Trial of High-dose Urso in Primary Sclerosing Cholangitis

Phase 2/3 Terminated 150 enrolled National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

RandomizedParallel-groupQuadruple-blindTreatment

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.

Timeline

Start: 2002-07
Primary completion: 2008-06
Completion: 2008-06

Outcome

Missed primary endpoint

Stopped: “DSMB reviewed interim analysis and terminated the study due to futility.”

paper the risk of a primary endpoint was 2.3 times greater for patients on UDCA than for those on placebo (P < 0.01) PMID 19585548 ↗

Publications

Results 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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ursodeoxycholic Acid	Small molecule	28 mg/kg	—
Subject	Ursodeoxycholic Acid	Small molecule	30 mg/kg	—