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    EudraCT Number:2021-000037-14
    Sponsor's Protocol Code Number:NEOD001-301
    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:2021-06-09
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2021-000037-14
    A.3Full title of the trial
    A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis
    Estudio de fase 3, aleatorizado, multicéntrico, doble ciego y controlado con placebo sobre la eficacia y seguridad de birtamimab añadido al tratamiento estándar comparado con placebo añadido al tratamiento estándar en pacientes con amiloidosis de cadena ligera (AL) en estadio IV de la Clínica Mayo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Global Phase 3 Double-Blind, Placebo-Controlled study to assess Efficacy and Safety of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis
    Estudio global de fase 3, doble ciego y controlado con placebo para evaluar la eficacia y seguridad de birtamimab añadido al tratamiento estándar comparado con placebo añadido al tratamiento estándar en pacientes con amiloidosis de cadena ligera (AL) en estadio IV de la Clínica Mayo
    A.4.1Sponsor's protocol code numberNEOD001-301
    A.5.4Other Identifiers
    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 SponsorProthena Biosciences Limited
    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 supportProthena Biosciences Limited
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProthena Biosciences Limited
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address77 Sir John Rogerson’s Quay, Block C
    B.5.3.2Town/ cityGrand Canal Docklands, Dublin 2
    B.5.3.3Post codeD02 T804
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1100
    D.3 Description of the IMP
    D.3.1Product nameBirtamimab
    D.3.2Product code Birtamimab
    D.3.4Pharmaceutical form Powder 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 INNBIRTAMIMAB
    D.3.9.1CAS number 1608108-91-3
    D.3.9.2Current sponsor codebirtamimab
    D.3.9.3Other descriptive nameHumanized IgG1 kappa antiamyloid
    D.3.9.4EV Substance CodeSUB198016
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    AL amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble, fibrillar deposits of abnormal AL protein (amyloid), in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac, renal, and hepatic dysfunction, gastrointestinal involvement and neuropathy and macroglossia
    Amiloidosis AL implica un trastorno hematológico causado por células plasmáticas clonales que producen cadenas ligeras de inmunoglobulina mal plegadas. Prod excesiva de cadenas ligeras mal plegadas da como resultado formas solubles y agregadas de cadenas ligeras y depósitos fibrilares insolubles de proteína AL anómala (amiloide) en tejidos y órganos. Puede causar diversos síntomas y disfunción orgánica, disfunción cardíaca, renal y hepática, afectación gastrointestinal, neuropatía y macroglosia
    E.1.1.1Medical condition in easily understood language
    Light chain (AL) amyloidosis is a blood disorder, which causes progressive organ damage as a result of the misfolding of proteins. This disease can produce a range of symptoms and organ dysfunction
    Amiloidosis de cadenas ligeras (AL) es un trastorno sanguíneo que causa daño orgánico progresivo consecuencia del mal plegado de proteínas.Esta enfermedad puede producir síntomas y disfunción orgánica
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10036673
    E.1.2Term Primary amyloidosis
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of birtamimab plus standard of care compared to placebo plus standard of care when administered intravenously in Mayo Stage IV subjects with AL amyloidosis by assessing time to all-cause mortality.
    Evaluar la eficacia del birtamimab más el tratamiento de referencia en comparación con el placebo más el tratamiento de referencia cuando se administra por vía intravenosa en pacientes afectados de amiloidosis AL en estadio IV de la Clínica Mayo, mediante la evaluación del tiempo transcurrido hasta la mortalidad por cualquier causa.
