Evaluation of Outcome Measures and Phase 2 Lessons from the Aberrant Behavior Scale

Panel Presentation
Friday, May 3, 2019: 10:30 AM
Room: 518 (Palais des congres de Montreal)
E. Anagnostou1, T. Bennett2, S. Jacob3, R. Nicolson4 and M. Woodbury-Smith5, (1)Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, Canada, (2)Offord Centre for Child Studies, McMaster University, Hamilton, ON, CANADA, (3)Department of Psychiatry, University of Minnesota, Minneapolis, MN, (4)University of Western Ontario, London, ON, Canada, (5)Newcastle University, Institute of Neuroscience, Newcastle upon Tyne, United Kingdom of Great Britain and Northern Ireland
Background: Suboptimal outcome measures have plagued the field of clinical trials in ASD. Measures currently used in trials present limitations in psychometric properties, sensitivity to change, placebo response, among others.

Methods: This abstract will set up the panel by reviewing the recommendations on outcome measures, based on a series of white papers produced by a think tank sponsored by Autism Speaks and NIH. We will then examine how these recommendations behaved in a series of phase 2 clinical trials in ASD in Canada (POND network and related networks)

Results: Sensitivity to change was documented for the ABC-Social withdrawal subscale. However the measure seems to also be associated with significant placebo response. Secondary measures discussed in the white papers will also be presented with data to confirm the limitations presented in the original reports (multiple constructs combined into a singe score, unusual distribution of items).

Conclusions: Some of the originally proposed clinical trials outcome measures by the Autism speaks/NIH think tank show sensitivity to change with a variety of compounds and targets, but are presenting with limitations related to placebo response and construct validity. Further refinement of existing measures and development of new measures is required to optimize endpoint measurement for clinical trials in ASD.