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    Summary
    EudraCT Number:2020-005855-19
    Sponsor's Protocol Code Number:BCX9930-211
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-08-05
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    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
    Estudio abierto, de seguridad, tolerabilidad, y prueba de concepto con la terapia oral BCX9930 en sujetos con glomerulopatía C3, nefropatía por inmunoglobulina A, o nefropatía 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
    A.3.2Name or abbreviated title of the trial where available
    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 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 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.3.8 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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product 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
    glomerulopatía del complemento 3 (C3G)
    nefropatía por inmunoglobulina A (IgAN)
    nefropatía membranosa primaria (PMN)
    E.1.1.1Medical condition in easily understood language
    C3 G
    IgA nephropathy
    primary membranous nephropathy
    glomerulopatía del complemento 3 (C3G)
    nefropatía por inmunoglobulina A (IgAN)
    nefropatía membranosa primaria (PMN)
    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
    • Evaluar el potencial terapéutico de BCX9930, analizado mediante la medición de 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
    Evaluar la seguridad y tolerabilidad de BCX9930
    Evaluar el potencial terapéutico de BCX9930 según la evaluación de otras medidas de beneficio clínico
    Evaluar los efectos de BCX9930 sobre los hallazgos morfológicos microscópicos ópticos, de inmunofluorescencia y ultraestructurales
    Caracterizar los efectos de BCX9930 sobre biomarcadores en sangre y orina de activación y consumo del complemento
    Evaluar la correlación de los cambios asociados con BCX9930 en los biomarcadores en sangre y orina de la activación y el consumo del complemento con los cambios en la 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
    PK / PD sub-study - within the main protocol
    farmacocinéticos (PK)/ farmacodinámicos(PD)
    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 collected ≥ 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. Aceptar y ser capaz de proporcionar un consentimiento informado por escrito (o, para los sujetos que no han alcanzado la mayoría de edad, aceptar y ser capaz de proporcionar un asentimiento por escrito con el consentimiento informado por escrito proporcionado adicionalmente por los padres o el tutor legal del sujeto).
    2. Sujetos varones o mujeres no embarazadas, no lactantes ≥ 18 años de edad.
    • Solo para la cohorte C3G (cuando esté permitido), sujetos varones o mujeres no embarazadas y no lactantes de 12 a < 18 años de edad.
    3. Peso ≥ 40 kg.
    4. Diagnóstico primario de C3G, IgAN o PMN confirmado por revisión patológica central de imágenes digitales e informes de patología de muestras de biopsia renal
    5. Solo para sujetos con C3G, documentación de la duración de la enfermedad de al menos 90 días mediante una biopsia previa obtenida ≥ 90 días antes del cribado que confirme un diagnóstico de C3G o un diagnóstico clínico de C3G con al menos una evaluación de proteinuria documentada ≥ 90 días antes de la visita de selección inicial.
    6. Solo para sujetos con C3G, proteinuria definida como ≥ 1 g de proteína urinaria por 24 horas en el cribado que no ha mostrado una disminución ≥ 25 % con respecto a la evaluación de proteinuria documentada más reciente, que se obtuvo ≥ 30 días antes y ≤ 180 días de visita de selección inicial.
    7. Solo para sujetos con IgAN, proteinuria definida como 1 g ≤ 4 g de proteína urinaria por 24 horas en el cribado que no ha mostrado una disminución ≥ 25 % de la evaluación de proteinuria documentada más reciente, que fue recolectada ≥ 30 días antes y ≤ 180 días de la visita de selección inicial.
    8. Solo para sujetos con PMN, un título de inmunoglobulina G (IgG) de anticuerpo anti-receptor de fosfolipasa A2 (aPLA2Rab) de ≥ 150 U/mL y de 3,5 g ≤ 11 g de proteína urinaria de 24 horas en el cribado que no ha mostrado un ≥ 25 % de disminución de la evaluación de proteinuria documentada más reciente, que se recopiló ≥ 30 días antes y ≤ 180 días de la visita de selección inicial.
    9. Una TFGe de ≥ 50 ml/min/1,73 m2
    10. Signos vitales en decúbito supino dentro de los siguientes rangos:
    • Presión arterial sistólica, 80 a 150 mm Hg inclusive, para adultos
    • Presión arterial sistólica por debajo del percentil 90, para adolescentes por sección 12.10.1
    • Presión arterial diastólica ≤ 90 mm Hg
    11. Tratamiento con una dosis estable, máxima recomendada o máxima tolerada de un inhibidor de la enzima convertidora de angiotensina (IECA) o bloqueador del receptor de angiotensina (BRA) durante al menos 60 días antes de la visita del día 1 y, en opinión del investigador, la expectativa de continuar la misma dosis y régimen para dicho tratamiento durante el estudio.
