o il tutore
    devono fornire il consenso informato e l’assenso in forma scritta (se
    applicabile) prima della partecipazione allo studio.
    E.4Principal exclusion criteria
    Exclusion Criteria:
    1. Younger than 12 years of age.
    2. Current history of the following conditions or any other
    health issues that make it, in the opinion of the Investigator,
    unsafe for study participation:
    - Inflammatory bowel disease if currently active, or
    prior resection of small intestine;
    - Heart disease (e.g. myocardial infarction, heart
    failure, unstable arrhythmias, or poorly controlled
    hypertension) within 90 days prior to Screening;
    -Active bleeding disorder within 90 days prior to
    Screening;
    - History of malignant disease within 2 years prior to
    Screening.
    3. Hemoglobin level of < 9 g/dL at Screening or, in the opinion
    of the Investigator, a hemoglobin level that would make it
    unsafe for study participation.
    4. Known hypersensitivity to cysteamine and penicillamine.
    5. Female subjects who are nursing, planning a pregnancy, or
    are known or suspected to be pregnant.
    6. Subjects who, in the opinion of the Investigator, are not able
    or willing to comply with study requirements.
    1. Pazienti di età inferiore ai 12 anni. 2. Anamnesi corrente delle
    seguenti patologie o di altri problemi di salute che, secondo lo
    sperimentatore, rendono pericolosa la partecipazione allo studio:
    patologia infiammatoria dell’intestino se attualmente attiva o pregressa
    resezione dell’intestino tenue; cardiopatia (ad esempio infarto del
    miocardio, insufficienza cardiaca, aritmie instabili o ipertensione non
    controllata) nei 90 giorni che precedono lo screening; disordine
    emorragico attivo nei 90 giorni che precedono lo screening; anamnesi di
    patologia maligna nei 2 anni che precedono lo screening. 3. livello di
    emoglobina di < 9 g/dl allo screening o, secondo lo sperimentatore, un
    livello di emoglobina che renderebbe pericolosa la partecipazione allo
    studio. 4. Nota ipersensibilità alla cisteamina e alla penicillamina. 5.
    Soggetti di sesso femminile in allattamento, che stanno programmando
    una gravidanza o in stato di gravidanza accertata o sospetta. 6. Soggetti
    che, secondo lo sperimentatore, non possono o non intendono
    E.5 End points
    E.5.1Primary end point(s)
    Comparison of steady-state cysteamine-trough WBC cystine
    levels between Cystagon® and RP103 over 3 months for each
    treatment period.

    Confronto dei livelli di cistina leucocitaria e di quelli di cisteamina allo
    stato stazionario (steady state) tra Cystagon e RP103 nei 3 mesi per ogni
    periodo di trattamento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Months 1,2 and 3 vs Months 5,6 and 7 (Comparing the Cystagon and RP103 3-month treatment periods)
    Mesi 1,2 e 3 vs mesi 5,6 e 7 (confronto tra i periodi di 3 mesi di trattamento tra CYSTAGON e RP103)
    E.5.2Secondary end point(s)
    Safety Endpoints:
    - The safety profile of RP103 will be investigated by changes
    from the last study visit as noted in the following safety
    assessments: physical examination, vital signs, ECG and
    clinical laboratory testing. Incidence of adverse event report
    (including attribution of treatment-emergent non-serious and
    serious adverse events) will be recorded.

    Pharmacokinetic Endpoint:
    - Halitosis Substudy participants only: Cmax, Tmax and AUCs
    for plasma cysteamine and expired air DMS concentrations
    will be determined.

    Pharmacodynamic Endpoint:
    -The WBC cystine content will be measured from samples
    collected within 15 minutes pre-dose under Cystagon® or 30
    minutes post-dose under RP103 at each study visit.

    Exploratory Endpoints:
    -Central laboratory versus local laboratory WBC cystine
    values.
    -The longitudinal change in the use of concomitant gastric
    acid reduction therapies, quality of life questionnaires, and
    VAS swallowing difficulty and sleep rating scale scores.
    Safety Endpoints: The safety profile of RP103 will be investigated by
    changes from the last study visit as noted in the following safety
    assessments: physical examination, vital signs, ECG and clinical
    laboratory testing. Incidence of adverse event report (including
    attribution of treatment-emergent non-serious and serious adverse
    events) will be recorded. Pharmacokinetic Endpoint: Halitosis Substudy
    participants only: Cmax, Tmax and AUCs for plasma cysteamine and
    expired air DMS concentrations will be determined. Pharmacodynamic
    Endpoint: The WBC cystine content will be measured from samples
    collected within 15 minutes pre-dose under Cystagon or 30 minutes
    post-dose under RP103 at each study visit. Exploratory Endpoints:
    Central laboratory versus local laboratory WBC cystine values. The
    longitudinal change in the use of concomitant gastric acid reduction
    therapies, quality of life questionnaires, and VAS swallowing difficulty
    and sleep rating scale scores.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety and secondary endpoints will be assesed following every pertinent study visit. Unless they discontinue the study, subjects will complete Screening, Day 1, an 8 bi-Monthly (every 2 week) visits, i.e. through Month 7 plus Termination Visit. If study drug is not yet approved , that will be followed by Quarterly visits, up to 24 Months.
    La sicurezza e gli endpoint secondari saranno valutati dopo ogni visita di studio pertinente. A meno che non si interrompa lo studio, i soggetti completeranno lo Screening, ilgiorno 1, 8 visite bi-mensili (ogni due settimane), vale a dire fino al Mese 7 più la Visita di fine studio. Se il farmaco in studio non è ancora stato approvato, che sarà seguita da visite trimestrali, fino a 24 mesi.
    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) 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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 EEA12
    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
    United Kingdom
    United States
    France
    Germany
    Belgium
    Italy
    Netherlands
    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
    last visit of last subject undergoing the trial.
    Ultima Visita Ultimo Paziente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months24
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months24
    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: 50
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 50
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    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 Yes
    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
    Adolescents 12<17 years
    Adolescenti (12-17 anni)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 60
    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
    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 Decision2013-06-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-02-12
    P. End of Trial
    P.End of Trial StatusCompleted
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