norUrsodeoxycholic Acid vs Placebo in PSC

Double-blind, Randomized, Placebo-controlled, Phase III Study Comparing norUrsodeoxycholic Acid Capsules With Placebo in the Treatment of Primary Sclerosing Cholangitis

Double-blind, randomized, multi-center, placebo-controlled, comparative, phase III trial. The study will be conducted with two treatment groups in the form of a parallel group comparison and will serve to compare oral treatment with either 1500 mg/d norursodeoxycholic acid capsules or placebo capsules for the treatment of Primary Sclerosing Cholangitis.

Study Overview

• Status: Recruiting
• Conditions: Primary Sclerosing Cholangitis
• Intervention / Treatment: Drug: norUrsodeoxycholic Acid

• Study Type: Interventional
• Enrollment (Anticipated): 300
• Phase: Phase 3

Contacts and Locations

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

Study Locations

• Austria
  • Vienna, Austria, 1090
    • 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
    • Contact: Emina Halilbasic, MD; Phone Number: 491210 0043 (0)1 40400; Email: emina.halilbasic@meduniwien.ac.at
• Germany
  • Lower Saxonia
    • Hannover, Lower Saxonia, Germany, 30623
      • Recruiting
      • Medical School Hannover
      • Contact: Heike Bantel, MD; Phone Number: 9514 +49 511 532; Email: bantel.heike@mh-hannover.de
      • Contact: Markus Cornberg, MD; Phone Number: 6821 +49 511 532; Email: cornberg.markus@mh-hannover.de

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Signed informed consent
• verified PSC
• Liver Biopsy available for Review
• If pre-treated with UDCA Patient must be on stable dose not exceeding 20mg/kg/bw
• Patients with or without concomittant IBD

Exclusion Criteria:

• History or presence of other concomitant liver diseases
• Presence of Cholangiocarcinoma
• Secondary causes of Sclerosing Cholangitis
• Small Duct Cholangitis in the absence of large duct disease
• Any known relevant infectious disease
• Abnormal renal function
• Any active malignant disease
• Known intolerance/hypersensitivity to study drug

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: norUrsodeoxycholic acid; norUrsodeoxycholic acid 250mg capsules, 6 capsules/day for 2 years	Drug: norUrsodeoxycholic Acid; oral treatment; Other Names: NUC
Placebo Comparator: Placebo to norUrsodeoxycholic acid; norUrsodeoxycholic acid 250mg Placebo-capsules, 6 capsules/day for 2 years	Drug: norUrsodeoxycholic Acid; oral treatment; Other Names: NUC

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC)	prevention of disease progression assessed by partial normalization of serum Alkaline Phosphatase (s-ALP) levels in patients with PSC	2 years
Show superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC)	prevention of disease progression assessed by liver histology	2 years

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): February 8, 2018
• Primary Completion (Anticipated): April 1, 2021
• Study Completion (Anticipated): December 1, 2023

Study Registration Dates

• First Submitted: March 8, 2019
• First Submitted That Met QC Criteria: March 11, 2019
• First Posted (Actual): March 13, 2019

Study Record Updates

• Last Update Posted (Actual): October 31, 2019
• Last Update Submitted That Met QC Criteria: October 29, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholangitis; Cholangitis, Sclerosing
• Other Study ID Numbers: NUC-5/PSC

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
• product manufactured in and exported from the U.S.: No