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    Summary
    EudraCT Number:2019-004103-12
    Sponsor's Protocol Code Number:TAK-079-1004
    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:2021-01-21
    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 number2019-004103-12
    A.3Full title of the trial
    A Phase II, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability and Efficacy of TAK-079 in Patients with Persistent/Chronic Primary Immune Thrombocytopenia
    Studio di fase 2, randomizzato, in doppio cieco, controllato con placebo per valutare la sicurezza, la tollerabilità e l’efficacia di TAK-079 in pazienti con trombocitopenia immune primaria persistente/cronica
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Safety, Tolerability and Efficacy of TAK-079 in Patients with Immune Thrombocytopenia
    Studio per valutare la sicurezza, la tollerabilità e l'efficacia di TAK-079 in pazienti con trombocitopenia immune
    A.3.2Name or abbreviated title of the trial where available
    /
    /
    A.4.1Sponsor's protocol code numberTAK-079-1004
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1245-3760
    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 SponsorMILLENNIUM PHARMACEUTICALS, 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 supportMillennium Pharmaceuticals, Inc. (Takeda)
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMillennium Pharmaceuticals, Inc. (Takeda)
    B.5.2Functional name of contact pointDrug Information Call Center
    B.5.3 Address:
    B.5.3.1Street Address40 Landsdowne Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number0015107402412
    B.5.5Fax number0018008816092
    B.5.6E-mailmedical@mlnm.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 nameTAK-079
    D.3.2Product code [TAK-079]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTAK-079
    D.3.9.2Current sponsor codeTAK-079
    D.3.9.4EV Substance CodeSUB178418
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namedifenidramina
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form 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 INNDIFENIDRAMINA CLORIDRATO
    D.3.9.1CAS number 147-24-0
    D.3.9.2Current sponsor codedifenidramina cloridrato
    D.3.9.3Other descriptive namedyphenhydramine hydrochloride
    D.3.9.4EV Substance CodeSUB01769MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    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 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.IMP: 3
    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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameparacetamolo
    D.3.2Product code [acetominofene]
    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 INNPARACETAMOLO
    D.3.9.1CAS number 103-90-2
    D.3.9.2Current sponsor codeparacetamolo
    D.3.9.3Other descriptive nameacetominophen
    D.3.9.4EV Substance CodeSUB09611MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number1000
    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 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.IMP: 4
    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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namemetilprednisolone
    D.3.2Product code [/]
    D.3.4Pharmaceutical form 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 INNMETILPREDNISOLONE
    D.3.9.1CAS number 83-43-2
    D.3.9.2Current sponsor codemetilprednisolone
    D.3.9.3Other descriptive namemethylprednisolone
    D.3.9.4EV Substance CodeSUB08872MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number20
    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 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 injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary immune thrombocytopenia
    Trombocitopenia immune primaria
    E.1.1.1Medical condition in easily understood language
    Low levels of the blood cells that prevent bleeding (platelets)
    Bassi livelli di cellule del sangue che prevengono il sanguinamento (piastrine)
    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 23.0
    E.1.2Level LLT
    E.1.2Classification code 10050245
    E.1.2Term Autoimmune thrombocytopenia
    E.1.2System Organ Class 100000004851
    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 is to evaluate the safety and tolerability of TAK-079 in patients with persistent/chronic primary ITP.
    L’obiettivo primario è valutare la sicurezza e la tollerabilità di TAK-079 in pazienti con ITP primaria persistente/cronica.
    E.2.2Secondary objectives of the trial
    The secondary objective is to assess the effects of TAK-079 administration on platelet counts in patients with persistent/chronic primary ITP.
    L’obiettivo secondario è valutare gli effetti della somministrazione ripetuta di TAK-079 sulla conta piastrinica in pazienti con ITP primaria persistente/cronica.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each patient must meet all the following inclusion criteria to be to be randomized to treatment:
    1. Age 18 years or older and able and willing to comply with study procedures.
    2. Diagnosed with ITP that has persisted for =3 months, diagnosed in accordance to The American Society of Hematology 2011 Evidence-based Practice Guideline for Immune Thrombocytopenia or the International Consensus Report on The Investigation and Management of Primary Immune Thrombocytopenia as locally applicable.
