Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44364   clinical trials with a EudraCT protocol, of which   7388   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-003443-31
    Sponsor's Protocol Code Number:NUT-3/NAS
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2023-08-21
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-003443-31
    A.3Full title of the trial
    Double-blind, randomised, placebo-controlled, phase IIb trial on the efficacy and safety of norursodeoxycholic acid tablets in patients with non-alcoholic steatohepatitis (NASH)
    Studio in doppio cieco, randomizzato, controllato con placebo, di fase IIb sull’efficacia e la sicurezza delle compresse di acido norursodesossicolico in pazienti affetti da steatoepatite non alcolica (NASH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial comparing the efficacy and safety of norursodeoxycholic acid tablets with placebo in the treatment of non-alcholic fatty liver disease with liver inflammation ("non-alcoholic steatohepatitis"[NASH])
    Studio clinico che confronta l'efficacia e la sicurezza delle compresse di acido norursodesossicolico con il placebo nel trattamento della steatosi epatica non alcolica con l'infiammazione del fegato ("steatoepatite non alcolica"[NASH])
    A.3.2Name or abbreviated title of the trial where available
    Norursodeoxycholic acid vs. placebo in NASH
    Acido norursodesossicolico vs placebo nella NASH
    A.4.1Sponsor's protocol code numberNUT-3/NAS
    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 pointDepartment of Clinical Research
    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 number004976115140
    B.5.5Fax number00497611514377
    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 nameacido norursodesossicolico (NorUDCA)
    D.3.2Product code [NA]
    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 INNacido norucolico
    D.3.9.1CAS number 9697-24-2
    D.3.9.2Current sponsor codenorUDCA
    D.3.9.3Other descriptive nameNOR-URSODEOXYCHOLIC ACID, NCA
    D.3.9.4EV Substance CodeSUB31746
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Non-alcoholic steatohepatitis (NASH)
    Steatoepatite non alcolica (NASH)
    E.1.1.1Medical condition in easily understood language
    Non-alcoholic steatohepatitis
    Steatoepatite non alcolica
    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 24.1
    E.1.2Level PT
    E.1.2Classification code 10053219
    E.1.2Term Non-alcoholic steatohepatitis
    E.1.2System Organ Class 10019805 - Hepatobiliary disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of norursodeoxycholic acid (norUDCA) 1500 mg vs. norUDCA 1000 mg vs. placebo for the treatment of NASH
    Valutare l'efficacia dell'acido norursodesossicolico (norUDCA) 1500 mg rispetto a norUDCA 1000 mg rispetto al placebo per il trattamento della NASH
    E.2.2Secondary objectives of the trial
    To study safety and tolerability (adverse events [AEs], laboratory parameters) of norUDCA
    Studiare la sicurezza e la tollerabilità (eventi avversi [AE], parametri di laboratorio) di norUDCA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Must be willing to participate in the study and provide written informed consent
    • Male or female patients = 18 and < 75 years
    • Centrally assessed histological evidence of NASH and liver fibrosis
    • Women of childbearing potential agree 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
    • Essere disposto a partecipare allo studio e fornire il consenso informato scritto
    • Pazienti maschi o femmine = 18 e < 75 anni
    • Evidenza istologica valutata centralmente di NASH e fibrosi epatica
    • Le donne in età fertile accettano di utilizzare un metodo contraccettivo altamente efficace durante l'intera durata dello studio e per 4 settimane dopo l'ultima dose del trattamento di prova
    E.4Principal exclusion criteria
    • Patients taking prohibited medications
    • Presence of liver cirrhosis
    • Type 1 diabetes or uncontrolled Type 2 diabetes
    • History or presence of any other significant concomitant liver diseases
    • History of liver transplantation
    • BMI >45 kg/m^2
    • 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
    • Any active malignant disease (except for basal cell carcinoma)
    • Existing or intended pregnancy or breast-feeding
    • Pazienti che assumono farmaci non accettati
    • Presenza di cirrosi epatica
    • Diabete di tipo 1 o diabete di tipo 2 non controllato
    • Storia o presenza di altre malattie epatiche concomitanti significative
    • Storia di trapianto di fegato
    • BMI >45 kg/m^2
    • Qualsiasi malattia infettiva rilevante nota (ad es. tubercolosi attiva, sindrome da immunodeficienza acquisita [AIDS] che definisce malattie)
    • Funzionalità renale anormale (velocità di filtrazione glomerulare stimata dalla cistatina C < 30 ml/min) alla visita di screening
    • Qualsiasi malattia maligna attiva (ad eccezione del carcinoma a cellule basali)
    • Gravidanza esistente o prevista o allattamento
    E.5 End points
    E.5.1Primary end point(s)
    Resolution of NASH, assessed by centrally scored liver histology, and no worsening of fibrosis from baseline to EOT/withdrawal visit
    AND/OR
    Improvement of fibrosis, and no worsening of NAS from baseline to EOT/withdrawal visit
    Risoluzione della NASH, valutata dall'istologia epatica con punteggio centrale e nessun peggioramento della fibrosi dal basale alla visita EOT/di sospensione
    E/O
    Miglioramento della fibrosi e nessun peggioramento del NAS dal basale alla visita EOT/ritiro
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 72 (EOT)/withdrawal visit
    settimana 72 (EOT)/visita di ritiro
    E.5.2Secondary end point(s)
    • Improvement of NASH and no worsening of fibrosis from baseline to EOT/withdrawal
    • Change in NAS from baseline to EOT/withdrawal visit
    • ALT = 0.8 ULN at EOT/withdrawal visit
    • Miglioramento della NASH e nessun peggioramento della fibrosi dal basale alla EOT/visita di ritiro
    • Modifica del NAS dalla linea di base alla EOT/visita di ritiro
    • ALT = 0,8 ULN alla EOT/visita di ritiro
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 72 (EOT)/withdrawal visit
    settimana 72 (EOT)/visita di ritiro
    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
    Due diverse dosi di IMP (1500 mg/die o 1000 mg/die norUDCA)
    Two different doses of IMP (1500 mg/d or 1000 mg/d norUDCA)
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA116
    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 Information not present in EudraCT
    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
    Ireland
    Austria
    Belgium
    Czechia
    Denmark
    France
    Georgia
    Germany
    Greece
    Hungary
    Israel
    Italy
    Latvia
    Netherlands
    Poland
    Portugal
    Russian Federation
    Spain
    Turkey
    United Kingdom
    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
    Ultima visita dell'ultimo paziente (UVUP)
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    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: 298
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 65
    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 Yes
    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 state23
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 240
    F.4.2.2In the whole clinical trial 363
    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 is left to investigator’s discretion.
    Il trattamento dopo la fine dello studio è lasciato alla discrezione dello sperimentatore.
    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 Decision2023-01-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-20
    P. End of Trial
    P.End of Trial StatusCompleted
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Wed Oct 29 10:36:46 CET 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA