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    Summary
    EudraCT Number:2014-003865-11
    Sponsor's Protocol Code Number:NEOD001-CL002
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-01
    Trial results View 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 number2014-003865-11
    A.3Full title of the trial
    A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care vs. Placebo Plus Standard of Care in Subjects with Light Chain (AL) Amyloidosis
    Un estudio de fase III, aleatorizado, multicéntrico, con doble enmascaramiento, controlado con placebo y con dos grupos de tratamiento, de la seguridad y la eficacia de NEOD001 más el tratamiento de referencia en comparación con placebo más el tratamiento de referencia en sujetos con amiloidosis de cadena ligera (AL).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to see how effective and safe NEO001 is when given together with the standard treatment in comparison to placebo together with the standard treatment in people with Light Chain (AL) Amyloidosis.
    Un estudio para ver cómo de efectivo y seguro es NEO001 cuando se administra junto con el tratamiento estándar en comparación con placebo junto con el tratamiento estándar, en personas con amiloidosis de cadena ligera (AL).
    A.4.1Sponsor's protocol code numberNEOD001-CL002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02312206
    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 Therapeutics Limited
    B.1.3.4CountryIreland
    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 Therapeutics Limited
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProthena Biosciences Inc
    B.5.2Functional name of contact pointSpencer Guthrie
    B.5.3 Address:
    B.5.3.1Street Address650 Gateway Blvd
    B.5.3.2Town/ citySouth San Francisco, California
    B.5.3.3Post code94080
    B.5.3.4CountryUnited States
    B.5.6E-mailspencer.guthrie@prothena.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1100
    D.3 Description of the IMP
    D.3.1Product nameNEOD001
    D.3.2Product code NEOD001
    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 INNN/A
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeNEOD001
    D.3.9.3Other descriptive nameHumanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody
    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.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
    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
    Light chain (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
    (AL)implica trastorno hemático provocado por células plasmáticas clonales q produce pliegue defectuoso d cadenas ligeras de inmunoglobulinas. Sobreproducción d cadenas ligeras mal plegadas por parte d células plasmáticas resulta en formas solubles agregadas d CL y depósitos insolubles fibrilares d proteínas AL anómalas n tejidos y órganos. Puede causar gran variedad de síntomas e insuficiencias orgánicas, q incluyen insuficiencias cardíacas, renales y hepáticas, (GI), 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 dysfuction
    AL es un trastorno de la sangre, q causa daños progresivos en órganos como resultado del mal plegamiento de las proteínas. Esta enfermedad puede producir una gama de síntomas y Mala Función de órganos
    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 18.0
    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 NEOD001 plus standard of care vs. placebo plus standard of care when administered intravenously in subjects with AL amyloidosis by assessing time to all- cause mortality or cardiac hospitalization
    Evaluar la eficacia de NEOD001 más el tratamiento de referencia en
    comparación con placebo más el tratamiento de referencia cuando se
    administra por vía intravenosa a sujetos con amiloidosis AL; para
    ello se evaluará el tiempo hasta la muerte por cualquier causa o la
    hospitalización por causas cardíacas
    E.2.2Secondary objectives of the trial
    In subjects with AL amyloidosis, comparing NEOD001 plus standard of care vs. placebo plus standard of care:
    - To evaluate the cardiac response rate as assessed by NT-proBNP
    - To evaluate time to cardiac mortality or cardiac hospitalization
    - To evaluate cardiac functional response using the 6 Minute Walk Test (6MWT)
    - To evaluate general health-related quality of life using the Short Form 36 questionnaire (SF-36)
    - To evaluate cardiac-specific quality of life using the Kansas City Cardiomyopathy Questionnaire (KCCQ)
    - To evaluate the renal response rate using established criteria (Palladini 2014)
    - To evaluate the hepatic response rate according to consensus criteria (Comenzo 2012)
    En sujetos con amiloidosis AL, comparar NEOD001 más el tratamiento de referencia con placebo más el tratamiento de referencia:
    - Evaluar la tasa de respuesta cardíaca, evaluada mediante la fracción aminoterminal del péptido natriurético cerebral (NTproBNP)
    - Evaluar el tiempo hasta la muerte por causas cardíacas o la hospitalización por causas cardíacas
    - Evaluar la respuesta funcional cardíaca mediante la prueba de deambulación de 6 minutos (PD6M).
    - Evaluar la calidad de vida relacionada con la salud general por medio del formulario breve Short Form-36 (SF-36).