    3. Presents with a mean platelet count of <30,000/µL for the 4 weeks before the first study dose. The mean platelet count is based on at least 2 platelet counts within 4 weeks of dosing, including the value obtained at screening. No individual platelet count >35,000/µL during these times is allowed.
    4. Has had 1 prior platelet response to any standard of care treatment to achieve a platelet count of =50,000/µL. Any standard treatment in this context may include therapies that are not
    permitted concomitant therapies during the study.
    5. If receiving standard background treatment for ITP, treatment should be stable in dose and frequency for at least 4 weeks before dosing.
    a) Permitted standard background treatments may include: 1 oral corticosteroid; ±1 immunosuppressant from the following list: azathioprine, danazol, dapsone, cyclosporine, mycophenolate mofetil, mycophenolate sodium; ±1 TPO-RA (romiplostim, eltrombopag, avatrombopag); ±fostamatinib. Corticosteroids, including dexamethasone, must be given as oral, daily therapy as opposed to pulse therapy. High-dose pulse steroid therapy is not
    allowed within 14 days before Day 1.
    b) The dose of any permitted standard background therapy must be expected to remain stable through the study, unless dose reduction is required because of toxicities.
    6. Female patients of childbearing potential are required to have a negative pregnancy test. Both female patients of childbearing potential and male patients must practice an effective, reliable,
    and approved contraceptive regimen during the study and for up to 90 days or 5 half-lives, whichever is longer, after discontinuation of treatment.
    7. Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care.
    Principali criteri di inclusione:
    1. Età pari o superiore ai 18 anni e in grado e disposto a rispettare le procedure dello studio.
    2. Diagnosi di ITP che ha si è mantenuta per = 3 mesi, diagnosticata in conformità alle linee guida della pratica basata sull’evidenza per la trombocitopenia immune della Società americana di ematologia 2011 o al rapporto sul consenso internazionale sulla sperimentazione e la gestione della trombocitopenia immune primaria applicabile a livello locale.
    3. Conta piastrinica media < 30.000/µl per le 4 settimane precedenti la prima dose dello studio. La conta piastrinica media è basata su almeno 2 conte piastriniche nelle 4 settimane di somministrazione, incluso il valore ottenuto allo screening. Non è consentita alcuna conta piastrinica > 35.000/µl durante questi periodi.
    4. Ha avuto una precedente risposta piastrinica a qualsiasi trattamento dello standard di cura per raggiungere una conta piastrinica = 50.000/µl. Qualsiasi terapia standard in questo contesto può includere terapie che non sono terapie concomitanti consentite durante lo studio.
    5. Sta ricevendo un trattamento di base standard per l’ITP, il trattamento deve essere stabile in dose e frequenza per almeno 4 settimane prima della somministrazione.
    a) I trattamenti di base standard consentiti possono includere: 1 corticosteroide orale, ± 1 immunosoppressore tra i seguenti: azatioprina, danazolo, dapsone, ciclosporina, micofenolato mofetile, micofenolato sodico; ± 1agonista del recettore della trombopoietina (romiplostim, eltrombopag, avatrombopag); ± fostamatinib. I corticosteroidi, compreso il desametasone, devono essere somministrati come terapia orale, giornaliera rispetto alla pulsoterapia. Una pulsoterapia steroidea ad alte dosi non è consentita nei 14 giorni prima del Giorno 1.
    b) La dose di qualsiasi terapia di base standard consentita deve essere rimanere stabile per tutta la durata dello studio, a meno non sia richiesta una riduzione della dose a causa della tossicità.
    6. Le pazienti di sesso femminile in età fertile devono avere un test di gravidanza negativo. Sia le pazienti di sesso femminile in età fertile che i pazienti di sesso maschile devono adottare un regime contraccettivo efficace, affidabile e approvato durante lo studio e fino a 90 giorni o 5 emivite, a seconda di quale periodo è più lungo, dopo l’interruzione del trattamento.
    7. Il consenso per iscritto volontario deve essere fornito prima dell’esecuzione di qualsiasi procedura correlata allo studio che non rientri nella terapia standard, con la consapevolezza che il consenso può essere ritirato dal paziente in qualsiasi momento, senza pregiudizio per le cure mediche future.
