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    Summary
    EudraCT Number:2020-004486-40
    Sponsor's Protocol Code Number:R3918-PNH-2021
    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:2022-03-11
    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 number2020-004486-40
    A.3Full title of the trial
    A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY
    ESTUDIO ALEATORIZADO, ABIERTO Y CONTROLADO CON RAVULIZUMAB PARA EVALUAR LA EFICACIA Y LA SEGURIDAD DEL TRATAMIENTO COMBINADO DE POZELIMAB Y CEMDISIRAN EN PACIENTES CON HEMOGLOBINURIA PAROXÍSTICA NOCTURNA QUE NO RECIBEN TRATAMIENTO CON INHIBIDORES DEL COMPLEMENTO O QUE NO HAN RECIBIDO RECIENTEMENTE TRATAMIENTO CON INHIBIDORES DEL COMPLEMENTO
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy
    ESTUDIO CONTROLADO CON RAVULIZUMAB PARA EVALUAR LA EFICACIA Y LA SEGURIDAD DEL TRATAMIENTO COMBINADO DE POZELIMAB Y CEMDISIRAN EN PACIENTES CON HEMOGLOBINURIA PAROXÍSTICA NOCTURNA QUE NO RECIBEN TRATAMIENTO CON INHIBIDORES DEL COMPLEMENTO O QUE NO HAN RECIBIDO RECIENTEMENTE TRATAMIENTO CON INHIBIDORES DEL COMPLEMENTO
    A.4.1Sponsor's protocol code numberR3918-PNH-2021
    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 SponsorRegeneron 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 supportRegeneron Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address777 Old Saw Mill River Road
    B.5.3.2Town/ cityTarrytown
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.4Telephone number0034900 834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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 namePozelimab
    D.3.2Product code REGN3918
    D.3.4Pharmaceutical form 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 INNPozelimab
    D.3.9.2Current sponsor codeREGN3918
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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: 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 nameCemdisiran
    D.3.2Product code ALN-CC5
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCEMDISIRAN
    D.3.9.2Current sponsor codeALN-CC5
    D.3.9.3Other descriptive nameCEMDISIRAN
    D.3.9.4EV Substance CodeSUB194793
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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: 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 ™ (ravulizumab-cwvz)
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Pharmaceuticals, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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.3.4Pharmaceutical form 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.3.9.3Other descriptive nameRavulizumab
    D.3.9.4EV Substance CodeSUB192773
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin 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: 4
    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 namePozelimab
    D.3.2Product code REGN3918
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPozelimab
    D.3.9.2Current sponsor codeREGN3918
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 (PNH)
    Hemoglobinuria paroxística nocturna (HPN)
    E.1.1.1Medical condition in easily understood language
    A rare disease causing the breakdown of red blood cells
    Enfermedad Rara que causa la descomposición de los glóbulos rojos
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    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 effect on hemolysis and red blood cell (RBC) transfusions over a 24-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor therapy
    Evaluar el efecto sobre la hemólisis y las transfusiones de eritrocitos durante un período de tratamiento de 24 semanas con la combinación de pozelimab y cemdisirán en comparación con el tratamiento con ravulizumab en pacientes con hemoglobinuria paroxística nocturna (HPN) activa sin tratamiento previo con inhibidores del complemento o que no los han recibido recientemente
    E.2.2Secondary objectives of the trial
    • Evaluate the effect of pozelimab and cemdisiran combination treatment versus ravulizumab treatment on the following:
    − Measures of hemolysis
    − Transfusion parameters
    − Hemoglobin levels
    − Fatigue as assessed by Clinical Outcome Assessments (COAs)
    − HRQoL as assessed by COAs
    − Safety and tolerability
    − Complement activation
    • To assess the concentrations of total pozelimab and total ravulizumab in serum and total cemdisiran and total complement factor 5 (C5) protein in plasma
    • To assess the immunogenicity of pozelimab and cemdisiran
    •Evaluar el efecto de la combinación de pozelimab y cemdisirán en comparación con el tratamiento con ravulizumab sobre los siguientes aspectos:
    -Medidas de la hemólisis.
    -Parámetros transfusionales.
    -Concentración de hemoglobina.
    -Fatiga evaluada mediante evaluaciones de resultados clínicos (ERC).
    -Calidad de vida relacionada con la salud (CVRS) evaluada mediante ERC.
