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    Summary
    EudraCT Number:2020-004438-39
    Sponsor's Protocol Code Number:BCX9930-202
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2021-07-23
    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 number2020-004438-39
    A.3Full title of the trial
    A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy
    Un estudio randomizado, abierto, multicéntrico, en grupos paralelos para evaluar la eficacia, seguridad y tolerabilidad del fármaco oral BCX9930 en monoterapia para el tratamiento de la Hemoglobinuria Paroxística Nocturna en sujetos con una respuesta inadecuada a la terapia inhibidora de C5
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BCX9930 treatment in patients with an inadequate response to C5 inhibitors
    BCX9930 tratamiento en sujetos con una respuesta inadecuada a la terapia inhibidora de C5
    A.3.2Name or abbreviated title of the trial where available
    REDEEM 1
    REDEEM 1
    A.4.1Sponsor's protocol code numberBCX9930-202
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBioCryst Pharmaceuticals Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBioCryst Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMS Advanced Medical Services
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Address26 -28 Hammersmith Grove, 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 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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Solaris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/03/166
    D.3 Description of the IMP
    D.3.1Product nameeculizumab
    D.3.2Product code eculizumab
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEculizumab
    D.3.9.1CAS number 219685-50-4
    D.3.9.2Current sponsor codeSoliris
    D.3.9.3Other descriptive nameSoliris
    D.3.9.4EV Substance CodeSUB25187
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product 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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Ultomiris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/16/1661
    D.3 Description of the IMP
    D.3.1Product nameRavulizumab
    D.3.2Product code ravulizumab
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRAVULIZUMAB
    D.3.9.2Current sponsor codeUltomiris
    D.3.9.4EV Substance CodeSUB192773
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product 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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal Nocturnal Hemoglobinuria
    Hemoglobinuria Paroxística Nocturna
    E.1.1.1Medical condition in easily understood language
    PNH
    HPN
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10034042
    E.1.2Term Paroxysmal nocturnal haemoglobinuria
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to C5 inhibitor therapy
    • To evaluate the long-term safety and tolerability of oral BCX9930 monotherapy administered over a 28- to 52 week treatment period in subjects with PNH with an inadequate response to C5 inhibitor therapy
    • Determinar la eficacia de la monoterapia oral BCX9930 administrada durante 24 semanas, en comparación con la terapia continua con inhibidores del componente 5 (C5) del complemento, en sujetos con hemoglobinuria paroxística nocturna (HPN) con una respuesta inadecuada a la terapia con inhibidores de C5
    • Evaluar la seguridad y tolerabilidad a largo plazo de la monoterapia oral BCX9930 administrada durante un período de tratamiento de 28 a 52 semanas en sujetos con HPN con una respuesta inadecuada a la terapia con inhibidor de C5
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of BCX9930 monotherapy administered for 24 weeks, as compared to continued C5 inhibitor therapy
    • To characterize the effects of BCX9930 monotherapy, as compared to continued C5 inhibitor therapy, using clinical and laboratory measurements, including complement and thrombosis biomarkers, PNH clone size, and C3-opsonization of red blood cells
    • To evaluate the effects of BCX9930 monotherapy, as compared to continued C5 inhibitor therapy, on the FACIT-Fatigue scale and other patient-reported outcomes (PROs)
    • To assess the effectiveness of BCX9930 monotherapy in the treatment of PNH
    • To characterize the effects of BCX9930 monotherapy using clinical and laboratory measurements, PNH clone size, and C3 opsonization of RBCs
    • To evaluate the effects of BCX9930 monotherapy on FACIT-Fatigue scale and other PROs
    • To characterize BCX9930 PK parameters
    Evaluar la seguridad y tolerabilidad de BCX9930 en monoterapia administrada durante 24 semanas, en comparación con la terapia continua con inhibidores de C5
    •Caracterizar los efectos de la monoterapia con BCX9930 en comparación con la terapia continua con inhibidor de C5, utilizando mediciones clínicas y de laboratorio, incluidos los biomarcadores del complemento y la trombosis, el tamaño del clon de HPN y la opsonización C3 de los RGR
    •Evaluar los efectos de la monoterapia con BCX9930, en comparación con la terapia continua con inhibidores de C5, en la escala FACIT y otros resultados comunicados por el paciente (PROs)
    • Evaluar la efectividad de la monoterapia BCX9930 en el tratamiento de la HPN
    • Caracterizar los efectos de la monoterapia BCX9930 utilizando mediciones clínicas y de laboratorio, el tamaño del clon de HPN y la opsonización C3
    •Evaluar los efectos de la monoterapia BCX9930 en la escala FACIT-F y otros PRO
    •Caracterizar de BCX9930 PK en sujetos con HPN
    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 - protocol, V2 dated 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. Treated with a stable regimen of eculizumab for ≥ 3 months prior to the screening visit or ravulizumab for ≥ 6 months prior to the screening visit.
