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    Summary
    EudraCT Number:2021-000621-27
    Sponsor's Protocol Code Number:RTRX-RE021-201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2021-07-27
    Trial 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 number2021-000621-27
    A.3Full title of the trial
    A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects with Selected Proteinuric Glomerular Diseases (EPPIK).

    P/024/2021
    Estudio de cohortes de fase 2, con un solo grupo, abierto para evaluar la seguridad, la eficacia y la
    farmacocinética del tratamiento con esparsentán en pacientes pediátricos con determinadas
    enfermedades glomerulares proteinúricas (EPPIK).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, efficacy and pharmacokinetics of sparsentan in pediatric subjects with selected kidney diseases.
    la seguridad, la eficacia y la farmacocinética del tratamiento con esparsentán en pacientes pediátricos con determinadas enfermedades de riñon
    A.3.2Name or abbreviated title of the trial where available
    EPPIK
    A.4.1Sponsor's protocol code numberRTRX-RE021-201
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/021/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorTravere Therapeutics, Inc.
    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 supportTravere Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTravere Therapeutics, Inc.
    B.5.2Functional name of contact pointTravere Therapeutics Call Center
    B.5.3 Address:
    B.5.3.1Street Address3611 Valley Centre Drive, Suite 300
    B.5.3.2Town/ citySan Diego, California
    B.5.3.3Post code92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1877659 5518
    B.5.6E-mailmedinfo@travere.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/3/15/1574 - FSGS EU/3/20/2345 - IGAN
    D.3 Description of the IMP
    D.3.1Product nameSparsentan
    D.3.2Product code RE-021
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNSparsentan
    D.3.9.1CAS number 254740-64-2
    D.3.9.2Current sponsor codeRE-021
    D.3.9.3Other descriptive nameSPARSENTAN
    D.3.9.4EV Substance CodeSUB168607
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Proteinuric glomerular diseases including:

    •Focal segmental glomerulosclerosis (FSGS)
    •Minimal change disease (MCD)
    •Immunoglobulin A nephropathy (IgAN)
    •Immunoglobulin A vasculitis (IgAV)
    •Alport syndrome (AS)
    •glomeruloesclerosis focal ysegmentaria (GEFS)
    •enfermedad por cambios mínimos (ECM)
    •nefropatía por inmunoglobulina A (IgAN)
    • vasculitis por inmunoglobulina A (IgAV)
    •síndrome de Alport (SA)
    E.1.1.1Medical condition in easily understood language
    Diseases of the filtering unit of the kidney (glomerulus).
    Enfermedades de la unidad de filtrado del riñón (glomérulo)
    E.1.1.2Therapeutic area Diseases [C] - Symptoms and general pathology [C23]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10067757
    E.1.2Term Focal segmental glomerulosclerosis
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10021263
    E.1.2Term IgA nephropathy
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.1
    E.1.2Level LLT
    E.1.2Classification code 10082959
    E.1.2Term IgA vasculitis
    E.1.2System Organ Class 100000004858
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10001843
    E.1.2Term Alport's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10058326
    E.1.2Term Minimal change disease
    E.1.2System Organ Class 10038359 - Renal and urinary 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 primary objects of the study are as follows:
    • Evaluate the safety and tolerability of sparsentan oral suspension
    • Assess changes in proteinuria after once-daily dosing of sparsentan oral suspension over the 108-week treatment period
    Los obetivos principales son los siguientes:
    •Evaluar la seguridad y tolerabilidad de la suspensión oral de esparsentán.
    •Evaluar los cambios en la proteinuria después de la administración de una vez al día de la
    suspensión oral de esparsentán durante el periodo de tratamiento de 108 semanas.
    E.2.2Secondary objectives of the trial
    The secondary objectives are as follows:
    • Assess the PK of sparsentan oral suspension in a pediatric population
    • Assess changes in estimated eGFR after once-daily dosing of sparsentan oral suspension over the 108-week treatment period
    Los obetivos decundarios son los siguientes:
    •Evaluar la farmacocinética (FC) de la suspensión oral de esparsentán en una población
    pediátrica.
