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    Summary
    EudraCT Number:2017-004084-12
    Sponsor's Protocol Code Number:EFC15392
    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:2018-06-06
    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 number2017-004084-12
    A.3Full title of the trial
    Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Estudio multicéntrico, aleatorizado, doble ciego, controlado con placebo en dos etapas para caracterizar la eficacia, seguridad, tolerabilidad y farmacocinética de GZ/SAR402671 en pacientes con Enfermedad Renal Poliquística Autosómica Dominante (ERPAD) en riesgo de progresión rápida
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Estudio de eficacia, seguridad, tolerabilidad y farmacocinética de GZ/SAR402671 en pacientes con Enfermedad Renal Poliquística Autosómica Dominante (ERPAD) en riesgo de progresión rápida
    A.3.2Name or abbreviated title of the trial where available
    SAVE-PKD
    SAVE-PKD
    A.4.1Sponsor's protocol code numberEFC15392
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1202-0775
    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 SponsorGenzyme Corporation
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationsanofi-aventis, s.a.
    B.5.2Functional name of contact pointUnidad Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street Addressc/ Josep Pl nº2, 4ª planta
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.3.4CountrySpain
    B.5.4Telephone number93 485 94 00
    B.5.6E-mailES-unidadestudiosclinicos@sanofi.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVenglustat
    D.3.2Product code SAR402671, GZ402671 or GZ/SAR402671
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVenglustat
    D.3.9.1CAS number 1401090-53-6
    D.3.9.2Current sponsor codeGZ/SAR402671
    D.3.9.3Other descriptive nameGenz-682452-AU
    D.3.9.4EV Substance CodeSUB166602
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVenglustat
    D.3.2Product code SAR402671, GZ402671 or GZ/SAR402671
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVenglustat
    D.3.9.1CAS number 1401090-53-6
    D.3.9.2Current sponsor codeGZ/SAR402671
    D.3.9.3Other descriptive nameGenz-682452-AU
    D.3.9.4EV Substance CodeSUB166602
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Congenital, hereditary and neonatal diseases
    Enfermedades congénitas, hereditarias y neonatales
    E.1.1.1Medical condition in easily understood language
    Congenital, hereditary and neonatal diseases
    Enfermedades congénitas, hereditarias y neonatales
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10036046
    E.1.2Term Polycystic kidney, autosomal dominant
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the effect of GZ/SAR402671 on the rate of total kidney volume (TKV) growth and eGFR in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Determinar el efecto de GZ/SAR402671 sobre la tasa de crecimiento del volumen renal total (VRT) en pacientes con riesgo de ERPAD en riesgo de progresión rápida
    E.2.2Secondary objectives of the trial
    -To determine the effect of GZ/SAR402671 on the rate of renal function decline (Stage 1) and on the rate of TKV growth (Stage 2).
    -To evaluate the pharmacokinetics (PK) of GZ/SAR402671 in ADPKD patients (Stages 1 and 2).
    -Safety/tolerability objective:
    -To characterize the safety profile of GZ/SAR402671 (Stages 1 and 2).
    -To evaluate the effect of GZ/SAR402671 on mood using Beck Depression Inventory II (BDI-II) (Stages 1 and 2).
    -To evaluate the effect of GZ/SAR402671 on the lens by ophthalmological examination (Stages 1 and 2).
    -To evaluate change in nocturia based on patient reported diary (Stages 1 and 2).
    -To evaluate the effect of GZ/SAR402671 on kidney concentrating ability by assessing urine osmolality (in patients not on diuretic) (Stage 2 only).
    Determinar el efecto de GZ/SAR402671 sobre la tasa de descenso de la función renal (Etapa 1) y sobre la tasa de crecimiento del VRT (Etapa 2)
    Evaluar la farmacocinética (FC) de GZ/SAR402671 en los pacientes con ERPAD. (Etapa 1 y 2)
    Objetivos de seguridad/tolerabilidad:
    Determinar el perfil de seguridad global de GZ/SAR402671 (Etapa 1 y 2)
    Evaluar el efecto de GZ/SAR402671 sobre el estado de ánimo mediante BDI-II (Beck Depression Inventory-II [Inventario de Depresión de Beck II]). (Etapa 1 y 2)
    Evaluar el efecto de GZ/SAR402671 sobre el cristalino en la exploración oftalmológica. (Etapa 1 y 2)
    Evaluar el cambio en la nicturia en función del diario del paciente. (Etapa 1 y 2)
    Evaluar el efecto de GZ/SAR402671 sobre la capacidad de concentración renal mediante la evaluación de la osmolalidad de la orina (en el caso de pacientes sin diuréticos). (Solo Etapa 2)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Measured Glomerular Filtration Rate Substudy
    The main objective is to determine the effect of GZ/SAR402671 on mGFR.