    5. Recorded the following results during screening:
    a. Hb of ≤ 105 g/L (≤ 10.5 g/dL).
    b. ARC of ≥ 100 × 109 cells/L (≥ 100,000 cells/µL; ≥ 100 G/L).
    c. Absolute neutrophil count of ≥ 0.75 × 109 cells/L (≥ 750 cells/µL; ≥ 0.75 × G/L).
    d. Platelet count of ≥ 30 × 109/L (≥ 30,000/µL; ≥ 30 G/L).
    e. Adequate iron reserve based on ferritin ≥ LLN or total iron binding capacity ≤ upper limit of the normal reference range (ULN).
    f. Estimated glomerular filtration rate of ≥ 50 mL/min/1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Levey and Stevens 2010).
    6. Contraception requirements: WOCBP and partners of male subjects to use highly effective contraception
    7. Documentation of current vaccinations against Neisseria meningitidis types A, C, W, and Y, and Streptococcus pneumoniae, or willingness to start vaccination series at least 14 days prior to Day 1.
    (Note: Vaccination for N. meningitidis type B and for H. influenzae type B (Hib) is strongly encouraged where authorized and available.)
    8. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study, including compliance with the BID dosing schedule for BCX9930.
    9. Willing and able to provide written informed consent
    1. Hombre o mujer, ≥ 18 años.
    2. Peso ≥ 40 kg.
    3. Diagnóstico documentado de HPN confirmado por citometría de flujo con un tamaño de clon de granulocitos o monocitos de HPN ≥ 10 % durante la selección.
    4. Tratados con un régimen estable de eculizumab durante ≥ 3 meses antes de la visita de selección o ravulizumab durante ≥ 6 meses antes de la visita de selección.
    5. Se registraron los siguientes resultados durante la selección:
    a. Hb de ≤ 105 g/L (≤ 10,5 g/dL).
    b. ARC de ≥ 100 × 109 células/L (≥ 100 000 células/µL; ≥ 100 G/L).
    c. Recuento absoluto de neutrófilos ≥ 0,75 x 109 células/L (≥ 750 células/µL; ≥ 0,75 × G/L).
    d. Recuento de plaquetas ≥ 30 ×109/L (≥ 30 000 / µL; ≥ 30 G / L).
    e. Reserva de hierro adecuada basada en ferritina ≥ (LIN) o capacidad total de unión al hierro ≤ límite superior del rango de referencia normal (LSN).
    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)(Levey and Stevens 2010).
    6. Requisitos de anticoncepción - las mujeres en edad fértil acepte utilizar un método anticonceptivo altamente efectivo durante todo el ensayo clínico y durante 30 días después de la última dosis del fármaco objeto del estudio.
    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 con N.meningitids tipo B y H.Influenzae tipo B (Hib) está extremamente recomendado cuando está 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 supplementation 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. Aspartate aminotransferase (AST; also serum glutamic-oxaloacetic transaminase [SGOT]) > 3 × ULN.
    (Note: Subjects may be enrolled with AST > 3 × ULN if explained by hemolysis.)
    c. Total serum bilirubin > 2 × ULN
    (Note: Subjects may be enrolled with total serum bilirubin > 2 × ULN if explained by hemolysis or Gilbert’s syndrome. In the case of hemolysis, total serum bilirubin must be < 5 × ULN and in the case of Gilbert’s syndrome, total serum bilirubin must be < 11 × ULN.)
    12. Current use of a prohibited concomitant medication within 7 days prior to Day 1 as detailed in Section 9.8.1.
    13. Positive serology for human immunodeficiency virus, or active infection with hepatitis B virus or hepatitis C virus, unless receiving antiviral therapy and viral load is undetectable.