    •Evaluar los cambios en la tasa de filtración glomerular estimada (TFGe) después de la
    administración una vez al día de la suspensión oral de esparsentán durante el periodo de
    tratamiento de 108 semanas.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For All Subjects (Both Populations)
    A subject must meet all of the following criteria to be eligible for participation in this study:
    1. The subject or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent, and where required, the subject is willing to provide assent before any screening procedures per local requirements.
    2. The subject has an eGFR ≥30 mL/min/1.73 m2 at screening.
    3. The subject has a mean seated blood pressure between the 5th and 95th percentile for age, sex, and height.

    For Population 1
    1. The subject is male or female ≥1 year at screening to <18 years of age at Day 1.
    2. The subject has a UP/C ≥1.5 g/g at screening AND one of the following:
    • Biopsy-proven FSGS or MCD histological patterns and clinical presentation consistent with primary FSGS or MCD and qualifying proteinuria at screening despite history or ongoing treatment with corticosteroids and/or other immunosuppressive disease-modifying agents.
    • Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD. Subjects with a documented podocytic mutation do not require kidney biopsy.
    • Biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion.

    For Population 2
    1. The subject is male or female ≥2 years to <18 years of age at screening.
    2. The subject has UP/C ≥1.0 g/g at screening AND one of the following:
    • Biopsy-confirmed IgAN or IgAV
    • AS (pathogenic X-linked COL4A5 mutation OR autosomal-recessive mutations in both alleles of COL4A3 and/or COL4A4 OR autosomal-dominant COL4A3 and/or COL4A4 and digenic mutations [ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes])
    Para Todos los Pacientes ( Ambas poblaciones)
    El paciente deben cumplir todos los criterios siguientes a fin de ser apto para participar en este estudio:
    1.El paciente o el progenitor/tutor legal (según corresponda) está dispuesto y puede proporcionar el consentimiento informado firmado y, cuando sea necesario, el paciente está dispuesto a proporcionar el asentimiento antes de cualquier procedimiento de selección según los requisitos locales.
    2. El paciente presenta una TFGe de ≥30 ml/min/1,73 m2 en la selección
    Para Poblacion 1
    1. El paciente es hombre o mujer de ≥1 año de edad en el momento de la selección y <18 años de
    edad en el día 1.
    2. El paciente tiene una UP/C ≥1,5 g/g en la selección Y uno de los siguientes:
     Patrones histológicos de la GEFS o la ECM demostrados mediante biopsia y presentación
    clínica compatible con la GEFS o la ECM primarias y un tipo de proteinuria de las
    admitidas para el estudio al momento de la selección a pesar de tener antecedentes de
    tratamiento o tratamiento en curso con corticoesteroides u otros fármacos
    inmunosupresores modificadores de la enfermedad.
     Documentación de una mutación genética en una proteína podocitaria asociada a la GEFS
    o la EMC. Los pacientes con una mutación podocitaria documentada no necesitan biopsia
    renal.
     Patrón histológico de la GEFS demostrado mediante biopsia con antecedentes médicos y
    presentación clínica compatible con la causa de inadaptación de la lesión.

    Para Poblacion 2
    1. El paciente es hombre o mujer de ≥2 a <18 años de edad en el momento de la selección.
    2. El paciente tiene una UP/C ≥1,0 g/g en la selección Y uno de los siguientes diagnósticos:
    • IgAN o IgAV confirmadas mediante biopsia
    •SA (mutación de COL4A5 patógena ligada al cromosoma X O mutaciones autosómicas recesivas en ambos alelos de COL4A3 y/o COL4A4 O mutaciones autosómicas dominantesde COL4A3 y/o COL4A4 y mutaciones digénicas [es decir, mutaciones simultáneas en 2 de los genes COL4A3, COL4A4 y COL4A5]).
