Biomarkers Consortium - The Autism Biomarkers Consortium for Clinical Trials (ABC-CT)

Autism Spectrum Disorders (ASD), affecting an estimated 1% of children worldwide, are a group of developmental disorders marked by impairments in reciprocal social interaction and the presence of stereotyped or repetitive behavior, interests or activities. ASD affects early brain development with signs and symptoms of autism emerging in the first two years of life. The pathologies remain unknown and diagnosis is complicated by an extremely heterogeneous clinical presentation, including variability in cognitive function that can range from severely impaired to above normal. Heterogeneity and the lack of objective, quantifiable measures of change in social functioning hinder attempts to develop interventions for the core social impairment of autism. There is a great need for biomarkers that can separate subjects into subgroups that share a common pathophysiology to maximize the ability of clinical trials to detect benefit, as well as for biomarkers that are sensitive to change in social functioning which may ultimately be strong predictors of treatment effects or provide evidence for choice of one class of treatment over another.

To address this need, the present project is focusing on evaluating the potential utility of EEG to measure brain function, eye tracking technology to measure visual attention and automated recording techniques to assess behavior and speech, children aged 6 to 11 will be monitored over a six-month period. In particular, the project will compare whether lab-based measures, versus clinician and caregiver assessments of specific domains of social impairment, are more sensitive indicators of clinical status. Additionally, the project will evaluate whether a well-justified set of eye tracking and EEG measures, individually or in combination, have potential utility as stratification biomarkers and/or sensitive and reliable measures of change in clinical trials. Finally, the project will collect blood DNA samples from all subjects, including the parents of the ASD subjects, for future genomic and other analyses.

Goals

  • To qualify a set of measures that can be used as stratification biomarkers and/or sensitive and reliable objective measures of social impairment in ASD clinical trials, which could serve as indicative markers of long-term clinical outcome.
  • To evaluate whether a well-justified set of eye tracking and EEG measures, individually or in combination, have potential utility as stratification biomarkers and/or sensitive and reliable measures of change in clinical trials.

Results & Accomplishments

The Feasibility Study has been completed and the full study has started.

Partners

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
European Autism Interventions – A Multicentre Study for Developing New Medications (EU-AIMS)
Food and Drug Administration
Janssen Research & Development, LLC
National Institute of Mental Health (NIMH)
National Institute of Neurological Disorders and Stroke (NINDS)
Simons Foundation
Yale University School of Medicine

Contact

Rosa Canet-Aviles, Scientific Program Manager, Neuroscience, rcanet-aviles@fnih.org