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    Summary
    EudraCT Number:2020-004403-14
    Sponsor's Protocol Code Number:BCX9930-203
    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:2021-07-14
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2020-004403-14
    A.3Full title of the trial
    A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria
    Un estudio randomizado, doble ciego, multicéntrico, con un grupo control con placebo, en grupos paralelos para evaluar la eficacia, seguridad y tolerabilidad del fármaco oral BCX9930 para el tratamiento de la Hemoglobinuria Paroxística Nocturna
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BCX9930 compared to placebo in the treatment of PNH
    BCX9930 en comparación con placebo para el tratamiento de la HPN
    A.3.2Name or abbreviated title of the trial where available
    REDEEM 2
    A.4.1Sponsor's protocol code numberBCX9930-203
    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/ cityLondon
    B.5.3.3Post codeW67BA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44208834 1144
    B.5.5Fax number+44208834 1156
    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
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal Nocturnal Hemoglobinuria
    Hemoglobinuria Paroxística Nocturna
    E.1.1.1Medical condition in easily understood language
    PNH
    HPN
    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 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 12 weeks, as compared to placebo, in subjects with paroxysmal nocturnal hemoglobinuria (PNH)
    • To evaluate the long-term safety and tolerability of oral BCX9930 monotherapy administered for up to 52 weeks in subjects with PNH
    • Determinar la eficacia de la monoterapia oral de BCX9930 administrada durante 12 semanas, en comparación con placebo, en sujetos con hemoglobinuria paroxística nocturna (HPN)
    • Evaluar la seguridad y tolerabilidad a largo plazo de la monoterapia oral BCX9930 administrada hasta 52 semanas en sujetos con HPN
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, in subjects with PNH
    • To characterize the effects of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, using clinical and laboratory measurements, including complement and thrombosis biomarkers, and PNH clone size
    • To evaluate the effects of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, on FACIT-Fatigue scale and other PROs
    • To assess the effectiveness of BCX9930 monotherapy administered for up to 52 weeks
    • To characterize the effects of BCX9930 monotherapy administered for up to 52 weeks using clinical and laboratory measurements, including complement and thrombosis biomarkers, and PNH clone size
    • To evaluate the effects of BCX9930 monotherapy administered for up to 52 weeks on FACIT-Fatigue scale and other PROs
    • To characterize BCX9930 plasma concentrations and pharmacokinetic (PK) parameters
    • Evaluar la seguridad y tolerabilidad de BCX9930 en monoterapia administrada 12 semanas, en comparación con placebo
    • Caracterizar los efectos de la monoterapia de BCX9930 administrada 12 semanas, en comparación con placebo, utilizando mediciones clínicas y de laboratorio, y el tamaño de la clona de HPN
    • Evaluar los efectos de la monoterapia con BCX9930 administrada 12 semanas, en comparación con placebo, en la escala de FACIT y otros PRO
    • Evaluar la efectividad de la monoterapia con BCX9930 administrada de 52 semanas
    • Caracterizar los efectos de la monoterapia BCX9930 administrada de 52 semanas, utilizando mediciones clínicas y de laboratorio, incluidos los biomarcadores de complemento y trombosis, y el tamaño de la clona de HPN
    • Evaluar los efectos de la monoterapia BCX9930 administrada durante un plazo máximo de 52 semanas en la escala FACIT-F y otros PRO
    • Caracterizar las concentraciones plasmáticas de BCX9930 y los parámetros PK a lo largo del tiempo
    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 substudy - within the same protocol V2, 27 May 2021
    PK / PD meta-estudio - protocolo, V2 dated 27 Mayo 2021
    E.3Principal inclusion criteria
    1. Male or female, aged ≥ 18 years old.
    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. Are either: (a) naïve to treatment with a complement inhibitor, or (b) have received no treatment with a complement inhibitor for at least 12 months prior to the screening visit.
