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    Summary
    EudraCT Number:2017-004605-41
    Sponsor's Protocol Code Number:021IGAN17001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-07-30
    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 number2017-004605-41
    A.3Full title of the trial
    A Randomized, Multicenter, Double-blind, Parallel-group, Active-control Study of the Efficacy and Safety of Sparsentan for the Treatment of Immunoglobulin A Nephropathy
    Estudio aleatorizado, multicéntrico, doble ciego, de grupos paralelos y controlado con principio activo, de la eficacia y la seguridad de esparsentán para el tratamiento de la nefropatía por inmunoglobulina A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effectiveness and Safety of Sparsentan as treatment for Immunoglobulin A Nephropathy (IgAN)
    Eficacia y seguridad de esparsentán para el tratamiento de la nefropatía por inmunoglobulina A
    A.4.1Sponsor's protocol code number021IGAN17001
    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 SponsorRetrophin, 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 supportRetrophin, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRetrophin, Inc.
    B.5.2Functional name of contact pointRetrophin Call Center
    B.5.3 Address:
    B.5.3.1Street Address3721 Valley Centre Drive, Suite 200
    B.5.3.2Town/ citySan Diego, CA
    B.5.3.3Post code92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34962339485
    B.5.6E-mailcallcenter@retrophin.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 nameSparsentan
    D.3.2Product code RE-021
    D.3.4Pharmaceutical form Capsule, hard
    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 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 milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 RoleComparator
    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 Yes
    D.2.1.1.1Trade name Irbesartan tablets (Approved in the USA. Reference listed drug Avapro) NDC # 43547-0374-03
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameover-encapsulated 150 mg Irbesartan Tablets
    D.3.4Pharmaceutical form Capsule, hard
    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 INNIrbesartan
    D.3.9.1CAS number 138402-11-6
    D.3.9.3Other descriptive nameIRBESARTAN
    D.3.9.4EV Substance CodeSUB08293MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    Immunoglobulin A Nephropathy (IgAN)
    nefropatía por inmunoglobulina A
    E.1.1.1Medical condition in easily understood language
    Also known as Berger’s disease; a form of glomerulonephritis or an inflammation of the glomeruli of the kidney.
    También conocida como enfermedad de Berger, una forma de glomerulonefritis (inflamación del glomérulo renal)
    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 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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The efficacy objective of the study is to determine the effect of sparsentan on proteinuria and renal function, as compared to an angiotensin receptor blocker, in patients with Immunoglobulin A Nephropathy (IgAN).

    The safety objective of the study is to assess the safety and tolerability of sparsentan by double-blind monitoring of safety endpoints.
    Eficacia: Determinar el efecto de esparsentán sobre la proteinuria y la función renal, en comparación con un bloqueador de los receptores de la angiotensina, en pacientes con nefropatía por inmunoglobulina A (NIgA).
    Seguridad: Evaluar la seguridad y la tolerabilidad de esparsentán mediante la supervisión con doble ciego de los criterios de valoración de la seguridad.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The patient is willing and able to provide signed informed consent.
    2. The patient is male or female, aged >=18 years.
    3. The patient has biopsy-proven primary IgAN. The biopsy may have been performed at any time in the past.
    4. The patient has a urine total protein value >=1.0 g/day at screening.
    5. The patient has an eGFR >=30 mL/min/1.73 m2 at screening.
    6. The patient has been on a stable dose of ACEI and/or ARB therapy for at least 12 weeks prior to screening that is:
    A. the patient’s maximum tolerated dose, AND
    B. at least one-half of the maximum labeled dose (according to approved labeling).
    7. In the Investigator’s opinion, the patient’s blood pressure has been managed in accordance with standard of care using ACEI and/or ARB therapy as described in Inclusion Criterion #6 and additional antihypertensive agents if needed. At screening, the patient’s systolic blood pressure must be <=150 mmHg and diastolic blood pressure must be <=100 mmHg.