    E.2.2Secondary objectives of the trial
    To evaluate birtamimab plus standard of care compared to placebo plus standard of care on the following:
    • Change from baseline to Month 9 in health-related quality of life using the Short Form-36 questionnaire Version 2 (SF-36v2)
    • Change from baseline to Month 9 in the 6-Minute Walk Test (6MWT) distance
    Evaluar el birtamimab más el tratamiento de referencia en comparación con el placebo más el tratamiento de referencia en los siguientes aspectos:
    • Cambio producido desde el inicio hasta el mes 9 en la calidad de vida relacionada con la salud, utilizando el cuestionario abreviado de 36 apartados, versión 2 (SF-36v2)
    • Cambio producido desde el inicio hasta el mes 9 en la distancia recorrida durante la prueba de marcha de 6 minutos (PM6M)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Aged ≥18 years
    2.Newly diagnosed and AL amyloidosis treatment naive
    3.Bone marrow demonstrating clonal plasma cells
    4.Confirmed diagnosis of AL amyloidosis by the following:
    -Histochemical diagnosis of amyloidosis determined by polarizing light microscopy of green birefringent material in Congo red-stained tissue specimens OR characteristic electron microscopy appearance
    -Confirmatory immunohistochemistry OR mass spectroscopy of AL amyloidosis
    5.Confirmed diagnosis of AL amyloidosis by mass spectrometry or immunoelectron microscopy of amyloid material in tissue biopsy if the subject meets any of the following:
    -Is black or African American
    -Is over 75 years of age with concurrent monoclonal gammopathy
    -Has a history of familial amyloidosis and has concurrent monoclonal gammopathy
    -If the subject meets any of the above 3 conditions and has echocardiographic evidence of amyloidosis, biopsy-proven amyloidosis with a monoclonal gammopathy and no tissue is available for mass spectrometry or immunoelectron microscopy, the subject must have gene sequencing consistent with transthyretin (TTR) wild type (e.g., no TTR mutation present) AND must score 0 in technetium-99m-3,3-diphosphono-1,2 propanodicarboxylic acid (99mTc DPD), hydroxymethylenediphosphonate (99mTc HMDP), or pyrophosphate (99mTc PYP) scintigraphy
    6.Cardiac involvement as defined by all of the following:
    -Past documented or presently noted clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure
    -Either an endomyocardial biopsy demonstrating AL amyloidosis or an echocardiogram demonstrating a mean left ventricular wall thickness at diastole >12 mm in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening
    7.Confirmed Mayo Stage IV as defined by:
    -NT-proBNP ≥1800 pg/mL and
    -Troponin-T >0.03 ng/mL and
    -dFLC ≥18 mg/dL
    8.Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly
    9.Adequate bone marrow reserve, hepatic function, and renal function, as demonstrated by:
    -Absolute neutrophil count ≥1.0 × 10e9/L
    -Platelet count ≥75 × 10e9/L
    -Hemoglobin ≥9 g/dL
    -Total bilirubin ≤ 2 × the upper limit of normal (ULN)
    -Aspartate aminotransferase (AST)/serum glutamic oxaloacetic transaminase ≤3 × ULN
    -Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase ≤3 × ULN
    -Alkaline phosphatase (ALP) ≤5 × ULN (except for subjects with hepatomegaly and isozymes specific to liver, rather than bone)
    -Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration equation
    10.Seated systolic blood pressure (BP) 90 to 180 mmHg
    11.Distance walked during each Screening 6MWT is >30 meters and <550 meters
    12.Women of childbearing potential (WOCBP) must have 2 negative pregnancy tests during Screening, the second within 24 hours prior to the first administration of study drug, and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
    13.Male subjects must be surgically sterile or must agree to use highly effective physician approved contraception from Screening to 90 days following the last study drug administration
    14.Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
    1. Edad ≥ 18 años
    2. Amiloidosis AL y de nuevo diagnóstico sin tratamiento anterior
    3. Médula ósea que muestre células plasmáticas clonales
    4. Diagnóstico de amiloidosis AL confirmado por los siguientes medios:
    • Diagnóstico histoquímico de amiloidosis determinado mediante microscopia de luz polarizada de material verde birrefringente en muestras de tejido con tinción de rojo Congo O BIEN aspecto característico de la microscopia electrónica
    • Inmunohistoquímica confirmatoria O BIEN espectroscopia de masas de amiloidosis AL
    5. Diagnóstico de amiloidosis AL confirmado mediante espectrometría de masas o inmunoelectromicroscopia de material amiloide en biopsia de tejido, si el paciente cumple alguno de los siguientes criterios:
    • Raza negra o afroamericana
    • Edad superior a 75 años y gammapatía monoclonal simultánea
    • Antecedentes de amiloidosis familiar y gammapatía monoclonal concurrente
    O BIEN
    • Si el paciente cumple alguna de las 3 condiciones anteriores y presenta signos ecocardiográficos de amiloidosis, amiloidosis confirmada mediante biopsia con gammapatía monoclonal y ausencia de tejido disponible para espectrometría de masas o inmunoelectromicroscopia, debe tener secuenciación génica compatible con el tipo natural de transtiretina (TTR) (por ejemplo, sin mutación de TTR presente) Y ADEMÁS una puntuación de 0 en la gammagrafía con tecnecio-99m-3,3-difosfono-1,2 ácido propanodicarboxílico (99mTc DPD; Rapezzi 2011), hidroximetilendifosfonato (99mTc HMDP; Galat 2015) o pirofosfato (99mTc PYP; Bokhari 2013).
