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    Summary
    EudraCT Number:2017-003539-12
    Sponsor's Protocol Code Number:BIVV009-04
    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:2021-01-15
    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 number2017-003539-12
    A.3Full title of the trial
    A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO ASSESS THE EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WITHOUT A RECENT HISTORY OF BLOOD TRANSFUSION
    STUDIO DI FASE 3, RANDOMIZZATO, IN DOPPIO CIECO, CONTROLLATO CON PLACEBO PER VALUTARE L'EFFICACIA E LA SICUREZZA DI BIVV009 IN PAZIENTI AFFETTI DA MALATTIA DA AGGLUTININE A FREDDO PRIMARIA CHE NON HANNO UN'ANAMNESI RECENTE DI TRASFUSIONI DI SANGUE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine the safety and effectiveness of study drug BIVV009 in patients with Primary Agglutinin Disease without recent history of blood
    transfusions
    Uno studio per determinare la sicurezza e l'efficacia del farmaco in studio BIVV009 in pazienti affetti da Malattia da Agglutinine Primaria che non hanno una storia recente di trasfusioni di sangue
    A.3.2Name or abbreviated title of the trial where available
    Cadenza
    Cadenza
    A.4.1Sponsor's protocol code numberBIVV009-04
    A.5.4Other Identifiers
    Name:BIVV009-04Number:BIVV009-04
    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 SponsorBIOVERATIV 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 supportBioverativ USA Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharm-Olam LLC
    B.5.2Functional name of contact pointClinical Trial Dept
    B.5.3 Address:
    B.5.3.1Street Address450 N. Sam Houston Parkway, E. Suite 250
    B.5.3.2Town/ cityHouston
    B.5.3.3Post codeTX77060
    B.5.3.4CountryUnited States
    B.5.4Telephone number0017135597900
    B.5.5Fax number0017818634989
    B.5.6E-mailtracy.klenk@pharm-olam.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 numberEMA/OD/177/15
    D.3 Description of the IMP
    D.3.1Product nameBIVV009
    D.3.2Product code BIVV009
    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 INNBIVV009
    D.3.9.2Current sponsor codeBIVV009
    D.3.9.3Other descriptive nameTNT009
    D.3.9.4EV Substance CodeSUB177476
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number18
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBIVV009
    D.3.9.2Current sponsor codeBIVV009
    D.3.9.3Other descriptive nameTNT009
    D.3.9.4EV Substance CodeSUB177476
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Cold Agglutinin Disease
    Malattia da Agglutinine a Freddo
    E.1.1.1Medical condition in easily understood language
    Autoimmune Disease against red blood cells (Haemolytic Anaemia)
    Malattia Autoimmune contro gli eritrociti (Anemia Emolitica)
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10073785
    E.1.2Term Autoimmune haemolytic anaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of Part A is to determine whether BIVV009 administration results in a = 1.5 g/dL increase in hemoglobin (Hgb) level
    and avoidance of transfusion in patients with primary CAgD without a
    recent history of blood transfusion.
    The primary objective of Part B is to evaluate the long-term safety and
    tolerability of BIVV009 in patients with primary CAgD.
    L'obiettivo primario della parte A ¿ quello di determinare se la somministrazione di BIVV009 produce un aumento dei livelli di emoglobina (Hgb) di = 1,5 g/dl ed evita le trasfusioni in pazienti affetti da CAgD primaria che non hanno un¿anamnesi recente di trasfusioni di sangue

    L'obiettivo primario della Parte B ¿ valutare la sicurezza a lungo termine e la tollerabilit¿ di BIVV009 in pazienti affetti da CAgD primaria.
    E.2.2Secondary objectives of the trial
    The secondary efficacy objectives of Part A are:
    To assess the effect of BIVV009 on clinical events and laboratory
    parameters related to hemolysis and anemia in patients with primary
    CAgD
    To assess the effect of BIVV009 on specific complications of CAgD
    To assess the effect of BIVV009 on quality of life (QOL) in patients with
    primary CAgD.
    The secondary objective of Part B is to investigate the durability of
    response during long-term treatment with BIVV009 in patients with
    primary CAgD.
