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    Summary
    EudraCT Number:2011-004915-22
    Sponsor's Protocol Code Number:A3921145
    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:2012-09-21
    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 number2011-004915-22
    A.3Full title of the trial
    A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF CP-690,550 FOR
    TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)
    STUDIO DI FOLLOW-UP A LUNGO TERMINE, IN APERTO, SU CP-690,550 PER IL TRATTAMENTO DELL'ARTRITE IDIOPATICA GIOVANILE (AIG)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long-term, open label, study for treatment of JIA
    Studio a lungo termine in aperto per il trattamento dell'AIG
    A.4.1Sponsor's protocol code numberA3921145
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01513902
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/162/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPFIZER 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 supportPfizer inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact pointClinical Trials.gov Call Center
    B.5.3 Address:
    B.5.3.1Street Address235 EAST 42ND STREET
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number001 800 7181021
    B.5.5Fax number001 303 7391119
    B.5.6E-mailclinicaltrials.govcallcenter@pfizer.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 nameTofacitinib
    D.3.2Product code CP-690,550
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNTofacitinib
    D.3.9.1CAS number 540737-29-9
    D.3.9.2Current sponsor codeCP-690,550-10
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 nameTofacitinib
    D.3.2Product code CP-690,550
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNTofacitinib
    D.3.9.1CAS number 540737-29-9
    D.3.9.2Current sponsor codeCP-690,550-10
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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
    JUVENILE IDIOPATHIC ARTHRITIS (JIA)
    ARTRITE IDIOPATICA GIOVANILE (AIG)
    E.1.1.1Medical condition in easily understood language
    JUVENILE IDIOPATHIC ARTHRITIS (formerly known as Juvenile Rheumatoid Arthritis)
    Artrite idiopatica giovanile (precedentemente nota come Artrite reumatoide giovanile)
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.0
    E.1.2Level SOC
    E.1.2Classification code 10028395
    E.1.2Term Musculoskeletal and connective tissue disorders
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.0
    E.1.2Level LLT
    E.1.2Classification code 10059176
    E.1.2Term Juvenile idiopathic arthritis
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to determine the long term safety and tolerability of
    CP-690,550 for treatment of the signs and symptoms of JIA.
    • L’obiettivo dello studio è quello di determinare la tollerabilità e la sicurezza a lungo termine del CP-690,550 per il trattamento dei segni e sintomi dell’AIG.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to evaluate the persistence of efficacy of
    CP-690,550 for treatment of the signs and symptoms of JIA.
    0901
    • L’obiettivo secondario dello studio è quello di valutare la durata dell’efficacia del CP-690,550 per il trattamento dei segni e sintomi dell’AIG.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following inclusion criteria to be eligible for enrollment into the
    study:
    1. Pediatric patients with JIA aged from 2 to less than 18 years who met entry criteria
    for the qualifying/index study and in the opinion of the investigator have sufficient
    evidence of RA disease activity to warrant use of CP-690,550 as a DMARD. Patients
    turning 18 years of age during participation in the qualifying/index study or
    subsequently will be eligible for participation in this study.
    2. The patient has discontinued disallowed concomitant medications for the required
    time prior to the first dose of study drug, as defined in Appendix 1, and is taking only
    those concomitant medications in doses and frequency allowed by the protocol.
    3. Fertility:
    a. Sterile male, or non sterile male. If the patient is a non sterile male on
    background medications (including DMARDs) that require male
    contraceptive precautions according to the local drug label and is sexually
    active with a female partner of child-bearing potential, he and his partner must
    be practicing effective contraceptive measures.
    b. Females of childbearing potential must be using a reliable means of
    contraception (abstinence being a possible option) throughout the study and
    and for at least one ovulatory cycle after CP-690,550 treatment is discontinued.
    4. For patients receiving methotrexate (MTX) treatment, MTX may be administered
    either orally or parenterally at doses up to the lesser of 20 mg/wk or 15 mg/m2/week.
    5. For patients receiving leflunomide treatment, leflunomide may be administered
    according to the following dosing scheme:
     10 mg every other day for patients weighing less than 20 kg,
     10 mg every day for patients weighing between 20 and 40 kg,
     20 mg every day for patients weighing over 40 kg;
    Or as according to local standards.
    6. A negative QuantiFERON-TB Gold In-Tube test4 performed within the 3 months
    prior to screening. A negative PPD test can be substituted for the
    QuantiFERON-TB Gold In-Tube test only if the central laboratory is unable to
    perform the test or cannot determine the results to be positive or negative and the
    Pfizer medical monitor approves it, on a case-by-case basis.