    - Evaluar la calidad de vida específica en cuanto a aspectos cardíacos utilizando el cuestionario de miocardiopatía de Kansas City (KCCQ)
    - Evaluar la tasa de respuesta renal por medio de criterios establecidos (Palladini 2014)
    - Evaluar la tasa de respuesta hepática conforme a los Criterios de consenso (Comenzo 2012)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria:
    1. Aged >= 18 years
    2. Newly diagnosed and AL amyloidosis treatment naive
    3. Bone marrow consistent with plasma cell dyscrasia within 42 days prior to Month 1-Day 1
    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 AND
    - Confirmatory immunohistochemistry OR mass spectroscopy of AL amyloidosis
    5. Confirmed diagnosis of AL amyloidosis by mass spectrometry of 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
    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
    - Echocardiogram demonstrating a mean left ventricular wall thickness > 12 mm in the absence of other cardiac cause to explain wall thickening (such as hypertension or cardiac valvular disease)
    - NT-proBNP >= 650 pg/mL in the absence of renal failure or atrial fibrillation with an uncontrolled ventricular rate
    7. Planned first-line chemotherapy contains bortezomib administered weekly and SC
    8. Adequate bone marrow reserve, hepatic and renal function, as demonstrated by: absolute neutrophil count (ANC) >= 1.0 x10^9/L
    - platelet count >=75 x 10^9/L
    - hemoglobin >=9 g/dL
    - total bilirubin <= 2 x upper limit of normal (ULN)
    - aspartate aminotransferase (AST) / serum glutamic oxaloacetic transaminase (SGOT) <=3 x ULN
    - alanine aminotransferase (ALT) / serum glutamic pyruvic transaminase (SGPT) <=3 x ULN
    - alkaline phosphatase (ALP) <= 5 x ULN (except for patients with hepatomegaly and isozymes specific to liver, rather than bone)
    - estimated glomerular filtration rate (eGFR) >=30 mL/min
    9. Seated systolic blood pressure 90-180 mmHg
    10. Distance walked during 6MWT is >=30 meters and <=550 meters
    11. Women of childbearing potential (WOCBP) must have two negative pregnancy tests during Screening, the second within 24 hours prior to the first administration of study drug and must agree to use physician-approved contraception from Screening to 30 days following the last study drug administration
    12. Male subjects must be surgically sterile or must agree to use physician-approved contraception from Screening to 30 days following the last study drug administration
    13. Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
    Criterios de inclusión (los sujetos deben cumplir todos los criterios siguientes):
    1. Edad >=18 años
    2. Con diagnóstico reciente y sin tratamiento previo para la amiloidosis AL
    3. Médula ósea indicativa de discrasia de células plasmáticas en los 42 días anteriores al día 1 del mes 1.
    4. Diagnóstico de amiloidosis AL confirmado mediante lo siguiente:
    - Diagnóstico histoquímico de amiloidosis determinado mediante microscopia de luz polarizada de material birrefringente verde en muestras de tejido teñidas de rojo Congo O BIEN aspecto característico en la microscopia electrónica Y TAMBIÉN
    - inmunohistoquímica confirmatoria O BIEN espectrometría de masas de amiloidosis AL.
    5. Diagnóstico confirmado de amiloidosis AL mediante espectrometría de masas de una biopsia tisular si el sujeto cumple alguno de los siguientes criterios:
    - es afroamericano o de raza negra
    - supera los 75 años de edad con gammapatía monoclonal concurrente
    - tiene antecedentes de amiloidosis familiar y presenta gammapatía monoclonal concurrente
    6. Afectación cardíaca definida por todos los siguientes:
    - Signos y síntomas clínicos documentados en el pasado o detectados en la actualidad indicativos de diagnóstico de insuficiencia cardíaca en el contexto de diagnóstico confirmado de amiloidosis AL en ausencia de una explicación alternativa para la insuficiencia cardíaca
    - Ecocardiografía que muestre un grosor medio de la pared ventricular izquierda > 12 mm en ausencia de otra causa cardíaca que explique el engrosamiento de la pared (como hipertensión o cardiovasculopatía)
    - NT-proBNP >= 650 pg/ml en ausencia de insuficiencia renal o fibrilación auricular con frecuencia ventricular no controlada
    7. La quimioterapia de primera línea prevista contiene bortezomib
    administrado semanalmente por vía subcutánea (s.c.)
