WP 04 – Clinical Research Development

Objectives

This workpackage has two main components:

1) WP04 includes two of the to-date largest and most comprehensively phenotyped patient cohorts (infant-at risk study, Longitudinal European Autism Project (LEAP)) to identify biomarkers that predict the development of ASD and that can be used to stratify patient groups.

EU-AIMS Longitudinal European Autism Project (EU-AIMS LEAP) - protocols and SOPs (download: zip-file):

  • Study Protocol: Baseline and Follow-up Phases
  • EU_AIMS_LEAP_SOP_clinical_assessments
  • EU_AIMS_LEAP_Eye_tracking_SOP
  • EU_AIMS_LEAP_hair_sample_collection_SOP
  • EU-AIMS_LEAP_ACC_LONG_MRI_SOP
  • EU-AIMS_LEAP_ACC LONG MRI QA SOP
  • EU-AIMS_LEAP Cognitive Testing SOP [including New ET Testing]
  • EU-AIMS_LEAP_ToM_Narrative_SOP
  • EU-AIMS_LEAP_fMRI_SOP
  • EU-AIMS_LEAP_EEG_SOP
  • EU-AIMS_Study_Sample_Collection_SOP

2) WP04 built up the largest network of clinical sites in Europe for ASD: total of 93 sites across 37 European countries. EU-AIMS provides the infrastructure, including instruments, regulatory framework, and network of clinical sites.

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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