Norursodeoxycholic Acid in the Treatment of Primary Sclerosing Cholangitis

Phase 2

Completed

• Conditions: Primary Sclerosing Cholangitis
• Interventions: Drug: norUDCA; Drug: Placebo

• Registration Number: NCT01755507
• Lead Sponsor: Dr. Falk Pharma GmbH

Brief Summary

Evaluation of the efficacy of different doses of nor UDCA vs. placebo for the treatment of Primary Sclerosing Cholangitis (PSC). Identification of optimal dose(s)for the treatment of PSC.

Detailed Description

Double-blind, randomized, multi-center, placebo-controlled, comparative exploratory phase II-finding trial.The study will be conducted with four treatment groups in the form of a parallel-group comparison and will serve to compare oral treatment with either 500 mg/d, 1000 mg/d or 1500 mg/d norursodeoxycholic acid capsules vs. Placebo capsules for the treatment of PSC.

Study Timeline

• Study Start: 2012-12
• Study First Submitted: 2012-12-19
• Study First Submitted QC: 2012-12-21
• Study First Posted: 2012-12-24
• Primary Completion: 2015-10
• Study Completion: 2015-12
• Status Verified: 2016-08
• Last Update Submitted: 2016-08-02
• Last Update Posted: 2016-08-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 159

Inclusion Criteria

1. Signed Informed Consent
2. Verified diagnosis of PSC
3. PSC patients with or without IBD
4. Women of childbearing potential have to apply during the entire duration of the study a highly effective method of birth control.

Exclusion Criteria

1. History or presence of other concomitant liver diseases
2. Treatment with UDCA within 8 weeks prior to baseline visit.
3. Child B/C liver cirrhosis
4. Total bilirubin > 3.0 mg/dl at screening or baseline.
5. Any relevant systemic disease
6. TSH>ULN at screening
7. any severe concomitant cardiovascular, renal, endocrine, or psychiatric disorder
8. Any active malignant disease
9. Known intolerance/hypersensitivity to study drug
10. Existing or intended pregnancy of brest feeding
11. Simultaneous participation in another clinical trial

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
C	norUDCA	norUDCA
placebo	Placebo	Placebo
A	norUDCA	norUDCA
B	norUDCA	norUDCA

Primary Outcome Measures

Name	Time	Method
Change in serum AP levels during treatment	12 weeks

Secondary Outcome Measures

Name	Time	Method
Proportion of patients with at least 50% reduction in s-ALP	12 weeks

Trial Locations

Locations (3)

Prof. M. Trauner; 🇦🇹 Wien, Austria

Prof. Michael Manns; 🇩🇪 Hannover, Germany

Kirsten Boberg; 🇳🇴 Oslo, Norway