    7. Evidence of a personally signed and dated informed consent document with assent as
    appropriate indicating that the patient (or a legally acceptable representative) has been
    informed of all pertinent aspects of the study.
    8. Patients who are willing and able to comply with all scheduled visits, treatment plan,
    laboratory tests, and other study procedures.
    I soggetti devono soddisfare tutti i seguenti criteri di inclusione per essere considerati idonei all’arruolamento nello studio:
    1. Pazienti pediatrici con AIG di almeno 2 anni e meno di 18 anni di età che soddisfano i criteri per lo studio indice/di qualificazione e che, secondo il giudizio dello sperimentatore, presentano evidenza sufficiente di attività patologica dell’AR da garantire l’uso del CP-690,550 come DMARD. I pazienti che compiono 18 anni durante la partecipazione allo studio indice/di qualificazione o successivamente saranno ritenuti idonei per la partecipazione al presente studio.
    2. Il paziente deve aver interrotto l’uso di farmaci concomitanti non consentiti per il tempo richiesto prima della prima somministrazione del farmaco di studio, come descritto nell’Appendice 1, ed assumere i farmaci concomitanti soltanto secondo i dosaggi e la frequenza consentiti dal protocollo.
    3. Fertilità:
    a. Pazienti di sesso maschile, sterili o non sterili. Se il paziente è un soggetto maschile non sterile in terapia di background con farmaci (DMARD compresi) che necessitano l’assunzione di precauzioni contraccettive, ed è sessualmente attivo con una partner in età fertile, il paziente e la sua partner devono intraprendere efficaci misure contraccettive conformemente a quanto indicato sull’etichettatura del farmaco locale.
    b. Le pazienti di sesso femminile in età fertile devono utilizzare un metodo contraccettivo affidabile (oppure praticare l’astinenza come alternativa possibile) nel corso dello studio e per almeno un ciclo di ovulazione dopo l’interruzione del trattamento con il CP-690,550.
    4. Per pazienti in terapia con il metotrexato (MTX), il MTX potrà essere somministrato per via orale o parenterale in dosaggi fino a meno di 20 mg/settimana oppure 15 mg/m2/settimana.
    5. Per pazienti in terapia con leflunomide, la leflunomide potrà essere somministrata secondo il seguente schema di somministrazione:
    • 10 mg a giorni alterni per pazienti con un peso inferiore a 20 kg,
    • 10 mg ogni giorno per pazienti con un peso compreso tra 20 e 40 kg,
    • 20 mg ogni giorno per pazienti con un peso superiore a 40 kg,
    Oppure secondo le regolamentazioni locali.
    6. Un test negativo di QuantiFERON-TB Gold In-Tube effettuato entro i 3 mesi precedenti lo screening. Un test della tubercolina negativo può sostituire il test QuantiFERON-TB Gold In-Tube4 solamente se il laboratorio centrale non è in grado di effettuare il test o se non può determinare la positività o negatività dei risultati e, caso per caso, con l’approvazione del monitor Pfizer.
    7. Documentazione di consenso informato firmato e datato personalmente con assenso in cui è opportunamente indicato che il/la paziente (o un rappresentante legale autorizzato) è stato/a informato/a di tutti gli aspetti relativi allo studio.
    8. Pazienti disposti e in grado di rispettare le visite schedulate, il programma di trattamento, gli esami di laboratorio e le altre procedure di studio.
    E.4Principal exclusion criteria
    1. Systemic JIA with active systemic features, persistent oligoarthritis, and
    undifferentiated JIA.
    2. Blood dyscrasias, including:
    a. Hgb <10 g/dL or Hct <33%.
    b. WBC <3.0 x 109/L.
    c. Neutrophil count <1.2 x 109/L.
    d. Platelet count <100 x 109/L.
    3. Estimated GFR <40 mL/min/1.73 m2 calculated using the Schwartz formula
    (Appendix 4) at the Screening Visit.
    4. Current or recent history of uncontrolled clinically significant renal, hepatic,
    hematological, gastrointestinal, endocrine, pulmonary, cardiac, or neurological
    disease.
    5. AST or ALT >/=1.5 times the upper limit of normal or any other clinically significant
    laboratory abnormality.
    For all patients:
    6. History of any other rheumatic autoimmune disease.
    7. History or current symptoms suggestive of any lymphoproliferative disorder, such as
    Epstein Barr Virus (EBV) related lymphoproliferative disorder, history of lymphoma,
    leukemia, or signs and symptoms suggestive of current lymphatic disease.
