NCT00059202

Brief Summary

This is a multicenter, randomized, controlled trial of high dose ursodiol versus placebo for patients with primary sclerosing cholangitis (PSC). The average duration of follow-up will be approximately five years with important clinical endpoints such as death, eligibility for liver transplantation, changes in histology and cholangiogram as well as liver biochemistries and quality of life data collected.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
150

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Jul 2002

Longer than P75 for phase_2

Geographic Reach
1 country

7 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2002

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

April 21, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 22, 2003

Completed
5.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2008

Completed
12.9 years until next milestone

Results Posted

Study results publicly available

April 8, 2021

Completed
Last Updated

May 3, 2021

Status Verified

April 1, 2021

Enrollment Period

5.9 years

First QC Date

April 21, 2003

Results QC Date

March 12, 2021

Last Update Submit

April 7, 2021

Conditions

Primary Sclerosing Cholangitis

Keywords

UrsoPSC

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Treatment Failure

    Treatment failure was defined as death, transplantation, meeting minimal listing criteria, development of varices, cholangiocarcinoma, or progression to cirrhosis

    5 years

Secondary Outcomes (15)

  • Number of Deaths

    5 years

  • Number of Participants Who Have a Liver Transplantation

    5 years

  • Number of Participants Meeting Minimal Listing Criteria for Liver Transplantation

    5 years

  • Number of Participants Who Developed Cholangiocarcinoma

    5 years

  • Number of Participants Who Developed Varices

    5 years

  • +10 more secondary outcomes

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo tablet that is identical (size, color, etc) to experimental ursodeoxycholic acid tablet.

Drug: Placebo

Ursodeoxycholic acid

EXPERIMENTAL

Ursodeoxycholic acid 28-30 mg/kg/day

Drug: Ursodeoxycholic Acid

Interventions

Ursodeoxycholic AcidDRUG

Ursodeoxycholic Acid 28-30 mg/kg/day in divided doses

Also known as: UDCA
Ursodeoxycholic acid
PlaceboDRUG

Placebo for Urso

Placebo

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
* Chronic cholestatic disease of at least six months' duration. * Serum alkaline phosphatase at least 1 ½ times the upper limits of normal. * Retrograde, operative, percutaneous, or magnetic resonance cholangiography demonstrating intrahepatic and/or extrahepatic biliary duct obstruction, beading, or narrowing consistent with PSC within one year of the study entry. * Liver biopsy in the previous one year which is available for review and compatible with the diagnosis of PSC. Compatible biopsy features include fibrous cholangitis, ductopenia with periportal inflammation and biliary fibrosis.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (7)

Mayo Clinic

Scottsdale, Arizona, United States

Location

Mayo Clinic

Jacksonville, Florida, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

St. Louis University

St Louis, Missouri, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, United States

Location

Medical College of Virginia

Richmond, Virginia, United States

Location

University of Washington

Seattle, Washington, United States

Location

Related Publications (1)

  • Lindor KD, Kowdley KV, Luketic VA, Harrison ME, McCashland T, Befeler AS, Harnois D, Jorgensen R, Petz J, Keach J, Mooney J, Sargeant C, Braaten J, Bernard T, King D, Miceli E, Schmoll J, Hoskin T, Thapa P, Enders F. High-dose ursodeoxycholic acid for the treatment of primary sclerosing cholangitis. Hepatology. 2009 Sep;50(3):808-14. doi: 10.1002/hep.23082.

    PMID: 19585548RESULT

MeSH Terms

Conditions

Cholangitis, Sclerosing

Interventions

Ursodeoxycholic Acid

Condition Hierarchy (Ancestors)

CholangitisBile Duct DiseasesBiliary Tract DiseasesDigestive System Diseases

Intervention Hierarchy (Ancestors)

Deoxycholic AcidCholic AcidsBile Acids and SaltsSteroidsFused-Ring CompoundsPolycyclic CompoundsCholanes

Results Point of Contact

Title
Dr. Keith Lindor
Organization
Mayo Clinic
507-284-2511

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 21, 2003

First Posted

April 22, 2003

Study Start

July 1, 2002

Primary Completion

June 1, 2008

Study Completion

June 1, 2008

Last Updated

May 3, 2021

Results First Posted

April 8, 2021

Record last verified: 2021-04

Locations

US(7)