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    Summary
    EudraCT Number:2015-001590-41
    Sponsor's Protocol Code Number:CLJN452X2201
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-01-13
    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 ConcernedGermany - BfArM
    A.2EudraCT number2015-001590-41
    A.3Full title of the trial
    A multi-part, randomized, double-blind, placebo-controlled study to assess the safety, tolerability and efficacy of tropifexor (LJN452) in patients with primary biliary cholangitis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multi-part, double blind study to assess safety, tolerability and efficacy of tropifexor (LJN452) in PBC patients
    A.3.2Name or abbreviated title of the trial where available
    Multi-part, double blind study to assess safety, tolerability and efficacy of LJN452 in PBC patients
    A.4.1Sponsor's protocol code numberCLJN452X2201
    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 SponsorNovartis Pharma Services AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma GmbH
    B.5.2Functional name of contact pointMedizinischer Infoservice (MCC)
    B.5.3 Address:
    B.5.3.1Street AddressRoonstrasse 25
    B.5.3.2Town/ cityNürnberg
    B.5.3.3Post code90429
    B.5.3.4CountryGermany
    B.5.4Telephone number+491802232300
    B.5.5Fax number+4991127312160
    B.5.6E-mailinfoservice.novartis@novartis.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code LJN452, 0,01 mg
    D.3.4Pharmaceutical form Capsule, hard
    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 INNtropifexor
    D.3.9.2Current sponsor codeLJN452
    D.3.9.3Other descriptive nameLJN452
    D.3.9.4EV Substance CodeSUB174477
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.01
    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.3.2Product code LJN452, 0,03 mg
    D.3.4Pharmaceutical form Capsule, hard
    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 INNtropifexor
    D.3.9.2Current sponsor codeLJN452
    D.3.9.3Other descriptive nameLJN452
    D.3.9.4EV Substance CodeSUB174477
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.03
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code LJN452, 0,1 mg
    D.3.4Pharmaceutical form Capsule, hard
    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 INNtropifexor
    D.3.9.3Other descriptive nameLJN452
    D.3.9.4EV Substance CodeSUB174477
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.1
    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 placeboCapsule, hard
    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 cholangitis
    E.1.1.1Medical condition in easily understood language
    primary biliary cholangitis
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10036680
    E.1.2Term Primary biliary cirrhosis
    E.1.2System Organ Class 100000004871
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To determine the safety and tolerability of daily dosing of LJN452 in patients with Primary Biliary Cholangitis
    • To determine the effect of LJN452 on cholestatic markers in patients with Primary Biliary Cholangitis. More specifically, in Part 2 to determine the dose-response relationship of LJN452 on GGT following 12 weeks of treatment
    E.2.2Secondary objectives of the trial
    •To determine the change in the itch domain of PBC40 questionnaire
    •To evaluate the change in overall disease specific quality of life
    •To evaluate the change in itch based on 100 mm visual analog score (VAS)
    •To evaluate the pharmacokinetics (PK) of LJN452 in patients with PBC
    • Part 2: To determine the dose-response relationship of LJN452 on ALP in patients with PBC following 12 weeks of treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ●Diagnosis of PBC as demonstrated by the presence of ≥ 2 of the
    following 3 diagnostic criteria: -History of Alkaline Phosphatase elevated
    above upper limit of normal for at least 6 months
    - Positive anti-mitochondrial antibodies (AMA) titer or if AMA negative or
    in low titer (<1:80) PBC specific antibodies (anti- GP210 and/or anti-
    SP100 and/or antibodies against the major M2 components (PDC-E2, 2-
    oxo-glutaric acid dehydrogenase complex)
    - Previous liver biopsy consistent with PBC
    ●At least 1 of the following markers of disease severity: - ALP ≥ 1.67×
    upper limit of normal (ULN) -  Total bilirubin > ULN but < 1.5 × ULN
    In addition, patients must meet the following biochemical criteria at
    enrollment
     ALT/AST ≤5 × ULN
     Total bilirubin ≤1.5 × ULN
     INR ≤ ULN
    ●Subjects must weigh at least 40 kg to participate in the study, and
    must have a body mass index (BMI) within the range of
    18 - 40 kg/m^2. BMI = Body weight (kg) / [Height (m)]^2.
    ●Able to communicate well with the investigator, to understand and
    comply with the requirements of the study.
    E.4Principal exclusion criteria
    ● Women of child-bearing potential, defined as all women physiologically
    capable of becoming pregnant, unless they are using highly effective
    methods of contraception for 30 days before randomization, during
    dosing and for 30 days following the end of treatment.
    Presence of other concomitant liver diseases.
    ● Cirrhosis with complications, including history or presence of:
    ● Variceal bleed
    ● Uncontrolled ascites
    ● Encephalopathy
    ● Spontaneous bacterial Peritonitis
    ● Significant hepatic impairment as defined by Child-Pugh classification
    of B or C, history of liver transplantation, current placement on a liver
    transplant list or current Model for End Stage Liver Disease (MELD)
    score ≥15.
    ● History of conditions that may cause increases in ALP (e.g., Paget's
    disease).
    ● Use of investigational drugs or immunosuppressive drugs at the time of
    enrollment, or within 5 half-lives or 30 days of randomization, whichever
    is longer; or longer if required by local regulations. Use of high dose oral
    steroids to treat co-morbid conditions (e.g., airways disease) will be
    allowed but must be properly documented as such in concomitant
    medications.
    ● Currently taking obeticholic acid or have taken obeticholic acid within
    30 days of randomization
    ● Previous participation in CLJN452X2201 and received study medication
    within three months of randomization (or longer if required by local
    regulations).
    E.5 End points
    E.5.1Primary end point(s)
    1. Change in cholestatic markers from baseline
    2. Number of patients with adverse events, serious adverse events and death
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. 28 days
    2. 86 days
    E.5.2Secondary end point(s)
    1. Change from baseline in disease specific quality of life
    2. Change from baseline in itch as determined by the itch domain in the PBC-40
    3. Change from baseline in itch as determined by 10mm VAS
    4. Area under the plasma concentration-time profile (AUCtau)
    5. Maximum plasma concentration of LJN452 (Cmax)
    6. Minimum plasma concentration of LJN452 (Cmin)
    7. Average steady state plasma concentration following multiple doses of LJN452 (Cav,ss)
    8. Time to reach maximum concentration after drug administration (Tmax)
    9. Apparent systemic clearance from plasma (CL/F)
    10. Accumulation ratio of LJN452 (Racc)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. BL, 28 days
    2. BL, 28 days
    3. BL, 28 days
    4. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    5. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    6. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    7. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    8. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    9. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    10. Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56
    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 No
    E.6.10Pharmacogenetic Yes
    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 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    China
    Germany
    Poland
    Russian Federation
    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
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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: 123
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 29
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 83
    F.4.2.2In the whole clinical trial 152
    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)
    Patients in part 1 of the trial take ursodeoxycholic acid (UDCA) additionally, however, it will not have a sufficient effect on the treatment. Patient in part 2 will not take UDCA during the trial, but they can start a treatment with UDCA as soon as the visit on day 56 has been done.
    Generally further treatment is left to the physician to assess the best adequate treatment for each patient.
    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 Decision2015-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-01-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-08-02
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