    8. The patient is willing to undergo a change in ACEI and/or ARB and antihypertensive medications.
    9. Women of childbearing potential (WOCBP) must agree to the simultaneous use of 2 medically accepted methods of contraception from randomization until 90 days after the last dose of study medication. At least one method of contraception must be highly reliable (ie, can achieve a failure rate of <1% per year), such as stable oral, implanted, transdermal, or injected contraceptive hormones associated with inhibition of ovulation, or an intrauterine device (IUD) in place for at least 3 months. The other method of contraception must be a barrier method, such as a diaphragm with spermicide or male partner’s use of male condom with spermicide. WOCBP are defined as those who are fertile, following menarche and until becoming postmenopausal unless permanently sterile; permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as amenorrhea for more than 24 consecutive months without an alternative medical cause; women on hormone replacement therapy must have a documented plasma follicle-stimulating hormone level >40 mIU/mL. All WOCBP must have a negative pregnancy test (urine, with positive results confirmed by serum) at screening and randomization.
    10. Males must be surgically sterile (more than 3 months post-vasectomy) or must agree to the use of medically accepted methods of contraception that are considered highly reliable from randomization until 90 days after the last dose of study medication.
    1. El paciente debe estar dispuesto y capacitado para otorgar su consentimiento informado firmado.
    2. El paciente es hombre o mujer de >=18 años de edad.
    3. El paciente tiene NIgA primaria probada mediante biopsia. La biopsia se puede haber realizado en cualquier momento del pasado.
    4. El paciente tiene un valor de proteína total en orina de >=1,0 g/día en la selección.
    5. El paciente tiene una FGe >=30 ml/min/1,73 m2 en la selección.
    6. El paciente ha estado recibiendo una dosis estable de un tratamiento con IECA y/o BRA durante al menos 12 semanas antes de la selección, y que sea:
    A. la dosis máxima tolerada del paciente y
    B. al menos la mitad de la dosis máxima recomendada (según la ficha técnica aprobada).
    7. En opinión del investigador, la tensión arterial del paciente se ha tratado de acuerdo con la práctica clínica habitual mediante tratamientos con IECA y/o BRA como se describe en el criterio de inclusión n.º 6 y antihipertensivos adicionales si es necesario. En la selección, la tensión arterial sistólica del paciente debe ser <=150 mmHg y la tensión arterial diastólica debe ser <=100 mmHg.
    8. El paciente está dispuesto a someterse a un cambio en las medicaciones hipertensivas IECA y/o BRA.
    9. Las mujeres en edad fértil (MEF) deben aceptar el uso simultáneo de 2 métodos anticonceptivos médicamente aceptados desde la aleatorización hasta 90 días después de la última dosis de la medicación del estudio. Al menos uno de los métodos anticonceptivos debe ser altamente fiable (es decir, puede alcanzar un índice de fallo de <1 % al año), como anticonceptivos hormonales orales, implantables, transdérmicos o inyectados estables asociados a la inhibición de la ovulación, o un dispositivo intrauterino (DIU) colocado durante al menos 3 meses. El otro método anticonceptivo debe ser un método de barrera, como el diafragma con espermicida o el uso por parte de la pareja masculina de un preservativo con espermicida. Las MEF se definen como las mujeres fértiles, después de la menarquia y hasta que alcanzan la posmenopausia, a menos que sean estériles de forma permanente. Los métodos de esterilización permanente incluyen la histerectomía, la salpingectomía bilateral y la ovariectomía bilateral. El estado posmenopáusico se define como una amenorrea que supera los 24 meses consecutivos sin una causa médica alternativa; las mujeres con tratamiento de restitución hormonal deben tener un nivel de folitropina plasmático documentado de >40 mIU/ml. Todas las MEF deben obtener un resultado negativo en la prueba de embarazo (en orina, con resultados positivos confirmados en suero) en la selección y la aleatorización.
    10. Los hombres deben estar esterilizados quirúrgicamente (más de 3 meses después de la vasectomía) o deben aceptar utilizar métodos anticonceptivos médicamente aceptados que se consideren altamente fiables desde la aleatorización hasta 90 días después de la última dosis de la medicación del estudio.
    E.4Principal exclusion criteria
    1. The patient has IgAN secondary to another condition (eg, systemic lupus erythematosus, liver cirrhosis) or Henoch-Schonlein purpura.
