Norursodeoxycholic Acid vs. Placebo in NASH

Study Overview
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Brief Summary
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This is a double-blind, randomised, multi-centre, placebo-controlled, comparative, phase IIb trial. The trial will be conducted with three treatment groups in the form of a parallel group comparison and will serve to compare oral treatment with either 1500 mg/d or 1000 mg/d norUDCA tablets vs. placebo tablets for the treatment of NASH.

Official Title
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Double-blind, Randomised, Placebo-controlled, Phase IIb Trial on the Efficacy and Safety of Norursodeoxycholic Acid Tablets in Patients With Non-alcoholic Steatohepatitis (NASH)

Conditions
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Nonalcoholic Steatohepatitis

Intervention / Treatment
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* Drug: norUrsodeoxycholic acid


Participation Criteria
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Eligibility Criteria
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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 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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 74 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: norUrsodeoxycholic acid 1500 mg/day<br>3 film-coated tablets once daily for 72 weeks containing in total norUrsodeoxycholic acid 1500 mg | Drug: norUrsodeoxycholic acid<br>* 500 mg norUrsodeoxycholic acid or placebo film-coated tablets; 3 tablets/day<br>* Other names: NCA;|
| Experimental: norUrsodeoxycholic acid 1000 mg/day<br>3 film-coated tablets once daily for 72 weeks containing in total norUrsodeoxycholic acid 1000 mg | Drug: norUrsodeoxycholic acid<br>* 500 mg norUrsodeoxycholic acid or placebo film-coated tablets; 3 tablets/day<br>* Other names: NCA;|
| Placebo Comparator: Placebo to norUrsodeoxycholic acid<br>3 film-coated tablets once daily for 72 weeks containing placebo to norUrsodeoxycholic acid | Drug: norUrsodeoxycholic acid<br>* 500 mg norUrsodeoxycholic acid or placebo film-coated tablets; 3 tablets/day<br>* Other names: NCA;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with resolution of NASH, assessed by centrally scored liver histology, and no worsening of fibrosis AND/OR improvement of fibrosis, and no worsening of NAS |  | 72 weeks |