Randomised clinical trial: the efficacy and safety of propionyl-L-carnitine therapy in patients with ulcerative colitis receiving stable oral treatment

Aliment Pharmacol Ther 2011; 34: 1088–1097

Summary

Background Ulcerative colitis (UC) is characterised by impaired fatty‐acid oxidation; l ‐carnitine has a key role in fatty‐acid metabolism and short‐chain fatty acids such as butyrate and propionate are important energy source for intestinal epithelial cells. Aim To evaluate efficacy and safety of colon‐release propionyl‐l ‐carnitine (PLC) in patients with mild‐to‐moderate UC receiving stable oral aminosalicylate or thiopurine therapy. Methods In a multicentre, phase II, double‐blind, parallel‐group trial, patients were randomised to receive PLC 1 g/day, PLC 2 g/day or placebo. Main inclusion criteria were as follows: age 18–75; disease activity index (DAI) score 3–10 inclusive, be under oral stable treatment with aminosalicylate or thiopurine. The primary endpoint was clinical/endoscopic response, defined as a decrease in DAI score ≥ 3 points or remission, defined as a DAI score ≤ 2 with no individual sub‐score > 1. Results Of 121 patients who were randomised, 57 of 79 (72%) patients receiving PLC (combined 1 g and 2 g cohort) had a clinical/endoscopic response vs. 20 of 40 (50%) receiving placebo (P = 0.02). Specifically, in PLC 1 g/day group, 30 of 40 (75%) patients had clinical/endoscopic response (P = 0.02 vs. placebo) and 27 of 39 (69%) in the PLC 2 g/day group (P = 0.08 vs. placebo). Rates of remission were 22/40 (55%), 19/39 (49%), 14/40 (35%) in the PLC 1 g, PLC 2 g, and placebo groups, respectively. PLC had a similar safety profile to placebo; the most common adverse events were gastrointestinal. Conclusion Propionyl‐l ‐carnitine 1 g/day should be investigated further as a co‐treatment for mild‐to‐moderate ulcerative colitis (NCT‐01026857).