    12. Requisitos de anticoncepción: WOCBP acepta usar un método anticonceptivo altamente eficaz
    13. Documentación de vacunación actual contra Neisseria meningitidis Tipos A, C, W y Y, y contra Streptococcus pneumoniae, o estar dispuesto a comenzar la serie de vacunación al menos 14 días antes del Día 1
    14. En opinión del investigador, se espera que el sujeto cumpla adecuadamente con todos los procedimientos requeridos y las restricciones para el ensayo clínico
    E.4Principal exclusion criteria
    1. Known congenital deficiency of C1s, C1r, C1q, C2, C4. Known variants in complement factor H, complement factor I, C3, and complement factor B or genomic rearrangements in the complement factor-H-related proteins are not exclusionary.
    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 (eg, infection-associated disease or associated with another systemic disease, anti-phospholipid antibody syndrome with significant clinical disease, immune complex glomerulonephritis, immunoglobulin A [IgA] vasculitis with nephritis [Henoch-Schönlein purpura] or morphologic features of secondary membranous nephropathy). Presence of C3 or C5 nephritic factors (eg, autoantibodies directed at C3 or C5 convertase), 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. Deficiencia congénita conocida de C1s, C1r, C1q, C2, C4. Las variantes conocidas en el factor de complemento H, el factor de complemento I, C3 y el factor de complemento B o los reordenamientos genómicos en las proteínas relacionadas con el factor de complemento no son excluyentes.
    2. Estar recibiendo hemodiálisis o diálisis peritoneal o tener previsto recibir diálisis durante la duración de este estudio.
    3. Antecedentes de trasplante de células hematopoyéticas o trasplante de órgano sólido o candidato anticipado para trasplante durante el ensayo clínico.
    4. Antecedentes de transfusión con sangre o productos sanguíneos, o plasmaféresis o recambio plasmático, dentro de los 30 días anteriores a la selección.
    5. Infarto de miocardio o accidente cerebrovascular en los 30 días anteriores a la selección, o afección cardiovascular o cerebrovascular clínicamente significativa actual y no controlada, incluida angina inestable, insuficiencia cardíaca congestiva grave, síncope inexplicable, arritmia y estenosis aórtica crítica.
    6. Antecedentes de malignidad en los 5 años anteriores a la visita de selección, con excepción de cáncer de vejiga superficial o de piel no melanoma tratado adecuadamente, carcinoma in situ del cuello uterino tratado curativamente u otro tumor sólido tratado curativamente que el investigador y el monitor médico consideren con bajo riesgo de recurrencia.
    7. Cualquier evidencia clínica o patológica de gammapatía monoclonal de importancia renal o poco clara, lupus u otra enfermedad autoinmune sistémica, u otras afecciones (p. Ej., Enfermedad asociada a infección o asociada con otra enfermedad sistémica, síndrome de anticuerpos antifosfolípidos con enfermedad clínica significativa, complejo inmune glomerulonefritis, vasculitis por inmunoglobulina A [IgA] con nefritis [púrpura de Henoch-Schönlein] o características morfológicas de nefropatía membranosa secundaria). La presencia de factores nefríticos C3 o C5 (p. ej., autoanticuerpos dirigidos a la convertasa C3 o C5), en ausencia de una infección conocida u otra enfermedad sistémica, no son excluyentes para este estudio.
    8. Tratamiento con azatioprina, canakinumab, ciclofosfamida, ciclosporina, eculizumab, everolimus, hidroxicloroquina, infliximab, sirolimus, ravulizumab, corticosteroides sistémicos, tacrolimus o cualquier otra terapia inmunosupresora o inmunomoduladora sistémica en un plazo de 90 días o en el plazo de 180 días en el caso de las terapias con anticuerpos anti-CD20, (por ejemplo, rituximab) antes de la visita de selección.
    a. Solo para sujetos con C3G, se permite el tratamiento continuo con un régimen posológico estable de micofenolato de mofetilo/micofenolato de sodio durante al menos 6 meses antes de la visita del día 1.
    9. Tratamiento con inhibidores de renina (p. ej., aliskiren) o inhibidor del cotransportador de sodio-glucosa 2 (SGLT2) en los 60 días anteriores al día 1.
    10. Participación en ese momento en cualquier otro ensayo de fármaco en investigación o participación en un ensayo de un fármaco en investigación en los 30 días anteriores a la visita de selección, o 5,5 semividas del fármaco que está siendo investigado, según el período que sea más largo.
