A dose finding design for seizure reduction in neonates

Moreno Ursino, Ying Yuan, Corinne Alberti, Emmanuelle Comets, Geraldine Favrais, Tim Friede, Frederike Lentz, Nigel Stallard and Sarah Zohar

Journal of the Royal Statistical Society Series C, 2019, vol. 68, issue 2, 427-444

Abstract: Clinical trials in vulnerable populations are extremely difficult to conduct. A sequential phase I–II trial aimed at finding the appropriate dose of levetiracetam for treating neonatal seizures was planned with a maximum sample size of 50 newborns. Three primary outcomes are considered: efficacy and two types of toxicity that occur at the same time but are measured at different time points. In the case of failure, physicians could add a second agent as a rescue medication. The primary outcomes were modelled via a logistic model for efficacy and a weighted likelihood with pseudo‐outcomes for the two toxicities taking into account the dependences under Bayesian inference. Simulations were conducted to assess the design properties.

Date: 2019
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https://doi.org/10.1111/rssc.12289

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Persistent link: https://EconPapers.repec.org/RePEc:bla:jorssc:v:68:y:2019:i:2:p:427-444

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