    Gli obiettivi secondari sull'efficacia della Parte A sono:
    ¿Valutare l'effetto di BIVV009 su eventi clinici e parametri di laboratorio correlati a emolisi e anemia in pazienti affetti da CAgD primaria
    ¿Valutare l'effetto di BIVV009 su specifiche complicanze di CAgD
    ¿Valutare l'effetto di BIVV009 sulla qualit¿ della vita (QOL) in pazienti affetti da CAgD primaria

    L'obiettivo secondario della parte B ¿ studiare la durata della risposta durante il trattamento a lungo termine con BIVV009 in pazienti affetti da CAgD primaria.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All patients must meet all the following inclusion criteria to be enrolled:
    1. Adult male and female patients = 18 years of age at Screening
    2. Body weight of = 39 kg at Screening
    3. Confirmed diagnosis of primary CAgD based on the following criteria:
    a. Chronic hemolysis
    b. Polyspecific direct antiglobulin test (DAT) positive
    c. Monospecific DAT strongly positive for C3d
    d. Cold agglutinin titer = 64 at 4¿C
    e. IgG DAT = 1+, and
    f. No overt malignant disease
    4. Hemoglobin level = 10.0 g/dL
    5. Bilirubin level above the normal reference range, including patients with Gilbert’s Syndrome
    6. Ferritin levels above the lower limit of normal. Concurrent treatment with iron supplementation is permitted if the patient has been on a stable dose during the previous 4 weeks.
    7. Presence of one or more of the following CAgD-related signs or symptoms within 3 months of Screening:
    a. Symptomatic anemia defined as:
    i. Fatigue
    ii. Weakness
    iii. Shortness of breath
    iv. Palpitations, fast heart beat
    v. Light headedness, and/or
    vi. Chest pain
    b. Acrocyanosis
    c. Raynaud’s syndrome
    d. Hemoglobinuria
    e. Disabling circulatory symptoms, and/or
    f. Major adverse vascular event (including thrombosis)
    8. Bone marrow biopsy within 6 months of Screening with no overt evidence of lymphoproliferative disease or other hematological malignancy. An additional bone marrow biopsy will be required if the prior bone marrow is deemed unsuitable for analysis by the Sponsor.
    9. Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitis, including serogroup B meningococcus where available, Haemophilus influenzae, and Streptococcus pneumoniae) within 5 years of enrollment or as specified in Section 6.1.1.1
    10. Patients must be willing to receive transfusions if they meet the eligibility criteria during the study treatment period. Patients who do not have a recent history of transfusion due to patient refusal or patient decision should not be enrolled if they do not agree to receive blood transfusions as needed.
    11. Adequate IV access
    12. If female, must be post-menopausal, surgically sterile, or be established on (= 3 months prior to Screening) and agree to continue to use the same highly effective methods of birth control throughout the study and for 9 weeks following administration of the last dose of study drug
    13. Males must be surgically sterile for at least 90 days or when sexually active with female partners of child-bearing potential will agree to use highly effective contraception from Day 0 until 9 weeks following administration of the last dose of study drug.
    14. Able to comprehend and give informed consent
    15. Able to comply with the requirements of the study and to complete the full sequence of protocol-related procedures
    Per essere arruolati, tutti i pazienti devono soddisfare tutti i seguenti criteri di inclusione:
    1. Pazienti adulti di sesso maschile e femminile di età = 18 anni allo Screening
    2. Peso corporeo = 39 kg allo Screening
    3. Diagnosi confermata di CAgD primaria sulla base dei seguenti criteri:
    a. Emolisi cronica,
    b. Test dell’antiglobulina diretto (DAT) polispecifico positivo,
    c. DAT monospecifico fortemente positivo per C3d,
    d. Titolo delle agglutinine a freddo =64 a 4¿C,
    e. DAT IgG = 1+, e
    f. Nessuna evidenza di patologia maligna
    4. Livello di emoglobina = 10,0 g/dL
    5. Livello di bilirubina al di sopra del normale intervallo di riferimento, inclusi i pazienti con la sindrome di Gilbert
    6. Livelli di ferritina sopra il piu’ basso limite di normalita’ .Il trattamento concomitante con supplemento di ferro e’ permesso se il paziente e’ha assunto una dose stabile durante le 4 settimane precedenti.
    7. Presenza di uno o più dei seguenti segni o sintomi correlati a CAgD entro 3 mesi dallo Screening:
    a. Anemia sintomatica definita come:
    i. Affaticamento
    ii. Debolezza
    iii. Respiro affannoso
    iv. palpitazioni, battito cardiaco accelerato
    v. stordimento e/o
    vi. dolore al petto
    b. acrocianosi
    c. Sindrome di Raynaud
    d. Emoglobinuria
    e. Sintomi circolatori invalidanti, e/o
    f. Eventi avversi vascolari maggiori (compresa trombosi)
    8. Biopsia del midollo osseo entro 6 mesi dallo Screening e nessuna palese evidenza di malattia linfoproliferativa o altre neoplasie ematologiche. Se il precedente campione di midollo osseo viene ritenuto non idoneo per l'analisi da parte dello Sponsor, sarà necessario eseguire una biopsia del midollo osseo aggiuntiva.
