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    Summary
    EudraCT Number:2020-005855-19
    Sponsor's Protocol Code Number:BCX9930-211
    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:2021-10-22
    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 number2020-005855-19
    A.3Full title of the trial
    An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects with Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, or Primary Membranous Nephropathy
    Studio open-label, proof of concept, sulla sicurezza e tollerabilità della terapia con BCX9930 somministrato per via orale in soggetti affetti da Glomerulopatia a depositi di C3, Nefropatia a depositi di IgA o Nefropatia Membranosa Primaria.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluating oral BCX9930 in renal diseases
    Valutazione del BCX9930 per via orale in patologie renali
    A.3.2Name or abbreviated title of the trial where available
    RENEW
    RENEW
    A.4.1Sponsor's protocol code numberBCX9930-211
    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, AMS
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW6 7BA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number88341144
    B.5.5Fax number88341156
    B.5.6E-mailoperations@ams-europe.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 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.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product 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 number100
    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
    complement 3 glomerulopathy
    immunoglobulin A nephropathy
    primary membranous nephropathy
    Glomerulopatia a depositi di C3,
    Nefropatia a depositi di IgA,
    Nefropatia Membranosa Primaria.
    E.1.1.1Medical condition in easily understood language
    C3 G
    IgA nephropathy
    primary membranous nephropathy
    C3 G
    Nefropatia IgA
    Nefropatia Membranosa Primaria.
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10021263
    E.1.2Term IgA nephropathy
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10027170
    E.1.2Term Membranous nephropathy
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077827
    E.1.2Term C3 glomerulopathy
    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 evaluate the therapeutic potential of BCX9930 as assessed by proteinuria measures
    Valutare il potenziale terapeutico del BCX9930 valutato mediante misurazioni di proteinuria
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of BCX9930
    To evaluate the therapeutic potential of BCX9930 as assessed by other measures of clinical benefit
    To evaluate effects of BCX9930 on light microscopic, immunofluorescence, and ultrastructural morphologic findings
    To characterize the effects of BCX9930 on blood and urine biomarkers of complement activation and consumption
    To evaluate the correlation of BCX9930-associated changes in blood and urine biomarkers of complement activation and consumption with changes in proteinuria
    • Valutare la sicurezza e la tollerabilità del BCX9930
    • Valutare il potenziale terapeutico del BCX9930 come valutato da altre misure di beneficio clinico
    • Valutare gli effetti del BCX9930 su microscopia ottica, immunofluorescenza e risultati morfologici ultrastrutturali
    • Caratterizzare gli effetti del BCX9930 sui biomarcatori ematici e urinari di attivazione e consumo del complemento
    • Valutare la correlazione dei cambiamenti associati a BCX9930 nei biomarcatori ematici e urinari di attivazione e consumo del complemento con cambiamenti in termini di proteinuria
    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

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Sottostudio PK/PD, all'interno dello stesso protocollo
    E.3Principal inclusion criteria
    1. Willing and able to provide written informed consent
    2. Male or non-pregnant, non-lactating female subjects >= 18 years of age
    3. Body weight >= 40 kg.
    4. Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review of digital images and pathology reports of renal biopsy samples
    5. For subjects with C3G only, documentation of duration of illness of at least 90 days by either a prior biopsy >= 90 days prior to screening confirming a diagnosis of C3G OR a clinical diagnosis of C3G with at least one documented proteinuria assessment >= 90 days prior to initial screening visit.
    6. For subjects with C3G only, proteinuria defined as >= 1 g of urinary protein per 24 hours at screening that has not shown a >= 25% decrease from the most recent documented proteinuria assessment, which was collected >= 30 days prior to and =< 180 days of initial screening visit.
    7. For subjects with IgAN only, proteinuria defined as 1 g to 4 g of urinary protein per 24 hours at screening that has not shown a >= 25% decrease from the most recent documented proteinuria assessment, which was collected >= 30 days prior to and =< 180 days of initial screening visit.