    Please refer to the clinical trial protocol EFC15392, version 1 dated 08-Feb-2018 (appendix A).
    Subestudio de la tasa de filtración glomerular medible
    El objetivo principal es determinar el efecto de GZ / SAR402671 en mGFR.
    Ver más detalles en el protocolo EFC15392, version 1 dated 08-Feb-2018 (appendix A).
    E.3Principal inclusion criteria
    -Male or adult female between ages of 18-50 years (both inclusive).
    -Diagnosis of ADPKD by unified Pei criteria.
    -Mayo Imaging Classification of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Class 1C, 1D or 1E.
    -Estimated glomerular filtration rate between 45-90 mL/min/1.73 m2 at screening (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]).
    -Stable treatment regimen of antihypertensive therapy for at least 30 days prior to screening visit for hypertensive patient.
    -Able to read, comprehend and respond to the study questionnaires.
    -Able to provide a signed written informed consent.
    - Patient does not have access to tolvaptan at the time of study start or tolvaptan is not indicated for treatment of patient according to treating physician (patient does not meet recommended criteria for treatment or does not tolerate treatment with tolvaptan).
    -The patient, if female of childbearing potential, must have a negative blood pregnancy test [betahuman chorionic gonadotropin (β-hCG)] at the screening visit and a negative urine pregnancy test at the baseline visit.
    -Sexually active female patients of childbearing potential and sexually mature male patients must agree to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception for the entire duration of the study and for at least 6
    weeks for females and 90 days for males following their last dose of study drug.
    - Pacientes adultos varones o mujeres de edad entre 18 y 50 años (ambas inclusive).
    - Diagnóstico de ERPAD según los criterios de Pei unificados.
    - Clase 1C, 1D, o 1E de ERPAD según la clasificación mediante imágenes de Mayo.
    - Tasa de filtración glomerular estimada entre 45 y 90 ml/min/1,73 m2 en la selección (CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration [Grupo de colaboración epidemiológica en insuficiencia renal crónica]).
    - Régimen de tratamiento estable con terapia antihipertensiva durante al menos 30 días antes de la visita de selección para los pacientes hipertensos.
    - Ser capaz de leer, comprender y responder los cuestionarios del estudio.
    - Ser capaz de proporcionar un consentimiento informado firmado.
    - El paciente no tiene acceso a tolvaptán en el momento del inicio del estudio o tolvaptán no está indicado para el tratamiento del paciente de acuerdo con el médico responsable del tratamiento (el paciente no cumple los criterios recomendados para el tratamiento o no tolera el tratamiento con tolvaptán).
    - El paciente, si es mujer en edad fértil, debe tener una prueba de embarazo en sangre negativa [gonadotropina coriónica betahumana (β-hCG)] en la visita de selección y una prueba de embarazo negativa en la orina en la visita basal.
    -Las pacientes mujeres sexualmente activas en edad fértil y los pacientes varones sexualmente maduros deben aceptar practicar la abstinencia verdadera de acuerdo con su estilo de vida preferido y habitual o utilizar 2 métodos anticonceptivos eficaces aceptables durante toda la duración del estudio y durante al menos 6 semanas para mujeres y 90 días para hombres después de su última dosis del medicamento del estudio.
    E.4Principal exclusion criteria
    -Systolic blood pressure >160 mm Hg at Run-in and Baseline visits.
    -History of administration of tolvaptan or other Polycystic Kidney Disease-modifying agents (somatostatin analogues) within 3 months prior to screening visit.
    -Current participation in another investigational interventional study or use of investigational medicinal product (IMP), within 3 months or 5 half lives, whichever is longer, before randomization.
    -The patient has a documented diagnosis of any of the following infections: hepatitis B, hepatitis C, human immunodeficiency virus 1 or 2. Patients with a positive hepatitis B surface antibody (HBsAb) test with a history of prior hepatitis B immunization are eligible if other criteria are met (ie, negative
    tests for: HBsAg, hepatitis B core antibody [HBcAb], and hepatitis C virus antibody [HCVAb]).
    -A history of drug and/or alcohol abuse within the past year prior to the screening visit.
    -The patient is scheduled for in-patient hospitalization including elective surgery, during the study.
