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    Summary
    EudraCT Number:2016-004664-18
    Sponsor's Protocol Code Number:NEOD001-OLE251
    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:2017-05-29
    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 number2016-004664-18
    A.3Full title of the trial
    A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)
    Estudio de extensión, abierto y de fase IIb para evaluar la eficacia y la seguridad a largo plazo de NEOD001 en sujetos con amiloidosis de cadena ligera (AL) incluidos anteriormente en el estudio NEOD001-201 (PRONTO).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201
    Estudio para evaluar la eficacia y seguridad a largo plazo de NEOD001 en sujetos con amiloidosis AL que han completado el estudio NEOD001-201.
    A.4.1Sponsor's protocol code numberNEOD001-OLE251
    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 pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address331 Oyster Point Boulevard
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34689772554
    B.5.5Fax number+1650837 8560
    B.5.6E-mailclinicaltrials@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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201
    El objetivo de este estudio es evaluar la eficacia y seguridad a largo plazo de NEOD001 en sujetos con amiloidosis AL que han completado el estudio NEOD001-201.
    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 cadena ligera es una enfermedad de la sangre que causa daño orgánico progresivo debido al plegamiento anómalo de las proteinas. Puede producir variedad de 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
    The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201
    El objetivo de este estudio es evaluar la eficacia y seguridad a largo plazo de NEOD001 en sujetos con amiloidosis AL que han completado el estudio NEOD001-201.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria (subjects must meet all of the following criteria):
    1. Completed the EOS Visit in Study NEOD001-201
    2. Adequate bone marrow reserve, hepatic and renal function, as demonstrated by:
    - Absolute neutrophil count (ANC) ≥1.0 × 109/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
    - Alkaline phosphatase (ALP) ≤5 × ULN (except for subjects with hepatomegaly and isozymes specific to liver, rather than bone)
    - Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation, or measured GFR ≥25 mL/min/1.73 m2
    3. Systolic blood pressure 80-180 mmHg
    4. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective physician-approved contraception (Appendix 2) from Screening to 90 days following the last study drug administration
    5. Male subjects must be surgically sterile or must agree to use highly effective physician-approved contraception (Appendix 2) from Screening to 90 days following the last study drug administration
    6. Ability to understand and willingness to sign an informed consent form (ICF) prior to initiation of any study procedures
    Criterios de inclusión (los pacientes deberán cumplir todos los criterios siguientes):
    1. Haber completado la visita de FdE en el estudio NEOD001-201.
    2. Reserva medular, función hepática y renal suficientes, demostrado por:
    o Recuento absoluto de neutrófilos (RAN) ≥ 1,0 × 109/l
    o Cifra de plaquetas ≥ 75 × 109/l
    o Hemoglobina ≥ 9 g/dl
    o Bilirrubina total ≤ 2 veces el límite superior de la normalidad (LSN)
    o Aspartato-aminotransferasa (AST) ≤ 3 × LSN
    o Alanina-aminotransferasa (ALT) ≤ 3 × LSN
    o Fosfatasa alcalina (FA) ≤ 5 × LSN (excepto para los sujetos con hepatomegalia e isoenzimas hepáticas, no óseas)
    o Filtración glomerular estimada (FGe) ≥ 25 ml/min/1,73 m2 calculada mediante la fórmula de Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) o FG medida ≥ 25 ml/min/1,73 m2
    3. Presión arterial sistólica 80-180 mm Hg
    4. Las mujeres en edad fértil deben obtener un resultado negativo en la prueba de embarazo de la selección y deben aceptar utilizar métodos anticonceptivos altamente eficaces autorizados por el médico (Anexo 2) desde la selección hasta 90 días después de la última administración del fármaco del estudio.
    5. Los sujetos varones deben estar esterilizados quirúrgicamente o deben aceptar utilizar métodos anticonceptivos altamente eficaces autorizados por el médico (Anexo 2) desde la selección hasta 90 días después de la última administración del fármaco del estudio.
