Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2017-003215-19
    Sponsor's Protocol Code Number:270-301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-01-28
    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 number2017-003215-19
    A.3Full title of the trial
    A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels < or = 1 IU/dL Receiving Prophylactic FVIII Infusions
    Studio di fase 3, in aperto, a braccio singolo volto a valutare l'efficacia e la sicurezza di BMN 270, un virus adeno-associato che funge da vettore del gene terapeutico che codifica il fattore VIII umano in pazienti affetti da emofilia A con livelli residui di fattore FVIII minore o uguale a 1 IU/dL in trattamento con infusioni profilattiche di FVIII
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 study to evaluate the efficacy and safety of BMN 270 gene transfer in patients with severe hemophilia A
    Studio clinico di Fase III per valutare l'efficacia e la sicurezza del trasferimento genico di BMN 270 in pazienti affetti da emofilia A di grado severo
    A.3.2Name or abbreviated title of the trial where available
    A Phase 3 study to evaluate the efficacy and safety of BMN 270 gene transfer in patients with severe
    Studio clinico di Fase III per valutare l'efficacia e la sicurezza del trasferimento genico di BMN 2
    A.4.1Sponsor's protocol code number270-301
    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.5.4Other Identifiers
    Name:NANumber:NA
    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 SponsorBIOMARIN PHARMACEUTICAL 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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceuticals Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.6E-mailmedinfo@bmrn.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/3/16/1622
    D.3 Description of the IMP
    D.3.1Product nameBMN 270
    D.3.2Product code BMN 270
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    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 INNvaloctocogene roxaparvovec
    D.3.9.1CAS number 1819334-78-5
    D.3.9.2Current sponsor codeBMN 270
    D.3.9.3Other descriptive nameAAV-HFVIII-SQ
    D.3.9.4EV Substance CodeSUB174200
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    Haemophilia A
    Emofilia A
    E.1.1.1Medical condition in easily understood language
    Bleeding Disorder
    Malattia emorragica
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10018937
    E.1.2Term Haemophilia A
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - Assess the efficacy of BMN 270 defined as FVIII activity, as measured by chromogenic substrate assay, during Weeks 49-52 following intravenous infusion of BMN 270
    Valutare l'efficacia di BMN 270, definita come l'attività di FVIII, misurato con un test cromogenico tra 49 e 52 settimane, dopo infusione endovenosa di BMN 270
    E.2.2Secondary objectives of the trial
    - Assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy from Week 5 to Week 52
    - Assess the impact of BMN 270 on the number of bleeding episodes requiring exogenous FVIII replacement therapy from Week 5 to Week 52
    Valutare l¿impatto di BMN 270 sull¿impiego della terapia sostututiva con FVIII esogeno dalla 5¿ alla 52¿ settimana dopo infusione di BMN 270
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males = 18 years of age with hemophilia A and residual FVIII levels = 1 IU/dL as evidenced by medical history, at the time of signing the informed consent.
    2. Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry. High-quality, well-documented historical data concerning bleeding episodes and FVIII usage over the previous 12 months must be available.
    3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs).
    4. Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study-related procedures.
    5. No previous documented history of a detectable FVIII inhibitor, and results from a Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) (or less than 1.0 BU for laboratories with a historical lower sensitivity cutoff for inhibitor detection of 1.0 BU) on 2 consecutive occasions at least one week apart
    within the past 12 months (at least one of which should be tested at the
    central laboratory).
    6. Sexually active participants must agree to use an acceptable method of effective contraception, either double-barrier contraception (ie, condom + diaphragm; or condom or diaphragm + spermicidal gel or foam) or their female partner either using hormonal contraceptives or having an intrauterine device. Participants must agree to contraception
    use for at least 12 weeks post-infusion; after 12 weeks, subjects may stop contraception use only if they have had 3 consecutive semen samples with no detectable viral vector DNA.
    7. Willing to abstain from alcohol consumption for at least the first 52 weeks following BMN 270 infusion.
    1. Soggetti di sesso maschile di età = 18 anni affetti da emofilia A con livelli residui del FVIII = 1 IU/dL nell’anamnesi al momento della firma del consenso informato.