    11. Cualquiera de los siguientes valores de laboratorio en la visita de selección: Hb < 8,5 g/dL; GB < 2,5 ×109/L; RAN < 1,0 ×109/L; recuento de plaquetas < 90 ×109/L; ALT, AST, ALP o bilirrubina total > 1,5 X LSN; albúmina sérica < 1,5 g/dL; o razón internacional normalizada (INR) > 1,4.
    a. Los sujetos con bilirrubina elevada de Grado 1 debido al síndrome de Gilbert podrán ser seleccionados.
    12. Cualquier parámetro de laboratorio en la selección que, a juicio del investigador, sea clínicamente significativo y represente un problema de seguridad.
    13. ECG anormal clínicamente significativo antes de la dosificación en la visita del día 1, que incluye, entre otros, un intervalo QTcF > 450 mseg en hombres y QTcF > 470 mseg en mujeres, o ventricular y / o contracciones auriculares prematuras que son más frecuentes que ocasionales, y / o como pares o más en agrupamiento.
    14. El uso actual de un medicamento concomitante prohibido dentro de los 7 días anteriores al Día 1
    15. Infección bacteriana, viral o fúngica grave activa o cualquier otra infección grave, incluida la infección por coronavirus [SARS-CoV-2]
    16. Serología positiva para el virus de VIH o infección activa con VHB o el VHC.
    17. Detección positiva de estupefacientes durante la selección, exceptuando aquellos con receta.
    18. Mujer embarazada, que planea quedar embarazada o amamantando.
    19. Hipersensibilidad conocida a BCX9930 o cualquiera de sus excipientes de formulación
    20. Antecedentes de hipersensibilidad grave a cualquier medicamento
    21. Cualquier afección médica o psiquiátrica, clínicamente significativa, pueda interferir con la capacidad del sujeto para participar en el ensayo, o aumentar el riesgo de participación para este sujeto.
    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
    Cambio en la excreción de proteínas en orina de 24 horas normalizada a creatinina en orina medida por el cambio porcentual en la relación proteína-creatinina en orina (uPCR) desde el inicio
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 24
    24 semanas
    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
    • Número y proporción de sujetos con una respuesta de uPCR definida como:
    - Remisión parcial, reducción ≥ 50 % desde el valor inicial
    - Remisión completa, ≤ 500 mg/g
    - Normalización, ≤ 200 mg/g
    • Cambio respecto al valor inicial en la excreción de proteínas urinarias de 24 horas, medido por el cambio porcentual en la proteína urinaria desde el valor inicial
    • Cambio respecto al valor inicial en la tasa de filtración glomerular estimada (TFGe)
    • Cambio respecto al valor inicial en la albúmina sérica
    • Número y proporción de sujetos con los siguientes parámetros:
    - Proteína ≥ 3,5 g en una toma de muestras de orina de 24 horas
    - Albúmina sérica ≤ 2,5 g/dL
    • Número y proporción de sujetos con una respuesta morfológica en cada una de las siguientes categorías evaluados mediante una escala de 0 a 4:
    - Disminución de la hipercelularidad endocapilar, hipercelularidad mesangial, semilunas activas (si están presentes)
    - Disminución de la lesión tubular aguda, inflamación intersticial, edema intersticial
    - Reducción de la tinción glomerular C3 y/o C4d
    - Reducción de la extensión de los depósitos, limpieza de los depósitos o ausencia de depósitos activos adicionales según lo evaluado por microscopía electrónica (EM)
    - Sin progresión de cambios crónicos (es decir, glomeruloesclerosis segmentaria global, atrofia tubular/fibrosis intersticial)
    • Cambio con respecto al valor inicial en los niveles sanguíneos constitutivos de biomarcadores del complemento, incluidos, entre otros, C3, factor Bb y C5b-9 soluble (sC5b-9)
    • Cambio con respecto al valor inicial en los niveles de orina de una sola micción de biomarcadores del complemento, incluidos, entre otros, Ba, C3a y sC5b-9 normalizados a creatinina en orina
    • Cambio con respecto al valor inicial en las mediciones de biomarcadores del complemento de los ensayos de estimulación ex vivo
    • Número y proporción de sujetos con un acontecimiento adverso aparecido durante el tratamiento (AAAT)
    • Número y proporción de sujetos que abandonan debido a un AAAT
    • Número y proporción de sujetos que experimentan un acontecimiento adverso grave aparecido durante el tratamiento (AAGAT)
    • Número y proporción de sujetos que experimentan un AAAT de grado 3 o 4 evaluado utilizando el Criterio de terminología común para acontecimientos adversos (CTCAA)
    • Número y proporción de sujetos que experimentan una anomalía de laboratorio según el CTCAA de grado 3 o grado 4 aparecida durante el tratamiento
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    24 semanas
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 No
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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
    Lo promotor tienen la intención de abrir un protocolo de acceso para los sujetos que reciben beneficios clínicos del tratamiento BCX9930 antes del final de la visita de la semana 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 Decision2021-10-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-17
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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