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    The EU Clinical Trials Register currently displays   29761   clinical trials with a EudraCT protocol, of which   4552   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2014-004693-42
    Sponsor's Protocol Code Number:747-206
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-12-23
    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 ConcernedUK - MHRA
    A.2EudraCT number2014-004693-42
    A.3Full title of the trial
    A Multicenter, Randomized, Open Label, Single- and Multiple-Dose, Dose Finding Study and Open-Label Extension to Assess the Effects of Obeticholic Acid in Pediatric Subjects with Biliary Atresia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body.
    A.4.1Sponsor's protocol code number747-206
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/175/2014
    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 organisationWainwright Associates Limited
    B.5.2Functional name of contact pointAssociate Director, RA
    B.5.3 Address:
    B.5.3.1Street AddressWessex House, Marlow Road
    B.5.3.2Town/ cityBourne End
    B.5.3.3Post codeSL8 5SP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number4401628530554
    B.5.6E-mailenquiries@wainwrightassociates.co.uk
    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 nameOCA 1.5mg
    D.3.2Product code OCA
    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
    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 number1.5
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameOCA 5mg
    D.3.2Product code OCA
    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
    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.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
    Biliary atresia
    E.1.1.1Medical condition in easily understood language
    liver condition with abnormal bile flow
    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 18.0
    E.1.2Level LLT
    E.1.2Classification code 10004653
    E.1.2Term Biliary atresia
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10004654
    E.1.2Term Biliary atresia, congenital
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Single-Dose, Multiple-Dose (SD MD) Phase
    To assess the effects of OCA in eligible pediatric subjects with biliary atresia on:
    •Safety and tolerability
    •PK of OCA and its conjugates

    Open-Label Extension (OLE)
    To assess the effects of long-term OCA treatment in eligible pediatric subjects with biliary atresia on:
    •Long-term safety and tolerability

    E.2.2Secondary objectives of the trial
    Single-Dose, Multiple-Dose (SD MD) Phase
    To assess the effects of OCA in eligible pediatric subjects with biliary atresia on:
    •Efficacy of OCA as measured by hepatobiliary indices of liver function
    •PD of OCA as measured by appropriate biomarkers
    •Exposure-response (PK/PD) and exposure-tolerability relationships

    Open-Label Extension (OLE)
    To assess the effects of long-term OCA treatment in eligible pediatric subjects with biliary atresia on:
    •Durability of efficacy as measured by hepatobiliary indices of liver function
    •PK, PD as measured by appropriate biomarkers
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male or female pediatric subjects, aged <18 years
    2.Diagnosis of biliary atresia, with successful HPE (also known as Kasai portoenterostomy) as defined by total bilirubin <2 mg/dL (34.2 μmol/L) at least 3 months post-HPE procedure.
    3.Gamma-glutamyl transferase (GGT) ≥2x ULN
    4.Able to take medications orally (ie, prior to the availability of the suspension formulation, able to swallow tablet(s) whole)
    5.Prior to the availability of the suspension formulation, this study will initially restrict subject enrollment by weight into the 3 OCA dose groups as follows (ie, subjects must meet these restrictions at Screening and Day 0):
    - Low Dose Group: ≥42 kg
    - Medium Dose Group: ≥20 kg
    - High Dose Group: ≥21 kg

    E.4Principal exclusion criteria
    Subjects with or experiencing:
    1. Prior liver transplant or active status on transplant list
    2.Complications of decompensated cirrhosis including:
    a. Total bilirubin ≥2 mg/dL (34.2 μmol/L)
    b. Platelets <40,000/µL
    c. INR ≥2
    d. Presence of known or large esophageal varices
    e. Uncontrolled ascites (diuretic resistant)
    f. Prior placement of portosystemic shunt
    g. Diagnosis of hepatopulmonary syndrome or portopulmonary hypertension
    3.Current intractable pruritus or those requiring systemic treatment for pruritus within 3 months (eg, with bile acid sequestrants or rifampicin)
    E.5 End points
    E.5.1Primary end point(s)
    SAFETY. Adverse events and laboratory values

    PK. Plasma concentrations of OCA and its conjugates (glycine 6α-ethyl-chenodeoxycholic acid [glyco-OCA] and taurine 6α-ethyl-chenodeoxycholic acid [tauro-OCA]) after single- and multiple-doses
    E.5.1.1Timepoint(s) of evaluation of this end point
    PK sampling on days 1, 2, 7, 42, 63, and 64 in the single dose and multi dose phases.
    E.5.2Secondary end point(s)
    EFFICACY. Alkaline phosphatase [ALP], aspartate aminotransferase [AST], alanine aminotransferase [ALT], gamma-glutamyl transferase [GGT], total and conjugated bilirubin, albumin, and international normalized ratio [INR]).

    PD. 7-hydroxy-4-cholesten-3-one [C4], and endogenous bile acids.

    PK/PD. Relationship of total OCA exposure to PD parameters, efficacy measures, Child-Pugh Score and adverse events
    E.5.2.1Timepoint(s) of evaluation of this end point
    C4 and endogenous bile acids taken on Days 1, 42 and 63.
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 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 Yes
    E.8.2.3.1Comparator description
    standard care
    E.8.2.4Number of treatment arms in the trial2
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    Canada
    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 years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 60
    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) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 15
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 22
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 23
    F.1.2Adults (18-64 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    All of the patients in this trial are children younger than 18 years.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 60
    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)
    Standard care for biliary atresia
    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 Decision2015-06-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-03-06
    P. End of Trial
    P.End of Trial StatusOngoing
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