    E.4Principal exclusion criteria
    Patients meeting any of the following exclusion criteria are not to be randomized to treatment:
    1. Use of anticoagulants or any drug with antiplatelet effect (such as aspirin) within 3 weeks before screening.
    2. History of any thrombotic or embolic event within 12 months before screening.
    3. History of splenectomy within 3 months before screening.
    4. Use of IVIg, subcutaneous immunoglobulin or anti-D treatment within 4 weeks of screening.
    5. Diagnosed with chronic obstructive pulmonary disease (COPD) or asthma, and a prebronchodilatory forced expiratory volume in 1 second (FEV1) <50% of predicted normal.
    Note: FEV1 testing is required for patients suspected of having COPD or asthma.
    6. Use of rituximab or any mAb for immunomodulation within 4 months before first dosing.
    Note: Patients with prior exposure to rituximab must have CD19 counts within the normal range at screening.
    7. Use of immunosuppressants (such as cyclophosphamide, vincristine) other than permitted oral immunosuppressants within 6 months before first dosing.
    8. Diagnosed with myelodysplastic syndrome.
    9. Has received vaccinations within 4 weeks before screening or has any vaccinations planned during the study.
    10. Currently experiencing any medical condition that, in the opinion of the investigator, might interfere with the patient’s participation in the study (such as significant cardiovascular, pulmonary, hematologic, gastrointestinal, endocrinologic, hepatic, renal, neurologic, malignancy, or infectious disease), that poses an added risk for the patient, or could confound the assessment of the patient.
    11. Pregnancy or lactation during screening period or on Day 1 before first dose of study drug.
    12. Participation in any other investigational drug study or exposure to other investigational agent within 4 weeks or 5 half-lives, whichever is longer, before Day 1.
    13. Has had an opportunistic infection =12 weeks before initial study dosing or is currently undergoing treatment for a chronic opportunistic infection, such as tuberculosis (TB), pneumocystis pneumonia, cytomegalovirus, herpes simplex virus, herpes zoster, or atypical mycobacteria.
    14. Exhibits clinically significant laboratory abnormalities at screening:
    a) Alanine aminotransferase or aspartate aminotransferase >3 times the upper limit of normal (ULN).
    b) Total bilirubin >1.5 × ULN (Note: Patients with a confirmed diagnosis of Gilbert syndrome that is documented in the patient’s medical record are not excluded based on this criterion).
    c) Absolute neutrophil count <1500/mm3.
    d) Hemoglobin <8 g/dL.
    e) IgG <500 mg/dL.
    f) Lymphocyte count <500/mm3.
    g) Creatinine clearance, CrCl, <30 mL/min (ie, CrCl =30 mL/min is allowed).
    15. Has a positive T-cell interferon-¿ release assay (TIGRA) (results through QuantiFERON TB Gold test or T-Spot/Elispot) at the screening visit, noting the following:
    a) A purified protein derivative (PPD) skin test may be used as a replacement, if TIGRA testing is not available.
    b) Patients with an indeterminate TIGRA result must meet the following criteria:
    – Has a negative PPD skin test (defined as <5 mm induration).
    – Is at low risk of acquiring TB (eg, avoids close contact with TB positive individual[s]), and/or chest x-ray =6 months before the screening visit that is consistent with no evidence of latent or active TB).
    16. Has a serious medical or psychiatric illness that could, in the investigator’s opinion, potentially interfere with the completion of treatment according to this protocol.
    Principali criteri di esclusione:
    1. L’uso di anticoagulanti o di qualsiasi farmaco con effetto antipiastrinico (quali aspirina) entro 3 settimane prima dello screening.
    2. Anamnesi di qualsiasi evento trombotico o embolico nei 12 mesi precedenti lo screening.
    3. Anamnesi di splenectomia nei 3 mesi precedenti lo screening.
    4. Uso di trattamento con IVIg, immunoglobuline per via sottocutanea o immunoglobuline anti-D nelle 4 settimane dallo screening.
    5. Diagnosi di broncopneumopatia cronica ostruttiva (BPCO) o asma e un volume espiratorio forzato prebroncodilatatorio in 1 secondo (FEV1) < 50% del normale previsto.
