Scientific study on the efficacy and safety of Nor-ursodeoxycholic acid tablets in the treatment of patients with non-alcoholic steatohepatitis
Summary description of the study
This clinical study investigates non-alcoholic steatohepatitis (NASH), a form of non-alcoholic fatty liver disease. This study aims to evaluate the efficacy and safety of Nor-ursodeoxycholic acid tablets (Nor-UDCA) in patients with NASH. There are 3 treatment groups: A: treatment with Nor-UDCA tablets 1500 mg, B: treatment with Nor-UDCA tablets 1000 mg, C: treatment with placebo tablets. Placebo tablets contain no active ingredient. The probability of being assigned to one of the groups is equal for all three groups (33.3 %). This study is double-blind, meaning that neither the study participant nor the physician knows which treatment the participant is receiving. The drug Nor-UDCA is not yet approved for the treatment of NASH. 363 patients from various countries in Europe will participate in the study. The study lasts approximately 76 weeks for each study participant with 72 weeks of treatment.
(BASEC)
Intervention under investigation
This clinical study aims to investigate whether the drug Nor-ursodeoxycholic acid (Nor-UDCA) is effective, tolerable, and safe for the condition of non-alcoholic steatohepatitis (NASH). Nor-UDCA is not yet approved for the treatment of NASH. The drug Nor-UDCA has been tested so far in approximately 470 individuals, including healthy individuals, patients with inflammation of the bile ducts, and patients with non-alcoholic fatty liver disease. Nor-UDCA is similar to the drug Ursodeoxycholic acid (UDCA, Ursofalk®), which has been approved for many years in many European countries for the treatment of a rare autoimmune liver disease and for the dissolution of gallstones.
(BASEC)
Disease under investigation
Non-alcoholic steatohepatitis (NASH)
(BASEC)
- Male or female patients aged between 18 and 75 years - Evidence of non-alcoholic steatohepatitis (NASH) through histological examination (BASEC)
Exclusion criteria
- Presence of serious pre-existing or comorbid conditions - Increased alcohol consumption - Existing or planned pregnancy or breastfeeding (BASEC)
Trial sites
Bern
(BASEC)
Sponsor
Sponsor: Dr. Falk Pharma GmbH Sponsor’s representative in Switzerland: Dr. Falk Pharma AG
(BASEC)
Contact
Contact Person Switzerland
Prof. Dr. med. Annalisa Berzigotti
+41316323391
annalisa.berzigotti@clutterinsel.chInselspital Bern
(BASEC)
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Bern
(BASEC)
Date of authorisation
17.09.2019
(BASEC)
ICTRP Trial ID
EUCTR2018-003443-31 (ICTRP)
Official title (approved by ethics committee)
Double-blind, randomised, placebo-controlled, phase IIb trial on the efficacy and safety of norursodeoxycholic acid tablets in patients with non-alcoholic steatohepatitis (NASH) (BASEC)
Academic title
Double-blind, randomised, placebo-controlled, phase IIb trial on the efficacy and safety of norursodeoxycholic acid tablets in patients with non-alcoholic steatohepatitis (NASH) - Norursodeoxycholic acid vs. placebo in NASH (ICTRP)
Public title
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]) (ICTRP)
Disease under investigation
Non-alcoholic steatohepatitis (NASH)
MedDRA version: 24.1Level: PTClassification code 10053219Term: Non-alcoholic steatohepatitisSystem Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] (ICTRP)
Intervention under investigation
Product Name: Norursodeoxycholic acid (NorUDCA)
Pharmaceutical Form: Tablet
INN or Proposed INN: Norursodeoxycholic acid
CAS Number: 9697-24-2
Current Sponsor code: norUDCA
Other descriptive name: NOR-URSODEOXYCHOLIC ACID
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
(ICTRP)
Type of trial
Interventional clinical trial of medicinal product (ICTRP)
Trial design
Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: yes Other specify the comparator: Two different doses of IMP (1500 mg/d or 1000 mg/d norUDCA) Number of treatment arms in the trial: 3 (ICTRP)
Inclusion/Exclusion criteria
Gender:
Female: yes
Male: yes
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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 298
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 65
(ICTRP)
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
Primary and secondary end points
Main Objective: To evaluate the efficacy of norursodeoxycholic acid (norUDCA) 1500 mg vs. norUDCA 1000 mg vs. placebo for the treatment of NASH ;Secondary Objective: To study safety and tolerability (adverse events [AEs], laboratory parameters) of norUDCA;Primary 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
;Timepoint(s) of evaluation of this end point: week 72 (EOT)/withdrawal visit (ICTRP)
Secondary 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;Timepoint(s) of evaluation of this end point: week 72 (EOT)/withdrawal visit (ICTRP)
Registration date
09.04.2019 (ICTRP)
Incorporation of the first participant
08.05.2019 (ICTRP)
Secondary sponsors
not available
Additional contacts
Department of Clinical Research, zentrale@drfalkpharma.de, +4976115140, Dr. Falk Pharma GmbH (ICTRP)
Secondary trial IDs
NUT-3/NAS, 2018-003443-31-CZ (ICTRP)
Results-Individual Participant Data (IPD)
not available
Further information on the trial
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-003443-31 (ICTRP)
Results of the trial
Results summary
not available
Link to the results in the primary register
not available