    -The patient has a medical condition, including significant intercurrent illness or any other extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
    -The patient, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (eg, has contraindications to pupillary dilation or unable to undergo magnetic resonance imaging (MRI ) [For example: patient’s weight exceeds weight capacity of the MRI, ferromagnetic metal prostheses, aneurysm clips, severe claustrophobia, large abdominal/back tattoos, etc]).
    -Any country-related specific regulation that would prevent the patient from entering the study.
    -The patients did not adhere to treatment (<70% compliance rate) in the run-in.
    -The patient has, according to World Health Organization (WHO) Grading, a cortical cataract >onequarter of the lens circumference (Grade cortical cataract-2 [COR-2]) or a posterior subcapsular cataract >2 mm (Grade posterior subcapsular cataract-2 [PSC-2]). Patients with nuclear cataracts will not be excluded.
    -The patient is currently receiving potentially cataractogenic medications, including a chronic regimen (more frequently than every 2 weeks) of any route of corticosteroids (including medium and high potency topical steroids) or any medication that may cause cataract, according to the Prescribing Information.
    -The patient has received strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to randomization. This also includes the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting GZ/SAR402671 administration.
    -The patient is pregnant, or lactating.
    -Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal unless the patient has the diagnosis of Gilbert syndrome.
    -Presence of severe depression as measured by BDI-II >28 and/or a history of a major affective disorder within 1 year of the screening visit.
    -Known hypersensitivity to GZ/SAR402671 or any component of the excipients.
    - PAS > 160 mmHg en las visitas de preinclusión e inicial.
    - Antecedentes de administración de tolvaptán o de otros fármacos modificadores de la poliquistosis renal (análogos de la somatostatina) en los 3 meses anteriores a la visita de selección.
    - Participación actual en otro ensayo o uso de medicamento en investigación (IMP), dentro de los 3 meses o 5 vidas medias, el que sea mayor, antes de la aleatorización.

    -El paciente tiene un diagnóstico documentado de cualquiera de las siguientes infecciones: hepatitis B, hepatitis C, virus de la inmunodeficiencia humana 1 o 2. Los pacientes con una prueba de anticuerpos de superficie de hepatitis B positiva (HBsAb) con antecedentes de inmunización previa contra la hepatitis B son elegibles si se cumplen otros criterios (es decir, pruebas negativas para: HBsAg, anticuerpo de núcleo de hepatitis B [HBcAb] y anticuerpo del virus de la hepatitis C [HCVAb]).

    -Una historia de abuso de drogas y / o alcohol durante el año anterior a la visita de selección.

    -El paciente está programado para la hospitalización del paciente, incluida la cirugía electiva, durante el estudio.

    - El paciente tiene una afección médica, incluidas enfermedades intercurrentes significativas o cualquier otra circunstancia atenuante que pueda interferir significativamente con el cumplimiento del estudio, incluidas todas las evaluaciones prescritas y las actividades de seguimiento.
    - El paciente, en opinión del Investigador, es incapaz de cumplir los requisitos del estudio o no puede someterse a las evaluaciones del estudio (p. ej., tiene contraindicaciones a la dilatación pupilar o no puede someterse a una resonancia magnética [RM] [Por ejemplo: el peso del paciente supera la capacidad de peso de la RM, prótesis de metal ferromagnético, clips de aneurisma, claustrofobia severa, tatuajes grandes en abdomen/espalda]).
    -Cualquier regulación específica relacionada con el país que evitaría que el paciente ingrese al estudio.
    -Los pacientes no se adhirieron al tratamiento (tasa de cumplimiento <70%) en el período inicial.
    - El paciente presenta, según la clasificación de la Organización Mundial de la Salud (OMS), una catarata cortical > 1 cuarto de la circunferencia del cristalino (catarata cortical de grado 2 [cortical cataract-2, COR-2]) o una catarata subcapsular posterior > 2 mm (catarata subcapsular posterior de grado 2 [posterior subcapsular cataract-2, PSC-2]). No se excluirá a los pacientes con cataratas nucleares.
    - El paciente está recibiendo actualmente medicación potencialmente cataratogénica, incluidas las pautas crónicas (con una frecuencia superior a cada 2 semanas) de corticoesteroides por cualquier vía (incluyendo corticoesteroides tópicos de potencia media o alta) o cualquier medicamento que pueda provocar cataratas, según la información de prescripción.
    - El paciente ha recibido inductores o inhibidores potentes o moderados de CYP3A4 en los 14 días o 5 semividas, lo que sea mayor, antes de la aleatorización. Esto también incluye el consumo de pomelo, zumo de pomelo o productos con pomelo durante las 72 horas previas a la administración de la primera dosis de GZ/SAR402671.
