Document Type
Article
Publication Date
6-1-2019
Identifier
PMCID: PMC6760680 DOI: 10.1038/s41390-018-0242-2
Abstract
Although seizures have a higher incidence in neonates than any other age group and are associated with significant mortality and neurodevelopmental disability, treatment is largely guided by physician preference and tradition, due to a lack of data from well-designed clinical trials. There is increasing interest in conducting trials of novel drugs to treat neonatal seizures, but the unique characteristics of this disorder and patient population require special consideration with regard to trial design. The Critical Path Institute formed a global working group of experts and key stakeholders from academia, the pharmaceutical industry, regulatory agencies, neonatal nurse associations, and patient advocacy groups to develop consensus recommendations for design of clinical trials to treat neonatal seizures. The broad expertise and perspectives of this group were invaluable in developing recommendations addressing: (1) use of neonate-specific adaptive trial designs, (2) inclusion/exclusion criteria, (3) stratification and randomization, (4) statistical analysis, (5) safety monitoring, and (6) definitions of important outcomes. The guidelines are based on available literature and expert consensus, pharmacokinetic analyses, ethical considerations, and parental concerns. These recommendations will ultimately facilitate development of a Master Protocol and design of efficient and successful drug trials to improve the treatment and outcome for this highly vulnerable population.
Journal Title
Pediatric research
Volume
85
Issue
7
First Page
943
Last Page
954
MeSH Keywords
Clinical Trial Protocols as Topic; Seizures; Infant, Newborn; Research Design
Recommended Citation
Soul JS, Pressler R, Allen M, et al. Recommendations for the design of therapeutic trials for neonatal seizures. Pediatr Res. 2019;85(7):943-954. doi:10.1038/s41390-018-0242-2
Included in
Bioethics and Medical Ethics Commons, Congenital, Hereditary, and Neonatal Diseases and Abnormalities Commons, Nervous System Diseases Commons, Neurology Commons, Pediatrics Commons
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Publisher's Link: https://www.nature.com/articles/s41390-018-0242-2