Outcome Measures in Clinical Trials for Fragile X Syndrome: The Search for Sensitive Neurocognitive Assays
Guy J., Scerif G.
© 2017 Elsevier Inc. All rights reserved. Clinical trials of multiple pharmacological agents have thus far had more limited success than hypothesized in demonstrating beneficial treatment effects for children; adolescents; and adults with fragile X syndrome (FXS). Other chapters in this edited volume have summarized potential reasons for these limited successes. Here we focus on evaluating the nature of outcome measures that have been used in clinical trials thus far. We emphasize how future trials of both pharmacological and nonpharmacological interventions will require more sensitive and objective measures of neurocognitive functioning; to be developed in close collaboration with stakeholder groups and researchers working on the cognitive; neural; and behavioral profile of individuals with FXS.