    5. Recorded the following results during screening:
    a. Hb ≤ 105 g/L (≤ 10.5 g/dL).
    b. LDH ≥ 2 × upper limit of normal reference range (ULN)
    c. ARC of ≥ 100 × 109 cells/L (≥ 100,000 cells/µL; ≥ 100 G/L).
    d. Absolute neutrophil count (ANC) of ≥ 0.75 × 109 cells/L (≥ 750 cells/µL; ≥ 0.75 G/L).
    e. Platelet count of ≥ 30 × 109/L (≥ 30,000/µL; ≥ 30 G/L).
    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:
    Female participants must meet at least one of the following requirements:
    a. Be a woman of nonchildbearing potential.
    b. Be a woman of childbearing potential who agrees to use a highly effective contraceptive method throughout the study and for a duration of 30 days after the last dose of study drug.
    c. Alternatively, true abstinence is acceptable for women of childbearing potential when it is in line with the subject’s preferred and usual lifestyle.
    Male participants must meet at least one of the following requirements:
    a. Males with a female partner of childbearing potential must use condoms throughout the study and for a duration of 90 days after the dose of study drug unless their partner is using a highly effective contraceptive method independent of the study.
    b. Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle
    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.
    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
    Hombre o mujer, ≥ 18 años.
    2. Peso ≥ 40 kg.
    3. Diagnóstico documentado de HPN confirmado por citometría de flujo con un tamaño de la clona de granulocitos o monocitos de HPN ≥ 10 % durante la selección.
    4. Son: (a) sujetos que nunca han recibido tratamiento con un inhibidor del complemento, o (b) sujetos que no han recibido tratamiento con un inhibidor del complemento durante al menos 12 meses antes de la visita de selección.
    5. Se registraron los siguientes resultados durante la selección:
    a. Hb < 105 g/L (< 10,5 g/dL) b. LDH ≥ 2 × límite superior de normalidad (LSN)
    c. RAR de ≥ 100 × 109 células/L (≥ 100.000 células/µL; ≥ 100 G/L).
    d. Recuento absoluto de neutrófilos (RAN) de (≥ 0,75 × 109 células / L (≥ 750 células / µL; ≥ 0,75 G/L).
    e. Recuento de plaquetas ≥ 30 ×109/L (≥ 30 000/µL; ≥ 30 G/L).
    f. Tasa de filtración glomerular estimada de ≥ 50 mL/min/1,73 m2 utilizando la ecuación de la Colaboración en Epidemiología de la Enfermedad Renal Crónica (CKD-EPI)
    6. Requisitos de anticoncepción:
    Las mujeres participantes deben cumplir al menos uno de los siguientes requisitos:
    a. Ser una mujer en edad no fértil.
    b. Ser una mujer en edad fértil que acepte utilizar un método anticonceptivo altamente efectivo durante todo el estudio y durante 30 días después de la última dosis del fármaco objeto del estudio.
    c. Como alternativa, la verdadera abstinencia es aceptable para las mujeres en edad fértil, siempre que esto pueda llevarse a cabo, teniendo en cuenta el estilo de vida preferido y habitual de la paciente.
    Los hombres que participen deben cumplir al menos uno de los siguientes requisitos de anticoncepción:
    a. Los hombres con una pareja femenina en edad fértil deben usar preservativos durante todo el ensayo clínico y durante un período de 90 días después de la dosis del fármaco del estudio, a menos que su pareja esté usando un método anticonceptivo altamente eficaz independiente del ensayo clínico.
    b. Como alternativa, la verdadera abstinencia es aceptable, siempre que esto pueda llevarse a cabo, teniendo en cuenta el estilo de vida preferido y habitual del paciente.
    Se proporcionan detalles adicionales en la Sección 11.2.1.
    7. Documentación de las vacunas actuales contra Neisseria meningitidis tipos A, C, W, y Y y Streptococcus pneumoniae o voluntad de comenzar la serie de vacunación al menos 14 días antes del Día 1.
    (Nota: la vacunación contra N.meningitidis tipo B y H.influenzae tipo B (Hib) se recomienda encarecidamente donde esté autorizado y disponible)
    8. En opinión del investigador, se espera que el sujeto cumpla adecuadamente con todos los procedimientos requeridos y las restricciones para el ensayo clínico, incluido el cumplimiento del programa de dosificación BID para BCX9930.
