Evaluation of the efficacy, safety, and tolerability of ezetimibe in primary hypercholesterolaemia: a pooled analysis from two controlled phase III clinical studies

Int J Clin Pract. 2003 Jun;57(5):363-8.


Ezetimibe is a novel cholesterol absorption inhibitor that blocks intestinal absorption of dietary and biliary cholesterol. Data from 1719 patients who participated in two multicentre, double-blind studies of ezetimibe were pooled to evaluate the drug's efficacy and safety in patients with primary hypercholesterolaemia. Following dietary stabilisation, a two- to 12-week washout period, and a four-week, single-blind, placebo lead-in period, patients were randomised to ezetimibe 10 mg or placebo once daily in the morning for 12 consecutive weeks. The primary efficacy endpoint was percent reduction in plasma low-density lipoprotein (LDL)-cholesterol from baseline at endpoint. Ezetimibe reduced LDL-cholesterol by a mean of 18.2% compared with an increase of 0.9% with placebo (p<0.01) and resulted in favourable, statistically significant changes in HDL-cholesterol, triglycerides and apo B. The response to ezetimibe was consistent across all subgroups analysed. Ezetimibe was well tolerated, with a safety profile similar to placebo.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Anticholesteremic Agents / adverse effects
  • Anticholesteremic Agents / therapeutic use*
  • Azetidines / adverse effects
  • Azetidines / therapeutic use*
  • Cholesterol, LDL / blood
  • Diet Records
  • Double-Blind Method
  • Ezetimibe
  • Female
  • Humans
  • Hypercholesterolemia / drug therapy*
  • Liver Function Tests
  • Male
  • Middle Aged
  • Patient Satisfaction
  • Single-Blind Method


  • Anticholesteremic Agents
  • Azetidines
  • Cholesterol, LDL
  • Ezetimibe