    8. Infections:
    a. Latent or active TB or any history of previous TB.
    b. Chronic infections.
    c. Any infection requiring hospitalization, parenteral antimicrobial therapy or
    judged to be opportunistic by the investigator within the 6 months prior to the
    first dose of study drug.
    d. Any treated infections within 2 weeks.
    e. A patient known to be infected with human immunodeficiency virus (HIV),
    hepatitis B or hepatitis C virus.
    f. History of infected joint prosthesis with prosthesis still in situ.
    9. History of recurrent (more than one episode) herpes zoster or disseminated (a single
    episode) herpes zoster or disseminated (a single episode) herpes simplex.
    10. Patients taking potent and moderate cytochrome P450 3A4 (CYP3A4) inhibitors
    (Appendix 5).
    11. Patients taking potent and moderate CYP3A4 inducers (Appendix 5).
    12. Participation in studies of investigational compounds (excluding qualifying/index
    study with CP-690,550) within 4 weeks or 5 half-lives (whichever is longer) prior to
    the first dose of study drug. Patients cannot participate in studies of other
    investigational compounds at any time during their participation in this study.
    Exposure to investigational biologics should be discussed with the Pfizer Medical
    Monitor.
    13. Any prior treatment with non B cell-specific lymphocyte depleting agents/therapies
    [eg, almetuzumab (CAMPATH), alkylating agents (eg, cyclophosphamide or
    chlorambucil), total lymphoid irradiation, etc]. Patients who have received rituximab
    or other selective B lymphocyte depleting agents (including experimental agents) are
    eligible if they have not received such therapy for at least 1 year prior to study
    baseline and have normal CD 19/20+ counts by FACS analysis.
    14. Pregnant or nursing females; females of childbearing potential who are unwilling or
    unable to use an acceptable method of contraception as outlined in this protocol
    during the study and for least one ovulatory cycle after the last dose of study
    medication.
    15. Intramuscular or intravenous corticosteroids in the 4 weeks preceding first dose of
    study medication.
    I pazienti che presentano qualsiasi dei seguenti aspetti non saranno inclusi nello studio:
    Per pazienti che non sono trasferiti al presente studio entro 14 giorni dopo la loro ultima visita dello studio indice/di qualificazione (1-5):
    1. AIG sistemica con caratteristiche sistemiche attive, oligoartrite persistente e AIG indifferenziata.
    2. Discrasia ematica, compresi:
    a. Hgb &lt;10 g/dl o Hct &lt;33%.
    b. WBC &lt;3,0 x 109/L.
    c. Conta dei neutrofili &lt;1,2 x 109/L.
    d. Conta delle piastrine &lt;100 x 109/L.
    3. VFG stimata &lt;40 ml/min/1,73 m2 calcolata utilizzando la formula di Schwartz (Appendice 4) nella Visita di Screening.
    4. Storia attuale o recente di patologia renale, epatica, ematologica, gastrointestinale, endocrina, polmonare, cardiaca o neurologica non controllata e clinicamente significativa.
    5. AST o ALT 1,5 volte il limite superiore della norma o altro valore di laboratorio clinicamente significativo che non rientra negli intervalli di normalità.
    Per tutti i pazienti:
    6. Storia di eventuali altre patologie reumatiche autoimmuni.
    7. Storia o sintomi in corso indicativi di possibili malattie linfoproliferative, come quelle correlate al virus di Epstein-Barr (EBV), oppure storia di linfoma, leucemia, o segni e sintomi indicativi di attuale patologia linfatica.
    8. Infezioni:
    a. TBC latente o attiva, oppure eventuale storia di precedente TBC.
    b. Infezioni croniche.
    c. Eventuali infezioni tali da richiedere ricovero ospedaliero, terapia antimicrobica parenterale o opportunistiche secondo il giudizio dallo sperimentatore entro i 6 mesi precedenti la prima somministrazione del farmaco di studio.
    d. Eventuali infezioni trattate entro 2 settimane.
    e. Paziente noto/a per aver contratto il virus dell’immunodeficienza umana (HIV), oppure il virus dell’epatite B o C.
    f. Storia di protesi articolare infetta con protesi ancora in situ.
    9. Storia di herpes zoster ricorrente (più di un episodio) o herpes zoster disseminato (un singolo episodio) o herpes simplex disseminato (un singolo episodio).
    10. Pazienti in terapia con potenti e moderati inibitori del citocromo P450 3A4 (CYP3A4) (Appendice 5).
    11. Pazienti in terapia con potenti e moderati induttori del CYP3A4 (Appendice 5).
    12. Partecipazione in studi su molecole sperimentali (escluso lo studio indice/di qualificazione sul CP-690,550) entro le 4 settimane o 5 emivite (qualunque sia il più lungo) prima della prima somministrazione del farmaco di studio. I pazienti non possono partecipare a studi con altre molecole in fase di sperimentazione durante la loro partecipazione a questo studio. L’esposizione a farmaci biologici in sperimentazione deve essere discussa con il monitor Pfizer.
