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    Summary
    EudraCT Number:2021-002844-66
    Sponsor's Protocol Code Number:INCB50465-309
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-10-15
    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 number2021-002844-66
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants with Primary Warm Autoimmune Hemolytic Anemia
    Estudio de fase III, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de parsaclisib en participantes con anemia hemolítica autoinmune primaria por anticuerpos calientes
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Efficacy and Safety of Parsaclisib in Participants with Primary Warm Autoimmune Hemolytic Anemia
    Estudio de la eficacia y seguridad de Parsaclisib en participantes con anemia hemolítica autoinmune primaria por anticuerpos calientes
    A.4.1Sponsor's protocol code numberINCB50465-309
    A.5.4Other Identifiers
    Name:INDNumber:147,208
    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 SponsorIncyte Corporation
    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 supportIncyte Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIncyte Corporation
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street Address1801 Augustine Cut-Off
    B.5.3.2Town/ cityWilmington
    B.5.3.3Post codeDE 19803
    B.5.3.4CountryUnited States
    B.5.6E-mailglobalmedinfo@incyte.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 nameParsaclisib (1 mg)
    D.3.2Product code INCB050465
    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 INNParsaclisib
    D.3.9.2Current sponsor codeINCB050465
    D.3.9.3Other descriptive nameINCB050465 HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB183790
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 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 nameParsaclisib (2.5 mg)
    D.3.2Product code INCB050465
    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 INNParsaclisib
    D.3.9.2Current sponsor codeINCB050465
    D.3.9.3Other descriptive nameINCB050465 HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB183790
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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
    D.8 Placebo: 2
    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
    Primary Warm Autoimmune Hemolytic Anemia
    anemia hemolítica autoinmune primaria por anticuerpos calientes
    E.1.1.1Medical condition in easily understood language
    Autoimmune disorder characterized by the premature destruction of healthy red blood cells
    Desorden autoinmune caracterizado por la destrucción prematura de globulos rojos sanos
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003825
    E.1.2Term Autoimmune hemolytic anemia
    E.1.2System Organ Class 100000004851
    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 efficacy of parsaclisib in the treatment of participants with wAIHA.
    Evaluar la eficacia de parsaclisib en el tratamiento de participantes con wAIHA.
    E.2.2Secondary objectives of the trial
    Key secondary objective:
    •To further evaluate parsaclisib in the treatment of participants with wAIHA.
    Other secondary objectives:
    •To further evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.
    •To evaluate the safety and tolerability of parsaclisib in participants with wAIHA.
    Exploratory objectives:
    •To further evaluate the efficacy of parsaclisib.
    •To evaluate the participant's quality of life and other PROs.
    •To characterize serum biomarkers and/or leukocyte profiles in participants with wAIHA treated with parsaclisib.
    •To evaluate PK of parsaclisib in participants with wAIHA.
    Objetivos Secundarios clave
    -Evaluar adicionalmente parsaclisib en el tratamiento de participantes con wAIHA.
    Otros objetivos secundarios
    -Evaluar adicionalmente la eficacia de parsaclisib en el tratamiento de participantes con wAIHA.
    -Evaluar la seguridad y la tolerabilidad de parsaclisib en participantes con wAIHA.
    Objetivos exploratorios
    -Evaluar adicionalmente la eficacia de parsaclisib
    -Evaluar la calidad de vida del participante y otros resultados percibidos por el paciente.
    -Caracterizar los biomarcadores séricos y/o los perfiles leucocitarios en participantes con wAIHA tratada con parsaclisib.
    -Evaluar la FC de parsaclisib en participantes con wAIHA.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Qualitative Exit Interview sub-study, included into Protocol v1 dated 27May2021
    Sub estudio de entrevista cualitativa de salida, incluida en el protocolo V1 de fecha 27May2021
    E.3Principal inclusion criteria
    1. Ability to comprehend and willingness to sign a written ICF for the study.
    2. Men or women, age ≥ 18 years at the time of signing the ICF.
    3. Diagnosis of primary wAIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the DAT positive for IgG only or IgG plus C3d.
    Note: Prior documentation of DAT testing is permitted.
    4. Participants who were inadequately controlled with, were intolerant to, or have a contraindication to other therapies. There is no limit to the number of prior treatment regimens.
    5. Hemoglobin ≥ 7 to < 10 g/dL with symptoms of anemia as assessed by the investigator at screening (Hgb as determined by local laboratory).
