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.
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.
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.
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.
*Janssen Research & Development, NJ, USA
†Newtown Clinical Research Centre, Johannesburg, South Africa
‡Nazaret Health Center, Valencia, Spain
§Barnsley Foundation Hospital NHS Trust, Barnsley, England.
Address correspondence and reprint requests to Gerhard Leitz, MD, PhD, Clinical Leader, Internal Medicine, Established Products, Janssen Research & Development, LLC, 1125 Trenton-Harbourton Road, Mail stop K20 905, Titusville, NJ 08560 (e-mail: GLeitz@its.jnj.com).
Received 3 December, 2018
Accepted 29 April, 2019
Funding Support: The study was funded by the marketing authorization holders of medicinal products containing domperidone that are part of the consortium, Domperidone PAES Collaboration Group, comprising Janssen Research & Development, Research and Development, Pierre Fabre for Pierre Fabre Médicament SAS, and Esteve Pharmaceuticals, S.A
Trial Registration: ClinicalTrial.gov number: NCT02699385
Conflicts of interest: Gerhard Leitz, Peter Hu, Carlos Appiani, and Qing Li are employees of Janssen Research & Development, LLD. Maria Garces-Sanchez has participated in advisory boards and speaker bureaus for GlaxoSmithKline, SPMSD, and Pfizer, for which payment is received; and she was a principal investigator in vaccine clinical trials for GlaxoSmithKline, SPMSD, Novartis, Wyeth, and Pfizer. Essack Mitha, and Rajeev Gupta have no conflict of interest to declare.
All authors participated in the original design of the studies, supervising recruitment and monitoring of data quality, and contributed to the data interpretation, development and review of this manuscript and confirm that they have read the Journal's position on issues involved in ethical publication and affirm that this report is consistent with those guidelines. All authors met ICMJE criteria and all those who fulfilled those criteria are listed as authors. All authors had access to the study data, provided direction and comments on the manuscript, made the final decision about where to publish these data and approved submission to this journal.
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