    -Seguridad y tolerabilidad.
    -Activación del complemento.
    •Determinar las concentraciones de pozelimab total y ravulizumab total en suero y de cemdisirán total y proteína C5 (factor 5 del complemento) total en plasma.
    •Evaluar la inmunogenicidad de pozelimab y cemdisirán.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    1. Future Biomedical Research (FBR) (Optional):
    Residual biomarker samples for study-related research, as well as unused PK and ADA samples, will be stored for up to 15 years after the final date of the database lock (or for a shorter time period if
    required per regional laws and regulations). The samples may be utilized for FBR that may or may not be directly related to the study, including being used as reference samples and assay development or validation.
    2. Pharmacogenomic Analysis (Optional):
    Whole blood samples for DNA extraction should be collected on day 1/baseline (predose), but can be collected at a later study visit. Whole blood samples for RNA extraction will be collected at time points according to Table 1 of protocol. DNA and RNA samples will be collected for pharmacogenomics analyses to understand the genetic
    determinants of efficacy and safety associated with the treatments in this study and the molecular basis of PNH and related diseases. The purpose of the pharmacogenomic analyses is to identify genomic associations with clinical or biomarker response to pozelimab and cemdisiran, other PNH clinical outcome measures and possible AEs. In addition, associations between genomic variants and prognosis or progression of PNH as well as related diseases may also be studied. These data may be used or combined with data collected from other studies to identify and validate genomic markers related to the study
    drug, target pathway, or PNH and related diseases. Analyses may include sequence determination or single nucleotide polymorphism studies of candidate genes and surrounding genomic regions. Other methods, including whole-exome sequencing, whole-genome sequencing, DNA copy number variation, and transcriptome sequencing (or other methods for quantitating RNA expression) may also be performed. The list of methods may be expanded to include novel methodology that may be developed during the course of this study or sample storage period.
    1.Investigación biomédica futura (FBR) (opcional):
    Las muestras de biomarcadores residuales para la investigación relacionada con el estudio, así como las muestras PK y ADA no utilizadas, se almacenarán hasta 15 años después de la fecha final del bloqueo de la base de datos (o por un período de tiempo más corto si requerido por las leyes y reglamentos regionales). Las muestras se pueden utilizar para FBR que pueden o no estar directamente relacionadas con el estudio, incluido el uso como muestras de referencia y desarrollo o validación de ensayos.
    2.Análisis Farmacogenómico (Opcional):
    Las muestras de sangre completa para la extracción de ADN se deben recolectar el día 1/basal (antes de la dosis), pero se pueden recolectar en una visita de estudio posterior. Las muestras de sangre completa para la extracción de ARN se recolectarán en puntos de tiempo de acuerdo con la Tabla 1 del protocolo. Se recolectarán muestras de ADN y ARN para análisis de farmacogenómica para comprender la genética determinantes de eficacia y seguridad asociados con los tratamientos en este estudio y la base molecular de la PNH y enfermedades relacionadas. El propósito de los análisis farmacogenómicos es identificar asociaciones genómicas con la respuesta clínica o de biomarcadores a pozelimab y cemdisiran, otras medidas de resultados clínicos de HPN y posibles eventos adversos. Además, también se pueden estudiar las asociaciones entre las variantes genómicas y el pronóstico o la progresión de la PNH, así como las enfermedades relacionadas. Estos datos pueden usarse o combinarse con datos recopilados de otros estudios para identificar y validar marcadores genómicos relacionados con el estudio fármaco, vía diana o PNH y enfermedades relacionadas. Los análisis pueden incluir la determinación de secuencias o estudios de polimorfismos de un solo nucleótido de genes candidatos y regiones genómicas circundantes. También se pueden realizar otros métodos, como la secuenciación del exoma completo, la secuenciación del genoma completo, la variación del número de copias del ADN y la secuenciación del transcriptoma (u otros métodos para cuantificar la expresión del ARN). La lista de métodos puede ampliarse para incluir una metodología novedosa que pueda desarrollarse durante el curso de este estudio o el período de almacenamiento de la muestra.
    E.3Principal inclusion criteria
    1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes described in the protocol
    2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms described in the protocol 3. LDH level ≥2 × ULN at the screening visit

    Note: Other protocol-defined Inclusion Criteria apply
    1.Diagnóstico de HPN confirmado mediante citometría de flujo de alta sensibilidad con granulocitos como se describe en el protocolo.