    8. Reserva medular, función renal y hepática adecuadas, como se
    demuestra mediante:
    - cifra absoluta de neutrófilos (CAN) >=1,0 x109/l
    - cifra de trombocitos >=75 x 109/l;
    - hemoglobina >=9 g/dl
    - bilirrubina total <=2 veces el límite superior de la normalidad (LSN)
    - aspartato aminotransferasa (ASAT)/transaminasa glutámico oxalacética sérica (SGOT) <=3 veces el LSN
    - alanina aminotransferasa (ALAT)/transaminasa glutámico pirúvica sérica (SGPT) <=3 veces el LSN
    - fosfatasa alcalina (ALP) <=5 veces el LSN (excepto los pacientes con hepatomegalia e isoenzimas específicas del hígado, en vez de del hueso) tasa de filtración glomerular estimada (TFGe) >=30 ml/min
    9. Tensión arterial sistólica en posición sedente de 90-180 mmHg
    10. Distancia caminada durante la PD6M >= 30 metros y <= 550 metros
    11. Las mujeres fértiles (MF) deben contar con dos pruebas de embarazo negativas durante la selección, la segunda en las 24 horas anteriores a la primera administración del fármaco del estudio y deben acceder a utilizar métodos anticonceptivos aprobados por el médico desde el momento de la selección hasta 30 días después de la última administración del fármaco del estudio
    12. Los varones deben estar esterilizados quirúrgicamente o acceder a utilizar un método anticonceptivo aprobado por el médico desde el momento de la selección hasta 30 días después de la última administración del fármaco del estudio
    13. Capacidad para entender y voluntad de firmar un formulario de
    consentimiento informado antes de iniciar ningún procedimiento del estudio
    E.4Principal exclusion criteria
    Subjects must meet none of the following criteria:
    1. Non-AL amyloidosis
    2. NT-proBNP > 8,500 pg/mL
    3. Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma (see Appendix 11)
    *Note that subjects who meet the IMWG definition of symptomatic multiple myeloma with signs and/or symptoms attributable only to associated amyloidosis and who do not otherwise meet the criteria for diagnosis of smoldering myeloma are potentially eligible upon approval of the Sponsor.
    4. Subject is eligible for and plans to undergo ASCT
    5. Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject´s ability to safely receive treatment or complete study assessments
    6. Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic 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
    Prior to randomization, any ECG screening abnormalities must be noted as not clinically significant by the Investigator
    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 IV 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
    - Stage I prostate cancer that does not require treatment
    - Any other cancer from which the subject has been disease-free for >= 2 years
    15. History of Grade >= 3 infusion-associated AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine (or an equivalent H1 antihistamine) or acetaminophen (or its equivalent, paracetamol)
    16. Uncontrolled, active human immunodeficiency virus (HIV), Hepatitis B or Hepatitis C infection
    17. Prior treatment with NEOD001
    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
    Criterios de exclusión (los sujetos no deben cumplir ninguno los criterios siguientes):
    1. Amiloidosis no AL
    2. NT-proBNP > 8500 pg/ml
    3. Cumple la definición de mieloma múltiple del grupo internacional de trabajo sobre el mieloma (International Myeloma Working Group, IMWG) (véase el apéndice 11)
    *Cabe señalar que los sujetos que cumplen la definición de mieloma múltiple sintomático del IMWG con signos y/o síntomas atribuibles sólo a la amiloidosis asociada y que por lo demás no cumplen los criterios de diagnóstico de mieloma quiescente son potencialmente elegibles con la aprobación del promotor.
    4. El sujeto es apto para ATHB y prevé someterse a uno
    5. Hipotensión ortostática sintomática que, a juicio del investigador, interferiría con la capacidad del sujeto de recibir tratamiento de forma segura o de completar las evaluaciones del estudio
    6. Infarto de miocardio, angina no controlada, arritmias ventriculares muy descontroladas, o indicios electrocardiográficos de isquemia aguda, en los 6 meses anteriores a la visita del día 1 del mes 1
    7. Estenosis valvular grave (p. ej., estenosis aórtica o mitral con 1 área de válvula < 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:
    - Bloqueo AV de primer grado
    - Bloqueo AV de segundo grado de tipo 1 (tipo Mobitz 1 / tipo Wenckebach)
    - Bloqueo de rama izquierda o derecha
    - Fibrilación auricular con frecuencia ventricular controlada.