    8. For subjects with PMN only, an anti-phospholipase A2 receptor antibody (aPLA2Rab) Immunoglobulin G (IgG) titer of >= 150 U/mL and 3.5 g to =<11 g of urinary protein per 24 hours at screening that has not shown a >= 25% decrease from the most recent documented proteinuria assessment, which was collected >= 30 days prior to and =< 180 days of initial screening visit.
    9. An eGFR >= 50 mL/min/1.73 m2 (or >= 30 mL/min/1.73 m2 after DMC recommendation)
    10. Resting supine vital signs within the following ranges:
    • Systolic blood pressure, 80 to 150 mm Hg, inclusive, for adults
    • Systolic blood pressure below the 90th percentile, for adolescents per Section 12.10.1
    • Diastolic blood pressure = 90 mm Hg
    11. Treatment with a stable, maximum recommended or maximum tolerated dose of an angiotensin converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) for at least 60 days prior to the Day 1
    12. Contraception requirements - WOCBP and female partners of male subjects to use highly effective contraception methods
    13. Documentation of current vaccination against N. meningitidis Types A, C, W and Y, and S. pneumoniae vaccine, or must be vaccinated or willingness to start vaccination series at least 14 days prior to Day 1
    14. In the opinion of the investigator, the subject is expected to comply adequately with all required study procedures and restrictions for the study
    1. Essere disposti e in grado di fornire il consenso informato scritto
    2. Essere soggetti di sesso maschile o soggetti di sesso femminile in assenza di gravidanza o allattamento di età >= 18 anni.
    3. Peso corporeo >= 40 kg.
    4. Diagnosi primaria di C3G, IgAN o PMN confermata dal patologo centrale dall'esame delle immagini digitali e dei referti patologici di campioni di biopsia renale.
    5. Solo per i soggetti con C3G, la documentazione della durata della malattia da almeno 90 giorni mediante una biopsia precedente raccolta >= 90 giorni prima dello screening a conferma di una diagnosi di C3G OPPURE una diagnosi clinica di C3G con almeno una valutazione documentata di proteinuria >= 90 giorni prima alla visita di screening iniziale.
    6. Solo per i soggetti con C3G, proteinuria definita come >= 1 g di proteine urinarie per 24 ore allo screening che non hanno evidenziato una diminuzione >= 25% dalla più recente valutazione documentata di proteinuria, che è stata raccolta >= 30 giorni prima e =< 180 giorni dalla visita di screening iniziale.
    7. Solo per i soggetti con IgAN, proteinuria definita come da 1 g a 4 g di proteine urinarie per 24 ore allo screening che non hanno evidenziato una diminuzione >= 25% dalla più recente valutazione della proteinuria documentata, che è stata raccolta >= 30 giorni prima e =< 180 giorni dalla visita di screening iniziale.
    8. Solo per i soggetti con PMN, il titolo dell'immunoglobulina G (IgG) dell’ anticorpo anti-recettore della fosfolipasi A2 (aPLA2Rab) di >= 150 U/ml e da 3,5 g a =< 11 g di proteine urinarie per 24 ore allo screening che non ha evidenziato un >= 25% di diminuzione rispetto alla più recente valutazione documentata della proteinuria, raccolta >= 30 giorni prima e =< 180 giorni della visita di screening iniziale.
    9. Un eGFR >= 50 ml/min/1,73 m2 (oppure >= 30 m/min/1,73 m 2 dopo la raccomandazione del DMC)
    10. Segni vitali in posizione supina a riposo entro i seguenti intervalli:
    • pressione arteriosa sistolica, compresa tra 80 e 150 mm Hg, per gli adulti
    • pressione arteriosa sistolica inferiore al 90º percentile, per gli adolescenti
    • pressione sanguigna diastolica =< 90 mm Hg
    11. Trattamento con un dosaggio stabile, massimo raccomandato o massimo tollerato di un inibitore dell'enzima di conversione dell'angiotensina (ACEi) o di un bloccante del recettore dell'angiotensina (ARB) per almeno 60 giorni prima del giorno1
    12. Misure contraccettive: donne in età fertile e partner femminili di pazienti maschi devono usare metodi di contraccezione altamente efficaci.
