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    Summary
    EudraCT Number:2019-001015-23
    Sponsor's Protocol Code Number:APHP180668
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-06-04
    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 ConcernedFrance - ANSM
    A.2EudraCT number2019-001015-23
    A.3Full title of the trial
    Double blind, multicentric, randomized, placebo-controlled trial, evaluating the efficacy of 24-month of bezafibrate in primary sclerosing cholangitis with persistent cholestasis despite ursodeoxycholic acid therapy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study evaluating the efficacy of bezafibrate for people suffering of primary sclerosing cholangitis
    A.3.2Name or abbreviated title of the trial where available
    BEZASCLER
    A.4.1Sponsor's protocol code numberAPHP180668
    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 SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    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 supportMinistère de la santé
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointSophie COURTIAL-DESTEMBERT
    B.5.3 Address:
    B.5.3.1Street AddressDRCI Hôpital St Louis, 1 av. Claude Vellefaux
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number+33140275591
    B.5.5Fax number+33144841701
    B.5.6E-mailsophie.courtial-destembert@aphp.fr
    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 BEFIZAL L.P. 400 mg, comprimé enrobé à libération prolongée
    D.2.1.1.2Name of the Marketing Authorisation holderARROW GENERIQUES
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 namebezafibrate
    D.3.2Product code C10AB02
    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 INNBEZAFIBRATE
    D.3.9.1CAS number 41859-67-0
    D.3.9.4EV Substance CodeSUB05810MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    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.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
    Adult patients with primary sclerosing cholangitis
    E.1.1.1Medical condition in easily understood language
    Primary sclerosing cholangitis
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    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 efficacy of 24-month treatment with bezafibrate (400 mg SR/d) versus placebo in addition to standard UDCA therapy in primary sclerosing cholangitis
    E.2.2Secondary objectives of the trial
    1. To compare between groups:
    a) Components of the primary composite outcome at M24
    b) Adverse effects including IBD activity hepatic, muscular, and kidney
    function.
    c) Quality of life and scores for pruritus and fatigue at M12 and M24
    d) Changes in liver tests between M0 and M24
    e) Occurrence of clinical events and transplant-free survival
    2. Exploratory objectives:
    - Changes in serum markers of fibrosis (ELF score and Pro-C3) and
    cholangiographic abnormalities between M0 and M24
    - Course of biomarkers (including microbiota) and correlation with
    observed effects between M0 and M24
    - Need for endoscopic procedures between M0 and M24
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Males or females ≥ 18 and ≤ 75 years
    • Large duct PSC verified by retrograde, operative, percutaneous or
    magnetic resonance cholangiography (MRC) demonstrating intrahepatic
    and /or extrahepatic biliary duct changes consistent with PSC
    • Colonoscopy within the last 5 years (or within 3 months if IBD is
    associated to PSC) with no cancer nor all-grade dysplasia
    • ALP ≥ 1.5 ULN at baseline
    • Treatment with UDCA (15-20 mg/kg/d) for ≥ 6 months before
    inclusion.
    • Using
    contraceptive in women of childbearing potential. Women of childbearing potential, i.e. fertile, following menarche and until becoming post-menopaused unless permanently sterile, who are sexually active have to apply a highly effective method of birth control with a low failure rate (i.e., less than 1% per year) when used constantly and correctly.
    • Affiliation to a social security system (AME excepted)
    • Signed informed consent
    E.4Principal exclusion criteria
    • Child-Pugh score B or C
    • Ascites or digestive hemorrhage (or history of)
    • Total bilirubin in the last 3 months > 50 μmole/L (3 mg/dl)
    • Gilbert syndrome defined as unconjugated bilirubinemia > 12 μmol/L
    • Albumin in the last 3 months < 35 g/L
    • Prothrombin index in the last 3 months < 70%
    • Platelets count in the last 3 months < 100000/mm3
    • ALT or AST > 5 ULN in the last 3 months
    • Prior liver transplantation
    • Treatment with a fibrate within the last 3 months inclusion or with a
    statin at inclusion
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with serum Alkaline Phosphatase < 1.5 ULN and a reduction of at least 15% from baseline at M24 and normal serum bilirubin and no increase of liver stiffness at M24 compared to Baseline (delta M24–M0 ≤ 0).
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months
    E.5.2Secondary end point(s)
    1. To compare between groups
    a) Components of the primary composite outcome analyzed separately:
    Proportion of patients with:
    • serum Alkaline Phosphatase < 1.5 ULN at M24 and at least 15% of
    decrease from baseline at M24
    • complete normalization of s-ALP at M24 (s-ALP ≤ 1.0 ULN at M24)
    • normal serum bilirubin
    • no increase in liver stiffness at M24
    b) Safety endpoint: Percentage of patients with clinical (including
    increased IBD activity) or biological adverse events (elevated creatinine,
    ALT, AST or CPK) during the study period;
    c) Quality of life (QMCF questionnaire – Questionnaire de la maladie
    chronique du foie) and scores for pruritus (measured by VAS and 5D
    pruritus scale) and fatigue (measured by adapted PBC-40 questionnaire
    (M0, M12 and M24))
    d) Changes in Patient-Reported Outcomes (PRO) specific for PSC (45).
    e) Changes in biochemical liver tests other than ALP, including total and
    conjugated bilirubin, GGT, AST, ALT, albumin, and INR, and in platelet
    count. Changes in total cholesterol, LDL, HDL and triglycerides.
    f) Survival rate without liver transplantation or hepatic events (ascites,
    variceal bleeding, encephalopathy, acute cholangitis,
    cholangiocarcinoma, hepatocellular carcinoma or serum total bilirubin >
    100 μmol/L for at least 3 months). PSC Prognostic scores including the
    MELD score, the Revised PSC Mayo Risk Score, the Hannover Score and
    the Amsterdam-Oxford prognostic model (M0, M12, M24)
    2. Exploratory endpoints (M0, M12, M24)
    • Course of:
    - serum markers of liver fibrosis (ELF score and Pro-C3)
    - cholangiographic abnormalities
    - biological markers of liver function
    - bile acids and cytokines
    - microbiota (M0-M12-M24) (dietetary questionnaire and questionnaire
    accompanying the stool sample)
    • Need for endoscopic treatment of biliary strictures
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 months
    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 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 concerned35
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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: 52
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 52
    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 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 state104
    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)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation French Network of Reference and Competence
    G.4.3.4Network Country France
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-05-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-02
    P. End of Trial
    P.End of Trial StatusOngoing
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