    6. Capacidad de entender y estar dispuestos a firmar el formulario de consentimiento informado (FCI) antes de empezar los procedimientos del estudio.
    E.4Principal exclusion criteria
    Exclusion Criteria (subjects must not meet any of the following criteria):
    1. Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiographic (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continuation or initiation of treatment with NEOD001 or participation in the study
    2. 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
    3. Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
    4. Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
    5. 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 (uncontrolled [i.e., >110 bpm] ventricular rate is not allowed [determined by an average of three beats in Lead II or 3 representative beats if Lead II is not representative of the overall ECG])
    6. Has not recovered (i.e., equivalent to a Common Terminology Criteria for Adverse Events [CTCAE] ≥Grade 2) from the clinically significant toxic effects of prior anticancer therapy. Exception: subjects who have received treatment with a proteasome inhibitor such as bortezomib may have CTCAE Grade 2 neuropathy.
    7. Received any of the following within the specified time frame prior to the Month 1-Day 1 Visit:
    - Oral or IV antibiotics, antifungals, or antivirals within 1 week, with the exception of prophylactic oral agents. Note: In the event that a subject requires the chronic use of antivirals, Medical Monitor permission is required for entry into the study.
    - Hematopoietic growth factors, transfusions of blood or blood products within 1 week
    - Chemotherapy, radiotherapy, HDAC inhibitors, or other plasma cell directed therapy within 2 weeks
    - ASCT within 4 weeks (i.e., ASCT is allowed if it occurred before enrollment in Study NEOD001-201 or after completion of Study NEOD001-201 if it was at least 4 weeks before Month 1-Day 1 of this study)
    - Major surgery within 4 weeks (or within 2 weeks following consultation with and approval of Medical Monitor)
    - Planned organ transplant during the study
    - Any investigational agent, other than NEOD001, within 4 weeks
    - Any experimental imaging agent directed at amyloid within 2 weeks
    8. 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 mg/mL
    - Any other cancer from which the subject has been disease-free for ≥2 years
    9. History of Grade ≥3 infusion-related adverse events (AEs) or hypersensitivity to NEOD001
    10. History of severe allergy to any of the components of NEOD001 such as histidine/L-Histidine, Trehalose, or Polysorbate 20
    11. Currently known uncontrolled bacterial, viral, fungal, HIV, hepatitis B, or hepatitis C infection
    12. Women who are breastfeeding
    13. 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
    14. Unable or unwilling to adhere to the study-specified procedures and restrictions
    15. Subject is under legal custodianship
    Criterios de exclusión (los sujetos no deberán cumplir ninguno de los criterios que se indican a continuación):
    1. Cualquier nueva contraindicación médica o anomalía clínicamente significativa en las exploraciones física y neurológica, los parámetros analíticos, las constantes vitales o el estudio electrocardiográfico (ECG) (p. ej., fibrilación auricular; con la excepción de los sujetos en los que la frecuencia ventricular esté controlada) que impida la continuación o la instauración del tratamiento con NEOD001 o la participación en el estudio.
    2. Hipotensión ortostática sintomática que, según el criterio médico del investigador, podría interferir en la idoneidad del sujeto para recibir tratamiento de forma segura o completar las evaluaciones del estudio.
    3. Infarto de miocardio, angina de pecho no controlada, arritmias ventriculares no controladas o signos ECG de isquemia aguda, en los 6 meses previos a la visita del día 1 del mes 1.
    4. Estenosis valvular grave (p. ej., estenosis mitral o aórtica con un área valvular < 1,0 cm2) o cardiopatía congénita importante.