    13. Documentazione delle attuali vaccinazioni contro Neisseria meningitidis tipo A, C, W e Y e Streptococcus pneumoniae, o disponibilità a sottoporsi alla serie di vaccinazioni almeno 14 giorni prima del giorno 1.
    14. A giudizio dello sperimentatore, il soggetto è in grado di rispettare adeguatamente tutte le procedure e le limitazioni dello studio richieste per lo studio.
    E.4Principal exclusion criteria
    1. Known congenital deficiency of C1s, C1r, C1q, C2, C4.
    2. Receiving hemodialysis or peritoneal dialysis or anticipated to receive dialysis during the duration of this study.
    3. History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
    4. History of transfusion with blood or blood products, or plasmapheresis or plasma exchange, within 30 days prior to screening.
    5. 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.
    6. History of malignancy within 5 years prior to the screening visit, with the 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.
    7. Any clinical or pathological evidence of monoclonal gammopathy of unclear or renal significance, lupus or other systemic autoimmune disease, or other conditions. Presence of C3 or C5 nephritic factors, in the absence of known infection or other systemic disease, are not exclusionary for this study.
    8. Treatment with azathioprine, canakinumab, cyclophosphamide, cyclosporine, eculizumab, everolimus, hydroxychloroquine, infliximab, sirolimus, ravulizumab, systemic corticosteroids, tacrolimus, or any other systemic immunosuppressive or immunomodulatory therapies within 90 days OR within 180 days for anti-CD20 antibody therapies (eg, rituximab) prior to the screening visit.
    a. For subjects with C3G only, ongoing treatment with a stable dosing regimen of mycophenolate mofetil/mycophenolate sodium for at least 6 months prior to Day 1 Visit is allowed.
    9. Treatment with renin inhibitors (eg, aliskiren) or sodium-glucose-cotransporter 2 (SGLT2) inhibitor within 60 days prior to Day 1.
    10. 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.
    11. Any of the following at screening: Hb < 8.5 g/dL; WBC < 2.5 × 109/L; ANC < 1.0 × 109/L; platelet count < 90 × 109/L; ALT, AST, ALP or total bilirubin > 1.5 × ULN; serum albumin < 1.5 g/dL; or international normalized ratio (INR) > 1.4.
    a. Subjects with Grade 1 elevated bilirubin due to Gilbert’s syndrome are allowed to enroll
    12. Any laboratory parameter at screening that is clinically significant and would represent a safety concern.
    13. Clinically significant abnormal electrocardiogram (ECG) prior to dosing at the Day 1 Visit, including a QT interval corrected (QTcF) > 450 msec in males and QTcF > 470 msec in females, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping.
    14. Current use of a prohibited concomitant medication within 7 days prior to Day 1
    15. Active serious bacterial, viral, or fungal infection or any other serious infection within 14 days of screening
    16. Positive serology for HIV, or active infection with HBV or HCV
    17. Positive drugs of abuse screen during screening
    18. Pregnant, planning to become pregnant, or breastfeeding.
    19. Known hypersensitivity to BCX9930 or any of its formulation excipients
    20. History of severe hypersensitivity to any medicinal product
    21. Any clinically significant medical or psychiatric condition that would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject
    1. Deficit congenito noto di C1s, C1r, C1q, C2, C4.
    2. In emodialisi o dialisi peritoneale o che si prevede riceva dialisi durante il presente studio.
    3. Anamnesi di trapianto di cellule ematopoietiche o trapianto di organo solido o candidato che si prevede riceverà un trapianto durante lo studio.
    4. Anamnesi di trasfusione con sangue o prodotti sanguigni, o plasmaferesi o scambio di plasma, entro 30 giorni prima dello screening.
    5. Infarto miocardico o accidente cerebrovascolare nei 30 giorni precedenti allo 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.
    6. Anamnesi di neoplasia nei 5 anni precedenti la visita di screening, a 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 in modo curativo ritenuto dallo sperimentatore e dal monitoraggio medico a basso rischio di recidiva.
    7. Qualsiasi evidenza clinica o patologica di gammopatia monoclonale di significato non chiaro o renale, lupus o altra malattia autoimmune sistemica o altre condizioni. La presenza di fattori nefritici C3 o C5, in assenza di infezione nota o altra malattia sistemica, non è esclusiva per questo studio.