    Antes de la aleatorización, el investigador deberá anotar cualquier anomalía electrocardiográfica detectada en la selección como «no significativa desde el punto de vista clínico»
    9. Neuropatía periférica evaluada como de grado 2 con dolor, grado 3 o grado 4 según los Criterios terminológicos comunes para acontecimientos adversos del National Cancer Institute (CTCAE del NCI)
    10. El sujeto está recibiendo antibióticos, antimicóticos o antivíricos por vía oral o i.v. en la semana anterior al día 1 del mes 1 a excepción de los profilácticos orales
    11. Tratamiento previo con factores de crecimiento hematopoyético,
    transfusiones de sangre o hemoderivados en la semana anterior al día 1 del mes 1
    12. Radioterapia previa en las 4 semanas anteriores al día 1 del mes 1
    13. Cirugía mayor en las 4 semanas anteriores al día 1 del mes 1 o cirugía mayor prevista durante el estudio
    14. Neoplasia maligna activa a excepción de cualquiera de los siguientes:
    - Carcinoma basocelular, carcinoma epidermoide, o cáncer de cuello de útero localizado adecuadamente tratados
    - Cáncer en estadio 1 adecuadamente tratado del que el sujeto esté actualmente en remisión y que haya estado en remisión durante 2 años
    - Cáncer de próstata en estadio 1 que no requiere tratamiento
    - Cualquier otro tipo de cáncer del que el sujeto lleve recuperado >= 2 años
    15. Antecedentes de AA de grado >= 3 asociados a la infusión o hipersensibilidad a otro anticuerpo monoclonal o hipersensibilidad conocida a la difenhidramina (o un antihistamínico H1 equivalente) o al acetaminofeno (o su equivalente, el parecetamol)
    16. Infección activa no controlada por el VIH o los virus de la hepatitis B o C
    17. Tratamiento previo con NEOD001
    18. Tratamiento con otro fármaco experimental en los 30 días anteriores al día 1 del mes 1.
    19. Pacientes embarazadas o en periodo de lactancia.
    20. Cualquier afección que pudiera interferir, o cuyo tratamiento pudiera interferir, con la realización del estudio o que, en opinión del investigador, aumentaría de forma inaceptable el riesgo del sujeto al participar en el estudio
    E.5 End points
    E.5.1Primary end point(s)
    Time to all-cause mortality or cardiac hospitalization
    Tiempo hasta la muerte por cualquier causa o la hospitalización por causas cardíacas.
    E.5.1.1Timepoint(s) of evaluation of this end point
    As adjudicated by the CEC
    Según validación por el Comité de acontecimientos clínicos [CAC]
    E.5.2Secondary end point(s)
    - NT-proBNP
    - Time to cardiac mortality or cardiac hospitalization
    - 6MWT
    - SF-36
    - KCCQ
    - Renal best response
    - Hepatic best response
    -NT-proBNP
    -Tiempo hasta la muerte por causas cardíacas o la hospitalización por causas cardíacas
    - PD6M
    - SF-36
    - Cambio en el KCCQ
    - Mejor respuesta renal
    - Mejor respuesta hepática
    E.5.2.1Timepoint(s) of evaluation of this end point
    - NT-proBNP best response from baseline to Month 9
    - Time to cardiac mortality or cardiac hospitalization as adjudicated by the CEC
    - Change from baseline to Month 9 in the 6MWT
    - Change from baseline to Month 9 in the SF-36
    - Change from baseline to Month 9 in the KCCQ
    - Renal best response from baseline to Month 9 (Palladini 2014)
    - Hepatic best response from baseline to Month 9 (Comenzo 2012)
    - Tiempo hasta la muerte por causas cardíacas o la hospitalización por causas cardíacas según la validación de CAC
    - Cambio en la PD6M desde el inicio hasta el mes 9
    - Cambio en el SF-36 desde el inicio hasta el mes 9
    - Cambio en el KCCQ desde el inicio hasta el mes 9
    - Mejor respuesta renal desde el inicio hasta el mes 9 (Palladini 2014)
    - Mejor respuesta hepática desde el inicio hasta el mes 9 (Comenzo 2012)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic 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.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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Australia
    Austria
    Belgium
    Canada
    France
    Germany
    Greece
    Israel
    Italy
    Netherlands
    Poland
    Spain
    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
    The study will end when approximately 156 primary endpoint events have occurred and EOS visits have been completed for all subjects who remain on study at that time. Events are defined as deaths due to any cause, or cardiac hospitalizations as adjudicated by the CEC
    El estudio finalizará cuando se hayan producido aproximadamente 156 episodios del criterio principal de valoración y se hayan completado las visitas de FE para todos los sujetos que continúen en el estudio en ese momento. Los acontecimientos se definen como muertes por cualquier causa y hospitalizaciones por causas cardíacas, según la validación del CAC.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 200
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 236
    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 (i.e., once approximately 156 primary endpoint events have been reached), subjects still on treatment will be invited to enrol in an open-label extension study of NEOD001.
    En el momento de la finalización del estudio (es decir, una vez que se hayan alcanzado aproximadamente 156 episodios del criterio principal de valoración), se invitará a los sujetos que sigan recibiendo tratamiento a que participen en un estudio abierto de ampliación de NEOD001.
    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 Decision2015-07-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-07-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-06-08
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