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A dose finding design for seizure reduction in neonates

Author

Listed:
  • Moreno Ursino
  • Ying Yuan
  • Corinne Alberti
  • Emmanuelle Comets
  • Geraldine Favrais
  • Tim Friede
  • Frederike Lentz
  • Nigel Stallard
  • Sarah Zohar

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.

Suggested Citation

  • Moreno Ursino & Ying Yuan & Corinne Alberti & Emmanuelle Comets & Geraldine Favrais & Tim Friede & Frederike Lentz & Nigel Stallard & Sarah Zohar, 2019. "A dose finding design for seizure reduction in neonates," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 68(2), pages 427-444, February.
    • Handle: RePEc:bla:jorssc:v:68:y:2019:i:2:p:427-444
    DOI: 10.1111/rssc.12289
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