    8. Trattamento con azatioprina, canakinumab, ciclofosfamide, ciclosporina, eculizumab, everolimus, idrossiclorochina, infliximab, sirolimus, ravulizumab, corticosteroidi sistemici, tacrolimus o qualsiasi altra terapia immunosoppressiva o immunomodulante sistemica nei 90 giorni OPPURE 180 giorni per terapie anticorpali CD20 (per esempio rituximab) precedenti alla visita di screening.
    a. Per i soggetti con solo C3G, è consentito il trattamento in corso con un regime di dosaggio stabile di micofenolato mofetile/micofenolato sodico per almeno 6 mesi prima della visita del giorno 1.
    9. Trattamento con inibitori della renina (per es., Aliskiren) o inibitore del cotrasportatore sodio-glucosio 2 (SGLT2) entro 60 giorni prima del giorno 1.
    10. Attuale partecipazione 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.
    11. Uno qualsiasi dei seguenti valori di laboratorio alla visita di screening: emoglobina <8,5 g/dl; globuli bianchi totali (WBC) 2,5 × 109 l; conta assoluta dei neutrofili (ANC) 1,0 × 109/l; conta piastrinica 90 × 109/l; alanina aminotransferasi (ALT), aspartato aminotransferasi (AST), fosfatasi alcalina (ALP) o bilirubina totale 1,5 volte superiore della norma (ULN); albumina sierica 1,5 g/dl; o rapporto internazionale normalizzato (INR) 1.4.
    a. I soggetti con bilirubina di Grado 1 elevata dovuta alla sindrome di Gilbert sono autorizzati a partecipare.
    12. Qualsiasi parametro di laboratorio allo screening che, a giudizio dello sperimentatore, sia clinicamente significativo e rappresenterebbe un problema di sicurezza.
    13. Elettrocardiogramma anormale clinicamente significativo (ECG) prima della somministrazione alla visita del giorno 1
    15. Infezione attiva grave batterica, virale o fungina o qualsiasi altra infezione grave entro 14 giorni dallo screening.
    16. Sierologia positiva per HIV o infezione in corso da HBV o HCV.
    17. Screening positivo per droghe da abuso.
    18. Incinta, sta pianificando una gravidanza o sta allattando.
    19. Nota ipersensibilità al BCX9930 o a uno qualsiasi degli eccipienti della sua formulazione.
    20. Anamnesi di grave ipersensibilità a qualsiasi medicinale.
    21. Qualsiasi altra condizione medica o psichiatrica clinicamente significativa che potrebbe interferire con la capacità del soggetto di partecipare allo studio o incrementare il rischio derivante dalla partecipazione da parte di tale soggetto.
    E.5 End points
    E.5.1Primary end point(s)
    Change in 24-hour urinary protein excretion normalized to urine creatinine as measured by percentage change in uPCR from baseline
    Variazione dell'escrezione urinaria di proteine nelle 24 ore normalizzata a creatinina urinaria misurata dalla variazione percentuale del rapporto proteine-creatinina nell'urina (uPCR) rispetto al basale
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 24
    Settimana 24
    E.5.2Secondary end point(s)
    • Number and proportion of subjects with a uPCR response defined as:
    - Partial remission, = 50% reduction from baseline
    - Complete remission, = 500 mg/g
    - Normalization, = 200 mg/g
    • Change from baseline in 24-hour urinary protein excretion as measured by percentage change in urinary protein from baseline
    • Change from baseline in estimated glomerular filtration rate (eGFR)
    • Change from baseline in serum albumin
    • Number and proportion of subjects with the following parameters:
    - Protein = 3.5 g in a 24-hour urine collection
    - Serum albumin = 2.5 g/dL
    • Number and proportion of subjects with a morphologic response in each of the following categories assessed using a 0-4 scale:
    - Decreased endocapillary hypercellularity, mesangial hypercellularity, active crescents (if present)
    - Decreased acute tubular injury, interstitial inflammation, interstitial edema
    - Reduction in C3 and/or C4d glomerular staining
    - Reduction in the extent of deposits, clearing of deposits, or no additional active deposits as assessed by electron microscopy (EM)
    - No progression of chronic changes (ie, global, segmental glomerulosclerosis, tubular atrophy/interstitial fibrosis)
    • Change from baseline in constitutive blood levels of complement biomarkers, including but not limited to C3, Factor Bb, and soluble C5b-9 (sC5b-9)
