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    Summary
    EudraCT Number:2015-004318-14
    Sponsor's Protocol Code Number:NEOD001-201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-03-18
    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 number2015-004318-14
    A.3Full title of the trial
    A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction
    Estudio en fase 2b, aleatorizado, de doble ciego y controlado con placebo sobre NEOD001 en pacientes tratados previamente con amiloidosis de cadena ligera (AL) que presentan insuficiencia cardiaca persistente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study is to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.
    Un estudio es evaluar si NEOD001 es seguro y efectivo en pacientes con amiloidosis de cadena ligera AL que les afecta al corazón.
    A.4.1Sponsor's protocol code numberNEOD001-201
    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.5.4Telephone number+16506152106
    B.5.5Fax number+16508378560
    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.3.4Pharmaceutical 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 drip use (Noncurrent)
    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 or primary systemic amyloidosis, involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains by plasma cells results in both soluble, aggregated forms of light chains and insoluble, fibrillar deposits of abnormal AL protein (amyloid), in the tissues and organs of individuals with AL amyloidosis.
    La amiloidosis de cadena ligera (AL) o amiloidosis sistémica primaria, implica un trastorno hematológico causado por células plasmáticas clonales que producen cadenas ligeras de inmoglobulina mal plegadas. La producción excesiva de cadenas ligeras mal plegadas por parte de las células plasmáticas resulta en formas agregadas de cadenas ligeras solubles y depósitos fibrilares de proteínas AL anómalas (amiloides) insolubles en los tejidos y órganos de personas con amiloidosis AL.
    E.1.1.1Medical condition in easily understood language
    AL Amyloidosis is a relatively rare blood disease in which deposits of abnormal proteins, called amyloid, can build up in your organs and cause progressive damage so that they no longer work properly.
    La amiloidosis AL es una enfermedad de la sangre relativamente rara por la que se pueden formar depósitos de proteínas anormales, denominadas amiloide, en los órganos, lo que podría causar en daños.
    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 18.1
    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
    The objective of this study is to determine the efficacy and safety of NEOD001 versus placebo in subjects with AL amyloidosis who have persistent cardiac dysfunction.
    El objetivo de este estudio es determinar la eficacia y la seguridad de NEOD001 frente al placebo en pacientes con amiloidosis de cadena ligera (AL) que presentan insuficiencia cardíaca persistente.
    E.2.2Secondary objectives of the trial
    • Change from baseline to Month 12 in the Physical Component Score of the Short Form-36 (SF-36)
    • Change from baseline to Month 12 in the 6-minute walk test (6MWT)
    • For renal-evaluable subjects, renal best response from baseline to Month 12
    • For subjects with peripheral neuropathy, change from baseline to Month 12 in their Neuropathy Impairment Score–Lower Limbs (NIS-LL) score
    • For subjects with painful peripheral neuropathy, change from baseline to Month 12 in their VASPI score
    • Change from baseline to Month 12 in the Kansas City Cardiomyopathy Questionnaire (KCCQ)
    • Progression free survival (combined cardiac and renal progression) at Month 12
    • Assessment of the safety and tolerability of NEOD001 as assessed by vital signs, duration of therapy
    • Cambio desde el valor basal al del Mes 12 en la puntuación del componente físico del Formulario Breve 36 (SF-36)
    • Cambio desde el valor inicial al del Mes 12 en el test de marcha de 6 minutos (6MWT)
    • En los pacientes a los que se les puede evaluar la función renal, mejor respuesta renal desde el valor inicial al del Mes 12
    • Para los pacientes con neuropatía periférica, cambio desde el valor inicial al del Mes 12 en su puntuación en disfunción por neuropatía, extremidades inferiores (NIS-LL)
    • Para los pacientes con neuropatía periférica dolorosa, cambio desde el valor inicial al del Mes 12 en su puntuación en VASPI
    • Cambio desde el valor inicial al del Mes 12 en el Cuestionario de Cardiomiopatía de Kansas City (KCCQ)
    • Supervivencia libre de progresión (progresión cardíaca y renal combinadas) en el Mes 12.
    • Evaluación de la seguridad y la tolerabilidad de NEOD001 evaluadas por las constantes vitales, la duración del tratamiento.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria:

    1. Age ≥18 years

    2. Confirmed diagnosis of systemic 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 electron microscopy immunohistochemistry OR mass spectroscopy of AL amyloidosis

    3. Confirmed diagnosis of AL amyloidosis by mass spectrometry of tissue biopsy (if archival tissue is not available, a biopsy is required) 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