    6. Afectación cardíaca definida por todos los siguientes elementos:
    • Signos y síntomas clínicos documentados u observados en la actualidad que respaldan un diagnóstico de insuficiencia cardíaca en el contexto de un diagnóstico confirmado de amiloidosis AL en ausencia de una explicación alternativa para la insuficiencia cardíaca
    • Biopsia endomiocárdica que muestre amiloidosis AL o un ecocardiograma que muestre un grosor medio de la pared ventricular izquierda en la diástole superior a 12 mm en ausencia de otras causas (por ejemplo, hipertensión grave o estenosis aórtica) que explicasen de manera satisfactoria el grado de engrosamiento de la pared
    7. Estadio IV confirmado de la Clínica Mayo, definido por los siguientes elementos:
    • NT-proBNP ≥ 1800 pg/ml,
    • Troponina-T > 0,03 ng/ml, y
    • dCLL ≥ 18 mg/dl
    8. La quimioterapia de primera línea prevista contiene bortezomib administrado por vía subcutánea semanalmente
    9. Reserva de médula ósea, actividad hepática y actividad renal suficientes, demostradas por los siguientes elementos:
    • Cifra absoluta de neutrófilos ≥ 1,0 × 109/l
    • Cifra de plaquetas ≥ 75 × 109/l
    • Concentración de hemoglobina ≥ 9 g/dl
    • Concentración de bilirrubina total ≤ 2 × límite superior de la normalidad (LSN)
    • Concentración de aspartato-aminotransferasa (transaminasa glutámico-oxaloacética en suero) ≤ 3 × LSN
    • Concentración de alanina-aminotransferasa (transaminasa glutámico-pirúvica en suero) ≤ 3 × LSN
    • Concentración de fosfatasa alcalina ≤ 5 × LSN (excepto en pacientes afectados de hepatomegalia que presenten isoenzimas específicas del hígado, en lugar de óseas)
    • Velocidad de filtración glomerular calculada ≥ 30 ml/min/1,73 m2 según la ecuación del Grupo de Colaboración de Epidemiología de la Enfermedad Renal Crónica (Chronic Kidney Disease Epidemiology Collaboration)
    10. Presión arterial sistólica en sedestación de entre 90 y 180 mmHg
    11. Distancia recorrida durante cada PM6M de la selección igual o superior a 30 metros e igual o inferior a 550 metros
    12. Las mujeres con capacidad de concebir deben tener 2 pruebas de embarazo negativas durante la selección, la segunda en las 24 horas anteriores a la primera administración del fármaco del estudio, y han de acceder a utilizar un método anticonceptivo de gran eficacia aprobado por el médico desde la selección hasta 90 días después de la última administración del fármaco
    13. Los pacientes varones deben ser quirúrgicamente estériles o aceptar el uso de métodos anticonceptivos de alta eficacia aprobados por el médico desde la selección hasta 90 días después de la última administración del fármaco
    14. Capacidad para comprender y firmar un documento de consentimiento informado antes del inicio de ninguna actividad del estudio
    E.4Principal exclusion criteria
    1.Non-AL amyloidosis
    2.NT-proBNP >8500 pg/mL
    3.Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma
    *Note that subjects who meet the IMWG definition of symptomatic multiple myeloma with signs and/or symptoms attributable only to associated amyloidosis are potentially eligible upon approval of the Sponsor.
