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Article Dans Une Revue Journal of the Royal Statistical Society: Series C Applied Statistics Année : 2019

A dose finding design for seizure reduction in neonates

Résumé

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.
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Dates et versions

hal-03862983 , version 1 (11-12-2023)

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Moreno Ursino, Ying Yuan, Corinne Alberti, Emmanuelle Comets, Geraldine Favrais, et al.. A dose finding design for seizure reduction in neonates. Journal of the Royal Statistical Society: Series C Applied Statistics, 2019, 68 (2), pp.427-444. ⟨10.1111/rssc.12289⟩. ⟨hal-03862983⟩
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