    E.4Principal exclusion criteria
    For All Subjects (Both Populations)
    A subject who meets any of the following criteria will be excluded from this study:
    1. The subject weighs <7.3 kg at screening.
    2. The subject has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies.
    3. The subject has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition; eg, systemic lupus erythematosus and liver cirrhosis).
    4. The subject has had an acute onset or presentation of glomerular disease or a diagnostic biopsy or a relapse of glomerular disease requiring new or different class of immunosuppressive treatment (including, but not limited to, systemic corticosteroids, calcineurin inhibitors and mycophenolate mofetil abatacept, cyclophosphamide, rituximab, ofatumumab, and ocrelizumab) within 6 months before screening.
    5. Subjects taking chronic immunosuppressive medications (including systemic steroids) not on a stable dose for ≥1 month before screening.
    6. The subject requires any of the prohibited concomitant medications as defined in the study protocol.
    7. The subject has undergone any organ transplantation, with the exception of corneal transplants.
    8. The subject has a documented history of congenital or acquired heart failure (modified Ross heart failure classification for children Class II to Class IV [Ross 2012]) and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema.
    9. The subject has hemodynamically significant cardiac valvular disease.
    10. The subject has clinically significant congenital vascular disease.
    11. The subject has jaundice, hepatitis, or known hepatobiliary disease, or alanine aminotransferase and/or aspartate aminotransferase >2 times the upper limit of the normal range at screening.
    12. The subject has a history of malignancy within the past 2 years.
    13. The subject has a screening hematocrit <27% or a hemoglobin value <9 g/dL.
    14. The subject has a screening potassium value >5.5 mEq/L.
    15. The subject has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant.
    16. The subject has a history of allergic response to any Ang II antagonist or ERA, including sparsentan, or has a hypersensitivity to any of the excipients in the investigational product.
    17. Female subjects of childbearing potential, beginning at menarche, who do not agree to use 1 highly reliable (ie, can achieve a failure rate of <1% per year) method of contraception from 7 days before the first dose of the investigational product until 90 days after the last dose of investigational product. Examples of highly reliable contraception methods include stable oral, implanted, transdermal, or injected contraceptive hormones associated with the inhibition of ovulation or an intrauterine device (IUD) in place for at least 3 months. One additional barrier method must also be used during sexual activity, such as a diaphragm, diaphragm with spermicide (preferred), or male partner’s use of male condom or male condom with spermicide (preferred), from Day 1/Randomization until 90 days after the last dose of investigational product. Female subjects of childbearing potential are defined as those who are fertile after menarche, unless permanently sterile; permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. All female subjects of childbearing potential must have a negative serum pregnancy test result at screening (Visit 1) and a negative urine pregnancy test result, with positive results confirmed by serum, at every study visit from Day 1 (Visit 2) and after.
    Note: Before menarche, pregnancy testing and contraceptive use are not required. However, subjects and their parents/legal guardians must be advised that, immediately upon menarche, subjects will be required to begin pregnancy testing and initiate contraceptive use. This requirement cannot be waived.
    18. The subject has participated in a study of another investigational product within 28 days before screening or plans to participate in such a study during the course of this study.
    19. The subject has had prior exposure to sparsentan.
    20. The subject or parent/legal guardian (as appropriate), in the opinion of the Investigator, is unable to adhere to the requirements of the study including but not limited to, a history of noncompliance and/or any other reason that causes the Investigator to believe the subject would not be a good candidate for the study.
    Subjects with a medical condition not listed above that may interfere with the evaluation of efficacy or safety-related outcomes for the study will be reviewed with the Sponsor’s Medical Monitor before consideration of the subject for enrollment.
