CNS Safety of Drugs in Late Phase Clinical Trials: Using Cognitive Information in Discussions with Regulators

March 30, 2022

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In clinical trials of new drugs that have direct or indirect effects on the central nervous system (CNS), regulatory and medical/scientific groups must be confident that application of the drug at its established clinical dose is associated with negative effects on brain function. Cognition is one important marker of CNS function. In late phase clinical trials, the nature and magnitude of changes in cognition can provide an accurate index of negative CNS effects of the new drugs being studied.

While information about the effects on cognition are used commonly to guide drug development in early phase studies, in late phase trials the design and execution of cognitive tests, and the analyses of the data arising are constrained by different factors. These factors can include greater heterogeneity in sample characteristics, less precision in assessment contexts, less time available for assessments, and the necessity for regulatory compliance. In addition to this data in late phase trials where there is concern about CNS safety, it may also be required to submit to data safety monitoring boards.

In this webinar, Dr. Paul Maruff reviews the aspects of outcomes considered important to regulators from Cogstate’s recent experience in late phase clinical trial programs investigating:

  • Esketamine in adults with major depressive disorder
  • Evolocumab in children with familial hypercholesterolemia
  • Lurasidone in adolescents with schizophrenia


Paul Maruff, PhD
Chief Innovation Officer

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