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    Summary
    EudraCT Number:2021-001431-56
    Sponsor's Protocol Code Number:NUT-2/PBC
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-11-03
    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 ConcernedGermany - BfArM
    A.2EudraCT number2021-001431-56
    A.3Full title of the trial
    Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of norucholic acid tablets with placebo in the treatment of primary biliary cholangitis in patients with an inadequate response to ursodeoxycholic acid
    Doppelblinde, randomisierte, placebokontrollierte Dosisfindungsstudie der Phase II zur Untersuchung von unterschiedlichen Dosen von Norucholsäure im Vergleich zu Placebo zur Behandlung von primärer biliärer Cholangitis bei Patienten mit unzurechendem Ansprechen auf Ursodesoxycholsäure
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study comparing two doses of norucholic acid against placebo in the treatment of a liver disease called primary biliary cholangitis in patients without sufficient response to ursodeoxycholic acid
    A.3.2Name or abbreviated title of the trial where available
    NCA vs. placebo in PBC
    A.4.1Sponsor's protocol code numberNUT-2/PBC
    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 SponsorDr. Falk Pharma GmbH
    B.1.3.4CountryGermany
    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 supportDr. Falk Pharma GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDr. Falk Pharma GmbH
    B.5.2Functional name of contact pointDept. of Clinic. Res. & Development
    B.5.3 Address:
    B.5.3.1Street AddressLeinenweberstr. 5
    B.5.3.2Town/ cityFreiburg
    B.5.3.3Post code79108
    B.5.3.4CountryGermany
    B.5.4Telephone number+4976115140
    B.5.5Fax number+497611514377
    B.5.6E-mailzentrale@drfalkpharma.de
    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 nameNorucholic acid (NCA) 500 mg
    D.3.2Product code NCA 500mg
    D.3.4Pharmaceutical form Film-coated 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 INNNorucholic acid
    D.3.9.1CAS number 9697-24-2
    D.3.9.2Current sponsor codeNCA
    D.3.9.3Other descriptive namenorucholic acid
    D.3.9.4EV Substance CodeSUB179704
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 placeboFilm-coated tablet
    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 is a rare chronic liver disease
    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 27.0
    E.1.2Level PT
    E.1.2Classification code 10080429
    E.1.2Term Primary biliary cholangitis
    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 evaluate the efficacy of two doses of norucholic acid vs. placebo for the treatment of primary biliary cholangitis (PBC) in patients with an inadequate response to ursodeoxycholic acid (UDCA).
    E.2.2Secondary objectives of the trial
    To identify efficacious norucholic acid dose(s) for the treatment of PBC for further evaluation in phase III,
    To study safety and tolerability (adverse events, laboratory parameters) of norucholic acid.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Signed informed consent,
    • Male or female patients ≥ 18 and ≤ 74 years at screening,
    • PBC verified by at least 2 out of the following 3 criteria at screening:
    - Chronic cholestatic disease of at least 12 months duration,
    - Positive anti-mitochondrial antibody (AMA) titer or, if AMA negative or in low titer (< 1:80), presence of 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]),
    - Liver biopsy available for review and compatible with the diagnosis of non-cirrhotic PBC,
    • Ursodeoxycholic acid (UDCA) treatment for at least 12 months (with a stable dose for ≥ 3 months) prior to screening,
    • Women of childbearing potential agreeing to use a highly effective method of birth control during the entire duration of the trial and for 4 weeks following the last dose of trial treatment, defined as those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptive methods, some intrauterine devices (IUD), sexual abstinence, or vasectomized partner. Women of non-childbearing potential (surgically sterile [e.g., hysterectomy, bilateral salpingectomy, bilateral oophorectomy], or postmenopausal with at least two years without spontaneous menses) may be included. The investigator is responsible for determining whether the patient has adequate birth control for trial participation.
    E.4Principal exclusion criteria
    • History or presence of any other relevant concomitant liver diseases.
    • Patients taking prohibited medications
    • Liver cirrhosis. NOTE: Patients with compensated cirrhosis and a Child-Pugh Score <8 are allowed to participate,
    • Any known relevant infectious disease (e.g., active tuberculosis, acquired immunodeficiency syndrome [AIDS]-defining diseases),
    • Abnormal renal function (glomerular filtration rate estimated from cystatin C < 30 ml/min) at screening visit,
    • Previous or concurrent cancer except cervical carcinoma in situ, treated basal cell carcinoma, or any cancer curatively treated < 3 years before screening,
    • Existing or intended pregnancy or breast-feeding
    E.5 End points
    E.5.1Primary end point(s)
    Mean relative change (%) in ALP between the baseline visit and the EOT visit (Last Observation Carried Forward, LOCF).
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of Treatment, 12 weeks after baseline
    E.5.2Secondary end point(s)
    1. Proportions of patients with at least 10%, at least 20%, and at least 40% reduction in ALP between baseline and End of treatment (LOCF),
    2. Proportion of patients with normalisation of ALP (< ULN) at any visit during the treatment phase,
    3. Proportion of patients with bilirubin levels <0.6x ULN at any visit during the treatment phase,
    4. Proportion of patients with partial normalisation of ALP (< 1.5x ULN) at any visit during the treatment phase,
    5. ALP at each trial visit (screening to follow-up),
    6. Absolute and relative changes (%) of ALP from baseline to each visit up to End of treatment (LOCF), and from End of treatment to the follow-up visit,
    7. Gamma-glutamyltransferase (γ-GT), AST, ALT, albumin, platelet count and total and conjugated bilirubin levels at each trial visit (screening to follow-up),
    8. Absolute and relative changes (%) of γ-GT, AST, ALT, albumin, platelet count and total and conjugated bilirubin levels from baseline to each visit up to End of treatment (LOCF), and from End of treatment to the follow-up visit.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. End of Treatment, 12 weeks after baseline
    2. any visit during the treatment phase
    3. any visit during the treatment phase
    4. any visit during the treatment phase
    5. at each trial visit
    6. End of treatment and follow-up visit
    7. at each trial visit
    8. End of treatment and follow-up visit
    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 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 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 Yes
    E.8.2.3.1Comparator description
    two different doses of IMP: 1500mg/d and 1000mg/d NCA
    E.8.2.4Number of treatment arms in the trial3
    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 concerned11
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA43
    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
    Switzerland
    United Kingdom
    Austria
    Belgium
    Denmark
    Finland
    France
    Germany
    Netherlands
    Norway
    Poland
    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 years2
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    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: 80
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 70
    F.4.2.2In the whole clinical trial 90
    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)
    Treatment after trial end (i.e., after FU [follow-up visit]) is left to the investigator’s discretion.
    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 Decision2021-12-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-12-12
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