    Para Todos los Pacientes (Ambas poblaciones)
    Un paciente que cumpla alguno de los requisitos siguientes será excluido de este estudio:1. El paciente pesa <7,3 kg en la selección.2. El paciente presenta un patrón histológico de GEFS o ECM secundario a infecciones víricas, toxicidades farmacológicas o neoplasias malignas.3. El paciente tiene depósitos glomerulares de IgA que no se encuentran enel contexto de la IgAN o IgAV primarias(es decir, secundarios a otra afección; p. ej., lupus eritematoso sistémico y cirrosis hepática).4. El paciente ha tenido una aparición o presentación aguda de enfermedad glomerular o una biopsia diagnóstica o una recidiva de enfermedad glomerular que requiere tratamiento inmunosupresor nuevo o de una clase diferente (incluidos, entre otros, corticoesteroides sistémicos, inhibidores de la calcineurina y micofenolato de mofetilo, abatacept, ciclofosfamida, rituximab, ofatumumab y ocrelizumab) en los 6 meses anteriores a la selección5. Pacientes que tomen inmunosupresores crónicos (incluidos corticoesteroides sistémicos) sin una dosis estable durante ≥1 mes antes de la selección.6. El paciente requiere medicación concomitante prohibida según lo especificado en el protocolo del estudio.7. El paciente se ha sometido a cualquier trasplante de órganos, con la excepción de los trasplantes de córnea.8. El paciente tiene antecedentes documentados de insuficiencia cardíaca congénita o adquirida (Clase II a clase IV en la clasificación de la insuficiencia cardíaca de Ross modificada para
    niños) y/o hospitalización previa por insuficiencia cardíaca o disnea inexplicable, ortopnea, disnea paroxística nocturna, ascitis y/o edema periférico.9. El paciente presenta valvulopatía cardiaca hemodinámicamente significativa.10. El paciente tiene una enfermedad vascular congénita clínicamente significativa.11. El paciente tiene ictericia, hepatitis o enfermedad hepatobiliar conocida, o alanina aminotransferasa y/o aspartato aminotransferasa >2 veces el límite superior de la normalidad en la selección.12. El paciente tiene antecedentes de neoplasia maligna en los últimos 2 años.13. El paciente presenta, en la selección, un hematocrito <27 % o un valor de hemoglobina <9 g/dl.14. El paciente tiene un valor de potasio en la selección >5,5 mEq/l.15. El paciente presenta valores anómalos en la selección para análisis clínicos que el investigador considera clínicamente significativos.16. El paciente tiene antecedentes de respuesta alérgica a cualquier antagonista de la angiotensina
    II o antagonista del receptor de la endotelina, incluido el esparsentán, o tiene hipersensibilidad
    a cualquiera de los excipientes del producto en investigación.17. Pacientes de sexo femenino con capacidad de concebir, empezando en la menarquia, que no acepten utilizar un método anticonceptivo altamente fiable (es decir, que pueda alcanzar una tasa de fallo <1 % al año) desde 7 días antes de la primera dosis del producto en investigación hasta 90 días después de la última dosis del producto en investigación. Son ejemplos de métodos anticonceptivos altamente fiables los siguientes: hormonas anticonceptivas orales,implantables, transdérmicas o inyectadas estables asociadas a la inhibición de la ovulación o a un dispositivo intrauterino implantado durante al menos 3 meses. También se debe utilizar un método de barrera adicional durante la actividad sexual, como un diafragma, diafragma con espermicida (preferentemente) o el uso por parte de la pareja masculina de preservativo masculino o preservativo masculino con espermicida (preferentemente) desde el día 1/aleatorización hasta 90 días después de la última dosis del producto en investigación. Las pacientes con capacidad de concebir se definen como aquellas fértiles después de la menarquia, a menos que sean estériles de forma permanente; los métodos de esterilización permanente incluyen la histerectomía, la salpingectomía bilateral y la ovariectomía bilateral. Todas las pacientes en edad fértil deben tener un resultado negativo en la prueba de embarazo en suero en la selección (visita 1) y un resultado negativo en la prueba de embarazo en orina,con resultados positivos confirmados en suero, en cada visita del estudio desde el día 1 (visita3) y después de esta. Nota: Antes de la menarquia, no es necesario realizar pruebas de embarazo ni utilizar anticonceptivos. Sin embargo, se debe advertir a las pacientes y a sus progenitores/tutores legales de que, inmediatamente después de la menarquia, las pacientes tendrán que comenzar las pruebas de embarazo e iniciar el uso de anticonceptivos. Nadie puede ser eximido de este requisito.
