Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials)
Véronique Crutel, Estelle Lambert, Pierre‐François Pénélaud, Cristina Albarrán Severo, Joaquín Fuentes, Antoine Rosier, Amaia Hervás, Stéphane Marret, Guiomar Oliveira, Mara Parellada, Simon Kyaga, Sylvie Gouttefangeas, Marianne Bertrand, Denis Ravel, Bruno Falissard
Abstract
There are currently no approved pharmacological treatments to improve social reciprocity and limit repetitive and rigid behaviors in autism spectrum disorder (ASD). We describe the design of two Phase III studies evaluating the efficacy/safety of bumetanide oral liquid formulation in ASD. These are international, multicenter, randomized, double-blind, placebo-controlled studies in children and adolescents with ASD aged 7 to 17 years (n = 200; study 1), or younger children with ASD aged 2 to 6 years (n = 200; study 2). The primary endpoint of each is change in Childhood Autism Rating Scale 2 total raw score after 6 months. These studies could contribute to the first pharmacological treatment to improve social reciprocity and limit repetitive and rigid behaviors in children and adolescents with ASD.