Servier and Neurochlore announce the results of two phase III clinical trials assessing bumetanide for autism spectrum disorder.

As a consequence, Servier and Neurochlore have decided, by mutual agreement, on an early termination of the two clinical studies .The studies in Phase III versus placebo, were initiated 4 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 6 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.

The results of these studies have not demonstrated the superiority of bumetanide compared with placebo. None of the effectiveness criteria, both primary (CARS – Childhood Autism Rating Scale) and secondary (SRS – Social Responsiveness Scale -, CGI Scale – Clinical Global Impression Scale -, Vineland) were reached after 6 months of treatment, not in children aged 2-6 years or in children and adolescents aged 7-17 years. The studies did not reveal any unexpected safety issue associated with the use of bumetanide.

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