Clinical trial NCT01755507: Norursodeoxycholic Acid in the Treatment of Primary Sclerosing Cholangitis
Official title: Double-blind,Randomized, Placebo-controlled, Phase II Dose-finding Study Comparing Different Doses of Norursodeoxycholic Acid Capsules With Placebo in the Treatment of Primary Sclerosing Cholangitis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Sclerosing Cholangitis
Keywords: PSC
Lead sponsor: Dr. Falk Pharma GmbH
Sponsor class: INDUSTRY
First posted: 2012-12-24
Enrollment: 159
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in serum AP levels during treatment
timeFrame: 12 weeks
Secondary outcomes:
measure: Proportion of patients with at least 50% reduction in s-ALP
timeFrame: 12 weeks
Other outcomes:
measure: Adverse Events
timeFrame: 12 weeks
Locations:
city: Wien
country: Austria
facility: Prof. M. Trauner
city: Hannover
country: Germany
facility: Prof. Michael Manns
city: Oslo
country: Norway
facility: Kirsten Boberg
Study officials:
affiliation: Med. Uni Wien
name: Michael Trauner, Prof
role: PRINCIPAL_INVESTIGATOR
affiliation: Med.Hochschule Hannover
name: Michael P Manns, Prof
role: PRINCIPAL_INVESTIGATOR
References:
citation: Fickert P, Hirschfield GM, Denk G, Marschall HU, Altorjay I, Farkkila M, Schramm C, Spengler U, Chapman R, Bergquist A, Schrumpf E, Nevens F, Trivedi P, Reiter FP, Tornai I, Halilbasic E, Greinwald R, Prols M, Manns MP, Trauner M; European PSC norUDCA Study Group. norUrsodeoxycholic acid improves cholestasis in primary sclerosing cholangitis. J Hepatol. 2017 Sep;67(3):549-558. doi: 10.1016/j.jhep.2017.05.009. Epub 2017 May 18.