Norursodeoxycholic Acid vs. Placebo in NASH

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

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.

Study Overview

• Status: Recruiting
• Conditions: Nonalcoholic Steatohepatitis
• Intervention / Treatment: Drug: norUrsodeoxycholic acid

• Study Type: Interventional
• Enrollment (Estimated): 363
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Michael Stiess, PhD; Phone Number: ++49-761-1514; Email: zentrale@drfalkpharma.de

Study Locations

• Austria
  • Vienna, Austria
    • Recruiting
    • Medical University of Vienna, Department of Internal Medicine III
    • Contact: Michael Trauner, MD; Phone Number: 47440 0043 (0)1 40400; Email: michael.trauner@meduniwien.ac.at

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 74 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Dr. Falk Pharma GmbH
• Investigators: Principal Investigator: Michael Trauner, MD, Medical University of Vienna, Department of Internal Medicine III

Study record dates

Study Major Dates

• Study Start (Actual): July 8, 2019
• Primary Completion (Estimated): January 1, 2025
• Study Completion (Estimated): April 1, 2025

Study Registration Dates

• First Submitted: October 6, 2021
• First Submitted That Met QC Criteria: October 6, 2021
• First Posted (Actual): October 19, 2021

Study Record Updates

• Last Update Posted (Actual): February 20, 2024
• Last Update Submitted That Met QC Criteria: February 16, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Liver Diseases; Fatty Liver; Non-alcoholic Fatty Liver Disease
• Other Study ID Numbers: NUT-3/NAS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: It is not yet decided which data will be made available. Neither a time frame can be indicated yet.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No