    6. FACIT-F score ≤ 43 at screening.
    7. Willingness to avoid pregnancy or fathering children based on the criteria below.
    a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children (with 99% certainty) from screening through 90 days after the last dose of study drug and must refrain from donating sperm during this period. Permitted methods in that are at least 99% effective preventing
    pregnancy should be communicated to the participants and their understanding confirmed.
    b. Female participants who are WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test before the first dose on Day 1 and must agree to take appropriate precautions to avoid pregnancy (with 99% certainty) from screening through 90 days after the last dose of study drug and must refrain from donating oocytes during this period. Permitted methods in that are at least 99% effective preventing pregnancy should be communicated to the participants and their understanding confirmed.
    c. A female participant not considered to be of childbearing potential is eligible.
    Note: This criterion does not apply to women of nonchildbearing potential (i.e., surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, defined as amenorrhea at least 12 months before screening, confirmed by FSH levels at screening).
    8. Willingness to receive PJP prophylaxis during the study period from Day 1 through at least 2 to 6 months after the last dose of study drug.
    1.Capacidad para comprender un FCI por escrito para el estudio y voluntad de firmarlo.
    2.Varones o mujeres ≥18 años de edad en la fecha de la firma del FCI.
    3.Diagnóstico de wAIHA primaria con base en la presencia de anemia hemolítica y la evidencia serológica de anticuerpos antieritrocitarios, detectable en PAD positiva para IgG solo o IgG más C3d.
    4.Participantes con un control inadecuado de la enfermedad con otros tratamientos, intolerancia a otros tratamientos o contraindicación para otros tratamientos. No hay límite para el número de pautas terapéuticas anteriores.
    5.Nivel de hemoglobinas de entre ≥ 7 y <10 g/dl con síntomas de anemias según lo evaluado por el investigador en la selección (Hgb según lo determinado por el laboratorio local).
    6.Puntuación FACIT-F de ≤43 en la selección.
    7.Voluntad de evitar el embarazo o engendrar un hijo con base en los criterios que figuran a continuación.
    a.Los participantes varones con potencial reproductivo deben acceder a tomar medidas de precaución adecuadas para evitar engendrar un hijo (con una fiabilidad del 99 %) desde la selección hasta 90 días después de la última dosis del fármaco del estudio y deben abstenerse de donar esperma durante dicho periodo. Los métodos permitidos que poseen una eficacia de al menos el 99 % de prevención del embarazo deben comunicarse a los participantes y debe confirmarse que los han comprendido.
    b.Las participantes con capacidad de quedarse embarazadas deben obtener un resultado negativo en una prueba de embarazo en sangre en la selección y otro en una prueba de embarazo en orina antes de la primera dosis del día 1, deben acceder a tomar medidas de precaución adecuadas para evitar el embarazo (con una fiabilidad del 99 %) desde la selección hasta 90 días después de la última dosis del fármaco del estudio y deben abstenerse de donar ovocitos durante dicho periodo. Los métodos permitidos que poseen una eficacia de al menos el 99 % de prevención del embarazo deben comunicarse a las participantes y debe confirmarse que los han comprendido.
    c.Una participante que no tiene capacidad de quedarse embarazada es elegible.
    Nota: Este criterio no resulta de aplicación en mujeres que no tienen capacidad de quedarse embarazadas (es decir, estériles quirúrgicamente por histerectomía y/u ooforectomía bilateral O posmenopáusica, definida como con amenorrea durante al menos 12 meses antes de la selección y confirmada por los niveles de FSH en la selección).
    8.Voluntad de recibir profilaxis para neumonía por PJ durante el periodo del estudio desde el día 1 hasta al menos 2-6 meses después de la última dosis del fármaco del estudio.
    E.4Principal exclusion criteria
    1. Women currently pregnant or breastfeeding or participants expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 90 days from the date of last dose of study drug.
    2. A diagnosis of other types of AIHA; CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria.
    3. Warm AIHA suspected to be secondary to a lymphoproliferative malignancy or secondary to an autoimmune disease (eg, systemic lupus erythematosus, Castleman's disease, Sjögren's syndrome, or other).
    4. A splenectomy less than 3 months before randomization.
    5. Concurrent conditions or history of other diseases:
    a. History or clinical manifestations of significant unstable metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, urological, neurological, or psychiatric disorders.
    b. Current or previous malignancy within 5 years of study entry, except basal or squamous cell skin cancer with removal considered to be curative, superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy without sponsor approval.
    c. Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, and/or cardiac conduction issues within 6 months of Day 1 visit.
    d. Current New York Heart Association Class II to IV congestive heart failure or uncontrolled arrhythmia.
    6. Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies as assessed by the investigator as a clinical risk for thrombosis.
