2024 - Developmental & Epileptic Encephalopathy (DEE) Trials: Optimizing Endpoints, Determining MCID, and Enhancing Trial Data Quality

Clinical trials in patients with developmental and epileptic encephalopathy (DEE) present considerable challenges for research teams, including the difficulty in identifying patients, the large geographic spread of patients and the need for measurements that can catch even the smallest degree of change in behavior or function.
One byproduct of these challenges is the difficulty that arises in site selection. It becomes a near impossible task to find sites that are in the appropriate geographies and have the level of expertise to administer the scales necessary for signal detection.
In this webinar, the expert speaker will address these obstacles and share solutions. She will describe approaches to collect data across large geographic areas and in locations that do not have specialized or experienced raters. She will share factors to consider when selecting cognitive and behavioral endpoints for DEE trials, including perspectives from regulatory agencies.
Moreover, she will describe the application of developmental assessments (e.g., Bayley-4) and behavioral scales (e.g., the Vineland-3), along with approaches for determining minimally clinically important difference (MCID) with these measures in DEE populations.
Register for this webinar to gain insights into the complex challenges of conducting clinical trials for developmental and epileptic encephalopathy.
Keywords: Clinical Trials, Rare Disease, Drug Development, Clinical Research, CRO, Epilepsy, Patient Recruitment, Clinical Data, Rare Disease Trials, Rare Disease Clinical Trials, Sites