    Nota: Il test FEV1 è richiesto per i pazienti con sospetta BPCO o asma.
    6. Uso di rituximab o di qualsiasi anticorpo monoclonale immunomodulante nei 4 mesi precedenti la prima somministrazione.
    Nota: I pazienti con precedente esposizione a rituximab devono avere una conta di CD19 nel range normale allo screening.
    7. Uso di agenti immunosoppressori (come ciclofosfamide, vincristina) diversi dagli immunosoppressori orali consentiti nei 6 mesi precedenti la prima somministrazione.
    8. Diagnosi di sindrome mielodisplastica.
    9. Ha ricevuto vaccinazioni nelle 4 settimane precedenti lo screening o è prevista una vaccinazione durante lo studio.
    10. Attualmente sta manifestando una condizione medica che, a giudizio dello sperimentatore, potrebbe interferire con la partecipazione paziente allo studio (come neoplasie significative cardiovascolari, polmonari, ematologiche, gastrointestinali, endocrinologiche, epatiche, renali, neurologiche o malattia infettiva), che comporta un rischio aggiuntivo per il paziente o potrebbe confondere la valutazione del paziente.
    11. Gravidanza o allattamento durante il periodo di screening o il Giorno 1 prima della prima dose di farmaco sperimentale.
    12. Partecipazione a qualsiasi altro studio su un farmaco sperimentale oppure esposizione a un altro agente sperimentale nelle 4 settimane o 5 emivite, a seconda di quale periodo è più lungo, prima del Giorno 1.
    13. Ha avuto un’infezione opportunistica = 12 settimane prima della somministrazione iniziale dello studio o è attualmente sottoposto a trattamento per un’infezione opportunistica cronica, come tubercolosi (TB), polmonite da Pneumocystis, citomegalovirus, virus dell’herpes simplex, herpes zoster o micobatteri atipici.
    14. Mostra anomalie di laboratorio clinicamente significative allo screening:
    a) Alanina aminotransferasi o aspartato aminotransferasi > 3 volte il limite superiore della norma (ULN).
    b) Bilirubina totale > 1,5 volte l’ULN (Nota: i pazienti con una diagnosi confermata di sindrome di Gilbert documentata nella cartella clinica del paziente non sono esclusi in base a questo criterio).
    c) Conta assoluta dei neutrofili < 1500/mm3.
    d) Emoglobina < 8 g/dl.
    e) IgG < 500 mg/dl.
    f) Conta linfocitaria < 500/mm3.
    g) Clearance della creatinina, CrCl, < 30 ml/min (ovvero, una CrCl = 30 ml/min è consentita).
    15. Ha un risultato positivo del test di rilascio di interferone ¿ delle cellule T (TIGRA) (risultati tramite il test QuantiFERON TB Gold o T Spot/Elispot) alla visita di screening, annotando quanto segue:
    a) Può essere utilizzato un test cutaneo con un derivato proteico purificato (PPD) in sostituzione se il test TIGRA non è disponibile.
    b) I pazienti con un risultato TIGRA indeterminato devono soddisfare i seguenti criteri:
    – Presentare un risultato negativo al test cutaneo PPD (definito come indurimento < 5 mm).
    – Presenta un basso rischio di contrarre la TB (per es. evita uno stretto contatto con individui positivi alla TB) e/o radiografia del torace = 6 mesi prima della visita di screening che è coerente con l’assenza di qualsiasi evidenza di TB latente o attiva.
    16. Presenta una malattia medica o psichiatrica grave che potrebbe, secondo il parere dello sperimentatore, interferire potenzialmente con il completamento del trattamento in conformità al presente protocollo.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the percentage of patients with TEAEs including Grade 3 or higher events, SAEs, and AEs leading to TAK-079 discontinuation.
    L’endpoint primario è la percentuale di pazienti con eventi avversi emergenti dal trattamento (TEAE) inclusi gli eventi di grado 3 o superiore, gli eventi avversi seri e gli eventi avversi (AE) che portano all’interruzione di TAK-079.
    E.5.1.1Timepoint(s) of evaluation of this end point
    TEAEs that occur after administration of the first dose of TAK-079 and through to the end of the SFP period will be tabulated and followed until resolution.