    9. Vaccinazioni documentate contro patogeni batterici incapsulati (Neisseria meningitis, incluso il meningococcus del sierogruppo B dove applicabile, Haemophilus influenzae e Streptococcus pneumoniae) entro 5 anni dall’arruolamento o come specificato nella Sezione 6.1.1.1
    10. I Pazienti devono esser disposti a ricevere trasfusioni se soddisfano i criteri di eleggibilita’ durante il periodo di trattamento dello studio. I pazienti che non presentano una storia recente di trasfusione dovuta al rifiuto del paziente o a decisione del paziente non dovrebbero esser arruolati nello studio se non sono d’accordo nel ricevere una trasfusione di sangue secondo necessita’.
    11. Accesso EV adeguato
    12. Se di sesso femminile, deve essere in post-menopausa, chirurgicamente sterile, o usare stabilmente (= 3 mesi prima dello Screening) ed accettare di continuare a utilizzare lo stesso metodo contraccettivo altamente efficace durante tutto lo studio e per 9 settimane dopo la somministrazione dell'ultima dose di farmaco in studio
    13. Gli uomini devono essere chirurgicamente sterili da almeno 90 giorni o, se sessualmente attivi con partner di sesso femminile in età fertile, dovranno accettare di usare metodi di contraccezione altamente efficaci dal Giorno 0 fino a 9 settimane dopo la somministrazione dell'ultima dose di farmaco in studio.
    14. In grado di comprendere e dare il proprio consenso informato
    15. In grado di soddisfare le esigenze dello studio e di completare l’intera sequenza delle procedure correlate al protocollo
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria will be excluded from the study:
    1. Cold agglutinin syndrome secondary to infection, rheumatologic disease, or active hematologic malignancy
    2. History of blood transfusion within 6 months of screening, or history of more than one blood transfusion within 12 months of screening
    3. Clinically relevant infection of any kind within the month preceding enrollment (eg, active hepatitis C, pneumonia)
    4. Clinical diagnosis of systemic lupus erythematosus (SLE); or other autoimmune disorders with anti-nuclear antibodies at Screening. Anti-nuclear antibodies of long standing duration without associated clinical symptoms will be adjudicated on a case-by-case basis during the Confirmatory Review of Patient Eligibility (Section 6.1.1.3).
    5. Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at Screening
    6. Positive human immunodeficiency virus (HIV) antibody at Screening
    7. Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (eg, with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment
    8. Concurrent treatment with corticosteroids other than a stable daily dose equivalent to
    = 10 mg/day prednisone for previous 3 months
    9. Erythropoietin deficiency. Concurrent treatment with erythropoietin is permitted if the patient has been on a stable dose for the previous 3 months.
    10. Concurrent usage of iron supplementation unless the patient has been on a stable dose for at least 4 weeks.
    11. Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the patient or compromise the quality of the data derived from his/her participation in this study (as determined by the Investigator [or designee]) at Screening
    12. Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half lives, whichever is greater, prior to treatment start
    13. Females who are pregnant, lactating, or, if having reproductive potential, are considered potentially unreliable with respect to contraceptive practice
    14. History of hypersensitivity to BIVV009 or any of its components.
    I pazienti che rispondono a qualsiasi dei seguenti criteri saranno esclusi dallo studio:
    1. Sindrome da agglutinine a freddo secondaria a infezione, malattia reumatologica o neoplasia ematologica attiva
    2. Anamnesi di trasfusione di sangue entro 6 mesi dallo screening o anamnesi di più di una trasfusione di sangue entro 12 mesi dallo screening
    3. Infezione clinicamente rilevante di qualsiasi genere entro il mese precedente l’arruolamento (ad es, epatite C attiva, polmonite)
    4. Diagnosi clinica di lupus eritematoso sistemico (SLE); o altre malattie autoimmuni con anticorpi anti-nucleo allo Screening. Gli anticorpi antinucleari di lunga durata senza sintomi clinici associati saranno giudicati caso per caso durante il riesame di conferma dell'ammissibilità dei pazienti (paragrafo 6.1.1.3).
    5. Quadro completo di analisi per l’epatite positivo (compresi antigene superficiale dell’epatite B e/o anticorpo del virus dell’epatite C) prima di o allo Screening
    6. Esito positivo per gli anticorpi del virus dell'immunodeficienza umana (HIV) allo Screening
    7. Trattamento con rituximab in monoterapia entro 3 mesi o in combinazione (ad es., con bendamustina, fludarabina, ibrutinib, o farmaci citotossici) entro 6 mesi prima dell'arruolamento
    8. Trattamento concomitante con corticosteroidi diverso da una dose giornaliera stabile equivalente a = 10 mg/die di prednisone per i 3 mesi precedenti
    9. Deficit di eritropoietina Il trattamento concomitante con eritropoietina è consentito se il/la paziente ha seguito una terapia con dosaggio stabile per i 3 mesi precedenti.
