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    The EU Clinical Trials Register currently displays   40626   clinical trials with a EudraCT protocol, of which   6627   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2018-003443-31
    Sponsor's Protocol Code Number:NUT-3/NAS
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-06-03
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    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)
    Dubbelblind, gerandomiseerd, placebo-gecontroleerd fase IIb onderzoek naar de doeltreffendheid en veiligheid van nor-ursodeoxycholzuur tabletten bij patiënten met niet-alcoholische steatohepatitis (NASH)
    Essai de phase IIb, en double aveugle, randomisé, contrôlé contre placebo évaluant l’efficacité et la sécurité de l’acide norursodéoxycholique en comprimés chez des patients présentant une stéatohépatite non alcoolique (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])
    Klinische proef die de doeltreffendheid en veiligheid van nor-ursodeoxycholzuur tabletten vergelijkt met placebo bij de behandeling van niet-alcoholische
    leververvettingsaandoening met lever ontsteking ("niet-alcoholische steatohepatitis"[NASH])
    Essai clinique comparant l’efficacité et la sécurité des comprimés d'acide norursodéoxycholique avec un placebo dans le traitement de la stéatose hépatique non alcoolique avec une inflammation du foie (« stéatohépatite non alcoolique"[NASH])
    A.3.2Name or abbreviated title of the trial where available
    Norursodeoxycholic acid vs. placebo in NASH
    Nor-ursodeoxycholzuur t.o.v. placebo bij NASH
    Acide norursodéoxycholique vs placebo dans le traitement de la 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.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.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.5.4Telephone number+4976115140
    B.5.5Fax number+497611514377
    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 nameNorursodeoxycholic acid (NorUDCA)
    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 INNNorursodeoxycholic acid
    D.3.9.1CAS number 9697-24-2
    D.3.9.2Current sponsor codenorUDCA
    D.3.9.3Other descriptive nameNOR-URSODEOXYCHOLIC ACID
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Non-alcoholic steatohepatitis (NASH)
    Niet-alcoholische steatohepatitis (NASH)
    Stéatohépatite non alcoolique (NASH)
    E.1.1.1Medical condition in easily understood language
    Non-alcoholic steatohepatitis
    Niet-alcoholische steatohepatitis
    Stéatohépatite non alcoolique
    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 22.0
    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
    De evaluatie van de doeltreffendheid van nor-ursodeoxycholzuur (nor-UDCA) 1500 mg t.o.v. nor-UDCA 1000 mg t.o.v. placebo voor de behandeling van NASH
    Évaluer l’efficacité de l’acide norursodéoxycholique (norUDCA) 1500 mg vs norUDCA 1000 mg vs placebo dans le traitement de la NASH
    E.2.2Secondary objectives of the trial
    To study safety and tolerability (adverse events [AEs], laboratory parameters) of norUDCA
    Onderzoek naar de veiligheid en verdraagbaarheid (bijwerkingen [AE's], laboratoriumparameters) van nor-UDCA
    Étudier la sécurité et la tolérance (événements indésirables [EI], paramètres de laboratoire) du 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
    • Alanine aminotransferase (ALT) > 0.8 upper limit of normal (ULN)
    • 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
    • Moeten bereid zijn deel te nemen in de studie en schriftelijke geïnformeerde toestemming geven
    • Mannelijke of vrouwelijke patiënten ≥ 18 en < 75 jaar
    • Centraal geëvalueerde histologische bevestiging van NASH en leverfibrose
    • Alanine-aminotransferase (ALAT) > 0,8 bovengrens van normaal (ULN)
    • Vrouwen die kinderen kunnen krijgen, moeten ermee instemmen om een zeer doeltreffende voorbehoedsmethode te gebruiken voor de hele duur van het onderzoek en nog 4 weken na de laatste dosis van de onderzoeksbehandeling
    • Doit être disposé à participer à l’étude et de fournir un consentement éclairé signé
    • Patients hommes ou femmes ≥ 18 et < 75 ans
    • Preuve histologique évaluée de manière centralisée d’une NASH et fibrose hépatique
    • Alanine aminotransférase (ALAT) > 0,8 fois la limite supérieure de la normale (LSN)
