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    Summary
    EudraCT Number:2011-004728-36
    Sponsor's Protocol Code Number:747-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-02-22
    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 number2011-004728-36
    A.3Full title of the trial
    A Phase 3, Double Blind, Placebo Controlled Trial and Long Term Safety
    Extension of Obeticholic Acid in Patients with Primary Biliary Cirrhosis
    Estudio de fase 3, doble ciego, controlado con placebo y extensión de seguridad a largo plazo con ácido obeticólico en pacientes con cirrosis biliar primaria.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of Obeticholic Acid versus Placebo in Patients with Primary Biliary Cirrhosis plus long term extension study only with Obeticholic Acid
    Un estudio con ácido obeticólico comparado con placebo en pacientes con cirrosis biliar primaria más extensión del estudio a largo plazo sólo con ácido obeticólico
    A.4.1Sponsor's protocol code number747-301
    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 SponsorIntercept Pharmaceuticals, 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 supportIntercept Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAccelsiors CRO and Consultancy Services
    B.5.2Functional name of contact pointClinical Trials Info
    B.5.3 Address:
    B.5.3.1Street Address50 Miskolci Utca
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1147
    B.5.3.4CountryHungary
    B.5.4Telephone number3612990091
    B.5.5Fax number3612990096
    B.5.6E-mailclinicaltrials@accelsiors.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/3/10/753
    D.3 Description of the IMP
    D.3.1Product nameOCA (INT-747)
    D.3.2Product code OCA (INT-747)
    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 INNObeticholic acid
    D.3.9.1CAS number 459789-99-2
    D.3.9.2Current sponsor codeOCA, 6-ECDCA or INT-747
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/753
    D.3 Description of the IMP
    D.3.1Product nameOCA (INT-747)
    D.3.2Product code OCA (INT-747)
    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 INNObeticholic acid
    D.3.9.1CAS number 459789-99-2
    D.3.9.2Current sponsor codeOCA, 6-ECDCA or INT-747
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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: 3
    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/3/10/753
    D.3 Description of the IMP
    D.3.1Product nameOCA (INT-747)
    D.3.2Product code OCA (INT-747)
    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 INNObeticholic acid
    D.3.9.1CAS number 459789-99-2
    D.3.9.2Current sponsor codeOCA, 6-ECDCA or INT-747
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 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
    Primary biliary cirrhosis
    Cirrosis biliar primaria
    E.1.1.1Medical condition in easily understood language
    Primary biliary cirrhosis (PBC) is a rare, chronic, autoimmune liver disease characterized by both liver and biliary tract lesions that progresses to cirrhosis and other complications.
    La cirrosis biliar primaria (CBP) es una enfermedad hepática autoinmune crónica y rara que se caracteriza por lesiones hepáticas y biliares que progresan a cirrosis y otras complicaciones
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level SOC
    E.1.2Classification code 10019805
    E.1.2Term Hepatobiliary disorders
    E.1.2System Organ Class 10019805 - Hepatobiliary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the effects of OCA in patients with PBC on:
    -Serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint
    -Safety
    Evaluar los efectos de ácido obeticólica (AOC) en pacientes con CBP sobre:
    - Fosfatasa alcalina (FA) y biliurrina total séricas, conjuntamente como variable compuesta
    - Seguridad
    E.2.2Secondary objectives of the trial
    To assess the effects of OCA in patients with PBC on:
    -Hepatocellular injury and liver function, including histology (inflammatory,
    structural [portal, parenchymal] and fibrotic assessments)
    -Disease specific symptoms
    -Biomarkers and noninvasive assessments of liver fibrosis
    -Bile acids (BA)
    -Other exploratory evaluations
    Evaluar los efectos de OAC en pacientes con CBP sobre:
    -Daño hepatocelular y función hepática, incluyendo la histología (evaluaciones inflamatorias, estructurales [portal, parenquimatosa] y fibróticas).
    -Síntomas específicos de la enfermedad.
    -Biomarcadores y evaluaciones no invasivas de la fibrosis hepática.
    -Ácidos biliares (AB).
    -Otras evaluaciones exploratorias
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional Genetics Research Study (A Phase 3, Double Blind, Placebo Controlled Trial and Long Term Safety Extension of Obeticholic Acid in Patients with Primary Biliary Cirrhosis)
    To study genes that may be involved in the development of Primary Biliary Cirrhosis (PBC) disease (and related conditions).
    Estudio de investigación genética opcional (Estudio de fase 3, doble ciego, controlado con placebo y extensión de seguridad a largo plazo con ácido obeticólico en pacientes con cirrosis biliar primaria) para el estudio de genes que pueden estar involucrados en el desarrollo de la cirrosis biliar primaria y afecciones relacionadas.
    E.3Principal inclusion criteria
    1. Definite or probable PBC diagnosis (consistent with AASLD and EASL Practice Guidelines; [Lindor 2009; EASL 2009]), as demonstrated by the presence of ? 2 of the following 3 diagnostic factors:
    -History of elevated ALP levels for at least 6 months prior to Day 0
    -Positive AMA titer or PBC specific antibodies
    -Liver biopsy consistent with PBC
    2. At least 1 of the following qualifying biochemistry values:
    - ALP?1.67x ULN
    -Total bilirubin > ULN but < 2x ULN
    3. Age ?18 years
    4. Taking UDCA for at least 12 months (stable dose for ? 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ? 3 months) prior to Day 0
    5. Contraception: Female patients of childbearing potential must use ?1 effective (?1% failure rate) method of contraception during the trial and until the End of Treatment (EOT) visit.
    6. Must provide written informed consent and agree to comply with the trial protocol.
    1.Diagnóstico confirmado o probable de CBP (compatible con las directrices de práctica de la AASLD y AEEH; [Lindor 2009; AEEH 2009]), demostrado por la presencia de al menos 2 de los siguientes 3 factores diagnósticos:
    -Antecedentes de niveles elevados de FA durante un mínimo de 6 meses antes del día 0.
    -Valor positivo de AAM o de anticuerpos específicos de la CBP.
    -Biopsia hepática compatible con CBP.
    2.Al menos uno de los siguientes valores bioquímicos habilitantes:
    -FA ? 1,67x LSN.
    -Bilirrubina total > LSN pero < 2x LSN.
    3.Edad ? 18 años.
    4.Tratamiento con AUDC durante al menos 12 meses (con una dosis estable durante ? al menos 3 meses) antes del día 0, o incapacidad de tolerar el AUDC (ausencia de AUDC durante ? al menos 3 meses) antes del día 0.
    5.Anticoncepción: las pacientes en edad fértil deben utilizar como mínimo un método anticonceptivo efectivo (?1 % de tasa de fracaso) durante el ensayo y hasta la consulta visita de fin de tratamientofinal del tratamiento (FDT).
    6.Haber otorgadodo su consentimiento informado por escrito y aceptar cumplir el protocolo del ensayo.
    E.4Principal exclusion criteria
    Patients will be excluded from the trial if they meet any of the following:
    1. History or presence of other concomitant liver diseases including:
    -Hepatitis B or C virus (HCV, HBV) infection
    -Primary sclerosing cholangitis (PSC)
    -Alcoholic liver disease
    -Definite autoimmune liver disease or overlap hepatitis
    -Nonalcoholic steatohepatitis (NASH)
    -Gilbert"s Syndrome (exclusion due to interpretability of bilirubin levels)
    2. Presence of clinical complications of PBC or clinically significant hepatic
    decompensation, including:
    -History of liver transplantation, current placement on a liver transplant list or current MELD score ?15
    -Portal hypertention and complications, including: known esophageal varices, poorly controlled or diuretic resistant ascites, history of variceal bleeds or related interventions (e.g., insertion of variceal bands or transjugular intrahepatic portosystemic shunt [TIPS]), hepatic encephalopathy
    -Cirrhosis with complications, including history or presence of: spontaneous
    -Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/dL (178 ?mol/L) bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN
    3. Patients with a history of severe pruritus requiring current or prior systemic treatment (e.g., with bile acid sequestrants [BAS] or rifampicin)
    4. Administration of the following medications is prohibited as specified below:
    -Prohibited 6 months prior to Day 0 and throughout the trial (i.e., to last dose and/or EOT): azathioprine, colchicine, cyclosporine, methotrexate,
    mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates;
    budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including ?-methyl-dopa, sodium valproic acid, isoniazide, or nitrofurantoin)
    -Prohibited 12 months prior to Day 0 and throughout the trial (i.e., to last dose and/or EOT): antibodies or immunotherapy directed against interleukins or other cytokines or chemokines
    5. Patients who have previously participated in a clinical trial of OCA will not be allowed to participate.
    6. History or presence of clinically concerning cardiac arrhythmias, or prolongation of Screening (pretreatment) QT or QTc interval of > 500 milliseconds (msec).
    7. If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
    Se excluirán del estudio aquellos pacientes que cumplan cualquiera de los siguientes criterios:
    1.Antecedentes o presencia de otras enfermedades hepáticas concurrentes, entre ellas:
    -Infección por el virus de la hepatitis B o C (VHB, VHC).
    -Colangitis esclerosante primaria (CEP).
    -Hepatitis alcohólica.
    -Enfermedad hepática autoinmune confirmada o síndrome de sobreposición
    -Esteatohepatitis no alcohólica (ENA).
    -Síndrome de Gilbert (exclusión debida a la interpretación de los niveles de bilirrubina).
    2.Presencia de complicaciones clínicas de la CBP o descompensación hepática clínicamente significativa, incluyendo:
    -Antecedentes de trasplante hepático, inclusión vigente en una lista de trasplante hepático o puntuación actual ?15 en la escala MELD.
    -Hipertensión portal y complicaciones portales, incluyendo: varices esofágicas conocidas, ascitis mal controlada o resistente a diuréticos, antecedentes de hemorragias de varices esofágicas o intervenciones relacionadas (p. ej., inserción de bandas varicosas o derivación intrahepática portosistémica transyugular [DIPST]), encefalopatía hepática.
    -Cirrosis con complicaciones, incluidos los antecedentes o la presencia de: peritonitis bacteriana espontánea, carcinoma hepatocelular, bilirrubina >2x LSN.
    -Síndrome hepatorrenal (tipo I o II) o creatinina sérica en el momento de la selección >2 mg/dl (178 ?mol/l).
    3.Pacientes con antecedentes de prurito intenso que requiere o ha requerido tratamiento polisistémico actual o anterior (p. ej., con secuestradores de ácidos biliares [SAB] o rifampicina).
    4.La administración de los siguientes medicamentos está prohibida en la medida indicada a continuación:
    -Medicación prohibida en los 6 meses anteriores al día 0 y durante todo el ensayo (hasta la última dosis y/o FDT): azatioprina, colchicina, ciclosporina, metotrexato, mofetil micofenolato, pentoxifilina, fenofibrato u otros fibratos, budesonida y otros corticoides sistémicos, fármacos potencialmente hepatotóxicos (entre ellos ?-metil-dopa, valproato sódico, isoniacida o nitrofurantoína).
    -Medicación prohibida en los 12 meses anteriores al día 0 y durante todo el ensayo (hasta la última dosis y/o FDT): anticuerpos o inmunoterapia dirigida contra interleucinas u otras citocinas o quimiocinas.
    5.No se permitirá la participación de los pacientes que hayan participado anteriormente en un ensayo clínico sobre el AOC.
    6.Antecedentes o presencia de arritmias cardíacas de interés clínico, o prolongación del intervalo QT o QTc >500 milisegundos (ms) en el momento de la selección (antes del tratamiento).
    7.En las mujeres: embarazo conocido, o prueba de embarazo positiva en orina (confirmada por una prueba de embarazo positiva en suero), o lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint (evaluated as a responder analysis):
    ALP < 1.67x ULN and total bilirubin within normal limits (WNL), and
    ALP decrease of ? 15% (to exclude clinically insignificant ALP changes)
    Variable principal (evaluado como análisis respondedor):
    FA<1.67x LSN y bilurribina total dentro de los límites de normalidad (DLN), y
    Disminución de FA ? 15% (para excluir cambios en FA clínicamente no significativos)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Double blind phase : Screening(? -1 to-8 Wks), Day 0, W2, M3, M6, M9, M12/LTSE D1, End of Treatment (if applicable), follow-up

    Long term safety extension: M3, M6, M9, M12, End of Treatment, follow up
    Fase doble ciego: selección ((? -1 a -8 semanas), Día 0, Semana 2, Mes 3, Mes 6, Mes 9, Mes 12/Día 1 de extensión a largo plazo, Fin de tratamiento (si aplica), seguimiento (si aplica)

    Extensión de seguridad a larga plazo: mes 3, Mes 6, Mes 9, Mes 12 (durante 5 años), Fin de tratamiento, seguimiento
    E.5.2Secondary end point(s)
    Analyses regarding secondary endpoints will be specified in the SAP and conducted for the following parameters. It is anticipated that additional disease prognostic algorithms will be published during the course of the trial. Where appropriate, secondary analyses will be conducted using such algorithms. At the blinded data review a determination will be made as to which algorithms will be evaluated statistically, as it is likely that there will be small numbers of patients for some algorithms and formal statistical analysis will not therefore be appropriate.

    - ALP response rates of 10%, 20% and 40% change
    - Disease Prognostic Risk (criteria in relevant patients):
    -ALP ? 3x ULN and AST ? 2x ULN and normal bilirubin
    - ALP ? 1.5x ULN and AST ? 1.5x ULN and bilirubin within normal limits
    - ALP ? 1.67x ULN and normal bilirubin
    - Normal bilirubin and normal albumin
    - Clinical laboratory values:
    - GGT, ALT, AST, total and conjugated bilirubin
    - Albumin, prothrombin time and INR
    -Liver biopsy/histology: Inflammatory, structural (portal, parenchymal) and fibrotic assessments
    - Disease Specific Symptoms:
    - PBC-40
    - 5-D Pruritus Questionnaire
    - Pruritus VAS
    - Biomarkers and non-invasive assessments of liver fibrosis
    - Fibrosis biomarkers (ELF)
    - TE (at selected trial sites)
    - Other analytes: TNF-?, TGF-?, IL-6, CK-18 and lysophosphatidic acid
    -Bile acids
    - Plasma OCA, other bile acids and conjugate concentrations
    - Bile and feces concentrations (at selected trial sites)
    Los análisis a realizar en relación a las variables secundarias se especificarán en el plan de análisis estadístico y se realizarán para los parámetros que se describen posteriormente. Se cree que durante el curso del estudio se publicarán nuevos algoritmos de pronóstico de la enfermedad, y donde sea apropiado, se realizarán análisis secundarios utilizando dichos algoritmos. En la revisión ciega de los datos se tomará la decisión de qué algoritmos se evaluarán estadísticamente ya que es probable que haya un pequeño número de pacientes para algunos algoritmos y entonces no estaría indicado un análisis estadístico formal.
    -Tasas de respuesta de la FA con un cambio de 10 %, 20 %, 40 % de cambio.
    -Riesgo pronóstico de enfermedad (criterios en pacientes relevantes):
    -FA ? 3x LSN y ASAT ? 2x LSN y bilirrubina normal
    -FA ? 1,5x LSN y ASAT? 1,5x LSN y bilirrubina dentro de los límites normales
    -FA ? 1,67x LSN y bilirrubina normal.
    -Bilirrubina normal y albúmina normal
    -Valores analíticos:
    -GT, ALAT, ASAT, bilirrubina total y conjugada.
    -Albúmina, tiempo de protrombina y CIN.
    -Biopsia/histología hepática: evaluaciones inflamatorias, estructurales (portal, parenquimatosa) y fibróticas
    -Síntomas específicos de la enfermedad:
    -Cuestionario 5-D
    -EAV del prurito
    -CBP-40.
    -Biomarcadores y evaluaciones no invasivas de fibrosis hepática:
    -Biomarcadores de fibrosis (FHM).
    -Elastografía transitoria (ET; en los centros del ensayo donde esté disponible).
    -Otros parámetros analíticos: FNT-?, FCT-?, IL-6, CC-18 y ácido lisofosfatídico.
    -Ácidos biliares
    -AOC plasmático, otros ácidos biliares y conjugados.
    -Concentraciones de ácidos biliares en la bilis y en las heces (en algunos centros del ensayo).
    E.5.2.1Timepoint(s) of evaluation of this end point
    At all study visits.
    En todas las visitas del estudio.
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) 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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 EEA40
    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
    Austria
    Belgium
    Canada
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Sweden
    Switzerland
    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
    Last visit of the last subject
    Última visita del último sujeto
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 years7
    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: 150
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 150
    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)
    Follow up visit occurring 4 weeks (± 7 days) after receiving the last dose of study medication.
    Visita de seguimiento a las 4 semanas ((± 7 días) tras recibir la última dosis de medicación del 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 Decision2012-02-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-12-17
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