    2. Soggetti trattati con terapia profilattica sostitutiva con FVIII per almeno 12 mesi prima dell’ingresso nello studio. Si deve disporre di dati storici ben documentati e di alta qualità relativi agli episodi emorragici e alla somministrazione del FVIII nei 12 mesi precedenti.
    3. Soggetti trattati con/esposti a concentrati o crioprecipitati di FVIII per almeno 150 giorni
    di esposizione (exposure day, ED).
    4. Disposti a e in grado di firmare il consenso informato scritto dopo che sarà stata loro
    illustrata la natura dello studio e prima dell’inizio di qualsiasi procedura legata allo studio.
    5. Titoli rilevabili di inibitore del FVIII, non precedentemente documentati in anamnesi e risultati di un test Bethesda o di un test Bethesda con modifica di Nijmegen inferiori a 0,6 Unità Bethesda (UB) ( oppure inferiori a 1,0 UB per i laboratori con una soglia di sensibilità inferiore storica per la rilevazione degli inibitori pari a 1,0 UB) in 2 occasioni consecutive ad almeno una settimana di distanza negli ultimi 12 mesi.
    partecipanti sessualmente attivi devono acconsentire a usare un metodo contraccettivo
    accettabile ed efficace, e cioè contraccezione a doppia barriera (ossia preservativo +
    diaframma oppure preservativo o diaframma + gel o schiuma spermicida) oppure utilizzo da
    parte della partner di contraccettivi ormonali o di una spirale. I partecipanti devono acconsentire a far uso di contraccettivi per almeno 12 settimane successive all’infusione; dopo 12 settimane i soggetti potranno sospendere l’utilizzo dei contraccettivi solo in seguito all’esecuzione di 3 esami consecutivi su campioni di sperma con assenza di DNA del vettore virale rilevabile.
    7. Soggetti disposti ad astenersi dal consumo di alcol per almeno le prime 52 settimane
    successive all’infusione di BMN 270.
    E.4Principal exclusion criteria
    1. Detectable pre-existing antibodies to the AAV5 capsid.
    2. Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
    3. Significant liver dysfunction with any of the following abnormal
    laboratory results:
    • ALT (alanine aminotransferase) > 1.25x ULN;
    • AST (aspartate aminotransferase) > 1.25x ULN;
    • GGT (gamma-glutamyltransferase) > 1.25x ULN;
    • Total bilirubin > 1.25x ULN;
    • Alkaline phosphatase > 1.25x ULN; or
    • INR (international normalized ratio) = 1.4.
    Subjects whose liver laboratory assessments fall outside of these ranges may undergo repeat testing of the entire liver test panel within the same Screening window and, if eligibility criteria are met on retest, may be enrolled after confirmation by the Medical Monitor 4. Prior liver biopsy showing significant fibrosis of 3 or 4 as rated on a scale of 0-4 on the Batts-Ludwig (Batts 1995) or METAVIR (Bedossa 1996) scoring systems, or an equivalent grade of fibrosis if an alternative scale is used.
    5. Evidence of any bleeding disorder not related to hemophilia A.
    6. Platelet count of < 100 x 10^9/L.
    7. Creatinine = 1.5 mg/dL.
    8. Liver cirrhosis of any etiology as assessed by liver ultrasound.
    9. Chronic or active hepatitis B as evidenced by positive serology testing (HBsAg, HBsAb, and HBcAb) and confirmatory HBV DNA testing. Refer to the Centers for Disease Control (CDC) table for the interpretation of serological test results in the Laboratory Manual.
    10. Active Hepatitis C as evidenced by detectable HCV RNA or currently on antiviral therapy.
    11. Active malignancy, except non-melanoma skin cancer.
    12. History of hepatic malignancy.
    13. History of arterial or venous thromboembolic events (eg, deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolus), with the exception of catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
    14. Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation.
    15. Treatment with any investigational product within 30 days or 5 halflives of the investigational product prior to the screening period. For subjects who have received a prior investigational product, all ongoing adverse events (AEs) experienced while receiving that investigational
    product must have resolved prior to screening for this study.
    16. Any condition that, in the opinion of the Investigator or Sponsor would prevent the patient from fully complying with the requirements of the study (including possible corticosteroid treatment outlined in the protocol) and/or would impact or interfere with evaluation and
    interpretation of subject safety or efficacy result.
    17. Prior treatment with any vector or gene transfer agent.
    18. Major surgery planned in the 52-week period following the infusion with BMN 270.
    Preesistenza di anticorpi rilevabili contro il capside dell’AAV5.
    2. Qualsiasi evidenza di infezione attiva o di disordini immunosoppressivi, inclusa l’infezione da
    HIV.
    3. Disfunzione epatica significativa con uno qualunque dei seguenti risultati anomali agli esami di
    laboratorio:
    ¿ ALT >1,25 volte l’ULN;
    ¿ AST >1,25 volte l’ULN;
    ¿ GGT >1,25 volte l’ULN;
    ¿ Bilirubina totale >1,25 volte l’ULN;
    ¿ Fosfatasi alcalina >1,25 volte l’ULN; oppure
    ¿ INR = 1,4.
    Nel caso di soggetti con risultati degli esami di laboratorio relativi alla funzionalità epatica non rientranti nei range sopra indicati è possibile ripetere l’intero pannello di esami epatici entro la
    stessa finestra di screening e, qualora i criteri di eleggibilità siano soddisfatti dai risultati dei nuovi
    esami, i soggetti in questione potranno essere arruolati in seguito a conferma da parte dell’addetto al monitoraggio medico.
    4. Biopsia epatica precedente che attesti la presenza di una fibrosi significativa classificata di grado 3 o 4 su una scala da 0 a 4 in base ai sistemi di punteggio di Batts-Ludwig (Batts 1995) o METAVIR
    (Bedossa 1996), o un grado di fibrosi equivalente qualora si utilizzi una scala alternativa.
    5. Evidenza di qualsiasi disturbo della coagulazione non correlato all’emofilia A.
    6. Conta piastrinica < 100 x 109/L.
    7. Creatinina = 1,5 mg/dL.
    8. Cirrosi epatica di qualsiasi eziologia valutata mediante ecografia epatica.
    9. Epatite B cronica o attiva evidenziata da risultati positivi dei test sierologici (antigene di superficie dell’epatite B [HBsAg], anticorpi anti-antigene di superficie dell’epatite B [HBsAb], anticorpi contro l’antigene del core dell’epatite B [HBcAb]) ed esame HBV DNA di conferma. Per l’interpretazione dei risultati dei test sierologici consultare la tabella per i centri per la prevenzione
    e il controllo delle malattie (Centers for Disease Control, CDC) inclusa nel manuale di laboratorio.
    10. Epatite C attiva evidenziata da HCV RNA rilevabile, o terapia antivirale in corso.
    11. Malignità attiva, a eccezione del cancro della cute non melanomatoso.
    12. Anamnesi di malignità epatica.
    13. Anamnesi di eventi tromboembolici arteriosi o venosi (ad es. trombosi venosa profonda, ictus non emorragico, embolia polmonare, infarto miocardico, embolia arteriosa), con l’eccezione della
    trombosi da catetere per la quale il paziente non sia attualmente sottoposto a trattamento
    antitrombotico.
    14. Trombofilia nota, ereditaria o acquisita, incluse condizioni associate ad aumentato rischio di eventi tromboembolici, come la fibrillazione atriale.
    15. Trattamento con farmaco sperimentale nei 30 giorni o nelle 5 emivite del farmaco sperimentale che precedono il periodo di screening. Nel caso di soggetti che abbiano ricevuto un farmaco sperimentale in precedenza, tutti gli eventi avversi (EA) in corso manifestatisi durante la
    somministrazione di tale farmaco sperimentale dovranno essere stati risolti prima dello screening
    per questo studio.
    16. Qualsiasi condizione che, a giudizio dello sperimentatore o del promotore, impedirebbe al paziente di soddisfare pienamente i requisiti dello studio (incluse le possibili terapie con corticosteroidi definite nel protocollo) e/o potrebbe influire o interferire con la valutazione e l’interpretazione dei risultati di sicurezza o efficacia relativi al paziente.
