Synergy in Action

Autism Spectrum Disorder Working Group Activity / Deliverables

ISCTM/ECNP Joint Working Group
Chairs: Valentina Mantua, MD, PhD; Tiffany Farchione, MD; Celso Arango, MD, PhD, 

Inaugural Working Group session2018 Autumn Conference 15,16 October 2018, Marina del Rey
View Subgroup Presentations: 
Molecular Targets
Trial Design 


There is a clear unmet need in the availability of medicinal products for the treatment of Autism. Despite the great amount of scientific investigation into the neurodevelopmental and biological causes of Autism, research has failed to deliver a therapy for the core features of Autism or a biomarker to stratify populations for potential treatments with different mechanism of action.

If a product were intended to be developed for the treatment of individuals with Autism Spectrum Disorder under the DSM5 definition, heterogeneity in phenotypes and clinical manifestations would be a major drawback for the planning of a clinical trial. In addition, the clinical presentational and the medical needs of the population vary largely with age; therefore, hardly any data could be extrapolated and age groups should be investigated independently. Despite the availability of tools for diagnosis and outcome assessment, the clinical meaningfulness of changes on these measures has yet to be defined.  It is not yet clear which outcome measure is most appropriate for use in clinical trials.   Strategies to address these challenges and others will be the focus of this working group. 

The ISCTM is a unique environment born out of the need to foster drug development in CNS disorder. ISCTM brings together multi-disciplinary expertise including industry, academia, regulators, patients, and family associations. For the purpose of optimizing a methodological framework for the conduct of clinical trials in Autism Spectrum Disorder wide participation of all stakeholders is needed, and therefore  ECNP has agreed to formally join ISCTM in supporting this effort.

Working Group Project Page

Work plan:
The first phase of the Working Group will be to build a common knowledge, which would form the basis of a clinical trial methodology proposal. Issues to be addressed should include: population definition and stratification (IQ, biomarkers, age), endpoints and tools for outcome assessment, clinical trial duration, non-pharmacological therapies as background and comorbidities.

The main deliverable of the first phase is to develop a session which will be presented at the Joint ISCTM/ECNP meeting 6 September 2019, in Copenhagen. The second phase would include the organization of the knowledge accumulated and the building of a consensus around the main issues addressed during the first phase.

Working Group Session: 15 October, 2018 -2018 Autumn Conference –  The Ritz Carlton, Marina del Rey, California
Working Group Session: 19 or 20 February 2019 – 14th Annual Meeting – The Fairmont, Washington DC
Group will also meet via regular telecons.