    18. El paciente ha participado en un estudio de otro producto en investigación en los 28 días anteriores a la selección o tiene previsto participar en dicho estudio durante el transcurso de este estudio
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events (AEs) leading to treatment discontinuation, and adverse events of interest (AEOIs)
    2. Change from baseline in urine protein/creatinine ratio (UP/C) over the 108-week treatment period
    1. Incidencia de acontecimientos adversos surgidos durante el tratamiento (AAST), acontecimientos adversos graves (AAG), acontecimientos adversos (AA) que provocan lainterrupción del tratamiento y acontecimientos adversos de interés (AADI).
    2.Cambio con respecto al valor inicial en el cociente proteína/creatinina en orina (UP/C)durante el periodo de tratamiento de 108 semanas.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. During the whole study period
    2. Baseline to all study visits to 108 weeks
    1. Durante todo el periodo del estudio
    2. Valor inicial de todas las visitas del estudio a las 108 semanas
    E.5.2Secondary end point(s)
    1. Observed plasma PK concentrations at scheduled timepoints and visits
    2. Steady-state PK parameters (area under the plasma concentration-time curve during a dosing interval [AUCτ], maximum steady-state plasma drug concentration during a dosage interval [Cmax_ss], and minimum steady-state plasma drug concentration [Cmin_ss]) derived from population PK analysis
    3. Change from baseline in urine albumin/creatinine ratio (UA/C) over the 108-week treatment period
    4. Change from baseline in eGFR over the 108-week treatment period
    5. The proportion of subjects with focal segmental glomerulosclerosis (FSGS) and/or minimal change disease (MCD) histological patterns achieving partial remission, defined as UP/C ≤1.5 g/g and >40% reduction in UP/C over the 108-week treatment period
    1. Concentraciones FC plasmáticas observadas en puntos temporales y visitas programadas.
    2.Parámetros FC en situación de equilibrio (área bajo la curva de concentración plasmática frente al tiempo durante un intervalo de dosis [ABCτ], concentración plasmática máxima del fármaco en situación de equilibrio durante un intervalo de dosis [Cmáx_eq] y concentración plasmática mínima del fármaco en situación de equilibrio [Cmín_eq]) derivados del análisis FC poblacional.
    3.Cambio con respecto al inicio en el cociente albúmina/creatinina en orina (UA/C) durante el periodo de tratamiento de 108 semanas.
    4.Cambio con respecto al inicio en la TFGe durante el periodo de tratamiento de 108 semanas.
    5.La proporción de pacientes con patrones histológicos de glomeruloesclerosis focal y segmentaria (GEFS) y/o de enfermedad por cambios mínimos (ECM) que logran una remisión parcial, definida como una reducción de la UP/C ≤1,5 g/g y >40 % en la UP/C durante el periodo de tratamiento de 108 semanas.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Day 1, day 2, week 12
    2. Day 1, day 2, week 12
    3. Baseline to all study visits to 108 weeks
    4. Baseline to all study visits to 108 weeks
    5. Baseline to all study visits to 108 weeks
    1. Dia 1, dia 2, semana 12
    2. Dia 1, dia 2, semana 12
    3. Valor inicial de todas las visitas del estudio a las 108 semanas
    4. Valor inicial de todas las visitas del estudio a las 108 semanas
    5. Valor inicial de todas las visitas del estudio a las 108 semanas
    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 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) 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 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
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Sweden
    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
    End of trial will be when the last subject completes his/her final visit.
    El fin de ensayo será cuando el último paciente complete su Visita Final
    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 months5
    E.8.9.1In the Member State concerned days14
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days20
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 57
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 38
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 13
    F.1.2Adults (18-64 years) No
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 57
    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 Investigator should resume standard of care treatment, including treatment with appropriate medications, as deemed appropriate.
    El investigador debe reanudar el tratamiento estándar de atención, incluido el tratamiento con los medicamentos adecuados, según se considere apropiado.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-10-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-22
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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