    I TEAE che si verificano dopo la somministrazione della prima dose di TAK-079 e fino al termine del periodo di SFP saranno riepilogati in tabelle e seguiti fino alla risoluzione.
    E.5.2Secondary end point(s)
    The secondary endpoint assesses the effects of TAK-079 administration on changes in the platelet count, through the following 4 analyses:
    1. Percentage of patients with a platelet response. Platelet response is defined as a platelet count =50,000/µL and =20,000/µL above baseline on at least 2 visits in the absence of any concomitant rescue therapy.
    2. The percentage of patients with a complete platelet response. Complete platelet response is defined as a platelet count =100,000/µL on at least 2 visits in the absence of any concomitant rescue therapy.
    3. The percentage of patients with a clinically meaningful platelet response. A clinically meaningful platelet response is defined as a platelet count =20,000/µL above baseline on at least 2 visits in the absence of any concomitant rescue therapy.
    4. The percentage of patients with a hemostatic platelet response. A hemostatic platelet response is defined for patients with a baseline platelet count of <15,000/µL who achieve a platelet count of =30,000/µL and =20,000/µL above baseline on at least 2 visits in the absence of any concomitant rescue therapy.
    Principali criteri di valutazione e analisi:
    L’endpoint primario è la percentuale di pazienti con eventi avversi emergenti dal trattamento (TEAE) inclusi gli eventi di grado 3 o superiore, gli eventi avversi seri e gli eventi avversi (AE) che portano all’interruzione di TAK-079.
    L’endpoint secondario valuta gli effetti della somministrazione di TAK-079 sulle variazioni nella conta piastrinica, attraverso le 4 analisi seguenti:
    1. Percentuale di pazienti con una risposta piastrinica. La risposta piastrinica è definita come una conta piastrinica = 50.000/µl e = 20.000/µl sopra al basale in almeno 2 visite in assenza di qualsiasi terapia di soccorso concomitante.
    2. Percentuale di pazienti con una risposta piastrinica completa. La risposta piastrinica completa è definita come una conta piastrinica = 100.000/µl in almeno 2 visite in assenza di qualsiasi terapia di soccorso concomitante.
    3. Percentuale di pazienti con una risposta piastrinica clinicamente significativa. Una risposta piastrinica clinicamente significativa è definita come una conta piastrinica = 20.000/µl sopra al basale in almeno 2 visite in assenza di qualsiasi terapia di soccorso concomitante.
    4. Percentuale di pazienti con una risposta piastrinica emostatica. Una risposta piastrinica emostatica è definita per i pazienti con una conta piastrinica al basale < 15.000/µl che raggiungono una conta piastrinica = 30.000/µl e = 20.000/µl sopra al basale in almeno 2 visite in assenza di qualsiasi terapia di soccorso concomitante.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study. Please refer to protocol Appendix A and Appendix B.
    Per tutta la durata dello studio. La preghiamo di fare riferimento all’Appendice A e all’Appendice B del protocollo.
    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 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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 trial3
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Ukraine
    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
    The analyses for the clinical study report will be conducted after all patients randomized in the study have completed the end-of-study visit (ie, Week 32 for TAK-079 patients, Week 20 for placebo patients who chose not to enter the OLE, or EW32 for placebo patients who participated in the OLE)
    Le analisi per la relazione dello studio clinico saranno condotte dopo che tutti i pazienti randomizzati nello studio avranno completato la visita di fine studio (ossia, Settimana 32 per i pazienti con TAK-079, Settimana 20 per i pazienti con placebo che scelgono di non entrare nell’OLE o SE32 per i pazienti con placebo che hanno partecipato all’OLE)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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 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: 18
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    If the subject is not capable of rendering adequate written informed consent, then the subject's legally acceptable representative may provide such consent for the subject in accordance with applicable laws and regulations.
    Se il soggetto non è in grado di esprimere il consenso informato scritto adeguato, il rappresentante legalmente accettabile del soggetto può fornire tale consenso per il soggetto in conformità alle leggi e alle normative applicabili.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 54
    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 Decision2020-07-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-05-21
    P. End of Trial
    P.End of Trial StatusOngoing
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