1. A dose finding design for seizure reduction in neonates.
- Author
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Ursino, Moreno, Zohar, Sarah, Yuan, Ying, Alberti, Corinne, Comets, Emmanuelle, Favrais, Geraldine, Friede, Tim, Lentz, Frederike, and Stallard, Nigel
- Subjects
SPASMS ,NEWBORN infants ,CLINICAL trials ,ONCOLOGY ,DRUGS - Abstract
Summary: 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. [ABSTRACT FROM AUTHOR]
- Published
- 2019
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