Recommendations for the design of therapeutic trials for neonatal seizures

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dc.contributor.authorSoul, Janet S.
dc.contributor.authorPressler, Ronit
dc.contributor.authorAllen, Marilee
dc.contributor.authorBoylan, Geraldine B.
dc.contributor.authorRabe, Heike
dc.contributor.authorPortman, Ron
dc.contributor.authorHardy, Pollyanna
dc.contributor.authorZohar, Sarah
dc.contributor.authorRomero, Klaus
dc.contributor.authorTseng, Brian
dc.contributor.authorBhatt-Mehta, Varsha
dc.contributor.authorHahn, Cecil
dc.contributor.authorDenne, Scott
dc.contributor.authorAuvin, Stephane
dc.contributor.authorVinks, Alexander
dc.contributor.authorLantos, John
dc.contributor.authorMarlow, Neil
dc.contributor.authorDavis, Jonathan M.
dc.contributor.funderU.S. Food and Drug Administrationen
dc.date.accessioned2019-11-20T06:07:08Z
dc.date.available2019-11-20T06:07:08Z
dc.date.issued2019
dc.description.abstractAlthough 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.en
dc.description.sponsorshipU18FD005320-01en
dc.description.statusPeer revieweden
dc.description.versionPublished Versionen
dc.format.mimetypeapplication/pdfen
dc.identifier.citationSoul, J.A., Pressler, R., Allen, M., Boylan, G., Rabe, H., Portman, R., Hardy, P., Zohar, S., Romero, K., Tseng, B. and Bhatt-Mehta, V., 2018. Recommendations for the design of therapeutic trials for neonatal seizures. Pediatric research. (11pp). DOI:10.1038/s41390-018-0242-2en
dc.identifier.doi10.1038/s41390-018-0242-2en
dc.identifier.eissn1530-0447
dc.identifier.endpage954en
dc.identifier.issn0031-3998
dc.identifier.issued7en
dc.identifier.journaltitlePediatric Researchen
dc.identifier.startpage943en
dc.identifier.urihttps://hdl.handle.net/10468/9158
dc.identifier.volume85en
dc.language.isoenen
dc.publisherSpringer Natureen
dc.relation.urihttps://www.nature.com/articles/s41390-018-0242-2
dc.rights© The Author(s) 2019en
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/en
dc.subjectTherapeutic trialsen
dc.subjectNeonatal seizuresen
dc.subjectWell-designed clinical trialsen
dc.titleRecommendations for the design of therapeutic trials for neonatal seizuresen
dc.typeArticle (peer-reviewed)en
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