    4. 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 consistent with AL amyloidosis OR an echocardiogram demonstrating a mean left ventricular wall thickness in diastole >12 mm in the absence of other causes (e.g., severe hypertension, aortic stenosis) which would adequately explain the degree of wall thickening
    - NT-proBNP ?650 pg/mL in the absence of renal failure defined by an eGFR <30 mL/min/1.73 m2 or atrial fibrillation with an uncontrolled ventricular rate, as defined by >110 beats per minute

    5. NT-proBNP <5000 pg/mL

    6. Received at least one prior systemic chemotherapeutic regimen, which may include stem cell transplant, for AL amyloidosis

    7. Achieved at least a partial hematologic response to a previous therapy resulting in a stable hematologic condition not currently requiring additional active treatment against the PCD component of their AL disease

    8. Adequate bone marrow reserve, hepatic and renal function, as demonstrated by:
    - Absolute neutrophil count (ANC) ≥1.0 x109/L
    - Platelet count ≥75 × 109/L
    - Hemoglobin ≥9 g/dL
    - Total bilirubin ≤2 × upper limit of normal (ULN)
    - Aspartate aminotransferase (AST) ≤3 × ULN
    - Alanine aminotransferase (ALT) ≤3 × ULN
    - Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation

    9. Systolic blood pressure (BP) 90-180 mmHg

    10. Distance walked during 6MWT is >100 meters and <600 meters

    11. 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

    12. 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

    13. Ability to understand and willingness to sign an ICF prior to initiation of any study procedures
    Los pacientes deben cumplir todos los criterios siguientes:

    1. Edad: ≥18 años

    2. Diagnóstico confirmado de amiloidosis AL por medio de los siguientes:
    o Diagnóstico histoquímico de amiloidosis determinado por la polarización en un microscopio óptico de material birrefringente verde en muestras de tejido con tinción roja del Congo O BIEN apariencia característica bajo microscopio electrónico.
    Y
    o Confirmación inmunohistoquímica al microscopio electrónico O BIEN espectroscopia de masas de amiloidosis AL.

    3. Diagnóstico de amiloidosis AL confirmado mediante espectrometría de masas de una biopsia de tejido (si no hay un archivo de tejido, se necesita realizar una biopsia) si el paciente cumple cualquiera de lo siguiente:
    o Es de raza negra o afroamericano
    o Tiene más de 75 años y gammapatía monoclonal concurrente
    o Tiene antecedentes familiares de amiloidosis y gammapatía monoclonal concurrente

    4. Implicación cardíaca definida por TODOS los siguientes:
    o Signos y síntomas documentados en el pasado u observados en la actualidad que indiquen un diagnóstico de insuficiencia cardíaca en el marco de un diagnóstico confirmado de amiloidosis AL en ausencia de una explicación alternativa para la insuficiencia cardíaca
    o Biopsia de endomiocardio coherente con la amiloidosis AL O BIEN un ecocardiograma que demuestre un grosor de la pared ventricular izquierda en diástole superior a 12 mm en ausencia de otras causas (como hipertensión grave, estenosis aórtica) que pudieran explicar adecuadamente el grado de engrosamiento de la pared
    o NT-proBNP ≥650 pg/ml en ausencia de insuficiencia renal definida por eGFR <30 ml/min/1,73 m2 o fibrilación auricular con una velocidad ventricular no controlada, definida por >110 latidos por minuto

    5. NT-proBNP <5000 pg/mL

    6. Ha recibido al menos un ciclo anterior de quimioterapia terapéutica, que puede incluir trasplante de células madre, para la amiloidosis AL

    7. Ha logrado al menos una respuesta hematológica parcial a un tratamiento anterior que haya dado como resultado un estado hematológico estable que en la actualidad no requiera de tratamiento activo adicional contra el componente de PCD de su enfermedad de AL

    8. Reserva de médula ósea y función hepática y renal adecuadas según se demuestre por:
    o Recuento de neutrófilos absoluto (ANC) ≥1,0 x109/l
    o Recuento plaquetario ≥75 × 109/l
    o Hemoglobina ≥9 g/dl
    o Bilirrubina total ≤2 × límite superior de la normalidad (ULN, por sus siglas en inglés)
    o Aspartato aminotransferasa (AST) ≤3 × ULN
    o Alanina aminotransferasa (ALT) ≤3 × ULN
    o Tasa calculada de filtración glomerular (eGFR, por sus siglas en inglés) ≥30 ml/min/1,73 m2 según se define en la ecuación de la Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)

    9. Tensión arterial sistólica comprendida entre 90 y 180 mmHg

    10. Distancia recorrida durante el test de marcha de 6 minutos >100 metros y <600 metros

    11. Las mujeres en edad fértil (WOCBP, por sus siglas en inglés) deben tener dos pruebas negativas de embarazo durante la selección, la segunda en las 24 horas anteriores a la primera administración del fármaco del estudio; y deben aceptar utilizar un método anticonceptivo de alta eficacia aprobado por un médico desde la selección, hasta 90 días después de la última administración del fármaco del estudio.

