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    Summary
    EudraCT Number:2020-004438-39
    Sponsor's Protocol Code Number:BCX9930-202
    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:2022-01-27
    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 number2020-004438-39
    A.3Full title of the trial
    A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy
    Uno studio randomizzato, in aperto, multicentrico, a gruppi paralleli per valutare l'efficacia, la sicurezza e la tollerabilità della monoterapia orale BCX9930 per il trattamento dell'emoglobinuria parossistica notturna in soggetti con risposta inadeguata alla terapia con inibitori C5
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BCX9930 treatment in PNH patients with an inadequate response to C5 inhibitors
    BCX9930 per il trattamento dell'emoglobinuria parossistica notturna in soggetti con risposta inadeguata alla terapia con inibitori C5
    A.3.2Name or abbreviated title of the trial where available
    REDEEM 1
    REDEEM 1
    A.4.1Sponsor's protocol code numberBCX9930-202
    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 SponsorBIOCRYST 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 supportBioCryst Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMS Advanced Medical Services
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Address26 -28 Hammersmith Grove
    B.5.3.2Town/ cityLondra
    B.5.3.3Post codeW6 7BA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442088341144
    B.5.5Fax number+442088341156
    B.5.6E-mailoperations@ams-europe.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name ULTOMIRIS - 300 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO (VETRO) 3 ML (100 MG / ML) - 1 FLACONCINO
    D.2.1.1.2Name of the Marketing Authorisation holderALEXION EUROPE S.A.S.
    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/3/16/1661
    D.3 Description of the IMP
    D.3.1Product nameravulizumab
    D.3.2Product code [ravulizumab]
    D.3.4Pharmaceutical form Concentrate for 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 INNRavulizumab
    D.3.9.2Current sponsor codeUltomiris
    D.3.9.3Other descriptive nameUltomiris
    D.3.9.4EV Substance CodeSUB192773
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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 RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML)
    D.2.1.1.2Name of the Marketing Authorisation holderALEXION EUROPE S.A.S.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/03/166
    D.3 Description of the IMP
    D.3.1Product nameEculizumab
    D.3.2Product code [Eculizumab]
    D.3.4Pharmaceutical form Concentrate for 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 INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.2Current sponsor codeSoliris
    D.3.9.3Other descriptive nameSoliris
    D.3.9.4EV Substance CodeSUB25187
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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: 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 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 nameBCX9930
    D.3.2Product code [BCX9930]
    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 INNBCX9930
    D.3.9.2Current sponsor codeBCX9930
    D.3.9.3Other descriptive nameBCX9930 hydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal Nocturnal Hemoglobinuria
    Emoglobinuria parossistica notturna
    E.1.1.1Medical condition in easily understood language
    PNH
    EPN
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10034042
    E.1.2Term Paroxysmal nocturnal haemoglobinuria
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to C5 inhibitor therapy
    • To evaluate the long-term safety and tolerability of oral BCX9930 monotherapy administered over a 28- to 52 week treatment period in subjects with PNH with an inadequate response to C5 inhibitor therapy
    • Per determinare l'efficacia della monoterapia orale BCX9930 somministrata per 24 settimane, rispetto alla terapia continua con inibitori della componente 5 del complemento (C5), in soggetti con emoglobinuria parossistica notturna (EPN) con una risposta inadeguata alla terapia con inibitori C5
    • Valutare la sicurezza e la tollerabilità a lungo termine della monoterapia orale con BCX9930 somministrata per un periodo di trattamento da 28 a 52 settimane in soggetti con EPN con una risposta inadeguata alla terapia con inibitori C5
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of BCX9930 monotherapy administered for 24 weeks, as compared to continued C5 inhibitor therapy
    • To characterize the effects of BCX9930 monotherapy, as compared to continued C5 inhibitor therapy, using clinical and laboratory measurements, including complement and thrombosis biomarkers, PNH clone size, and C3-opsonization of red blood cells
    • To evaluate the effects of BCX9930 monotherapy, as compared to continued C5 inhibitor therapy, on the FACIT-Fatigue scale and other patient-reported outcomes (PROs)
    • To assess the effectiveness of BCX9930 monotherapy in the treatment of PNH
    • To characterize the effects of BCX9930 monotherapy using clinical
    and laboratory measurements, including complement and thrombosis biomarkers, PNH clone size, and C3 opsonization of RBCs
    • To evaluate the effects of BCX9930 monotherapy on FACIT-Fatigue scale and other PROs
    • To characterize BCX9930 PK parameters
    • Per valutare la sicurezza e la tollerabilità della monoterapia BCX9930 somministrata per 24 settimane, rispetto alla terapia continuata con un inibitore C5
    • Per caratterizzare gli effetti della monoterapia BCX9930, rispetto alla terapia continua con inibitore C5, utilizzando misurazioni cliniche e di laboratorio
    • Per valutare gli effetti della monoterapia BCX9930, rispetto alla terapia continuata con inibitori C5, sulla scala FACIT e altri risultati riferiti dai pazienti (PRO)
    • Valutare l'efficacia della monoterapia BCX9930 nel trattamento della EPN per un periodo di trattamento da 28 a 52 settimane
    • Per caratterizzare gli effetti della monoterapia BCX9930 utilizzando misurazioni cliniche e di laboratorio, in soggetti con EPN per un periodo di trattamento da 28 a 52 settimane
    • Per valutare gli effetti della monoterapia BCX9930 sulla scala FACIT e altri PRO in soggetti con EPN da 28 a 52 settimane
    • Caratterizzare le concentrazioni di BCX9930 e i parametri PK
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: PK / PD substudy - within the same protocol, V2 dated 27 May 2021

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: PK/PD V2 27 Maggio 2021
    E.3Principal inclusion criteria
    2. Body weight = 40 kg.
    3. Documented diagnosis of PNH confirmed by flow cytometry with a PNH granulocyte or monocyte clone size of = 10% during screening.
    4. Treated with a stable regimen of eculizumab for = 3 months prior to the screening visit or ravulizumab for = 6 months prior to the screening visit.
    5. Recorded the following results during screening:
    a. Hb of = 105 g/L (= 10.5 g/dL).
    b. ARC of = 100 × 109 cells/L (= 100,000 cells/µL; = 100 G/L).
    c. Absolute neutrophil count of = 0.75 × 109 cells/L (= 750 cells/µL; = 0.75 × G/L).
    d. Platelet count of = 30 × 109/L (= 30,000/µL; = 30 G/L).
    e. Adequate iron reserve based on ferritin = LLN or total iron binding capacity = upper limit of the normal reference range (ULN).
    f. Estimated glomerular filtration rate of = 50 mL/min/1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Levey and Stevens 2010).
    6. Contraception requirements: WOCBP and partners of male subjects to use highly effective contraception
    7. Documentation of current vaccinations against Neisseria meningitidis types A, C, W, and Y, and Streptococcus pneumoniae, or willingness to start vaccination series at least 14 days prior to Day 1. (Note: Vaccination for N. meningitidis type B and for H. influenzae type B (Hib) is strongly encouraged where authorized and available.)
    8. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study, including compliance with the BID dosing schedule for BCX9930.
    9. Willing and able to provide written informed consent
    1. Maschio o femmina, di età = 18 anni.
    2. Peso corporeo = 40 kg.
    3. Diagnosi documentata di EPN confermata mediante citometria a flusso con dimensione del clone di granulociti EPN o di monociti = 10% durante lo screening.
    4. Trattato con un regime stabile di eculizumab per = 3 mesi prima della visita di screening o ravulizumab per = 6 mesi prima della visita di screening.
    5. Ha registrato i seguenti risultati durante lo screening:
    a. Hb di = 105 g/L (= 10,5 g/dL).
    b. ARC di = 100 × 109 cellule/L (= 100.000 cellule / µL; = 100 G/L).
    c. Conta assoluta dei neutrofili di = 0,75 × 109 cellule/L (= 750 cellule / µL; = 0,75 × G/L).
    d. Conta piastrinica di = 30 × 109/L (= 30.000/µL; = 30 G/L).
    e. Riserva di ferro adeguata basata su ferritina = LLN o capacità totale di legare il ferro = limite superiore del range di riferimento normale (ULN).
    f. Velocità di filtrazione glomerulare stimata di = 50 mL/min/1,73 m2 utilizzando l'equazione della Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) (Levey and Stevens 2010).
    6. Contraccezione: WOCBP utilizzare un metodo contraccettivo altamente efficace durante lo studio
    7. Documentazione delle attuali vaccinazioni contro Neisseria meningitidis di tipo A, C, W e Y e Streptococcus pneumoniae o disponibilità a iniziare le serie di vaccinazioni almeno 14 giorni prima del Giorno 1.
    (Nota: la vaccinazione contro N. meningitidis tipo B e H. influenzae tipo B (Hib) è fortemente raccomandata dove autorizzato e disponibile.)
    8. Secondo il parere dello sperimentatore, il soggetto è in grado di rispettare adeguatamente tutte le procedure di studio e le restrizioni richieste per lo studio, inclusa la conformità con lo schema di dosaggio BID per BCX9930.
