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Effect of the DGAT1 Inhibitor Pradigastat on Triglyceride and ApoB48 Levels in Patients with Familial Chylomicronemia Syndrome

Meyers, Charles and Tremblay, Karine and Amer, Ahmed and Chen, Jin and Jiang, Liewen and Gaudet, Daniel (2015) Effect of the DGAT1 Inhibitor Pradigastat on Triglyceride and ApoB48 Levels in Patients with Familial Chylomicronemia Syndrome. Lipids in Health and Disease, 14 (8).

Abstract

This pilot study of pradigastat, a diacylglycerol acyltransferase-1 (DGAT1) inhibitor, was conducted to assess its effects on lipid parameters and tolerability after multiple dose administrations in patients with familial chylomicronemia syndrome (FCS), a rare inherited form of severe hypertriglyceridemia. Six patients underwent a 1-week dietary run-in period, followed by an open-label, fixed-sequence treatment with oral pradigastat at 20, 40, and 10 mg once daily for 3 weeks each, with an interlacing ≥4-week wash-out period. Fasting and postprandial triglyceride (TG) and apolipoprotein (apo) B48 levels with lipoprotein fractions were assessed after an FCS-adapted test meal. Pradigastat 20 and 40 mg led to 40 and 70% reduction in fasting TG at Week 3. Postprandial TG drop was significantly greater for both the 20 and 40 mg arms relative to the 10 mg group which did not reduce TG levels (p<0.05 for all parameters at each dose). Favorable effects were noted on lipoprotein fractions including reductions in chylomicron TG content. Mild gastrointestinal events were the most common adverse events. In conclusion, the novel DGAT1 inhibitor pradigastat led to substantial reductions in fasting and postprandial TG in patients with FCS, particularly reducing the number and TG content of chylomicron particles.

Item Type: Article
Keywords: lipoprotein lipase deficiency, type 1 hyperlipoproteinemia, orphan disease, LCQ908, clinical trial
Date Deposited: 27 Apr 2016 23:45
Last Modified: 27 Apr 2016 23:45
URI: https://oak.novartis.com/id/eprint/20414

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