NCT03872921

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

Active, enrolled Phase 3 Last updated 5 August 2024

What this trial tests

Phase 3 trial testing norUrsodeoxycholic Acid in Primary Sclerosing Cholangitis in 303 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

8 February 2018

Primary endpoint
1 January 2025

1 June 2026

Quick facts

Lead sponsor	Dr. Falk Pharma GmbH
Phase	Phase 3
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	303
Start date	8 February 2018
Primary completion	1 January 2025
Estimated completion	1 June 2026
Sites	2 locations across Austria, Germany

Drugs / interventions tested

• norUrsodeoxycholic Acid — full drug profile →

Conditions studied

• Primary Sclerosing Cholangitis — all drugs for Primary Sclerosing Cholangitis →

Sponsor

Dr. Falk Pharma GmbH — full company profile →

Who can join

Adults 16 to 75, any sex, with Primary Sclerosing Cholangitis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

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

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Combination therapy for non-alcoholic steatohepatitis: rationale, opportunities and challenges.
   Dufour JF, Caussy C, Loomba R. · · 2020 · cited 145× · PMID 32381514 · DOI 10.1136/gutjnl-2019-319104
2. Novel therapeutic targets for cholestatic and fatty liver disease.
   Trauner M, Fuchs CD. · · 2022 · cited 141× · PMID 34615727 · DOI 10.1136/gutjnl-2021-324305
3. New targets for NAFLD.
   Parlati L, Régnier M, Guillou H, Postic C. · · 2021 · cited 128× · PMID 34667947 · DOI 10.1016/j.jhepr.2021.100346
4. Bile acids and their receptors: modulators and therapeutic targets in liver inflammation.
   Bertolini A, Fiorotto R, Strazzabosco M. · · 2022 · cited 99× · PMID 35415765 · DOI 10.1007/s00281-022-00935-7
5. Nonalcoholic Fatty Liver Disease (NAFLD). Mitochondria as Players and Targets of Therapies?
   Di Ciaula A, Passarella S, Shanmugam H, Noviello M, et al · · 2021 · cited 93× · PMID 34065331 · DOI 10.3390/ijms22105375
6. A Current Understanding of Bile Acids in Chronic Liver Disease.
   Farooqui N, Elhence A, Shalimar. · · 2022 · cited 61× · PMID 35068796 · DOI 10.1016/j.jceh.2021.08.017
7. Primary Biliary Cholangitis and Primary Sclerosing Cholangitis: Current Knowledge of Pathogenesis and Therapeutics.
   Park JW, Kim JH, Kim SE, Jung JH, et al · · 2022 · cited 41× · PMID 35740310 · DOI 10.3390/biomedicines10061288
8. Primary sclerosing cholangitis.
   Rabiee A, Silveira MG. · · 2021 · cited 40× · PMID 33824933 · DOI 10.21037/tgh-20-266