    17. Precedente trattamento con qualsiasi vettore o agente di trasferimento genico.
    18. Intervento chirurgico importante programmato per il periodo di 52 settimane successivo all’infusione di BMN 270.
    E.5 End points
    E.5.1Primary end point(s)
    Change of the hFVIII activity, as measured by chromogenic substrate assay, during Weeks 49-52 post-BMN 270 infusion from baseline. Each subject's hFVIII activity during Weeks 49-52 is defined as the median of the values obtained during this 4-week window. Values for hFVIII activity will be excluded if obtained within 72 hours since the last infusion of exogenous FVIII protein concentrates.
    Variazione dell’attività del hFVIII, misurata mediante saggio con substrato cromogeno,
    durante le Settimane 49-52 post-infusione di BMN 270 rispetto al basale. L’attività del hFVIII
    di ciascun soggetto durante le Settimane 49-52 è definita come la mediana dei valori ottenuti
    durante tale finestra temporale di 4 settimane. I valori relativi all’attività del hFVIII saranno
    esclusi qualora siano ottenuti entro 72 ore dall’ultima infusione di concentrati proteici di FVIII
    esogeno.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During Weeks 49-52 post-BMN 270 infusion
    Dalla 49° alla 52° settimana dopo infusione di BMN 270
    E.5.2Secondary end point(s)
    - Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy during Week 5 to Week 52 post-BMN 270 infusion from the baseline utilization of exogenous FVIII replacement therapy calculated using subjects' historical medical records during the year prior to enrollment.
    - Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment during Week 5 to Week 52 of the study post-BMN 270 infusion from the baseline ABR calculated using subjects' historical medical records during the year prior to enrollment.
    - Variazione dell utilizzo annuo (IU/kg) della terapia sostitutiva con FVIII esogeno nel periodo compreso tra la Settimana 5 e la Settimana 52 post-infusione di BMN 270 rispetto all utilizzo al basale della terapia sostitutiva con FVIII esogeno, calcolata facendo riferimento alle cartelle cliniche dei soggetti relative all¿anno precedente all¿arruolamento.
    - Variazione del numero annuo di episodi emorragici richiedenti la terapia sostitutiva con
    FVIII esogeno durante le Settimane 5-52 dello studio post-infusione di BMN 270 rispetto all ABR basale, calcolata facendo riferimento alle cartelle cliniche dei soggetti relative all anno precedente all arruolamento.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Utilization (IU/kg) of exogenous FVIII replacement therapy will be evaluated during Week 5 to Week 52 post-BMN 270 infusion from the baseline utilization of exogenous FVIII replacement therapy calculated
    using subjects' historical medical records during the year prior to enrollment
    - The number of bleeding episodes requiring exogenous FVIII replacement treatment will be evaluated during Week 5 to Week 52 post-BMN 270 infusion from the baseline ABR calculated using subjects' historical medical records during the year prior to enrollment.
    - l utilizzo della terapia sostitutiva con FVIII esogeno (IU/kg) sara valutato durante le settimane 5-52 dopo infusione di BMN 270 rispetto all utilizzo di terapia sostitutiva con FVIII esogeno a baseline calcolata sulla base delle cartelle cliniche dei soggetti durante l¿anno precedente l¿arruolamento.
    - Il numero di episodi di sanguinamenti che richiedono la terapia sostitutiva con FVIII esogeno sara valutato durante il periodo dalla 5 alla 52 settimana dopo infusione di BMN 270 rispetto al tasso annuo di sanguinamenti (ABR) a baseline calcolato sulla base delle cartelle cliniche dei soggetti durante l anno precedente l arruolamento.
    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 No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised 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 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
    subjects' historical annualized bleeding rate and annualized FVIII utilization
    subjects' historical annualized bleeding rate and annualized FVIII utilization
    E.8.2.4Number of treatment arms in the trial1
    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 EEA19
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Brazil
    Israel
    Japan
    Korea, Republic of
    South Africa
    Taiwan
    United States
    Belgium
    France
    Germany
    Italy
    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
    LVLS
    LSLS
    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 months1
    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 months1
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 134
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 17
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 134
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    Nessuno
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-07-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-09
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat May 04 04:31:57 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA