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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7272   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2016-002965-67
    Sponsor's Protocol Code Number:57877.018.016
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-03-29
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-002965-67
    A.3Full title of the trial
    An open, non-randomized, parallel-group pharmokinetic and -dynamic, investigator-initiated study on effects of obeticholic acid (OCA) in bile of patients with primary biliary cholangitis (PBC) and non-alcoholic steatohepatitis (NASH) in comparison to healthy controls
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study on the effect of obetihcolic acid on bile composition in patients with PBC (chronic liver disease of the small bile ducts), NASH (chronic liver disease as a result of fatty liver) and healthy volunteers
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code number57877.018.016
    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 SponsorAMC
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAMC
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMC
    B.5.2Functional name of contact pointE.S. de Vries
    B.5.3 Address:
    B.5.3.1Street AddressMeibergdreef 9
    B.5.3.2Town/ cityAmsterdam Zuidoost
    B.5.3.3Post code1105 AZ
    B.5.4Telephone number00310205667375
    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.3.11 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary biliairy cholangitis (PBC)
    Non-alcoholic steatohepatitis (NASH)
    E.1.1.1Medical condition in easily understood language
    PBC = chronic liver disease which affects the small bile ducts
    NASH = chronic liver disease due to fatty liver
    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 20.0
    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 explore the exact working mechnism of OCA in PBC / NASH patients in comparison with healthy volunteers -->

    Assess the effect of obeticholic acid (OCA) in patients with PBC [in combination with long-term UDCA treatment (15 mg/kg/d)], patients with NASH and healthy volunteers on the bile acid and lipid composition of bile and serum,
    E.2.2Secondary objectives of the trial
    Assess the effect of obeticholic acid (OCA) in patients with PBC [in combination with long-term UDCA treatment (15 mg/kg/d)], patients with NASH and healthy volunteers on the biliary and serum levels of cytokines (e.g., IL-6, IL-8, TNFα), chemokines and enterohepatic hormones (e.g., FGF-19)

    Assess the effect of obeticholic acid (OCA) in patients with PBC [in combination with long-term UDCA treatment (15 mg/kg/d)], patients with NASH and healthy volunteers on the bile acid composition and microbiome in faeces

    Assess the effect of obeticholic acid (OCA) in patients with PBC [in combination with long-term UDCA treatment (15 mg/kg/d)], patients with NASH and healthy volunteers on the expression of duodenal transport proteins and enzymes contributing to the biliary and intestinal detoxification machinery
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female patients / volunteers of 18 to 75 years of age.
    - Body mass index within 18 to 40 kg/m2
    - Positive AMA (anti-mitochondrial antibody) testing (for PBC patients).
    - Proven non-cirrhotic liver disease compatible with PBC stage I, II, III or NASH, no reliable signs of portal hypertension such as esophageal varices or ascites and/or pylephlebectasia > 15 mm, and a fibroscan < 15 kPa.
    - The diagnosis of NASH must be confirmed by a liver biopsy (e.g. according Brunt criteria) in the last 24 months
    E.4Principal exclusion criteria
    - Existing cardiac / hematological / renal / gastrointestinal diseases which might interfere with the drugs' safety, tolerability, absorption, pharmacokinetics and / or endoscopy.
    - Other acute or chronic diseases which might affect absorption or metabolism of OCA
    - Existing disorders of the coagulation system or treatment with anticoagulants or agents inhibiting thrombocyte aggregation
    - Positive anti-HIV-test, HBsAg-test or anti-HCV-test.
    - Acute inflammation of the gallbladder.
    - Cholecystectomy
    - Histologically proven cirrhotic liver disease or total bilirubin > 2 mg/dl or reliable signs of portal hypertension such as esophageal varices or ascites and/or pylephlebectasia > 15 mm and / or fibroscan > 15 kPa.
    E.5 End points
    E.5.1Primary end point(s)
    - Bile acids: Concentrations of UDCA, cholic acid (CA), chenodeoxycholic acid (CDCA), deoxycholic acid (DCA), lithocholic acid (LCA), glycoursodeoxycholic acid (GUDCA), glycocholic acid (GCA), glycochenodeoxycholic acid (GCDCA), glycodeoxycholic acid (GDCA), glycolithocholic acid (GLCA), tauroursodeoxycholic acid (TUDCA), taurocholic acid (TCA), taurochenodeoxycholic acid (TCDCA), taurodeoxycholic acid (TDCA), taurolithocholic acid (TLCA) and C4 will be determined in plasma and bile by liquid chromatography tandem mass spectrometry according to the principles of good laboratory practice.

    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 0, 29/30, day 35
    E.5.2Secondary end point(s)
    - Lipids: The biliary lipid profile will be analyzed (before and 4 weeks after start of treatment with OCA) using a recently developed AMC lipidomics platform which allows to detect and quantify ~1000 different lipids in bile.

    - Cyto- and chemokines and hormones: A panel of 27 cyto- and chemokines will be analyzed in bile after dilution (50-100 fold) using Luminex® technology (Merck Millipore, Milliplex Map Human Cytokine/Chemokine Panel). IL6, IL8 and TNFα levels in bile will be quantified by ELISA (R&D Systems, #DY-206, #DY-208 and #DY-210). FGF19 levels in bile and serum will be determined by sandwich ELISA; bile samples will be tested for proteolytic activity by spiking with FGF19.

    Serum: CK-18 (a biomarker correlating with hepatocyte apoptosis)

    - Total biliary levels of bile salts, phospholipids and cholesterol will be determined by enzymatic methods. Total bilirubin will be quantified by HPLC.

    - Faeces: microbiome, bile acid composition
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 0, 29/30, day 35
    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 No
    E.6.5Efficacy No
    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) 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 33
    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 Yes
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state33
    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)
    After terminating the study, patients will have the opportunity to participate in a long term safety extension for 2 years for which OCA would be supplied by Intercept. Follow up visits would be scheduled in intervals of 3 months for clinical and biochemical evaluation
    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 Decision2017-07-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-02-27
    P. End of Trial
    P.End of Trial StatusOngoing
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