Cholic Acid for Hepatic Steatosis in Lipodystrophy

Phase 2 Completed 18 enrolled University of Texas Southwestern Medical Center FDA Office of Orphan Products Development · collab

RandomizedCrossoverTriple-blindTreatment

Summary

To evaluate the efficacy and safety of cholic acid therapy in treating lipodystrophy patients with hepatic steatosis. This is a randomized, double-blind, placebo-controlled cross-over study.

Timeline

Start: 2006-04
Primary completion: 2011-04
Completion: 2011-04

Outcome

Missed primary endpoint

registry analysis (superiority test); Cholic Acid vs Placebo; p = 0.42; Mean Difference (Net) 13.8 (95% CI -19.7 to 61.1); Mixed Models Analysis NCT00457639 ↗

paper Compared with placebo, CA did not reduce (median (interquartile range) hepatic TG content (14.8% (9.4-19.0%) vs 15.9% (10.5-26.5%) respectively; P=0.42) PMID 23447519 ↗

Publications

Results Ahmad Z, Subramanyam L, Szczepaniak L, Simha V, Adams-Huet B, Garg A. Cholic acid for hepatic steatosis in patients with lipodystrophy: a randomized, controlled trial. Eur J Endocrinol. 2013 Apr 15;168(5):771-8. doi: 10.1530/EJE-12-0969. Print 2013 May.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cholic Acid	Small molecule	15 mg/kg	Oral