Recommendations for the design of therapeutic trials for neonatal seizures

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dc.contributor.author Soul, Janet S.
dc.contributor.author Pressler, Ronit
dc.contributor.author Allen, Marilee
dc.contributor.author Boylan, Geraldine
dc.contributor.author Rabe, Heike
dc.contributor.author Portman, Ron
dc.contributor.author Hardy, Pollyanna
dc.contributor.author Zohar, Sarah
dc.contributor.author Romero, Klaus
dc.contributor.author Tseng, Brian
dc.contributor.author Bhatt-Mehta, Varsha
dc.contributor.author Hahn, Cecil
dc.contributor.author Denne, Scott
dc.contributor.author Auvin, Stephane
dc.contributor.author Vinks, Alexander
dc.contributor.author Lantos, John
dc.contributor.author Marlow, Neil
dc.contributor.author Davis, Jonathan M.
dc.date.accessioned 2019-11-20T06:07:08Z
dc.date.available 2019-11-20T06:07:08Z
dc.date.issued 2019
dc.identifier.citation Soul, 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/s41390-018-0242-2 en
dc.identifier.volume 85 en
dc.identifier.issued 7 en
dc.identifier.startpage 943 en
dc.identifier.endpage 954 en
dc.identifier.issn 0031-3998
dc.identifier.uri http://hdl.handle.net/10468/9158
dc.identifier.doi 10.1038/s41390-018-0242-2 en
dc.description.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. en
dc.description.sponsorship U18FD005320-01 en
dc.format.mimetype application/pdf en
dc.language.iso en en
dc.publisher Springer Nature en
dc.relation.uri https://www.nature.com/articles/s41390-018-0242-2
dc.rights © The Author(s) 2019 en
dc.rights.uri https://creativecommons.org/licenses/by/4.0/ en
dc.subject Therapeutic trials en
dc.subject Neonatal seizures en
dc.subject Well-designed clinical trials en
dc.title Recommendations for the design of therapeutic trials for neonatal seizures en
dc.type Article (peer-reviewed) en
dc.internal.authorcontactother Geraldine Boylan, Department of Paediatrics and Child Health, University College Cork, Cork, Ireland. +353-21-490-3000 Email: g.boylan@ucc.ie en
dc.internal.availability Full text available en
dc.description.version Published Version en
dc.contributor.funder U.S. Food and Drug Administration en
dc.description.status Peer reviewed en
dc.identifier.journaltitle Pediatric Research en
dc.internal.IRISemailaddress g.boylan@ucc.ie en
dc.identifier.eissn 1530-0447


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