    2.Enfermedad activa, definida por la presencia de uno o más signos o síntomas relacionados con la HPN como se describe en el protocolo.
    3.Concentración de LDH ≥ 2 veces el LSN en la visita de selección.

    Nota: Aplican los otros Criterios de Inclusión definidos en el protocolo
    E.4Principal exclusion criteria
    1. Prior treatment with a complement inhibitor within 6 months prior to screening visit, unless patient was treated with eculizumab or ravulizumab and has documented C5 variant R885H/C in which case there is no exclusion of such patients
    2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
    3. Body weight <40 kilograms at screening visit
    4. Planned use of any complement inhibitor therapy other than study drugs during the treatment period
    5. Not meeting meningococcal vaccination requirements for ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit
    6. Any contraindication for receiving Neisseria meningitidis vaccination
    7. Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab prescribing information, where available, or national guidelines/local practice or if necessary when vaccination is less than 2 weeks from study treatment initiation)
    8. Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
    9. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

    Note: Other protocol-defined Exclusion Criteria apply
    1.Tratamiento previo con un inhibidor del complemento en los 6 meses anteriores a la visita de selección, a menos que el paciente haya sido tratado con eculizumab o ravulizumab y tenga documentado el C5 variante R885H/C, en cuyo caso no se excluirá a tales pacientes.
    2.Recepción de un trasplante de órgano, antecedentes de trasplante de médula ósea u otro trasplante hematológico.
    3.Peso corporal < 40 kg en la visita de selección.
    4. Uso previsto de cualquier tratamiento inhibidor del complemento distinto de los fármacos del estudio durante el período de tratamiento.
    5. Incumplimiento de los requisitos de vacunación antimeningocócica para ravulizumab de conformidad con la ficha técnica local vigente (cuando esté disponible) y con una documentación mínima de la vacunación antimeningocócica en los 5 años previos a la visita de selección.
    Nota: los pacientes sin vacunación previa serán elegibles siempre que estén dispuestos a vacunarse antes del comienzo del tratamiento del estudio y la vacunación se documente antes de la aleatorización.
    6.Cualquier contraindicación para recibir la vacuna contra Neisseria meningitidis.
    7.Incapacidad para tomar antibióticos para la profilaxis antimeningocócica (si lo exige la ficha técnica local de ravulizumab, cuando esté disponible, o las directrices nacionales o la práctica local o si es necesario cuando la vacunación tenga lugar menos de 2 semanas después del inicio del tratamiento del estudio).
    8.Cualquier infección activa en curso o una infección reciente que requiera tratamiento sistémico con antibióticos, antivirales o antimicóticos en las 2 semanas previas a la selección o durante el período de selección.
    9. Antecedentes documentados de enfermedades autoinmunitarias sistémicas en curso, no controladas y activas.


    Nota: Aplican los otros Criterios de Exclusión definidos en el protocolo
    E.5 End points
    E.5.1Primary end point(s)
    1. Proportion of patients with adequate control of hemolysis
    2. Proportion of patients with transfusion avoidance
    1.Proporción de pacientes con control adecuado de la hemólisis
    2.Proporción de pacientes con evitación de transfusiones
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Between week 4 and week 24, inclusive