    9. Estar dispuesto y ser capaz de proporcionar un consentimiento informado por escrito.
    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.
    (Note: Suspected or confirmed coronavirus disease [COVID-19]; persistent or recurrent positive test(s) for severe acute respiratory syndrome coronavirus 2 [SARS CoV 2] nucleic acids or antigens; and worsening of dyspnea not due to PNH, vasculitic rash, and persistent fever or other symptoms consistent with multisystem inflammatory syndrome in adults [MIS A] are exclusionary.)
    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 with an unstable dose in the 28 days prior to the screening visit.
    10. Clinically significant abnormal electrocardiogram (ECG) at the screening visit.
    (Note: This includes, but is not limited to, a QT interval corrected using Fridericia’s method [QTcF] of > 450 msec in males or > 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.)
    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. AST (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
    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. Antecedentes conocidos o diagnóstico existente de deficiencia hereditaria del complemento.
    2. Antecedentes de trasplante de células hematopoyéticas o trasplante de órgano sólido o candidato anticipado para trasplante durante el ensayo clínico.
    3. 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.
    4. 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 u otro tumor sólido tratado curativamente que el investigador y el monitor médico consideren con bajo riesgo de recurrencia.
    5. Infección bacteriana, viral o fúngica activa o cualquier otra infección grave dentro de los 14 días previos a la selección.
    (Nota: La infección por coronavirus sospechada o confirmada [COVID-19]; pruebas positivas persistentes o recurrentes de síndrome respiratorio agudo coronavirus 2 [SARS-CoV-2] ácido nucleico o antígenos; y empeoramiento de la disnea que no se deba a HPN, erupción vasculítica y fiebre persistente u otros síntomas compatibles con el síndrome inflamatorio multisistémico en adultos [MIS-A] son excluyentes.
    6. 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.
    7. Tratamiento con globulina antitimocítica dentro de los 180 días previos a la visita de selección.
    8. Inicio del tratamiento con un agente estimulante de la eritropoyesis (p. ej., eritropoyetina), agonistas del receptor de trombopoyetina (p. ej., eltrombopag) o danazol dentro de los 28 días anteriores a la visita de selección.
    (Nota: el tratamiento con estos medicamentos iniciado en un período > 28 días antes de la visita de selección no es excluyente, si la dosis es estable y existe una expectativa razonable de que el tratamiento continuará).
    9. Recibir suplementos de hierro con una dosis inestable en los 28 días anteriores a la visita de selección.
    10. Electrocardiograma (ECG) anormal clínicamente significativo en la visita de selección.
    (Nota: Esto incluye, entre otros, un intervalo QT corregido con el método de Fridericia [QTcF] de > 450 mseg en hombres o> 470 mseg en mujeres, o contracciones prematuras ventriculares y/o auriculares que son más frecuentes que ocasionales, y/o con una agrupación en duplas o superior.)
    11. Sujetos con alguno de los siguientes resultados en la visita de selección:
    a. Alanina aminotransferasa (ALT, también transaminasa glutámico-pirúvica sérica [SGPT] > 3 × LSN.
    b. AST (SGOT) > 3 × LSN
    (Nota: los sujetos pueden incluirse con AST > 3 × LSN si esto fuera causado por hemólisis).
    c. Bilirrubina sérica total > 2 × LSN
    (Nota: Los sujetos pueden incluirse con bilirrubina sérica total > 2 × LSN si fuera por hemólisis o síndrome de Gilbert. En el caso de hemólisis, la bilirrubina sérica total debe ser < 5 × LSN y en el caso del síndrome de Gilbert, la bilirrubina sérica total debe ser < 11 × LSN).
    12. Uso actual de un medicamento concomitante prohibido en los 7 días anteriores al Día 1 tal y como se detalla en la Sección 9.8.1.
    13. Serología positiva para el virus de inmunodeficiencia humana o infección activa con el virus de la hepatitis B o el virus de la hepatitis C , a menos que se reciba terapia antiviral y la carga viral sea indetectable.