    2. The patient has cellular glomerular crescents present in >25% of glomeruli on renal biopsy within 6 months prior to screening.
    3. The patient has a history of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus (hemoglobin A1c [HbA1c] >8%), or nonfasting blood glucose >180 mg/dL at screening.
    4. The patient has undergone any organ transplantation, with the exception of corneal transplants.
    5. The patient requires any of the prohibited concomitant medications.
    6. The patient has been taking any systemic immunosuppressive medications (including corticosteroids) for >2 weeks within 3 months prior to screening.
    7. The patient has a documented history of heart failure (New York Heart Association Class II-IV) and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema.
    8. The patient has clinically significant cerebrovascular disease (transient ischemic attack or stroke) and/or coronary artery disease (hospitalization for myocardial infarction or unstable angina, new onset of angina with positive functional tests, coronary angiogram revealing stenosis, or a coronary revascularization procedure) within 6 months prior to screening.
    9. The patient has jaundice, hepatitis, or known hepatobiliary disease (including asymptomatic cholelithiasis), or alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >2 times the upper limit of the normal range at screening.
    10. The patient has a history of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years.
    11. The patient has a screening hematocrit value <27% or hemoglobin value <9 g/dL.
    12. The patient has a screening potassium value of >5.5 mEq/L.
    13. The patient has a history of alcohol or illicit drug use disorder (as defined in the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition).
    14. The patient has a history of serious side effect or allergic response to any angiotensin II antagonist or endothelin receptor antagonist, including sparsentan or irbesartan, or has a hypersensitivity to any of the excipients in the study medications.
    15. The female patient is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
    16. The male patient plans to father a child during the course of the study.
    17. The patient has participated in a study of another investigational product within 28 days prior to screening, or plans to participate in such a study during the course of this study.
    18. The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study, including the ability to swallow the study medication capsules whole.
    1. El paciente tiene NIgA secundaria a otra afección (por ejemplo, lupus eritematoso sistémico, cirrosis hepática) o púrpura de Henoch-Schonlein.
    2. El paciente presenta semilunas glomerulares celulares >25 % de los glomérulos en la biopsia renal en los 6 meses antes de la selección.
    3. El paciente tiene antecedentes de diabetes mellitus tipo 1, diabetes mellitus tipo 2 no controlada (hemoglobina A1c [HbA1c] >8 %) o glucemia en condición de no ayuno >180 mg/dl en la selección.
    4. El paciente se ha sometido a algún trasplante de órganos, a excepción de trasplantes de córnea.
    5. El paciente necesita tomar alguna de las medicaciones concomitantes prohibidas (consulte la sección 15.2.1).
    6. El paciente ha estado tomando medicamentos inmunodepresores sistémicos (incluidos corticoesteroides) durante >2 semanas en los 3 meses previos a la selección.
    7. El paciente tiene antecedentes documentados de insuficiencia cardíaca (clase II-IV de la Asociación del corazón de Nueva York [New York Heart Association]) o de hospitalización previa por insuficiencia cardíaca o disnea sin explicar, ortopnea, disnea paroxística nocturna, ascitis y/o edema periférico.
    8. El paciente tiene una enfermedad cerebrovascular clínicamente significativa (accidente isquémico transitorio o accidente cerebrovascular) y/o arteriopatía coronaria (hospitalización por infarto de miocardio o angina inestable, nueva aparición de angina con pruebas funcionales positivas, angiograma coronario que revela estenosis o un procedimiento de revascularización coronaria) en los 6 meses previos a la selección.
    9. El paciente tiene ictericia, hepatitis o enfermedad hepatobiliar conocida (incluida la colelitiasis asintomática) o niveles de alanina aminotransferasa (ALT) y/o de aspartato aminotransferasa (AST) >2 veces el límite superior de la normalidad en la selección.
    10. El paciente tiene antecedentes de neoplasia maligna diferente al carcinoma basocelular o epidermoide o carcinoma cervicouterino en los últimos 2 años.
    11. El paciente tiene un nivel de hematocrito en la selección de <27 % o de hemoglobina <9 g/dl.
    12. El paciente tiene un nivel de potasio en la selección de >5,5 mEq/l.
    13. El paciente tiene antecedentes de trastorno por abuso de alcohol o de drogas ilegales (tal y como lo define el Manual diagnóstico y estadístico de trastornos mentales, 5.ª edición).
    14. El paciente tiene antecedentes de efecto secundario o respuesta alérgica graves a los antagonistas de angiotensina II o a los antagonistas del receptor de endotelina, incluidos esparsentán o irbesartán, o bien tiene hipersensibilidad a cualquiera de los excipientes de las medicaciones del estudio.
    15. La paciente está embarazada, piensa quedarse embarazada durante el transcurso del estudio o está en periodo de lactancia.
    16. El paciente piensa concebir un hijo durante el transcurso del estudio.
    17. El paciente ha participado en un estudio de otro producto en investigación en los 28 días previos a la selección o prevé participar en un estudio de ese tipo durante el transcurso de este estudio.
    18. El paciente, en opinión del investigador, es incapaz de cumplir con los requisitos del estudio, incluida la capacidad para tragar las cápsulas enteras de la medicación del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy End points:
    The primary efficacy endpoint is the change from baseline in the urine protein/creatinine ratio (UP/C), based on a 24-hour urine sample, at Week 36, assessed on the log scale.

    Safety End points:
    • Changes from baseline in body weight, vital signs, physical examinations, peripheral edema, and clinical laboratory parameters
    • The incidence of TEAEs
    El criterio de valoración principal de la eficacia es el cambio desde el inicio en el cociente proteínas/creatinina (p/C) en orina, en función de la muestra de orina de 24 horas, en la semana 36, evaluado en la escala logarítmica.
    Las evaluaciones de seguridad incluyen:
    • Cambios respecto al valor inicial del peso corporal, las constantes vitales, las exploraciones físicas, el edema periférico y los parámetros de análisis clínicos
    • La incidencia de acontecimientos adversos (AA)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary efficacy endpoints will be evaluated at Week 36. The primary safety endpoint will be evaluated from randomization to Week 114.
    La eficacia será evaluada en la semana 36
    La seguridad será evaluada en la semana 114 desde la randomización
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints:
    The secondary efficacy endpoints with Type I error control are:
    • The rate of change in eGFR over a 52-week (approximately 1-year) period following the initial acute effect of randomized therapy (the initial acute effect of randomized therapy is defined as the first 6 weeks of randomized treatment with study medication; thus, the analysis is from 6 weeks post randomization to 58 weeks post randomization)
    • The rate of change in eGFR over a 104-week (approximately 2-year) period following the initial acute effect of randomized therapy (the initial acute effect of randomized therapy is defined as the first 6 weeks of randomized treatment with study medication; thus, the analysis is from 6 weeks post randomization to 110 weeks post randomization)
    • Achievement of urinary protein excretion, based on a 24-hour urine sample, <0.3 g/day at Week 36

    Other Efficacy Endpoints:
    • The mean change from baseline over time in selected proteinuria variables, based on a 24-hour urine sample (eg, total urine protein, total urine albumin, urine albumin/creatinine ratio [UA/C]), up to Week 110
    • The proportion of patients reaching a confirmed 40% change in eGFR, end-stage renal disease (ESRD), or death. (ESRD is defined as initiation of renal replacement therapy [RRT], kidney transplantation, or sustained eGFR <15 mL/min/1.73 m2.)