    10. Utilizzo concomitante di integratori di ferro a meno che il/la paziente non abbia seguito una terapia con dosaggio stabile da almeno 4 settimane.
    11. Anamnesi medica clinicamente significativa o malattia cronica in corso che metterebbe a rischio la sicurezza del/della paziente o comprometterebbe la qualità dei dati derivati dalla sua partecipazione a questo studio (come stabilito dallo sperimentatore [o da un suo incaricato]) allo Screening
    12. Trattamento concomitante con altri farmaci sperimentali o partecipazione ad un altro studio clinico con qualsiasi farmaco sperimentale entro 30 giorni o 5 emivite, a seconda di quale dei due sia il periodo più lungo, prima di iniziare il trattamento
    13. Donne in gravidanza o allattamento o, se in età fertile, considerate potenzialmente inaffidabili rispetto alla pratica contraccettiva
    14. Storia di ipersensibilita’a BIVV009 o a qualsiasi dei suoi componenti
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the responder rate:
    A patient will be considered a responder if he or she did not receive a
    blood transfusion from Week 5 through Week 26 (EOT) and did not
    receive treatment for CAgD beyond what is permitted per protocol.
    Additionally, the patient's Hgb level must meet the following criterion:
    •Hgb increase = 1.5 g/dL from baseline (defined as the last Hgb value
    before administration of the first dose of study drug) at treatment
    assessment endpoint (defined as mean value from Weeks 23, 25, and
    26)
    L'endpoint primario sull'efficacia è il tasso di risposta:
    Un/a paziente sarà considerato/a un responder se non ha ricevuto una trasfusione di sangue dalla Settimana 5 alla Settimana 26 (EOT) e non ha ricevuto trattamento per CAgD oltre quanto consentito dal protocollo. Inoltre, il livello di Hgb del/della paziente deve soddisfare i seguenti criteri:
    • Aumento di Hgb = 1,5 g/dl rispetto alla baseline (definito come ultimo valore di Hgb prima della somministrazione della prima dose di farmaco in studio) all’endpoint di valutazione del trattamento (definito come valore medio dalle Settimane 23, 25 e 26)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 26
    Settimana 26
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints (Part A) Mean change from baseline in Hgb at treatment assessment endpoint (mean of values at Week 23, 25, and 26) Mean change from baseline in bilirubin (excluding patients with Gilbert's Syndrome) at treatment assessment endpoint Mean change from baseline in QOL, as assessed by the change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scores at the treatment assessment endpoint Mean change from baseline in lactate dehydrogenase (LDH) at the treatment assessment endpoint Incidence of solicited symptomatic anemia at EOT Efficacy Endpoints (Part B) The following parameters of disease activity will be assessed: ¿Hemoglobin ¿Bilirubin (total) ¿QOL assessments (FACIT-fatigue, EQ-5D-5L, SF-12, and PGIC Scale) ¿LDH ¿Transfusion requirements ¿Haptoglobin
    Total healthcare resource utilization at EOT
    Gli Endpoints di efficacia secondari (Parte A) Variazione media rispetto al baseline di Hgb all¿endpoint di valutazione del trattamento (media dei valori alle Settimane 23, 25 e 26) Variazione media rispetto al baseline di bilirubina (esclusi i pazienti con sindrome di Gilbert), all¿endpoint di valutazione del trattamento Variazione media rispetto al baseline della QOL, valutata mediante la variazione dei punteggi del questionario Functional Assessment of Chronic Illness Therapy FACIT-Fatigue, all¿endpoint di valutazione del trattamento Variazione media rispetto al baseline dei livelli di lattato deidrogenasi (LDH) all¿endpoint di valutazione del trattamento Incidenza di anemia sintomatica indotta alla EOT Endopints di efficacia secondari (Parte B) Saranno valutati i seguenti parametri di attivit¿ della malattia: ¿Emoglobina ¿Bilirubina (totale) ¿Valutazioni QOL (scala FACIT-Fatigue, EQ-5D-5L, SF-12, e scala PGIC) ¿LDH ¿Requisiti di trasfusione ¿Aptoglobina
    Utilizzazione totale delle risorse sanitarie all¿EOT
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 23, 25 and 26
    Settimana 23, 25 e 26
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tollerabilit¿
    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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA37
    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
    Austria
    Belgium
    Canada
    Denmark
    France
    Germany
    Hungary
    Israel
    Italy
    Japan
    New Zealand
    Norway
    Poland
    Spain
    Switzerland
    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
    LSLV
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months5
    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: 4
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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 No
    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 27
    F.4.2.2In the whole clinical trial 40
    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)
    standard of care
    standard of care
    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 Decision2018-03-19
    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
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