    14. Detección positiva de estupefacientes, exceptuando aquellos con receta.
    15.Mujer embarazada, que planea quedar embarazada o amamantando.
    16. Hipersensibilidad conocida a BCX9930 o cualquiera de sus excipientes de formulación.
    17. Antecedentes de hipersensibilidad grave a cualquier medicamento, que se asoció con
    hinchazón, erupción cutánea grave que requiera tratamiento/hospitalización o anafilaxia.
    18. Cualquier otra condición médica o psiquiátrica clínicamente significativa que, en opinión del
    investigador o promotor pueda interferir con la capacidad del sujeto para participar en el ensayo, o
    aumentar el riesgo de participación de ese sujeto.
    E.5 End points
    E.5.1Primary end point(s)
    Part1
    • Change from baseline (CFB) in hemoglobin (Hb)
    Part2
    • 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 treatment-emergent CTCAE Grade 3 or Grade 4 laboratory abnormality
    Parte 1
    • Cambio desde el valor basal (CFB, por sus siglas en inglés) en hemoglobina (Hb)
    Parte 2
    • Número y proporción de sujetos con AAAT
    • Número y proporción de sujetos que abandonan debido a un AAAT
    • Número y proporción de sujetos que experimentan un AAGAT
    • Número y proporción de sujetos que experimentan un AAAT de grado 3 o un AAGAT de grado 4
    • Número y proporción de sujetos que experimentan una anomalía de laboratorio de grado 3 o grado 4 de AAGAT surgidos del tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part 1 - Week 12
    Part 2 - week 52
    Parte 1 - semana 12
    Parte 2 - semana 52
    E.5.2Secondary end point(s)
    Part 1
    1. Proportion of subjects who are transfusion-free
    2. Number of units of packed red blood cells (pRBCs) transfused
    3. Percent CFB in lactate dehydrogenase (LDH)
    4. CFB in FACIT-Fatigue scale score
    Part 2
    • CFB in Hb
    • Proportion of subjects with Hb ≥ 12 g/dL
    • Proportion of subjects achieving Hb stabilization (avoidance of a > 2 g/dL decrease in the absence of transfusion)
    • Proportion of subjects who are transfusion-free
    • Percent reduction in rate of pRBCs transfused
    • Percent CFB in LDH
    • CFB in ARC
    • Proportion of subjects with ARC in the normal range
    • CFB in haptoglobin
    • Proportion of subjects with haptoglobin ≥ LLN
    • CFB in total PNH RBC clone size
    • CFB in total PNH RBC clone size relative to PNH WBC clone size
    Parte 1
    • Proporción de sujetos que no reciben transfusión [desde el Día 14 a la Semana 12]
    • Número de unidades de concentrado de glóbulos rojos (pRBC) transfundidas [ desde el día 14 hasta la semana 12]
    • Porcentaje de CFB en lactato deshidrogenasa (LDH) [a la Semana 12]
    • CFB en la puntuación de la escala FACIT-F
    Parte 2
    • CFB en Hb
    • Proporción de sujetos con una Hb > 12 g/dL
    • Proporción de sujetos que alcanzaron una estabilización de la Hb
    • Proporción de sujetos que no recibieron transfusión
    • Porcentaje de reducción en la tasa de pRBC transfundidos
    • Porcentaje de CFB en LDH
    • CFB en ARC
    • Proporción de sujetos con ARC dentro de rango normal
    • CFB en haptoglobina
    • Proporción de sujetos con haptoglobina ≥ LLN
    • CFB en total de la medida de la clona en RBC en HPN
    • CFB en el tamaño total de la clona de RBC en HPN en relación con el tamaño de la clona de WBC en HPN
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1 - Week 12
    Part 2 - week 52
    Parte 1 - semana 12
    Parte 2 - semana 52
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Albania
    Azerbaijan
    Brazil
    Canada
    China
    Serbia
    South Africa
    Turkey
    United States
    European Union
    Argentina
    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
    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: 52
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 57
    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
    Los sujetos que continúen obteniendo beneficios clínicos podrán continuar el tratamiento con BCX9930 mediante la inscripción en el estudio de transferencia 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 Decision2021-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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