Workpackage 16 – Clinical trials


A large proportion of available antiepileptic drugs (AEDs) have no regulatory approval for use in children, Moreover, for most rare disabling epilepsies there are no approved treatments. There is an urgent need to collect standardized information on outcomes associated with specific treatments (individual AEDs, AED combinations, and non- pharmacological treatments such as diets, neurostimulation or surgical therapies) in epilepsy patients with well characterized rare syndromes and rare aetiologies. Moreover, there is a clear need to identify/develop novel treatments targeting specific aetiologies. Establishment of an epilepsy ERN offers unique opportunities to address these needs.


  • Explore relationships between applied treatments (including non-pharmacological treatments) and clinical outcomes in well characterized cohorts of epilepsy patients with rare epilepsies
  • Identify candidate treatments to be tested in rare epilepsies with rare syndromes/specific aetiologies;
  • Explore orphan drug development with Pharma (as seen with fenfluramine in Dravet syndrome)
  • Conduct well designed studies, including randomized controlled trials, to evaluate the potential efficacy and the safety of novel treatments in the above cohorts;
  • Contribute to discovery and development of novel chemical entities and other novel therapies by using precision medicine approaches


  • D16.1 Report on eCRF utilisation 
  • D V.3 Identification candidate treatments prioritized for testing in selected patient cohorts at month 24
  • D V.4 Report of exploratory studies and of controlled trials


Pr. Rima Nabbout, MD, PhD

Hôpital Necker – Enfants Malades

Paris, France

Pr. Rima Nabbout, MD, PhD

Pr. Emilio Perucca, MD