    13. Qualsiasi trattamento precedente con farmaci/terapie per la deplezione aspecifica dei linfociti B [per es. alemtuzumab (CAMPATH), agenti alchilanti (per es., ciclofosfamide o clorambucile), irradiazione linfonodale totale, ecc.] I pazienti che hanno assunto rituximab o un altro farmaco per la deplezione selettiva dei linfociti B (compresi i farmaci sperimentali) sono idonei se non sono stati sottoposti a tale terapia per almeno 1 anno prima della visita basale dello studio e presentano un’espressione normale CD 19/20+ all’analisi citofluorimetrica (FACS).
    14. Pazienti di sesso femminile in gravidanza o in allattamento; pazienti di sesso femminile in età fertile non disponibili o non in grado di utilizzare un metodo contraccettivo accettabile come richiesto dal protocollo durante lo studio e per almeno un ciclo di ovulazione dopo l’ultima somministrazione del farmaco di studio.
    15. Corticosteroidi per via intramuscolare o endovenosa nelle 4 settimane precedenti la prima somministrazione del farmaco di studio.
    E.5 End points
    E.5.1Primary end point(s)
    Standard laboratory safety data and adverse event (AE) reports. Body weight, height
    and Tanner Stages will collected to assess growth and physical development.
    • Dati standard di laboratorio sulla sicurezza e segnalazioni degli eventi avversi. Per valutare la crescita e lo sviluppo fisico si registreranno peso corporeo, altezza e stadi di Tanner.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary endpoints consist of 4 items:
    1) Standard Lab safety data
    2) AE Assessment
    3) body weight and height
    4) Tanner stage
    Gli endpoint primari sono costituiti da 4 elementi:
    1) Dati standard di Lab di sicurezza
    2) Valutazione EA
    3) il peso corporeo e altezza
    4) stadio di Tanner
    E.5.2Secondary end point(s)
     Physician global evaluation of disease activity.
     Number of joints with active arthritis.
     Number of joints with limitation of motion.
     Index of inflammation (C-reactive protein [CRP]).
     Juvenile Arthritis Multidimensional Assessment Report (JAMAR).
     Parent’s or child evaluation of overall wellbeing (JAMAR Visual Analog Scale
    [VAS] component).
     Functional ability (JAMAR).
     Health related quality of life (JAMAR).
     American College of Rheumatology (ACR) pediatric response and flare criteria.
     Inactive disease status parameters.
    I seguenti parametri di efficacia saranno valutati:
    • Valutazione globale del medico sull’attività della patologia.
    • Numero di articolazioni con artrite attiva.
    • Numero di articolazioni con limitati di movimento.
    • Indice di infiammazione (proteina C reattiva [CRP]).
    • Questionario multidimensionale per la valutazione dell’artrite giovanile (JAMAR).
    • Valutazione del benessere generale da parte del genitore o del bambino stesso (questionario JAMAR su scala analogico-visica [VAS]).
    • Capacità funzionale (JAMAR).
    • Qualità di vita correlata alla salute (JAMAR).
    • Risposta pediatrica e criteri di riacutizzazione dell’American College of Rheumatology (ACR).
    • Parametri dello stato inattivo della patologia.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The secondary endpoints (section 2.2.2)will be assessed at Baseline, and Months 1,3,6,9 and 12. During study years 2-10 the assessments will be completed at 6 month intervals.
    Gli endpoint secondari (sezione 2.2.2) saranno valutati al basale e Mesi 1,3,6,9 e 12. Durante gli anni di studio 2-10 le valutazioni saranno completate ad intervalli di 6 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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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
    Tolerability
    Tollerabilità
    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 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 Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial0
    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 concerned2
    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
    Argentina
    Mexico
    Russian Federation
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 years7
    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: 24
    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: 16
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    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 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
    Minors incapable of giving informed consent
    I minori incapaci di dare il consenso informato
    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 18
    F.4.2.2In the whole clinical trial 24
    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)
    Subjects may participate in extention up to 10 years. It is expected that drug will be approved when subjects' participation ends.
    I soggetti possono partecipare allo studio di estensione fino a 10 anni. Si prevede che il farmaco sarà approvato quando la partecipazione dei soggetti finisce.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation PRINTO
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-07-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-07-24
    P. End of Trial
    P.End of Trial StatusOngoing
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