    12. Los pacientes masculinos deben ser quirúrgicamente estériles o aceptar utilizar un método anticonceptivo de alta eficacia aprobado por un médico desde la selección, hasta 90 días después de la última administración del fármaco del estudio.

    13. Capacidad para comprender y voluntad para firmar un formulario de consentimiento informado antes de iniciar cualquier procedimiento del estudio.
    E.4Principal exclusion criteria
    Subjects must not meet any of the following criteria:

    1. Diagnosis of non-AL amyloidosis

    2. Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma

    3. 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

    4. Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit

    5. Severe valvular stenosis (e.g. aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease

    6. ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
    - First degree atrioventricular (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
    Note: Prior to randomization, any ECG screening abnormalities must be reviewed and designated as either "clinically significant" or "not clinically significant" by the Investigator

    7. Has not recovered (i.e., equivalent to a CTCAE Grade 1 [mild] or better) from the clinically significant toxic effects of prior anticancer therapy. Exception: subjects with prior bortezomib treatment may have CTCAE Grade 2 neuropathy.

    8. Received any of the following within the specified time frame prior to the Month 1-Day 1 Visit:
    - Oral or intravenous antibiotics, antifungals, or antivirals within 1 week, with the exception of prophylactic oral agents
    - Hematopoietic growth factors, transfusions of blood or blood products within 1 week
    - Chemotherapy, radiotherapy, or other plasma cell directed therapy within 6 months
    - ASCT within 12 months
    - Major surgery within 4 weeks or planned major surgery during the study
    - Any other investigational agent within 4 weeks
    - Prior treatment with NEOD001, 11-1F4, anti-serum amyloid P antibody, or other investigational treatment directed at amyloid

    9. 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

    10. History of Grade ≥3 infusion-related AEs or hypersensitivity to another monoclonal antibody

    11. Known prior allergic or infusion reaction to any of the study drug excipients

    12. Uncontrolled bacterial, viral, fungal, HIV, hepatitis B, or hepatitis C infection

    13. Women who are breastfeeding

    14. 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

    15. Unable or unwilling to adhere to the study-specified procedures and restrictions

    16. Subject is under legal custodianship
    Los pacientes no deben cumplir ninguno de los criterios siguientes:

    1. Diagnóstico de no amiloidosis AL

    2. Cumplir la definición de mieloma múltiple definido por el Myeloma Working Group (IMWG)

    3. Hipotensión ortostática sintomática que, a juicio médico del investigador, pudiera interferir con la capacidad del paciente para recibir de forma segura el tratamiento o completar las evaluaciones del estudio.

    4. Infarto de miocardio, angina sin controlar, arritmias ventriculares sin controlar o evidencia electrocardiográfica de isquemia aguda, en los 6 meses anteriores a la visita del Día 1 del Mes 1.

    5. Estenosis vascular grave (p. ej., estenosis aórtica o mitral con un área de válvula <1,0 cm2) o enfermedad cardíaca congénita grave

    6. Evidencias ECG de isquemia aguda o anomalías del sistema de conducción activa con la excepción de cualquiera de lo siguiente:
    o Bloqueo auriculoventricular (AV) de primer grado
    o Bloqueo AV de segundo grado (de Mobitz de tipo 1/o de tipo Wenckebach)
    o Bloqueo de rama derecha o izquierda
    o Fibrilación auricular con una velocidad ventricular controlada
    Nota: Antes de la aleatorización, cualquier anomalía en el ECG de selección se debe revisar y designar como "clínicamente significativo" o "no clínicamente significativo" por el investigador

    7. No se ha recuperado (es decir, equivalente a un CTCAE de grado 1 [leve] o superior) de los efectos tóxicos clínicamente significativos del tratamiento contra el cáncer anterior Excepción: los pacientes con tratamiento de bortezomib anterior pueden tener neuropatía de grado 2 según la CTCAE.

