Servier's Phase III bumetanide study fails endpoints

French privately-held pharma major Servier and Neurochlore have announced that no sign of effectiveness was observed in their two Phase III clinical studies assessing bumetanide versus placebo in the treatment of autism spectrum disorders (ASD) in children and adolescents.

As a consequence, Servier and Neurochlore have decided, by mutual agreement, on an early termination of the two clinical studies in progress.

These two double-blind randomized Phase III studies versus placebo were initiated four years ago in 14 countries, including 11 in Europe, within the framework of the Pediatric Investigation Plan (PIP) granted by the European Agency of Medicine (EMA). Thus, 422 children and adolescents with moderate-to-severe ASD, shared between the two studies, each with 211 participants according to their age (2-6 years and 7-17 years), were followed over six months in a double-blind placebo-controlled study, before being followed on bumetanide for an additional 6 months. It is the largest Phase III European program dedicated to the treatment of core symptoms of autism in pediatrics.