Other Publication Details
Mandatory Fields
Reviews
Soul, JS;Pressler, R;Allen, M;Boylan, G;Rabe, H;Portman, R;Hardy, P;Zohar, S;Romero, K;Tseng, B;Bhatt-Mehta, V;Hahn, C;Denne, S;Auvin, S;Vinks, A;Lantos, J;Marlow, N;Davis, JM
2019
June
Recommendations for the design of therapeutic trials for neonatal seizures
Validated
1
WOS: 33 ()
Optional Fields
HYPOXIC-ISCHEMIC ENCEPHALOPATHY GROSS MOTOR FUNCTION OFF-LABEL USE CEREBRAL-PALSY NEUROLOGIC EXAMINATION ANTIEPILEPTIC DRUGS BOVINE SURFACTANT CLINICAL-TRIALS ILAE COMMISSION ETHICAL-ISSUES
Although seizures have a higher incidence in neonates than any other age group and are associated with significant mortality and neurodevelopmental disability, treatment 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 at 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, 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 improve the treatment and outcome for this highly vulnerable population.
NEW YORK
NATURE PUBLISHING GROUP
0031-3998
943
954
10.1038/s41390-018-0242-2
Grant Details