    9. Capacità e volontà di fornire un consenso informato scritto
    E.4Principal exclusion criteria
    1. Known history of or existing diagnosis of hereditary complement deficiency.
    2. History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
    3. Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
    4. History of malignancy within 5 years prior to the screening visit, with exception of adequately treated non-melanoma skin or superficial bladder cancer, curatively treated carcinoma in situ of the cervix, or other curatively treated solid tumor deemed by the investigator and medical monitor to be at low risk for recurrence.
    5. Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening.
    6. Current participation in any other investigational drug study or participation in an investigational drug study within 30 days prior to the screening visit, or 5.5 half-lives of the investigational drug, whichever is longer.
    7. Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
    8. Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit. (Note: Treatment with these medications initiated > 28 days prior to the screening visit is not exclusionary, if the dose is stable and there is a reasonable expectation that treatment will be continued.)
    9. Receiving iron supplementation with an unstable dose in the 28 days prior to the screening visit.
    10. Clinically significant abnormal electrocardiogram (ECG) at the screening visit.
    11. Subjects with any of the following results at the screening visit:
    a. Alanine aminotransferase (ALT; also serum glutamic-pyruvic transaminase [SGPT]) > 3 × ULN.
    b. Aspartate aminotransferase (AST; also serum glutamic-oxaloacetic transaminase [SGOT]) > 3 × ULN.
    (Note: Subjects may be enrolled with AST > 3 × ULN if explained by hemolysis.)
    c. Total serum bilirubin > 2 × ULN
    (Note: Subjects may be enrolled with total serum bilirubin > 2 × ULN if explained by hemolysis or Gilbert's syndrome. In the case of hemolysis, total serum bilirubin must be < 5 × ULN and in the case of Gilbert's syndrome, total serum bilirubin must be < 11 × ULN.)
    12. Current use of a prohibited concomitant medication within 7 days prior to Day 1 as detailed in Section 9.8.1.
    13. Positive serology for human immunodeficiency virus, or active infection with hepatitis B virus or hepatitis C virus, unless receiving antiviral therapy and viral load is undetectable.
    14. Positive drugs of abuse screen, unless by prescription.
    15. Pregnant, planning to become pregnant, or breastfeeding.
    16. Known hypersensitivity to BCX9930 or any of its formulation excipients.
    17. History of severe hypersensitivity to any medicinal product, which was associated with swelling, severe rash requiring treatment/hospitalization, or anaphylaxis.
    18. Any other clinically significant medical or psychiatric condition that, in the opinion of the investigator or sponsor, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject.
    1. Storia nota o diagnosi esistente di deficit ereditario del complemento.
    2. Storia di trapianto di cellule ematopoietiche o trapianto di organo solido o candidato previsto per il trapianto durante lo studio.
    3. Infarto miocardico o accidente cerebrovascolare entro 30 giorni prima dello screening, o condizione cardiovascolare o cerebrovascolare clinicamente significativa e non controllata, inclusa angina instabile, grave insufficienza cardiaca congestizia, sincope inspiegabile, aritmia e stenosi aortica critica.
    4. Anamnesi di neoplasia nei 5 anni precedenti la visita di screening, ad eccezione di carcinoma cutaneo non melanoma o della vescica superficiale adeguatamente trattato, carcinoma in situ della cervice trattato in modo curativo o altro tumore solido trattato curativamente ritenuto dallo sperimentatore e dal monitor medico a basso rischio di recidiva.
    5. Infezione batterica, virale o fungina attiva o qualsiasi altra infezione grave entro 14 giorni prima dello screening.
    6. Partecipazione attuale a qualsiasi altro studio di un farmaco sperimentale o partecipazione a studio di un farmaco sperimentale nei 30 giorni precedenti alla visita di screening, o 5,5 emivite del farmaco sperimentale, a seconda di quale sia il periodo più lungo.
    7. Trattamento con globulina anti-timociti nei 180 giorni precedenti alla visita di screening.
    8. Inizio del trattamento con un agente stimolante l'eritropoiesi (es. Eritropoietina), un agonista del recettore della trombopoietina (es. Eltrombopag) o danazolo entro 28 giorni prima della visita di screening. (Nota: il trattamento con questi farmaci iniziato > 28 giorni prima della visita di screening non è esclusorio, se la dose è stabile e c'è una ragionevole aspettativa che il trattamento venga continuato.)