    14. Positive drugs of abuse screen, unless by prescription.
    15. Pregnant, planning to become pregnant, or breastfeeding.
    16. Known hypersensitivity to BCX9930 or any of its formulation excipients.
    17. History of severe hypersensitivity to any medicinal product, which was associated with swelling, severe rash requiring treatment/hospitalization, or anaphylaxis.
    18. Any other clinically significant medical or psychiatric condition that, in the opinion of the investigator or sponsor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject.
    1. 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 tratado curativamente 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 o sospecha de infección por coronavirus [COVID-19]; tests positivos persistentes o recurrentes por síndrome respiratorio agudo grave por antígenos o ácidos nucleicos de coronavirus 2 [SARS-CoV-2] ; 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 investigación con medicamento o participación en un ensayo de un medicamento en investigación en los 30 días anteriores a la visita de selección, o 5,5 semividas del medicamento que está siendo investigado, según el período que sea más largo.
    7. Tratamiento con globulina anti-timocitos 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 continúe).
    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 la transaminasa glutámica pirúvica sérica [SGPT]) > 3 × LSN
    b. Aspartato transaminasa (AST): también transaminasa glutámica-oxoloacética [SGOT]) > 3 × ULN
    (Nota los sujetos pueden reclutarese con AST > 3 × LSN si es explicado por hemolisis)
    c. Bilirrubina sérica total > 2 × LSN
    (Nota: Los sujetos pueden inscribirse 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. El uso actual de medicación concomitante prohibidaen 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 la inmunodeficiencia humana o infección activa por el virus de la hepatitis B o el virus de la hepatitis C, a menos que se esté recibiendo terapia antiviral y la carga vírica sea indetectable.
    14. Detección positiva de estupefacientes, exceptuando aquellos con receta.
    15. Mujer embarazada, que planea quedarse embarazada o amamantando.
    16. Hipersensibilidad conocida a BCX9930 o cualquiera de sus excipientes de su formulación.
    17. Antecedentes de hipersensibilidad grave a cualquier medicamento, que se asoció con hinchazón, erupción cutánea grave que requirió tratamiento/hospitalización o anafilaxia.
    18. Cualquier afección 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 para este sujeto
    E.5 End points
    E.5.1Primary end point(s)
    • Change from baseline (CFB) in hemoglobin (Hb)
    • Number and proportion of subjects with a TEAE
    • Number and proportion of subjects who discontinue due to a TEAE
    • Number and proportion of subjects who experience a TESAE
    • Number and proportion of subjects who experience a CTCAE Grade 3 or Grade 4 TEAE
    • Number and proportion of subjects who experience a treatment-emergent CTCAE Grade 3 or Grade 4 laboratory abnormality
    • Cambio con respecto al Estado Basal (CEB) en la hemoglobina (Hb)
    • Número y proporción de sujetos con un acontecimiento adverso aparecido durante el tratamiento (AAAT)
    • Número y proporción de sujetos que interrumpen el tratamiento 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 experimentaron un AAAT de grado 3 o grado 4 evaluados utilizando las escalas de calificación de Criterios de terminología comunes para acontecimientos adversos (CTCAA)
    • 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
    1 - mean of values at Weeks 12, 16, 20, and 24
    others - week 52
    1 - media de los valores en las semanas 12, 16, 20 y 24
    oltre - semana 52
    E.5.2Secondary end point(s)
    1. Proportion of subjects who are transfusion-free
    2. Number of units of packed red blood cells (pRBCs) transfused
    3. CFB in FACIT-Fatigue scale score
    1. Proporción de sujetos que no han recibido transfusiones
    2. Número de unidades de concentrado de glóbulos rojos (pRBCs) transfundidas
    3. CEB en la puntuación de la escala FACIT-F
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. - week 24
    2. - week 24
    3. - mean of values at Weeks 12, 16, 20, and 24
    1 - semana 24
    2 - semana 24
    3 - media de los valores en las semanas 12, 16, 20 y 24
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA 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
    Brazil
    Canada
    China
    Colombia
    Japan
    Korea, Republic of
    Taiwan
    Turkey
    United States
    European Union
    United Kingdom
    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
    LPLV
    LPLV
    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
    E.8.9.2In all countries concerned by the trial months3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 81
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who are experiencing clinical benefit will be offered the opportunity to continue to receive BCX9930 via an access protocol BCX9930-201 EudraCT: 2020-000501-93
    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 Decision2021-11-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-29
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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