    • Les femmes aptes à procréer acceptent d’utiliser une méthode de contraception très efficace pendant toute la durée de l’essai, et pendant 4 semaines après l’administration de la dernière dose du médicament à l’essai
    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
    • Patiënten die verboden medicijnen nemen
    • Aanwezigheid van levercirrose
    • Type 1 diabetes of ongecontroleerde type 2 diabetes
    • Voorgeschiedenis of aanwezigheid van alle andere beduidende gelijktijdige leveraandoeningen
    • Geschiedenies van een levertransplantatie
    • BMI > 45 kg/m^2
    • Elke bekende relevante infectieziekte (bv. actieve tuberculose, aandoeningen die wijzen op verworven immunodeficiëntiesyndroom [aids])
    • Abnormale nierfunctie (glomerulaire filtratiesnelheid geschat uit cystatine C < 30 ml/min) bij het screeningbezoek
    • Elke actieve maligne aandoening (behalve basaalcelcarcinoom)
    • Bestaande of geplande zwangerschap of borstvoeding
    • Patients qui prennent des médicaments interdit
    • Présence d’une cirrhose du foie
    • Diabète de type 1 or non contrôlé diabète de type 2
    • Antécédents ou présence de toute autre maladie hépatique concomitante significative
    • Histoire d’une greffe hépatique
    • IMC > 45 kg/m^2
    • Toute maladie infectieuse connue ayant de l’importance dans le cadre de cet essai (par ex. tuberculose active, maladies définissant le syndrome d’immunodéficience acquise [SIDA])
    • Fonction rénale anormale (taux de filtration glomérulaire estimé à partir de la cystatine C < 30 mL/min) lors de la visite de sélection
    • Toute maladie maligne active (sauf le carcinome à cellules basales)
    • Grossesse ou allaitement actuel ou envisagé
    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
    Improvement of fibrosis, and no worsening of NAS from baseline to EOT/withdrawal visit
    Verdwijning van NASH, geëvalueerd door een centraal bepaalde score voor leverhistologie, en geen verergering van fibrose van baseline tot EOT/terugtrekkingsbezoek
    Verbetering van fibrose, en geen verergering van NASH, van baseline tot EOT/terugtrekkingsbezoek
    Résolution de la NASH, évaluée de manière centralisée par l’histologie hépatique, et aucune aggravation de la fibrose entre la visite de baseline et la
    visite EOT/de retrait
    Amélioration de la fibrose, et aucune aggravation de la NASH, entre la visite de baseline et la visite EOT/de retrait
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 72 (EOT)/withdrawal visit
    week 72 (EOT)/terugtrekkingsbezoek
    Semaine 72 (EOT)/visit de retrait
    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
    • Verbetering van NASH en geen verergering van fibrose van baseline tot EOT/terugtrekkingsbezoek
    • Verandering in NAS van baseline tot EOT/terugtrekkingsbezoek
    • ALAT ≤ 0,8 ULN bij EOT/terugtrekkingsbezoek
    • Amélioration de la NASH et aucune aggravation de la fibrose entre la visite de baseline et la visite EOT/de retrait
    • Variation du score NAS entre la visite de baseline et la visite EOT/de retrait
    • ALAT ≤ 0,8 fois la LSN à la visite EOT/de retrait
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 72 (EOT)/withdrawal visit
    week 72 (EOT)/terugtrekkingsbezoek
    Semaine 72 (EOT)/visit de retrait
    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. 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. description
    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 concerned11
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA89
    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
    Czech Republic
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 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 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 state45
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 353
    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.
    Behandeling na studie einde is overgelaten aan de onderzoeker.
    Traitement après que la fin du procès est laissée à la discrétion de l’investigateur.
    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 Decision2019-06-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-31
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
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