    4.Subject is eligible for and plans to undergo ASCT or organ transplant during the study
    5.Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with the subject’s ability to safely receive treatment or complete study assessments
    6.Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
    7.Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
    8.ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
    -First degree AV-block
    -Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
    -Right or left bundle branch block
    -Atrial fibrillation with a controlled ventricular rate (uncontrolled [>110 bpm] ventricular rate is not allowed [determined by an average of 3 beats in Lead II or 3 representative beats if Lead II is not representative of the overall ECG])
    9.Peripheral neuropathy assessed as National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) Grade 2 with pain, Grade 3, or Grade 4
    10.Subject is receiving oral or intravenous antibiotics, antifungals, or antivirals within 1 week of Month 1-Day 1 with the exception of prophylactic oral agents
    11.Prior treatment with hematopoietic growth factors, transfusions of blood or blood products within 1 week of Month 1-Day 1
    12.Prior radiotherapy within 4 weeks of Month 1-Day 1
    13.Major surgery within 4 weeks of Month 1-Day 1 or planned major surgery during the study
    14.Active malignancy with the exception of any of the following:
    -Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer
    -Adequately treated Stage I cancer from which the subject is currently in remission and has been in remission for 2 years
    -Low-risk prostate cancer with Gleason score <7 and prostate specific antigen <10 ng/mL
    -Any other cancer from which the subject has been disease-free for ≥2 years
    15.History of severe allergy to any of the components of birtamimab such as histidine/L histidine hydrochloride monohydrate, trehalose dehydrate, or polysorbate 20 or history of Grade ≥3 infusion-related AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine (or an equivalent H1 antihistamine) or acetaminophen (or its equivalent, paracetamol)
    16.Known or history of uncontrolled, active HIV, hepatitis B or hepatitis C, or SARS-CoV-2 infection
    17.Prior treatment with plasma cell-directed chemotherapy, birtamimab, daratumumab, 11 1F4, anti-serum amyloid P antibody, doxycycline for amyloid, or other investigational treatment directed at amyloid
    18.Treatment with another investigational agent within 30 days of Month 1-Day 1
    19.Women who are pregnant or lactating
    20.Any condition which could interfere with, or the treatment for which might interfere with, the conduct of the study or which would, in the opinion of the Investigator, unacceptably increase the subject’s risk by participating in the study
    21.Subject is under legal custodianship
    22.History of epilepsy or seizure disorder with the exception of childhood febrile seizures
    23.Waldenström's macroglobulinemia and/or immunoglobulin M monoclonal gammopathy
    1. Amiloidosis no AL
    2. NT-proBNP > 8500 pg/ml
    3. Cumplimiento de la definición de mieloma múltiple del Grupo Internacional de Trabajo sobre el Mieloma (International Myeloma Working Group, IMWG)
    * Téngase en cuenta que los pacientes que cumplan la definición del IMWG de mieloma múltiple sintomático con signos o síntomas atribuibles solo a la amiloidosis asociada son potencialmente aptos, con autorización del promotor.
    4. El paciente es apto para someterse a un TACP o trasplante de órganos y tiene previsto hacerlo durante el estudio.
    5. Hipotensión ortostática sintomática que, según el criterio médico del investigador, interferiría en la capacidad del paciente para recibir tratamiento de forma segura o para efectuar las evaluaciones del estudio
    6. Infarto de miocardio, angina no estabilizada, arritmias ventriculares graves no estabilizadas o signos electrocardiográficos (ECG) de isquemia aguda, en los 6 meses anteriores a la consulta del día 1 del mes 1
    7. Estenosis valvular grave (por ejemplo, estenosis aórtica o mitral con un área valvular <1,0 cm2) o cardiopatía congénita grave
    8. Indicios electrocardiográficos de isquemia aguda o anomalías activas en el sistema de conducción, a excepción de cualquiera de los siguientes casos:
    • Bloqueo AV de primer grado
    • Bloqueo AV de segundo grado de tipo 1 (tipo Mobitz 1 o tipo Wenckebach)
    • Bloqueo de rama derecha o izquierda
    • Fibrilación auricular con frecuencia ventricular estabilizada (no se permite la frecuencia ventricular no estabilizada [>110 lpm, determinada por un promedio de 3 latidos en la derivación II o 3 latidos representativos si la derivación II no es representativa del ECG general])
    9. Neuropatía periférica evaluada como de grado 2, con dolor, según los criterios terminológicos comunes para acontecimientos adversos (CTCAA) del National Cancer Institute (Instituto Nacional del Cáncer estadounidense, NCI), de grado 3 o de grado 4
    10. El paciente está recibiendo antibióticos, antimicóticos o antivíricos por vía oral o intravenosa en la semana anterior al día 1 del mes 1, a excepción de los profilácticos orales
    11. Tratamiento anterior con factores de crecimiento hematopoyético, transfusiones de sangre o hemoderivados en la semana anterior al día 1 del mes 1
    12. Radioterapia anterior en las 4 semanas precedentes al día 1 del mes 1
    13. Operación de cirugía mayor en las 4 semanas anteriores al día 1 del mes 1 o intervención de esta índole prevista durante el estudio
    14. Neoplasia maligna activa, a excepción de cualquiera de los siguientes casos:
    • Carcinoma basocelular, carcinoma epidermoide o cáncer cervicouterino localizado tratados adecuadamente
    • Cáncer en estadio I tratado adecuadamente del que el paciente esté actualmente en remisión y del que lleve así 2 años
    • Cáncer de próstata de riesgo bajo con puntuación de Gleason <7 y antígeno prostático específico <10 ng/ml
    • Cualquier otro cáncer del que el paciente lleve recuperado ≥2
    15. Antecedentes de alergia grave a alguno de los componentes del birtamimab, como histidina o clorhidrato de L-histidina monohidratado, trehalosa deshidratada o polisorbato 20, o antecedentes de acontecimientos adversos (AA) relacionados con la infusión de grado ≥ 3, o hipersensibilidad a otro anticuerpo monoclonal, o hipersensibilidad conocida a la difenhidramina (o a un antihistamínico H1 equivalente) o al paracetamol (o a su equivalente, el acetaminofeno).
