Safety and Efficacy of Low Dose Domperidone for Treating Nausea and Vomiting Due to Acute Gastroenteritis in Children.

BACKGROUND This study was conducted based on a request from the European Medicines Agency to generate robust data on domperidone efficacy in children in the relief of symptoms of nausea and vomiting by assessing the effect of a low-dose and short treatment duration. METHODS In this randomized, double-blind, phase 3 study, children aged 6-months to 12-years with acute gastroenteritis randomly (1:1) received oral domperidone 0.25-mg/kg plus oral rehydration therapy (ORT) or matching placebo thrice-daily for 2-7 days. The proportion of patients with no vomiting episodes (primary endpoint) and patients aged ≥4 years with no nausea episodes (key secondary endpoint) within 48-hours of first treatment administration were evaluated. RESULTS The study was terminated early following futility analysis. At early termination, 292 patients randomly received domperidone (n = 147) or placebo (n = 145). The proportion of patients with no vomiting episodes within 48-hours of first treatment administration was similar between domperidone (32.0%) and placebo groups (33.8%). Similarly, there was no significant difference in proportion of patients aged ≥4 years with no nausea episodes within 48-hours of first treatment administration between domperidone (35.7%) and placebo (38.6%). Total 13 patients (domperidone, 3.4% [5/147] versus placebo, 5.5% [8/145]) reported ≥1 treatment-emergent adverse events. No deaths or adverse events of special interest (extrapyramidal symptoms and QT prolongation) were reported. CONCLUSION Low-dose of domperidone plus ORT did not significantly differ from placebo in reducing vomiting and nausea episodes in pediatric patients with AG, and the safety profile was similar between both groups.