    2. Day 1 through week 24
    1. Entre las semanas 4 y 24, inclusive.
    2. Desde el día 1 hasta la semana 24
    E.5.2Secondary end point(s)
    1. Proportion of patients with breakthrough hemolysis
    2. Proportion of patients with hemoglobin stabilization
    3. Proportion of patients with normalization of LDH
    4. Percent change in LDH
    5. Change in fatigue as measured by the FACIT-Fatigue Scale
    6. Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30)
    7. Change in global health status (GHS)/QoL scale score on the EORTC-QLC-C30
    8. Rate of RBC transfusion per protocol algorithm
    9. Number of units of RBC transfusion per protocol algorithm
    10. Time to first LDH ≤1.5 × ULN
    11. Time to first LDH ≤1.0 × ULN
    12. Percentage of days with LDH ≤1.5 × ULN
    13. Change in hemoglobin levels
    14. Incidence and severity of treatment emergent serious adverse events (SAEs)
    15. Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
    16. Incidence and severity of TEAE leading to treatment discontinuation
    17. Change in total CH50
    18. Percent change in total CH50
    19. Concentration of total C5 in plasma
    20. Concentrations of total pozelimab in serum
    21. Concentrations of total cemdisiran in plasma
    22. Concentrations of total ravulizumab in serum
    23. Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
    24. Incidence of treatment emergent ADAs to cemdisiran
    1. Proporción de pacientes con hemólisis intercurrente
    2. Proporción de pacientes con estabilización de la hemoglobina
    3. Proporción de pacientes con normalización de la LDH
    4. Variación porcentual de la LDH
    5. Variación de la fatiga medida mediante la escala FACIT
    6. Variación de la puntuación de función física (FF) en el cuestionario EORTC QLQ-C30 (Cuestionario de calidad de vida-Módulo básico de 30 apartados de la Organización Europea para la Investigación y el Tratamiento del Cáncer)
    7. Variación de la puntuación en la escala de estado general de salud (EGS)/calidad de vida (CdV) del cuestionario EORTC QLC-C30
    8. Frecuencia de eritrocitos transfundidOs conforme al algoritmo del protocolo
    9. Número de unidades de eritrocitos transfundidas conforme al algoritmo del protocolo
    10. Tiempo transcurrido hasta la primera LDH ≤1,5 LNS
    11. Tiempo transcurrido hasta la primera LDH ≤1,0 LNS
    12. Porcentaje de días con una LDH ≤1,5 LNS
    13. Variación de la concentración de hemoglobina
    14. Incidencia e intensidad de los acontecimientos adversos graves (AAG)
    15. Incidencia e intensidad de los acontecimientos adversos surgidos durante el tratamiento (AAST) de interés especial
    16. Incidencia e intensidad de los acontecimientos adversos surgidos durante el tratamiento (AAST) que motiven la suspensión del tratamiento
    17. Variación total del complemento (CH50)
    18. Variación porcentual del complemento (CH50)
    19. Concentración plasmática de C5
    20. Concentración sérica de pozelimab total
    21. Concentración plasmática de cemdisirán total
    22. Concentración sérica de ravulizumab total
    23. Incidencia de anticuerpos contra el fármaco (ACF) antipozelimab
    24. Incidencia de ACF anticemdisirán
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Post-baseline day 1 through week 24
    2. Day 1 (post-baseline) through week 24
    3. Between week 4 through week 24, inclusive
    4-5. From baseline to week 24
    6. Baseline to week 24
    7. From baseline to week 24
    8-9. Day 1 through week 24
    10-11. Up to Week 24
    12. Between week 4 and week 24, inclusive
    13. From baseline to week 24
    14-16. Up to 24 weeks
    17-18. From baseline to week 24
    19-20. Up to 50 weeks
    21. Up to 20 weeks
    22. Up to 34 weeks
    23-24. Up to 50 weeks
    1. Desde el día 1 (después del momento basal) hasta la semana 24.
    2. Desde el día 1 (después del momento basal) hasta la semana 24
    3. Entre las semanas 4 y 24, inclusive.
    4-5. Desde momento basal hasta la semana 24.
    6. Entre el momento basal y la semana 24.
    7. Desde el momento basal hasta la semana 24.
    8-9. Desde el día 1 hasta la semana 24.
    10-11. Hasta la semana 24.
    12. Entre las semanas 4 y 24, inclusive.
    13. Desde el momento basal hasta la semana 24.
    14-16. Durante 24 semanas
    17-18. Desde el momento basal hasta la semana 24
    19-20. Durante 50 semanas
    21. Durante 20 semanas
    22. Durante 34 semanas
    23-24. Durante 50 semanas
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Jordan
    Korea, Republic of
    Malaysia
    Mexico
    Peru
    Singapore
    South Africa
    Taiwan
    Turkey
    United States
    France
    Greece
    Italy
    Netherlands
    Poland
    Portugal
    Romania
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    Última visita último Paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months11
    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: 108
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 16
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 34
    F.4.2.2In the whole clinical trial 124
    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)
    All patients who complete the study will be offered to participate in the follow-up long-term, OLE
    study of the combination treatment.
    A todos los pacientes que completen el tratamiento de estudio se les ofrecerá participar en el estudio de extensión abierto a largo plazo del tratamiento combinado.
    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 Decision2022-05-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-06
    P. End of Trial
    P.End of Trial StatusOngoing
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