    5. Signos ECG de isquemia aguda o anomalías activas del sistema de conducción, a excepción de lo siguiente:
    o Bloqueo auriculoventricular (AV) de primer grado.
    o Bloqueo AV de segundo grado tipo 1 (tipo 1 de Mobitz/tipo Wenckebach).
    o Bloqueo de rama izquierda o derecha.
    o Fibrilación auricular con una frecuencia ventricular controlada (no se permite una frecuencia ventricular no controlada, es decir, > 110 lpm [determinada como la media de tres latidos en la derivación II o tres latidos representativos si la derivación II no es representativa del ECG global]).
    6. No haberse recuperado (es decir, equivalente a un grado ≥ 2 según los criterios terminológicos comunes para acontecimientos adversos [CTCAE]) de los efectos tóxicos clínicamente significativos del tratamiento antineoplásico previo. Excepción: los sujetos que han recibido tratamiento con un inhibidor del proteasoma, como el bortezomib, pueden presentar neuropatía de grado 2 según los CTCAE.
    7. Haber recibido cualquiera de los siguientes fármacos en el intervalo de tiempo especificado antes de la visita del día 1 del mes 1:
    o Antibióticos, antifúngicos o antivíricos orales o intravenosos en 1 semana previa, a excepción de profilácticos orales.
    Nota: en caso de que el sujeto necesite el uso crónico de antivíricos, es necesario el permiso del monitor médico para la inclusión en el estudio.
    o Factores de crecimiento hematopoyético, transfusiones de sangre o hemoderivados en la semana previa.
    o Quimioterapia, radioterapia, inhibidores de las histonas deacetilasas (HDAC) o cualquier tratamiento dirigido a las células plasmáticas en los 2 meses previos.
    o ATCM en las 4 semanas previas (es decir, se permite el ATCM si se ha realizado antes de la inclusión en el estudio NEOD001-201 o después de la finalización del estudio NEOD001-201 si se produjo al menos 4 semanas antes del día 1 del mes 1 de este estudio).
    o Cirugía mayor en las 4 semanas previas (o en las 2 semanas previas, si el monitor médico lo autoriza).
    o Trasplante de órganos programado durante el estudio.
    o Cualquier otro fármaco en investigación, distinto de NEOD001, en las 4 semanas previas.
    o Un biomarcador de imagen experimental dirigido al amiloide en las 2 semanas previas.
    8. Neoplasia maligna activa, a excepción de los siguientes casos:
    o Carcinoma basocelular, epidermoide o cáncer cervical in situ tratados de manera suficiente.
    o Cáncer en estadio I tratado de manera suficiente y actualmente en remisión. El sujeto presenta remisión desde hace ≥ 2 años.
    o Cáncer de próstata de bajo riesgo con puntuación de Gleason < 7 y antígeno prostático específico < 10 mg/ml.
    oSin indicios de cualquier otro tipo de cáncer desde hace ≥ 2 años.
    9. Antecedentes de acontecimientos adversos (AA) relacionados con la infusión o hipersensibilidad a NEOD001 de grado ≥ 3.
    10. Antecedentes de alergia grave a cualquiera de los componentes de NEOD001, tales como histidina/L-histidina, trehalosa o polisorbato 20.
    11. Infección bacteriana, vírica, fúngica o por el VIH, hepatitis B o hepatitis C no controlada y conocida en curso.
    12. Mujeres en periodo de lactancia.
    13. Sujeto con cualquier otro trastorno o tratamiento para cualquier trastorno que pudiera interferir en la realización del estudio, o que, en opinión del investigador, aumentara de forma inaceptable el riesgo del sujeto por su participación en el estudio.
