A placebo-controlled trial of itopride in functional dyspepsia

N Engl J Med. 2006 Feb 23;354(8):832-40. doi: 10.1056/NEJMoa052639.


Background: The treatment of patients with functional dyspepsia remains unsatisfactory. We assessed the efficacy of itopride, a dopamine D2 antagonist with anti-acetylcholinesterase [corrected] effects, in patients with functional dyspepsia.

Methods: Patients with functional dyspepsia were randomly assigned to receive either itopride (50, 100, or 200 mg three times daily) or placebo. After eight weeks of treatment, three primary efficacy end points were analyzed: the change from baseline in the severity of symptoms of functional dyspepsia (as assessed by the Leeds Dyspepsia Questionnaire), patients' global assessment of efficacy (the proportion of patients without symptoms or with marked improvement), and the severity of pain or fullness as rated on a five-grade scale.

Results: We randomly assigned 554 patients; 523 had outcome data and could be included in the analyses. After eight weeks, 41 percent of the patients receiving placebo were symptom-free or had marked improvement, as compared with 57 percent, 59 percent, and 64 percent receiving itopride at a dose of 50, 100, or 200 mg three times daily, respectively (P<0.05 for all comparisons between placebo and itopride). Although the symptom score improved significantly in all four groups, an overall analysis revealed that itopride was significantly superior to placebo, with the greatest symptom-score improvement in the 100- and 200-mg groups (-6.24 and -6.27, vs. -4.50 in the placebo group; P=0.05). Analysis of the combined end point of pain and fullness showed that itopride yielded a greater rate of response than placebo (73 percent vs. 63 percent, P=0.04).

Conclusions: Itopride significantly improves symptoms in patients with functional dyspepsia. (ClinicalTrials.gov number, NCT00272103.).

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Benzamides / administration & dosage
  • Benzamides / adverse effects
  • Benzamides / therapeutic use*
  • Benzyl Compounds / administration & dosage
  • Benzyl Compounds / adverse effects
  • Benzyl Compounds / therapeutic use*
  • Dopamine D2 Receptor Antagonists*
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Dyspepsia / blood
  • Dyspepsia / drug therapy*
  • Female
  • Humans
  • Male
  • Middle Aged
  • Prolactin / blood
  • Quality of Life


  • Benzamides
  • Benzyl Compounds
  • Dopamine D2 Receptor Antagonists
  • itopride
  • Prolactin

Associated data

  • ClinicalTrials.gov/NCT00272103