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    EudraCT Number:2018-002261-19
    Sponsor's Protocol Code Number:PRN1008-012
    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:2019-01-18
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2018-002261-19
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor PRN1008 in Moderate to Severe Pemphigus
    Estudio aleatorizado, doble ciego, controlado con placebo, multicéntrico para evaluar la eficacia y seguridad del inhibidor oral de BTK PRN1008 en el pénfigo moderado a grave
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the efficacy and safety of PRN1008 in patients with pemphigus
    Estudio para evaluar la eficacia y seguridad de PRN1008
    A.4.1Sponsor's protocol code numberPRN1008-012
    A.5.4Other Identifiers
    Name:IND #Number:134595
    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 SponsorPrincipia Biopharma
    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 supportPrincipia Biopharma
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPrincipia Biopharma, Inc.
    B.5.2Functional name of contact pointChief Medical Officer
    B.5.3 Address:
    B.5.3.1Street Address220 East Grand Ave
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+ 1650 416 7700
    B.5.5Fax number+ 1650 443-3001
    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 numberEMA/OD/157/17
    D.3 Description of the IMP
    D.3.1Product namePRN1008
    D.3.2Product code PRN1008
    D.3.4Pharmaceutical form Tablet
    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 INNnot available
    D.3.9.1CAS number 1575596
    D.3.9.2Current sponsor codePRN1008
    D.3.9.3Other descriptive namePRN1008
    D.3.9.4EV Substance CodeSUB182379
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 placeboTablet
    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
    pemphigus vulgaris [PV] or pemphigus foliaceus [PF]
    Pénfigo Vulgar o Pénfigo foliáceo
    E.1.1.1Medical condition in easily understood language
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10052802
    E.1.2Term Pemphigus vulgaris
    E.1.2System Organ Class 100000004858
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10057054
    E.1.2Term Pemphigus foliaceous
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Efficacy objectives
    • To evaluate the efficacy of PRN1008 in achieving durable CR on low to zero doses of oral corticosteroid (CS) and on the timecourse of quantitative disease activity scores
    • To assess the ability of PRN1008 to reduce CS exposure and the adverse effects of CS
    • To evaluate the time to specified clinical endpoints
    • To assesss the longer term durability of CR

    Safety Objectives
    • To evaluate the safety of PRN1008
    • To evaluate differences in potentially CS-related adverse events
    Objetivos de eficacia
    •Evaluar la eficacia de PRN1008 para lograr una RC duradera con dosis nulas o bajas de corticoesteroides (CE) orales y en el curso temporal de las puntuaciones cuantitativas de actividad de la enfermedad.
    •Evaluar la capacidad de PRN1008 de disminuir la exposición a los CE y los efectos adversos de los CE.
    •Evaluar el tiempo hasta alcanzar los criterios de valoración clínicos especificados.
    •Evaluar la durabilidad de la RC a más largo plazo.
    E.2.2Secondary objectives of the trial
    PK/PD Objectives
    • To evaluate the population pharmacokinetics (PK) of PRN1008
    • To evaluate pharmacodynamic (PD) effects of PRN1008 on anti-desmoglein (anti-dsg) autoantibody titers (anti-dsg1 and anti-dsg3)

    Exploratory Objectives
    • To examine the effects of PRN1008, if any, of the baseline covariates on PK and/or PD, and the relationship between PK, PD, and efficacy
    • To examine the effect of PRN1008 on the costs of hospitalizations, outpatient medical visits, adverse events, concomitant medication use and other relevant health economic outcomes
    • To examine the temporal relationship of change from baseline in Pemphigus Disease Area Index (PDAI) total activity score and quality of life and health economic measures
    Objetivos de FC/FD
    •Evaluar la farmacocinética (FC) poblacional de PRN1008
    •Evaluar los efectos farmacodinámicos (FD) de PRN1008 en los títulos de autoanticuerpos antidesmogleína (anti-dsg) (anti-dsg1 y anti-dsg3)
    Objetivos exploratorios
    •Examinar los efectos de PRN1008, en su caso, de las covariables iniciales en la FC y/o FD, así como la relación entre la FC, la FD y la eficacia
    •Examinar el efecto de PRN1008 en el coste de las hospitalizaciones, las visitas médicas ambulatorias, los acontecimientos adversos, el uso de medicamentos concomitantes y otros resultados pertinentes de economía sanitaria
    •Examinar la relación temporal del cambio desde el inicio en la puntuación de la actividad total del Índice del área de enfermedad del pénfigo (PDAI) y las medidas de calidad de vida y de economía sanitaria
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients, aged 18 to 80 years old with moderate to severe, newly diagnosed or relapsing PV or PF, with a clinical presentation and histopathology consistent with PV or PF.
    2. Positive circulating anti-dsg1 or 3 autoantibody titer
    3. PDAI score of at least 9 points for relapsing patients (diagnosed > 6 months prior to Screening) or at least 15 points for newly diagnosed patients (diagnosed ≤ 6 months prior to Screening)
    4. Body mass index (BMI) > 17.5
    5. Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥ 1.5 × 109/L, hemoglobin (Hgb) > 9 g/dL, platelet count ≥ 100 × 109/L, aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤ 1.5 × upper limit of normal (ULN), albumin ≥ 3 g/dL, creatinine ≤ 1.5 × ULN
    6. Female patients who are of reproductive potential must agree for the duration of the study to use an effective means of contraception (e.g., hormonal contraception methods that inhibit ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal ligation, vasectomized partner or condoms). Unless surgically sterile, postmenopausal females should have menopause confirmed by follicle-stimulating hormone (FSH) testing
    7. Able to provide written informed consent and agreeable to the schedule of assessments
    1.Pacientes varones o mujeres, con una edad comprendida entre los 18 y los 80 años, con PV o PF moderado a grave recién diagnosticado o recidivante, con una presentación clínica e histopatología congruentes con PV o PF.
    2.Títulos de autoanticuerpos anti-dsg1 o 3 circulantes positivos.
    3.Puntuación PDAI de al menos 9 puntos para pacientes recidivantes (diagnosticados >6 meses antes de la selección) o al menos 15 puntos para los pacientes recién diagnosticados (diagnosticados ≤6 meses antes de la selección).
    4.Índice de masa corporal (IMC) >17,5.
    5.Función hematológica, hepática y renal adecuadas (recuento absoluto de neutrófilos)
    ≥1,5 × 109/l, hemoglobina (Hgb) >9 g/dl, recuento de plaquetas ≥100 × 109/l, aspartato aminotransferasa (AST) y/o alanina aminotransferasa (ALT) ≤1,5 × límite superior de la normalidad (LSN), albúmina ≥3 g/dl, creatinina ≤1,5 × LSN.
    6.Las pacientes del sexo femenino que estén en edad fértil deben acceder a utilizar un método anticonceptivo eficaz (p. ej., métodos anticonceptivos hormonales que inhiban la ovulación, dispositivo intrauterino, sistema intrauterino de liberación de hormonas, ligadura de trompas bilateral, pareja vasectomizada o preservativos). A menos que sean quirúrgicamente estériles, se debe confirmar la menopausia en las mujeres posmenopáusicas mediante la prueba de la hormona estimulante de folículos (FSH).
    7.Ser capaces de dar su consentimiento informado por escrito y aceptar el calendario de evaluaciones.
    E.4Principal exclusion criteria
    1. Suspected paraneoplastic pemphigus and other forms of pemphigus that are not pemphigus vulgaris or pemphigus foliaceus
    2. Previous use of a Bruton’s tyrosine kinase (BTK) inhibitor
    3. Pregnant or lactating women
    4. Electrocardiogram (ECG) findings of QT corrected for heart rate (QTc) > 450 msec (males) or > 470 msec (females), poorly controlled atrial fibrillation (i.e., symptomatic patients or a ventricular rate above 100 beats/min on ECG), or other clinically significant abnormalities
    5. A history of malignancy of any type, other than surgically excised non-melanoma skin cancers or in situ cervical cancer within 5 years before Day 1
    6. Use of immunologic response modifiers as concomitant medication and with the following washout periods:
    A) stop at least 2 weeks prior to Screening: mycophenolate mofetil, azathioprine, methotrexate, cyclosporine, dapsone, intravenous immunoglobulin (IVIG), Kinaret (anakinra), Enbrel (etanercept) or any other immunosuppressant not mentioned in this exclusion criterion;
    B) 12 weeks prior to Screening: Remicade (infliximab), Humira (adalimumab), Simponi (golimumab), Orencia (abatercept), Actemra (tocilizumab), Cimzia (certolizumab), Cosentyx (secukinumab), plasmapheresis;
    C) 6 months prior to Screening (or shorter if there is documented B cell reconstitution for anti-CD20 drugs): antiCD20 drugs such as rituximab, ofatumumab, other long-acting biologics
    7. Use of proton pump inhibitor drugs such as omeprazole and esomeprazole within 3 days (It is acceptable to change patient to H2 receptor blocking drugs prior to Day 1.)
    8. Concomitant use of known strong-to-moderate inducers or inhibitors of CYP3A within 3 days or 5 half-lives (whichever is longer) of Day 1 (Appendix 8)
    9. Use of CYP3A-sensitive substrate drugs with a narrow therapeutic index within 3 days or 5 half-lives (whichever is longer) of Day 1 and for the remainder of the trial including, but not limited to alfentanil, astemizole, cisapride, cyclosporine, dihydroergotamine, ergotamine, fentanyl, pimozide, quinidine, sirolimus, tacrolimus, or terfenadine
    10. Has received any investigational drug (or is currently using an investigational device) within the 30 days before Day 1, or at least 5 times the respective elimination half-life time (whichever is longer)
    11. History of drug abuse within the previous 12 months
    12. Alcoholism or excessive alcohol use, defined as regular consumption of more than approximately 3 standard drinks per day
    13. Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant bowel resection that would preclude adequate PRN1008/placebo absorption
    14. Donation of a unit or more of blood or blood products within 4 weeks prior to Day 1
    15. History of solid organ transplant
    16. Positive at Screening for human immunodeficiency virus (HIV), hepatitis B (surface and core antibodies unrelated to vaccination, surface antigen), or hepatitis C (anti-HCV antibody confirmed with Hep C RNA)
    17. Positive interferon-gamma release assay (IGRA) (e.g., QuantiFERON®-TB Gold or T spot TB® Test) at Screening. Unless, all of the following 3 conditions are true:
    a. Chest X-ray does not show evidence suggestive of active tuberculosis (TB) disease
    b. There are no clinical signs and symptoms of pulmonary and/or extra-pulmonary TB disease
    c. Documented receipt of a course of prophylactic TB treatment of at least 6 months
    18. History of serious infections requiring intravenous therapy with the potential for recurrence
    19. Live vaccine within 28 days prior to Day 1 or plan to receive one during the trial
    20. Any other clinically significant disease, condition, or medical history that, in the opinion of the Investigator, would interfere with patient safety, trial evaluations, and/or trial procedures
    1.Sospecha de pénfigo paraneoplásico y otros tipos de pénfigo que no sean pénfigo vulgar o pénfigo foliáceo.
    2.Uso previo de un inhibidor de la tirosina quinasa de Bruton (BTK).
    3.Mujeres embarazadas o en periodo de lactancia.
    4.Hallazgos en los electrocardiogramas (ECG) de QT corregido para frecuencia cardíaca (QTc) >450 mseg (varones) o >470 mseg (mujeres), fibrilación auricular mal controlada (es decir, pacientes sintomáticos o una tasa ventricular superior a 100 latidos/min en el ECG) u otras anomalías clínicamente significativas.
    5.Antecedentes de neoplasia maligna de cualquier tipo, excepto cánceres de piel no melanoma extirpados quirúrgicamente o cáncer de cuello uterino in situ en los 5 años anteriores al día 1.
    6.Uso de modificadores de la respuesta inmunológica como medicación concomitante y con los siguientes periodos de lavado: A) interrupción al menos 2 semanas antes de la selección: micofenolato mofetil, azatioprina, metotrexato, ciclosporina, dapsona, inmunoglobulina intravenosa (Ig i.v.), Kinaret (anakinra), Enbrel (etanercept) o cualquier otro inmunosupresor que no se mencione en este criterio de exclusión; B) 12 semanas antes de la selección: Remicade (infliximab), Humira (adalimumab), Simponi (golimumab), Orencia (abatercept), Actemra (tocilizumab), Cimzia (certolizumab), Cosentyx (secukinumab), plasmaféresis; C) 6 meses antes de la selección (o menos si hay reconstitución de linfocitos B documentada para los fármacos anti-CD20): fármacos anti-CD20 como rituximab, ofatumumab y otros biológicos de acción prolongada.
    7.Uso de fármacos inhibidores de la bomba de protones, como el omeprazol y el esomeprazol en un plazo de
    3 días (es aceptable cambiar al paciente a bloqueadores de receptores de H2 antes del día 1).
    8.Uso concomitante de inductores o inhibidores del CYP3A pontentes a moderados conocidos en los 3 días o 5 semividas (lo que sea más largo) posteriores al día 1 (apéndice 8).
    9.Uso de fármacos sustrato sensibles al CYP3A con un índice terapéutico estrecho en los 3 días o 5 semividas (lo que sea más largo) posteriores al día 1 y durante el resto del ensayo, incluidos, sin carácter exclusivo, alfentanilo, astemizol, cisaprida, ciclosporina, dihidroergotamina, ergotamina, fentanilo, pimozida, quinidina, sirolimús, tacrolimús o terfenadina.
    10.Han recibido el fármaco en fase de investigación (o están usando actualmente un dispositivo en fase de investigación) en los 30 días anteriores al día 1, o al menos 5 veces el tiempo respectivo de la vida media de eliminación (lo que sea más largo).
    11.Antecedentes de abuso de fármacos en los últimos 12 meses.
    12.Alcoholismo o consumo excesivo de alcohol, definido como el consumo habitual de más de unas 3 bebidas estándar al día.
    13.Náuseas y vómitos refractarios, mala absorción, derivación biliar externa o resección intestinal significativa que impediría una absorción adecuada de PRN1008/placebo.
    14.Donación de una unidad o más de sangre o hemoderivados en las 4 semanas anteriores al día 1.

    15.Antecedentes de trasplante de órganos sólidos.
    16.Resultado positivo en la prueba de detección del virus de inmunodeficiencia humana (VIH), hepatitis B (anticuerpos de superficie y núcleo no relacionados con la vacunación, antígeno de superficie) o hepatitis C (anticuerpo anti-VHC confirmado con ARN de hepatitis C).
    17.Resultado positivo en el ensayo de liberación de interferón-gamma (IGRA) (p. ej., prueba QuantiFERON®-TB Gold o T-spot TB®) en la selección. A menos que sean ciertas las tres condiciones siguientes:
    a.La radiografía de tórax no muestra evidencias que sugieran tuberculosis (TB) activa
    b.No hay signos y síntomas clínicos de TB pulmonar y/o extrapulmonar
    c.Recepción documentada de un ciclo de tratamiento preventivo contra la TB de al menos 6 meses
    18.Antecedentes de infecciones graves que requieren tratamiento intravenoso con potencial de recidiva.
    19.Haber recibido una vacuna viva en los 28 días anteriores al día 1 o tener previsto recibirla durante el ensayo.
    20.Cualquier otra enfermedad, afección o antecedente médicos clínicamente significativos que, según la opinión del investigador, interferiría en la seguridad del paciente, las evaluaciones del ensayo y/o los procedimientos del ensayo.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy endpoint: The proportion of patients who are in CR from Week ≤ 29 to Week 37 with a daily corticosteroid dose of ≤ 5 mg/day

    Safety Endpoints
    • Nature, frequency, and severity of adverse events, including serious adverse events, adverse events leading to discontinuation and possible corticosteroid-related adverse effects
    • Change from baseline in vital signs and clinical laboratory test results (including complete blood count and blood chemistry)
    Criterio de valoración principal de la eficacia:La proporción de pacientes que están en RC desde la semana ≤29 hasta la semana 37 con una dosis de CE de ≤5
    Criterios de valoración de la seguridad
    •Naturaleza, frecuencia y gravedad de los acontecimientos adversos, incluidos los acontecimientos adversos graves, los acontecimientos adversos que obligan a la interrupción y los posibles efectos adversos relacionados con los corticoesteroides
    •Cambio desde el inicio en las constantes vitales y los resultados de las pruebas de laboratorio clínico (incluidos el hemograma completo y la bioquímica sanguínea)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy endpoints: at every visit between week 29 and Week 37
    Safety endpoints: at every visit
    Criterios de valoración de eficacia: En cada visita entre la semana 29 y la 37
    Criterios de valoración de seguridad: En cada visita
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoints
    • Cumulative CS dose over first 36 weeks (to Week 37)
    • Time to the beginning of the CR event used to define success for patients reaching the primary endpoint
    • Proportion of patients with CR from Week ≤ 29 to Week 37 with a daily CS dose of ≤ 10 mg/day

    Other Secondary Endpoints
    • GTI score at Week 37
    • Change in EuroQOL-5 Dimension 5 Level (EQ-5D-5L) score from baseline to Week 37
    • Change in EQ-5D-5L score from baseline to Weeks 5, 13, 25, and 61
    • Change in Autoimmune Bullous Disease Quality of Life (ABQOL) score from baseline to Weeks 5, 13, 25, 37 and 61
    • Change in PDAI score from baseline to Weeks 5, 13, 25, 37 and 61
    • Change in Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) score from baseline to Weeks 5, 13, 25, 37, and 61
    • Proportion of patients in CR for ≥ 8 weeks on zero CS at Week 61 by original PRN1008/placebo group assignment and overall
    • Proportion of patients in CR at Week 37 to maintain CR continuously to Week 61
    • Proportion of patients not in CR at Week 37 to achieve CR at Week 61
    • Proportion of patients to achieve CR of any duration at any time up to Week 37
    • Duration of CR
    Criterios de valoración secundarios clave de la eficacia
    •Dosis acumulada de CE durante las primeras 36 semanas (hasta la semana 37)
    •Tiempo hasta el inicio del acontecimiento de RC utilizado para definir el éxito de los pacientes que alcanzan el criterio de valoración principal.
    •Proporción de pacientes con RC desde la semana ≤29 hasta la semana 37 con una dosis de CE de
    ≤10 mg/día
    Otros criterios de valoración secundarios
    •Puntuación GTI en la semana 37
    •Cambio en la puntuación del EuroQOL-5 de 5 niveles y 5 dimensiones (EQ-5D-5L) desde el inicio hasta la semana 37
    •Cambio en la puntuación del EQ-5D-5L desde el inicio hasta las semanas 5, 13, 25 y 61
    •Cambio en la puntuación de la calidad de vida de la enfermedad ampollosa autoinmunitaria (ABQOL) desde el inicio hasta las semanas 5, 13, 25, 37 y 61
    •Cambio en la puntuación PDAI desde el inicio hasta las semanas 5, 13, 25, 37 y 61
    •Cambio en la puntuación de la calidad de vida durante el tratamiento de la enfermedad ampollosa autoinmunitaria (TABQOL) desde el inicio hasta las semanas 5, 13, 25, 37 y 61
    •Proporción de pacientes con RC durante ≥8 semanas sin CE en la semana 61 según
    la asignación original a PRN1008/placebo y en general
    •Proporción de pacientes con RC en la semana 37 que mantienen la RC de forma continua hasta la semana 61
    •Proporción de pacientes sin RC en la semana 37 que consiguen la RC en la semana 61
    •Proporción de pacientes que consiguen una RC de cualquier duración en cualquier momento hasta la semana 37
    •Duración de la RC
    E.5.2.1Timepoint(s) of evaluation of this end point
    at every visit
    En cada visita
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. 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 Yes
    E. trial design description
    double-blind for the first 36 weeks, then open label from week 37 to 61
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA49
    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
    United Arab Emirates
    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
    UVUP (última Visita último paciente)
    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 months4
    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 months4
    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) 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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 59
    F.4.2.2In the whole clinical trial 120
    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 patients will continue standard of care after completing the study.
    Los pacientes continuaran su tratamiento standar después de completar el estudio
    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 Decision2019-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-07
    P. End of Trial
    P.End of Trial StatusOngoing
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