    14. Incapaz o poco dispuesto a seguir los procedimientos y restricciones especificados en el estudio.
    15. El sujeto se encuentra bajo custodia legal.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints:
    • Long-term safety and tolerability as assessed by vital signs, 12 lead ECGs, routine clinical laboratory assessments, and AEs
    • Immunogenicity

    Efficacy Endpoints:
    • N-terminal pro-brain natriuretic peptide (NT-proBNP):
    - Response (Appendix 3)
    - Best response from baseline
    - Change from baseline
    • Change from baseline in troponin T
    • Change from baseline in the Short Form-36 Health Survey version 2 (SF-36v2) Physical Component Score (PCS), Mental Component Score (MCS), and the 8 subscales
    • Change from baseline in the 6-Minute Walk Test (6MWT) distance (meters)
    • Progression-free survival
    • For renal-evaluable subjects:
    - Renal response (Appendix 3)
    - Renal best response from baseline
    - Change from baseline in creatinine, proteinuria, and eGFR
    - Time to eGFR:
    - ≤15 mL/min/1.73 m2 (Chronic Kidney Disease [CKD] Stage 5)
    - <30 mL/min/1.73 m2 (CKD Stage 4)
    - <60 mL/min/1.73 m2 (CKD Stage 3)
    - Time to any worsening in CKD Stage
    - Time to 40% reduction in eGFR
    - Time to doubling of creatinine
    • For peripheral neuropathy-evaluable subjects:
    - Change from baseline in Neuropathy Impairment Score–Lower Limbs (NIS-LL) total score
    - Peripheral neuropathy best response
    - Peripheral neuropathy response (Appendix 3)
    - For subjects with painful peripheral neuropathy in Study NEOD001-201 (i.e., baseline Visual Analog Scale – Pain Intensity [VASPI] score >0) change from baseline in the VASPI score
    • For hepatic-evaluable subjects:
    - Hepatic response (Appendix 3)
    - Hepatic best response from baseline
    • Time to all-cause mortality (overall survival)
    • Frequency and duration of hospitalizations over the course of the study
    • Change from baseline in the Kansas City Cardiomyopathy Questionnaire (KCCQ) subscores and overall summary score
    • Time to progression for each organ (cardiac/NT-proBNP, renal, peripheral neuropathy, hepatic) separately and to any organ progression
    • Eastern Cooperative Oncology Group (ECOG) Performance Status and New York Heart Association (NYHA) Class at each visit including any changes from baseline
    • Change from baseline in serum free light chains (sFLCs), serum and 24-hour urine protein electrophoresis (PEP), and serum and urine immunofixation electrophoresis (IFE)
    • Severity of disease-related symptoms including any changes from baseline
    • Pharmacokinetics (PK)
    Criterios de valoración de la seguridad:
    • Seguridad y tolerabilidad a largo plazo evaluadas mediante las constantes vitales, ECG de 12 derivaciones, evaluaciones analíticas clínicas rutinarias y AA.
    • Inmunogenia.

    Criterios de valoración de la eficacia:
    • Prohormona N-terminal del péptido natriurético cerebral (NT-proBNP):
    o Respuesta (Anexo 3)
    o Mejor respuesta respecto al periodo basal
    o Cambio respecto al periodo basal
    • Cambio respecto al periodo basal en el valor de la troponina T.
    • Cambio respecto al periodo basal en la puntuación del componente físico (Physical Component Score, PCS), la puntuación del componente mental (Mental Component Score, MCS) y las ocho subescalas de la versión 2 del cuestionario abreviado de salud de 36 ítems (Short Form-36 Health Survey version 2, SF-36v2).
    • Cambio con respecto al periodo basal en la distancia recorrida (en metros) en la prueba de marcha de 6 minutos (6-Minute Walk Test, 6MWT).
    • Supervivencia libre de progresión.