    7. Hepatitis B (HBV) or hepatitis C (HCV) infection: Participants who are positive for the hepatitis B surface antibody or hepatitis B core antibody will be eligible if they are negative for HBV-DNA; these participants should be considered for prophylactic antiviral therapy. Participants who are positive for the anti-HCV antibody will be eligible
    if they are negative for HCV-RNA.
    8. Known HIV infection or positivity on immunoassay.
    9. History or presence of an abnormal ECG that, in the investigator's opinion, is clinically meaningful. Participants with screening QTc interval > 470 milliseconds for males and > 480 milliseconds for females (corrected by Fridericia) are excluded. In the event that a
    single QTcF is > 470 milliseconds for males or > 480 milliseconds for females, the participant may enroll if the average QTcF for 3 ECGs is < 470 milliseconds for males or < 480 milliseconds for females.
    10. Use of the following medications:
    a. Treatment with rituximab within 3 months of the Day 1 visit.
    b. Use of immunosuppressive therapy within 28 days of the Day 1 visit.
    Immunosuppressive therapy includes, but is not limited to, cyclosporine A, azothioprine, mycophenolate mofetil, cyclophosphamide, or high-dose corticosteroids.
    Note: Participants receiving corticosteroids must be at a stable dose level ≤ 20 mg/day (prednisone or equivalent corticosteroid dose) within 14 days of the Day 1 visit.
    c. Use of IVIG or epoetin alfa within 2 weeks of the Day 1 visit.
    d. Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine within 30 days of the Day 1 visit.
    e. Use or expected use during the study of any prohibited medications, including potent CYP3A4 inhibitors or inducers, within 14 days or 5 half-lives (whichever is longer) before the Day 1 visit.
    11. Current treatment or treatment within 30 days or 5 half-lives (whichever is longer) before the Day 1 visit with another investigational medication, or current enrollment in another
    investigational drug and/or device protocol.
    12. Known hypersensitivity or severe reaction to parsaclisib or its excipients.
    13. Unable to swallow oral medication, malabsorption syndrome, disease significantly affecting gastrointestinal function, total resection of the stomach or small bowel, ulcerative colitis, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.
    14. Current alcohol or drug use that, in the opinion of the investigator, will interfere with the participant's ability to comply with the dose regimen and study evaluations.
    15. Participants who, in the opinion of the investigator, are unable or unlikely to comply with the dose regimen and study evaluations.
    16. Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
    17. Prior treatment with parsaclisib or another PI3Kδ, or a pan-PI3K inhibitor for any indication.
    18. Participants with exclusionary laboratory values at screening
    1.Mujeres embarazadas o en lactancia o participantes que esperan concebir durante el transcurso estudio,desde la visita de selecc hasta 90 días después de la fecha de la última dosis del fármaco
    2.Diagnóstico de otros tipos de AIHA;enfermedad de crioaglutininas,síndrome de las crioaglutininas,AIHA mixto o hemoglobinuria paroxística por anticuerpo frío
    3.AIHA por anticuerpo caliente que se sospecha secundaria a neoplasia maligna linfoproliferativa o secundaria a enfermedad autoinmune(p. ej.lupus eritematoso sistémico,enfermedad de Castleman,síndrome de Sjögren u otras)
    4.Esplenectomía menos de 3 meses antes de la aleatorización
    5.Afecciones concurrentes o antecedentes de otras enfermedades:
    a.Antecedentes o manifestaciones clínicas de trastornos psiquiátricos,neurológicos,urológicos, gastrointestinales,cardiovasculares,pulmonares, hematológicos,renales,hepáticos o metabólicos inestables y significativos.
    b.Neoplasia maligna actual o previa en 5 años previos a la inclusión en el estudio,salvo cáncer de piel de células escamosas o basales con extirpación considerada curativa,cáncer de vejiga superficial,neoplasia intraepitelial de próstata,carcinoma in situ del cuello uterino u otra no invasiva o indolora sin aprobación del promotor.
    c.Enfermedad cardiaca clínicamente significativa,incluso angina,infarto de miocardio agudo y/o trastornos de conducción cardiaca insostenibles en 6 meses previos a visita día 1.
    d.Insuficiencia cardíaca congestiva actual de clase II-IV de la NY Heart Association o arritmia no controlada
    6.Positivo para anticuerpos antifosfolípidos o nivel alto de anticuerpos antiestreptolisina según lo evaluado por el investigador como riesgo de trombosis.