    -La paciente está embarazada o periodo de lactancia
    -Enzimas hepáticas (alanina aminotransferasa [ALT] / aspartato aminotransferasa [AST]) o bilirrubina total> 2 veces el límite superior de la normal a menos que el paciente tenga el diagnóstico del síndrome de Gilbert.
    -Presencia de depresión severa medida por BDI-II> 28 y / o antecedentes de un trastorno afectivo importante dentro de 1 año de la visita de selección.
    -Hipersensibilidad conocida a GZ / SAR402671 o cualquier componente de los excipientes.
    E.5 End points
    E.5.1Primary end point(s)
    1. Annualized rate of change in total kidney volume (TKV) based on magnetic resonance Imaging (MRI) from baseline to 18 months (Stage 1)
    2. Annualized rate of change in estimated glomerular filtration rate (eGFR) (Chronic Kidney Disease
    Epidemiology Collaboration [CKD-EPI] equation) from baseline to 24 months (Stage 2)
    La tasa anualizada del cambio del VRT según la RM desde el inicio hasta los 18 meses. (Etapa 1)
    La tasa anualizada del cambio de la TFGe (ecuación de CKD-EPI) desde el inicio hasta los 24 meses. (Etapa 2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. From baseline to 18 months
    2. From baseline to 24 months
    1. Desde el inicio hasta los 18 meses
    2. Desde el inicio hasta los 24 meses
    E.5.2Secondary end point(s)
    1. Annualized rate of change in eGFR (CKD-EPI equation) from baseline to 18 months (Stage 1) : From baseline to 18 months
    2. Annualized rate of change in TKV based on MRI from baseline to 24 months (Stage 2) : From baseline to 24 months
    3. Change in urine osmolality from baseline to 24 months (in patients not on diuretic) (Stage 2) : From baseline to 24 months
    4. Change in nocturia from baseline to 18 months (Stage 1) and from baseline to 24 months (Stage 2) :
    Stage 1: From baseline to 18 months
    Stage 2: From baseline to 24 months
    5. Number of adverse events (Stages 1 and 2) :
    Stage 1: From baseline to 18 months
    Stage 2: From baseline to 24 months
    6. Assessment of single time-point plasma concentration (Stages 1 & 2) : Stage 1: Day 1, Months 1, 6, and 18
    Stage 2: Months 6 and 24
    7. Change in the lens clarity from baseline by ophthalmological examination (Stages 1 and 2) :
    Stage 1: From baseline to 18 months
    Stage 2: From baseline to 24 months
    1. La tasa anualizada del cambio de la TFGe (ecuación de CKD-EPI) desde el inicio hasta los 18 meses. (Etapa 1): desde el inicio hasta los 18 meses
    2. La tasa anualizada del cambio del VRT según la RM desde el inicio hasta los 24 meses. (Etapa 2): desde el inicio hasta los 24 meses

    3. Cambio en la osmolalidad urinaria desde el inicio hasta 24 meses (en pacientes que no toman diurético) (Etapa 2): desde el inicio hasta los 24 meses
    4. Cambio en la nicturia desde el inicio hasta los 18 meses (Etapa 1) y desde el inicio hasta los 24 meses (Etapa 2):
    Etapa 1: desde el inicio hasta los 18 meses
    Etapa 2: desde el inicio hasta los 24 meses
    5. Número de acontecimientos adversos (Etapas 1 y 2):
    Etapa 1: desde el inicio hasta los 18 meses
    Etapa 2: desde el inicio hasta los 24 meses
    6. Evaluación de la concentración plasmática en un único punto de tiempo (Etapas 1 y 2): Etapa 1: Día 1, Meses 1, 6 y 18
    Etapa 2: Meses 6 y 24
    7. Cambio en la claridad de la lente desde la línea de base por examen oftalmológico (Etapas 1 y 2):
    Etapa 1: desde el inicio hasta los 18 meses
    Etapa 2: desde el inicio hasta los 24 meses
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to the section E.5.2.
    Ver sección E.5.2.
    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 Yes
    E.6.10Pharmacogenetic Yes
    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 concerned3
    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
    Belgium
    Canada
    China
    Czech Republic
    Denmark
    France
    Germany
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Portugal
    Romania
    Spain
    Switzerland
    Taiwan
    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
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial 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: 836
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 836
    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)
    None
    Ninguno
    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 Decision2018-06-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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