WP 04 – Clinical Research Development

Objectives

This workpackage has two main components. The first is to conduct clinical studies in high-risk infants to identify biomarkers which precede onset of clinical symptoms and establish, validate biomarkers in children and adults with ASD and identify biomarkers involved in drug response, and the second is to provide for the infrastructure, including instruments, regulatory framework, network of clinical sites and trained professionals necessary to perform clinical trials.The objectives of this workpackage are:

  • Identify biomarkers of ASD which precede onset of clinical symptoms
  • Validate biomarkers of ASD in children and adults
  • Identify molecular-physiologic pathways of drug response
  • Develop clinical infrastructure
  • Develop new standardized clinical and cognitive assessment methods for use in clinical trials, and regulatory framework
  • Assess clinical standards and outcome of ASD individuals in the EU, and develop education programs

WP Leads

Academic Lead: Professor Dr. Jan Buitelaar, Radboud University Nijmegen Medical Centre, The Netherlands

EFPIA Lead: Dr. Xavier Liogier d'Ardhuy, F. Hoffmann - La Roche, Switzerland

Workpackage partners

  • Radboud University Nijmegen Medical Centre
  • F. Hoffmann- La Roche
  • King's College London
  • Cambridge University
  • University Medical Centre
  • Karolinska Institutet
  • Eli Lilly and Company Ltd.
  • Janssen Pharmaceutica
  • Vifor Pharma
  • Birkbeck, University of London
  • University "Campus Bio-Medico"
  • Pfizer Limited
  • Autism Speaks

Workpackage outcomes

This workpackage 

  • will establish translationally validated, reliable, systems-level biomarkers that can be used across species and in healthy, high-risk and affected human subjects for stratification and characterization, response prediction and early efficacy studies.

  • will identify molecular biomarkers with an established link to heritable risk for ASD that can be deployed in affected individuals; and for the development and early validation of new compounds.

  • will yield usable individual mode and multimodal biomarkers for drug development and response prediction.

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