The European Medicines Agency requested that a study must be undertaken that will help to develop solid evidence on domperidone effectiveness in children for the alleviation of nausea and vomiting symptoms by investigating the effect of a low-dose and short treatment period. In this randomized, double-blind, phase 3 research, children aged 6 months to 12 years were randomly (1:1) assigned to receive oral domperidone 0.25 mg/kg with ORT or matched placebo thrice daily for 2 to 7 days. Within 48 hours of first medication administration, the proportion of patients with no vomiting episodes (primary goal) and patients aged 4 years with no nausea episodes (important secondary endpoint) was assessed.
Following a futility analysis, the research was ended early. At the time of early termination, 292 patients were randomly assigned to either domperidone (n=147) or placebo (n=145). The proportion of patients who had no vomiting episodes after 48 hours of starting therapy was comparable in the domperidone (32.0%) and placebo groups (33.8%). Similarly, there was no significant difference between domperidone (35.7%) and placebo in the proportion of kids aged 4 years who had no nausea episodes after 48 hours of first therapy administration (38.6% ). One treatment-emergent adverse event was recorded by 13 patients (domperidone, 3.4% [5/147] versus placebo, 5.5% [8/145]). There were no reported fatalities or serious adverse events (extrapyramidal symptoms or QT prolongation).
In pediatric patients with acute gastroenteritis (AG), a low dose of domperidone with ORT did not substantially differ from placebo in terms of lowering vomiting and nausea episodes, and the safety profile was identical in both groups.