    7.Infección por hepatitis B(VHB)o C(VHC):Los que obtengan resultado positivo para anticuerpos de superficie de hep B o anticuerpos del núcleo de hep B serán elegibles si obtienen un resultado negativo en prueba de ADN-VHB;deberá sopesarse que dichos participantes reciban tratamiento antiviral profiláctico.Los participantes positivos para anticuerpos anti-VHC serán elegibles si son negativos para ARN-VHC
    8.Infección por VIH o resultado positivo en inmunoanálisis
    9.Antecedentes o presencia de ECG anómalo que,a juicio del investigador,sea clínicamente significativo.Se excluyen participantes con intervalo QTc en selección>470 mseg en varones y>480 mseg en mujeres(corregido con fórmula de Fridericia).En caso de que un único intervalo QTcF sea>470 mseg en varones y >480 mseg en mujeres,podrá incluirse al participante si la media de QTcF de 3 ECG es<470 mseg en varones y <480 mseg en mujeres
    10.Uso de:
    a.rituximab en 3 meses anteriores a visita día 1.
    b.tratamiento inmunodepresor en 28 meses anteriores a visita día 1. Entre los ttos inmunodepresores se incluyen aquellos con ciclosporina A,Azatioprina,micofenolato de mofetilo,ciclofosfamida o corticoesteroides en dosis altas
    Nota:Los que reciban corticoesteroides deben mantener un nivel de dosis estable≤20mg/día(dosis de prednisona o corticoesteroide equivalente)los 14 días previos a visita día 1.
    c.Uso de IG i.v. o epoetina alfa en 2 semanas previas a visita día 1.
    d.Enfermedad infecciosa activa actual o crónica que requiera tto sistémico con antibióticos,antifúngicos,antivirales o exposición a vacuna con microbios vivos en 30 días previos a visita día 1.
    e.Uso actual o previsto durante el estudio de cualquier medicamento prohibido(apartado 6.6.3),incluso inductores o inhibidores potentes de CYP3A4,en 14 días o 5 semividas(el periodo de mayor duración)antes de visita 1
    11.Tto actual o tratamiento en 30 días o 5 semividas(el periodo de mayor duración)antes de visita día 1 con otro medicamento en fase de investigación o persona participando en otro protocolo de un producto y/o fármaco en investigación.
    12.Hipersensibilidad o reacción grave conocida a parsaclisib o excipientes
    13.Imposibilidad de tragar medicación por vía oral,síndrome de malabsorción,enfermedad que afecte significativamente a la función gastrointestinal,resección total del estómago o intestino delgado, colitis ulcerosa,enfermedad intestinal inflamatoria sintomática u obstrucción completa o parcial del intestino
    14.Uso actual de sustancias tóxicas o alcohol que,a juicio del investigador, pudiera interferir en la capacidad del participante para cumplir la pauta posológica y las evaluaciones del estudio
    15.Participantes que a juicio del investigador no puedan o no quieran cumplir la pauta posológica y completar las evaluaciones del estudio
    16.Cualquier afección que,a juicio del investigador,pudiera interferir en la plena participación en el estudio,incluso en la administración del fármaco del estudio y en la asistencia a las visitas; pudiera constituir un riesgo significativo para el participante;o pudiera interferir en la interpretación de los datos
    17.Tto previo con parsaclisib u otro inhibidor de PI3Kδ o pan-PI3K para cualquier indicación
    18.Participantes con valores analíticos en selección definidos en Tabla 7
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants attaining a durable hemoglobin response, defined as hemoglobin ≥ 10 g/dL with an increase from baseline of
    ≥ 2 g/dL not attributed to rescue therapy at ≥ 3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.
    Proporción de participantes que logran una respuesta de hemoglobina duradera, definida como hemoglobina ≥ 10 g / dL con un aumento desde el inicio de
    ≥ 2 g / dl no atribuido a la terapia de rescate en ≥ 3 de las 4 visitas disponibles en la semana 12 y / o más tarde durante el período de tratamiento doble ciego de 24 semanas.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12 and/or later during the 24-week double-blind treatment period
    Semana 12 y / o más tarde durante el período de tratamiento doble ciego de 24 semanas
    E.5.2Secondary end point(s)
    Proportion of participants with a ≥ 3-point increase from baseline in FACIT-F score at Week 24.
    Proporción de participantes con un aumento ≥ 3 puntos desde el inicio en la puntuación FACIT-F en la semana 24.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    Semana 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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    To evaluate the participant's quality of life and other PROs.
    Evaluar la calidad de vida del participante y otros PRO
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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
    Austria
    Belgium
    Canada
    France
    Germany
    Israel
    Italy
    Japan
    Netherlands
    Poland
    Spain
    Ukraine
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    Ultima visita ultimo paciente
    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 months3
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days10
    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: 80
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 57
    F.4.2.2In the whole clinical trial 100
    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)
    The patients will continue standard of care after completing the study.
    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 Decision2022-02-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-22
    P. End of Trial
    P.End of Trial StatusOngoing
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