    16. Infección activa por el VIH, el virus de la hepatitis B o C o el SARS-CoV-2 conocida o no estabilizada
    17. Tratamiento anterior con quimioterapia dirigida a las células plasmáticas, birtamimab, daratumumab, 11-1F4, anticuerpos contra el componente amiloide P en suero, doxiciclina para la amiloidosis u otro tratamiento experimental dirigido a la amiloidosis
    18. Tratamiento con otro fármaco experimental en los 30 días anteriores al día 1 del mes 1
    19. Mujeres embarazadas o en período de lactancia
    20. Cualquier afección o su tratamiento que pudieran interferir en la realización del estudio, o que, a juicio del investigador, pudieran incrementar de manera inaceptable el riesgo que corriese el paciente por participar en el estudio
    21. El paciente está sometido a tutela legal
    22. Antecedentes de epilepsia o trastorno convulsivo, a excepción de crisis febriles infantiles
    23. Macroglobulinemia de Waldenström o gammapatía monoclonal de inmunoglobulina M
    E.5 End points
    E.5.1Primary end point(s)
    Time to all-cause mortality
    Tiempo transcurrido hasta la mortalidad por cualquier causa
    E.5.1.1Timepoint(s) of evaluation of this end point
    -Interim Efficacy Analysis will be conducted when approximately 50% (or 24) of the events have occurred
    -Primary Analysis - for all-cause mortality, all deaths occurring after the first infusion of study drug (i.e., Study Day 1) through the study’s last subject last visit will be included.
    - Análisis de eficacia provisional se realizará cuando se hayan producido aproximadamente el 50 % (o 24) de los acontecimientos.
    - Análisis principal: mortalidad por cualquier causa, en la que se incluirán todas las muertes que se produzcan después de la primera infusión del fármaco del estudio (es decir, el día 1 de la investigación) hasta la última consulta del último paciente.
    E.5.2Secondary end point(s)
    •Change from baseline to Month 9 in the Physical Component Summary (PCS) score of the SF-36v2
    •Change from baseline to Month 9 in the 6MWT distance (meters)
    • Cambio producido desde el inicio al mes 9 en la puntuación obtenida en el resumen del componente físico (PCS) del cuestionario SF-36v2
    • Cambio producido desde el inicio hasta el mes 9 en la distancia recorrida durante la PM6M (en metros)
    E.5.2.1Timepoint(s) of evaluation of this end point
    from baseline until Month 9
    Desde el inicio hasta el mes 9
    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 No
    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 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. 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 No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned13
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA74
    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
    Korea, Republic of
    United States
    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
    The study will end when approximately 47 primary endpoint events have occurred or at least 135 subjects have completed 9 months of treatment and EOT Visits have been completed for all subjects who remain on study at that time. Events are defined as deaths due to any cause.
    El estudio finalizará cuando se hayan producido aproximadamente 47 acontecimientos del criterio de valoración principal o cuando un mínimo de 135 pacientes hayan completado 9 meses de tratamiento y todos los pacientes que permanezcan en el estudio en ese momento hayan completado las visitas de fin de tratamiento (FdT). Los acontecimientos se definen como muertes por cualquier causa
    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 days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 75
    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 91
    F.4.2.2In the whole clinical trial 150
    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)
    At the time of study completion (when approximately 47 events have been reached or at least 135 subjects have completed 9 months of treatment), all subjects still on study (subjects still receiving study drug treatment [birtamimab or placebo], subjects who discontinue study drug early but agree to return for assessments after the ETD Visit, and subjects participating in the Vital Status Assessment Follow-up) may be considered for entry into an open-label extension study of birtamimab.
    En la finalización del estudio (cuando se hayan alcanzado aprox 47 acontecimientos o al menos 135 pacs hayan completado 9 M de Tº) participantes que sigan en el estudio (que continúen recibiendo el Tº investigado [birtamimab o placebo] los que interrumpan el fármaco de forma prematura pero accedan a volver para las eval después de la visita de IAT y los que participen en el seg de la eval del estado vital) podrán ser considerados para participar en estudio de ampliación en abierto del birtamimab
    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 Decision2021-07-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-28
    P. End of Trial
    P.End of Trial StatusOngoing
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