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    Summary
    EudraCT Number:2011-004728-36
    Sponsor's Protocol Code Number:747-301
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-04-20
    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 ConcernedPoland - Office for Medicinal Products
    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
    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
    A.4.1Sponsor's protocol code number747-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01473524
    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 Yes
    D.2.1.1.1Trade name Ocaliva
    D.2.1.1.2Name of the Marketing Authorisation holderIntercept Pharma Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.9.4EV Substance CodeSUB91981
    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 Yes
    D.2.1.1.1Trade name Ocaliva
    D.2.1.1.2Name of the Marketing Authorisation holderIntercept Pharma Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.9.4EV Substance CodeSUB91981
    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.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
    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.
    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 assess the effects of OCA in patients with PBC on:
    -Serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint
    -Safety
    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
    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).
    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
    -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 for 30 days after the End of Treatment (EOT) visit.
    6. Must provide written informed consent and agree to comply with the trial protocol.
    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 C virus (HCV) infection; patients with active hepatitis B (HBV) infection will be excluded, however, patients who have seroconverted
    (HbsAg and Hbe Ag negative) may be included after consultation with the medical monitor.
    -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 with complications, including: known gastric or
    large esophageal varices, poorly controlled or diuretic resistant ascites,
    history of variceal bleeds or related therapeutic or prophylactic
    interventions (e.g., beta blockers, insertion of variceal bands or
    transjugular intrahepatic portosystemic shunt [TIPS]), or 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 μmol/L) bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN
    3. Patients with severe pruritus or those requiring systemic treatment
    for pruritus (e.g., with bile acid sequestrants [BAS] or rifampicin) within
    2 months of Day 0 will be excluded.
    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 α-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 likely
    to affect survival during the trial, 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
    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)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Time expected for all patients to be enrolled: Approximately 12 months

    Duration of individual patient participation: 12 months during the DB
    phase; Up to 5 years (60 months) in the LTSE

    Total duration of trial (excluding data collection and analysis):
    DB phase: 24 months (2 years) with open label LTSE phase: 84 months
    (7 years)
    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-α, TGF-β, 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)
    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 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 concerned5
    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
    Australia
    Canada
    Switzerland
    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 years7
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years7
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 150
    F.4.2.2In the whole clinical trial 180
    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 at the end of the double-blind phase.
    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 Decision2012-05-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-04-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-12-17
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