    • Para los sujetos en los que se evalúa la función renal:
    o Respuesta renal (Anexo 3)
    o Mejor respuesta renal respecto al periodo basal
    o Cambio respecto al periodo basal en los valores de creatinina, proteinuria y FGe
    o Tiempo transcurrido hasta obtener una FGe de:
     ≤ 15 ml/min/1,73 m2 (nefropatía crónica [NC] en estadio 5)
     < 30 ml/min/1,73 m2 (NC en estadio 4)
     < 60 ml/min/1,73 m2 (NC en estadio 3)
    o Tiempo transcurrido hasta el empeoramiento del estadio de la NC
    o Tiempo transcurrido hasta una reducción del 40 % en la FGe
    o Tiempo transcurrido hasta duplicarse la concentración de creatinina
    • Para sujetos en los que se evalúa la neuropatía periférica:
    o Cambio respecto al periodo basal en la puntuación total de la escala de afectación de la neuropatía en los miembros inferiores (Neuropathy Impairment Score-Lower Limbs, NIS-LL)
    o Mejor respuesta de la neuropatía periférica
    o Respuesta de la neuropatía periférica (Anexo 3)
    o Para sujetos con neuropatía periférica dolorosa en el estudio NEOD001-201 (es decir, puntuación basal en la escala analógica visual de intensidad del dolor [Visual Analog Scale – Pain Intensity, VASPI] > 0), cambio respecto al periodo basal en la puntuación VASPI
    • Para los sujetos en los que se evalúa la función hepática:
    o Respuesta hepática (Anexo 3)
    o Mejor respuesta hepática respecto al periodo basal
    • Tiempo transcurrido hasta la mortalidad por cualquier causa (supervivencia global).
    • Frecuencia y duración de las hospitalizaciones a lo largo del estudio.
    • Cambios con respecto al periodo basal en las subpuntuaciones y en la puntuación total de resumen del Cuestionario de cardiomiopatía de Kansas City (Kansas City Cardiomyopathy Questionnaire, KCCQ).
    • Tiempo transcurrido hasta la progresión para cada órgano (cardíaca/NT-proBNP, renal, neuropatía periférica, hepática) por separado y hasta la progresión para cualquier órgano.
    • Estado funcional del Eastern Cooperative Oncology Group (ECOG) y clase de la New York Heart Association (NYHA) en cada visita, incluidos los cambios respecto al periodo basal.
    • Cambio respecto al periodo basal en las cadenas ligeras libres en suero (CLLs), electroforesis de proteínas (EP) en suero y en orina de 24 horas, y electroforesis por inmunofijación (EI) en suero y en orina.
    • Intensidad de los síntomas relacionados con la enfermedad, incluidos los cambios respecto al periodo basal.
    • Farmacocinética (FC).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 38 months or until the study is terminated. Refer to Study Protocol, Table 1 Schedule of Assessments.
    Hasta 38 meses o hasta que el estudio haya terminada. Consúltese el protocolo del estudio, tabla 1 "Schedule of Assessments".
    E.5.2Secondary end point(s)
    Not applicable
    No aplica.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    No aplica.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic 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.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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    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 EEA17
    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
    France
    Germany
    Greece
    Israel
    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
    The study is expected to be completed approximately 3 years after the last subject is enrolled. The study is considered completed with the last assessment for the last subject participating in the study or if the study is terminated, whichever occurs first.
    Se espera completar el estudio aproximadamente 3 años despés del reclutamiento del úlitmo paciente. El estudio se considerará completado en la última revisión del último paciente que participe en el estudio o si el estudio decide terminarse, lo que ocurra antes.
    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 months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    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: 100
    F.1.3Elderly (>=65 years) No
    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 state11
    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)
    The study is expected to be completed approximately 3 years after the last subject is enrolled. The study is considered completed with the last assessment for the last subject participating in the study or if the study is terminated, whichever occurs first.

    The Sponsor reserves the right to terminate the study, participation of an individual Investigator, or a study site at any time for any reason. Conditions that may warrant termination are included in Section 4.6 of the protocol.
    Se espera completar el estudio alrededor de 3 años después del reclutamiento del úlitmo paciente. El estudio se considerará completado en la última revisión del último paciente que participe en el estudio o si el mismo decide terminarse, lo que ocurra antes.

    El promotor se reserva el derecho a terminar el estudio, la participación de un investigador o un centro en cualquier momento y por cualquier razón. Las condiciones para la terminación del estudio están en la sección 4.6 del protocolo.
    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 Decision2017-07-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-27
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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