    • Change from baseline in single void urine levels of complement biomarkers, including but not limited to Ba, C3a, and sC5b-9 normalized to urine creatinine
    • Change from baseline in complement biomarker measurements of ex vivo stimulation assays
    • Number and proportion of subjects with a treatment-emergent adverse event (TEAE)
    • Number and proportion of subjects who discontinue due to a TEAE
    • Number and proportion of subjects who experience a treatment-emergent serious adverse event (TESAE)
    • Number and proportion of subjects who experience a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE
    • Number and proportion of subjects who experience a CTCAE treatment-emergent Grade 3 or 4 laboratory abnormality
    • Numero e proporzione di soggetti con una risposta uPCR definita come:
    - remissione parziale, riduzione = 50% rispetto al basale
    - remissione completa, = 500 mg / g
    - normalizzazione, = 200 mg / g
    • Variazione rispetto al basale nell'escrezione urinaria di proteine nelle 24 ore misurata come variazione percentuale delle proteine urinarie rispetto al basale
    • Variazione rispetto al basale della velocità di filtrazione glomerulare stimata (eGFR)
    • Variazione rispetto al basale dell'albumina sierica
    • Numero e proporzione di soggetti con i seguenti parametri:
    - proteine = 3,5 g in una raccolta di urine nelle 24 ore
    - albumina sierica = 2,5 g / dL
    • Numero e proporzione di soggetti con una risposta morfologica in ciascuna delle seguenti categorie valutate utilizzando una scala da 0 a 4:
    - ridotta ipercellularità endocapillare, ipercellularità mesangiale, semilune attive (se presenti)
    - ridotta lesione tubulare acuta, infiammazione interstiziale, edema interstiziale
    - riduzione della colorazione glomerulare C3 e/o C4d
    - riduzione dell'entità dei depositi, eliminazione dei depositi o assenza di depositi attivi aggiuntivi come valutato mediante microscopia elettronica (EM)
    - assenza di progressione dei cambiamenti cronici (vale a dire, glomerulosclerosi segmentale, globale, atrofia tubulare/fibrosi interstiziale)
    • Variazione rispetto al basale dei livelli ematici costitutivi dei biomarcatori del complemento, inclusi ma non limitati a C3, Fattore Bb e C5b-9 solubile (sC5b-9)
    • Variazione rispetto al basale nell’urina a singola minzione dei livelli dei biomarcatori del complemento, inclusi ma non limitati al Fattore Ba, C3a e sC5b-9 normalizzati a creatinina urinaria
    • Variazione rispetto al basale nelle misurazioni dei biomarcatori del complemento degli esami di stimolazione ex vivo
    • Numero e proporzione di soggetti che presentano un evento avverso emergente da trattamento (TEAE)
    • Numero e proporzione di soggetti che interrompono lo studio a causa di un TEAE
    • Numero e proporzione di soggetti che registrano un evento avverso grave emergente da trattamento (TESAE)
    • Numero e proporzione di soggetti che presentano un TEAE di grado 3 o 4 come defiito da Common Terminology Criteria for Adverse Events (CTCAE)
    • Numero e proporzione di soggetti che registrano un'anormalità dei valori di laboratorio emergente da trattamento CTCAE di grado 3 o 4
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    Settimana 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 Yes
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA 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
    France
    Germany
    Italy
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    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 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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 42
    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)
    It is the sponsors intention to open an access protocol for subjects receiving clinical benefit from BCX9930 treatment before the end of the Week 24 visit
    é intenzione del Promotore attivare un protocollo di accesso per i soggetti che derivano benefici clinici dal trattamento prima della fine della visita della settimana 24
    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 Opinion2021-09-15
    P. End of Trial
    P.End of Trial StatusOngoing
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