    8. Ha recibido alguno de los siguientes dentro del periodo especificado antes de la visita del Día 1 del Mes 1:
    o Antibióticos orales o intravenosos, antimicóticos o antivíricos la semana anterior, con la excepción de agentes profilácticos orales.
    o Factores de crecimiento hematopoyético, transfusiones de sangre o hemoderivados la semana anterior
    o Quimioterapia, radioterapia u otro tratamiento dirigido a las células plasmáticas los 6 meses antes
    o ASCT los 12 meses antes
    o Cirugía mayor las 4 semanas antes o cirugía mayor prevista durante el estudio
    o Cualquier agente en investigación las 4 semanas antes
    o Antes del tratamiento con NEOD001, anticuerpo P amiloide antisuero 11-1F4 u otro tratamiento en investigación dirigido al amiloide

    9. Tumor maligno activo con la excepción de cualquiera de lo siguiente:
    o Carcinoma de células basales tratado adecuadamente, carcinoma de células escamosas o cáncer cervical in situ
    o Cáncer en estadio I tratado adecuadamente que esté actualmente en remisión o lleve en remisión ≥2 años.
    o Cáncer de próstata de estadio 1 que no requiera tratamiento
    o Cualquier otro cáncer del que el paciente lleve libre ≥2 años

    10. Antecedentes de AA de grado 3 o superior relacionados con la perfusión o hipersensibilidad a otro anticuerpo monoclonal

    11. Alergia o reacción anterior conocida a la perfusión de cualquiera de los excipientes del fármaco del estudio

    12. Infección bacteriana, vírica, fúngica, por VIH, hepatitis B o hepatitis C sin controlar

    13. Mujeres en periodo de lactancia

    14. Cualquier afección que pudiera interferir, o que el tratamiento de la misma pueda interferir con la realización del estudio o que pudiera, a juicio del investigador, aumentar de forma inaceptable el riesgo del paciente al participar en el estudio

    15. Incapacidad o falta de voluntad para cumplir los procedimientos y las restricciones especificadas por el estudio

    16. El paciente se encuentra bajo tutela legal
    E.5 End points
    E.5.1Primary end point(s)
    NT-proBNP best response
    Mejor respuesta del NT-proBNP
    E.5.1.1Timepoint(s) of evaluation of this end point
    NT-proBNP best response from baseline to Month 12
    Mejor respuesta del NT-proBNP desde el valor basal al del Mes 12
    E.5.2Secondary end point(s)
    - SF-36
    - 6MWT
    - Renal best response
    - Peripheral neuropathy score
    - VASPI score
    - KCCQ
    - Progression free survival (combined cardiac and renal progression)
    - Assessment of the safety and tolerability of NEOD001 as assessed by vital signs
    • SF-36
    • 6MWT
    • Mejor respuesta renal
    • Disfunción por neuropatía
    • Puntuación en VASPI
    • KCCQ
    • Supervivencia libre de progresión
    • Evaluación de la seguridad y la tolerabilidad de NEOD001 evaluadas por las constantes vitales
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Change from baseline to Month 12 in the Physical Component Score of the SF-36
    - Change from baseline to Month 12 in the 6MWT
    - For renal-evaluable subjects, renal best response from baseline to Month 12
    - For subjects with peripheral neuropathy, change from baseline to Month 12 in their NIS-LL score
    - VASPI score: baseline to Month 12
    - KCCQ: baseline to Month 12
    - Progression free survival at Month 12
    - Assessment of the safety and tolerability of NEOD001 as assessed by vital signs, duration of therapy, 12-lead ECGs, routine laboratory assessments, and frequency and severity of AEs as assessed by CTCAE grade
    • Cambio desde el valor basal al del Mes 12 en la puntuación del componente físico del SF-36
    • Cambio desde el valor inicial al del Mes 12 en 6MWT
    • En los pacientes a los que se les puede evaluar la función renal, mejor respuesta renal desde el valor inicial al del Mes 12
    • Para los pacientes con neuropatía periférica, cambio desde el valor inicial al del Mes 12 en su puntuación en NIS-LL
    • VASPI: valor inicial al del Mes 12
    • KCCQ: valor inicial al del Mes 12
    • Supervivencia libre de progresión en el Mes 12
    • Evaluación de la seguridad y la tolerabilidad de NEOD001 evaluadas por las constantes vitales, la duración del tratamiento, ECG de 12 derivaciones, pruebas de laboratorio rutinarias, frecuencia y gravedad de los AA evaluados por el CTCAE
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    France
    Germany
    Greece
    Italy
    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
    LVLS

    The study will end when the last subject completes 12 months of study treatment and the 30-day safety follow-up period (i.e., EOS Visit). After completing 12 months of treatment and the EOS Visit, a subject may be considered for entry into an open-label extension study, during which subjects will receive active treatment and may receive concurrent chemotherapy.
    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 months6
    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: 93
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 7
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 55
    F.4.2.2In the whole clinical trial 100
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After completing the Month 12 Visit, a subject may be considered for entry into an open-label extension study, during which subjects may receive concurrent chemotherapy.
    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 Decision2016-03-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-17
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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