    Exploratory endpoints are:
    • The change from baseline in eGFR at 6 weeks post randomization (ie, the acute effect of randomized therapy)
    • The change from end-of-treatment (EOT; ie, Week 110) in eGFR 4 weeks following cessation of treatment (ie, at Week 114)
    • The proportion of patients with hematuria at each visit
    • The proportion of patients requiring immunosuppressive medication during the study
    • Mean changes from baseline in quality of life (QOL), measured via patient-reported outcome (PRO) at each visit beginning with Week 36
    • Frequency and duration of hospitalizations (for any reason and for reasons related to the kidney)
    • Trough plasma PK concentrations
    Los criterios de valoración secundarios de la eficacia con control de error de tipo I son:
    • La tasa de cambio de FGe en un periodo de 52 semanas (aproximadamente 1 año) tras el efecto agudo inicial del tratamiento aleatorizado (el efecto agudo inicial del tratamiento aleatorizado se define como las primeras 6 semanas de tratamiento aleatorizado con la medicación del estudio; por tanto, el análisis es desde las 6 semanas tras la aleatorización hasta las 58 semanas tras la aleatorización)
    • La tasa cambio de la FGe en un periodo de 104 semanas (aproximadamente 2 años) tras el efecto agudo inicial del tratamiento aleatorizado (el efecto agudo inicial del tratamiento aleatorizado se define como las primeras 6 semanas de tratamiento aleatorizado con la medicación del estudio; por tanto, el análisis es desde 6 semanas tras la aleatorización hasta las 110 semanas tras la aleatorización)
    • Logro de la excreción de proteínas urinarias, basado en una muestra de orina de 24 horas, <0,3 g/día en la semana 36
    Otros criterios de valoración secundarios de la eficacia son:
    • El cambio medio desde el inicio con el tiempo en variables de proteinuria seleccionadas, en función de una muestra de orina de 24 horas (p. ej., proteína total en orina, albúmina total en orina, cociente albúmina/creatinina [a/C] en orina), hasta la semana 110
    • La proporción de pacientes que logran un cambio confirmado del 40 % en la FGe, enfermedad renal terminal (ERT) o la muerte. (La ERT se define como el inicio del tratamiento renal sustitutivo [TRS], trasplante renal o una FGe sostenida <15 ml/min/1,73 m2 durante el estudio.
    Los criterios de valoración exploratorios son:
    • El cambio desde el inicio en la FGe en las 6 semanas tras la aleatorización (es decir, el efecto agudo del tratamiento aleatorizado)
    • El cambio desde el final del tratamiento (FDT; es decir, la semana 110) en la FGe 4 semanas tras la interrupción del tratamiento (es decir, en la semana 114)
    • Las proporciones de pacientes con hematuria en cada visita
    • La proporción de pacientes que necesitan medicación con inmunodepresores durante el estudio
    • Cambios medios respecto al valor inicial de la calidad de vida (CdV), medidos mediante los resultados comunicados por el paciente (RCP) en cada una de las visitas empezando en la semana 36
    • Frecuencia y duración de las hospitalizaciones (por cualquier motivo y por motivos relacionados con el riñón)
    • Concentraciones FC plasmáticas mínimas
    E.5.2.1Timepoint(s) of evaluation of this end point
    The subsequent key secondary endpoint analysis of 6- to 110-week eGFR rate of change will be assessed at the 4% two-sided alpha level if the 6- to 58-week eGFR rate of change analysis fails to achieve p<0.01; otherwise the 6- to 110-week eGFR rate of change will be assessed at the 5% two-sided alpha level.
    El análisis posterior del criterio de valoración secundario clave de la tasa de cambio de la FGe en las semanas 6 a 110 se evaluará al nivel alfa bilateral del 4 % si el análisis de la tasa de cambio de la FGe en las semanas 6 a 58 no logra el p < 0,01; en caso contrario se evaluará la tasa de cambio de la FGe en las semanas 6 a 110 al nivel alfa bilateral del 5 %.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group 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) Yes
    E.8.2.2Placebo No
    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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA79
    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
    Croatia
    Czech Republic
    Estonia
    France
    Germany
    Hong Kong
    Hungary
    Italy
    Korea, Republic of
    Lithuania
    Malaysia
    New Zealand
    Poland
    Portugal
    Spain
    Turkey
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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: 260
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 160
    F.4.2.2In the whole clinical trial 280
    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. The Investigator should resume standard of care treatment, including treatment with appropriate medications, as deemed appropriate.
    Ninguno. El investigador debe reanudar la práctica clínica habitual, incluido el tratamiento con medicamentos apropiados, según lo 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 Decision2018-09-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-18
    P. End of Trial
    P.End of Trial StatusOngoing
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