    9. Ricevere un'integrazione di ferro con una dose instabile nei 28 giorni precedenti la visita di screening.
    10. Elettrocardiogramma anormale clinicamente significativo (ECG) alla visita di screening.
    11. Soggetti con uno dei seguenti risultati alla visita di screening:
    a. Alanina aminotransferasi (ALT; anche transaminasi glutammicopiruvica sierica [SGPT]) > 3 × ULN.
    b. Aspartato aminotransferasi (AST; anche transaminasi glutammicoossalacetica sierica [SGOT]) > 3 × ULN
    (Nota: i soggetti possono essere arruolati con ALT o AST > 3 × ULN se spiegato dall'emolisi.)
    c. Bilirubina sierica totale > 2 × ULN
    (Nota: i soggetti possono essere arruolati con bilirubina sierica totale > 2 × ULN se dovuta all'emolisi o alla sindrome di Gilbert. In caso di emolisi, la bilirubina sierica totale deve essere < 5 × ULN e nel caso della sindrome di Gilbert, la bilirubina sierica totale deve essere < 11 × ULN.)
    12. Uso corrente di un farmaco concomitante proibito entro 7 giorni prima del Giorno 1 come dettagliato nella Sezione 9.8.1.
    13. Sierologia positiva per virus dell'immunodeficienza umana o infezione attiva con virus dell'epatite B o virus dell'epatite C, a meno che non stia ricevendo una terapia antivirale e la carica virale non sia rilevabile.
    14. Screening positivo per droghe da abuso, salvo prescrizione medica.
    15. Incinta, sta pianificando una gravidanza o sta allattando.
    16. Nota ipersensibilità al BCX9930 o ad uno qualsiasi degli eccipienti della sua formulazione.
    17. Storia di grave ipersensibilità a qualsiasi medicinale, che è stata associata a gonfiore, a un'eruzione cutanea grave, che ha richiesto un trattamento/ricovero in ospedale, oppure ad anafilassi.
    18. Qualsiasi altra condizione medica o psichiatrica clinicamente significativa che, a giudizio dello sperimentatore o dello sponsor, potrebbe interferire con la capacità del soggetto di partecipare allo studio o aumentare il rischio risultante dalla partecipazione da parte di tale soggetto
    E.5 End points
    E.5.1Primary end point(s)
    • Change from baseline (CFB) in hemoglobin (Hb)
    • Number and proportion of subjects with a TEAE
    • Number and proportion of subjects who discontinue due to a TEAE
    • Number and proportion of subjects who experience a TESAE
    • Number and proportion of subjects who experience a CTCAE Grade 3 or Grade 4 TEAE
    • Number and proportion of subjects who experience a treatmentemergent CTCAE Grade 3 or Grade 4 laboratory abnormality
    • Variazione rispetto al basale (CFB) dell'emoglobina (Hb)
    • Numero e percentuale di soggetti che presentano un evento avverso emergente da trattamento (TEAE)
    • Numero e percentuale di soggetti che interrompono lo studio a causa di un evento avverso emergente da trattamento
    • Numero e percentuale di soggetti che registrano un evento avverso grave emergente da trattamento (TESAE)
    • Numero e percentuale di soggetti che registrano un evento avverso emergente da trattamento di grado 3 o 4 del CTCAE
    • Numero e percentuale di soggetti che registrano un'anormalità dei valori di laboratorio emergente dal trattamento di grado 3 o 4 del CTCAE
    E.5.1.1Timepoint(s) of evaluation of this end point
    1 - mean of values at Weeks 12, 16, 20, and 24
    others - week 52
    1 - media dei valori alle settimane 12, 16, 20 e 24
    altri - sett. 52
    E.5.2Secondary end point(s)
    1. Proportion of subjects who are transfusion-free
    2. Number of units of packed red blood cells (pRBCs) transfused
    3. CFB in FACIT-Fatigue scale score
    1. Proporzione di soggetti che sono liberi da trasfusioni
    2. Numero di unità di globuli rossi concentrati (pRBC) trasfuse
    3. CFB nel punteggio della scala FACIT
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. - week 24
    2. - week 24
    3. - mean of values at Weeks 12, 16, 20, and 24
    1 - sett. 24
    2 - sett. 24
    3 - media dei valori alle settimane 12, 16, 20 e 24
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response 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 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) Yes
    E.8.2.2Placebo No
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Argentina
    Brazil
    Canada
    China
    Colombia
    European Union
    Japan
    Korea, Republic of
    Taiwan
    Turkey
    United Kingdom
    United States
    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
    LVLS
    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 months0
    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 months3
    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: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 81
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who are experiencing clinical benefit will be offered the opportunity to continue to receive BCX9930 via an access protocol BCX9930-201 EudraCT: 2020-000501-93
    Ai soggetti che traggono un beneficio clinico sarà offerta l'opportunità di continuare a ricevere BCX9930 attraverso l'accesso al protocollo BCX9930-201 EudraCT: 2020-000501-93
    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 Decision2021-10-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-10
    P. End of Trial
    P.End of Trial StatusOngoing
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