Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials)
Year: 2020
Type: article
Study design: Study Protocol
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.
Authors: Véronique Crutel, Estelle Lambert, Pierre-François Penelaud, Cristina Albarrán Severo, Joaquin Fuentes, Antoine Rosier, Amaia Hervás, Stéphane Marret, Guiomar Oliveira, Mara Parellada, Simon Kyaga, Sylvie Gouttefangeas, Marianne Bertrand, Denis Ravel, Bruno Falissard
Institutions: Institut de Recherches Internationales Servier (France), Policlínica Gipuzkoa, Centre Hospitalier du Rouvray, University Hospital Mútua de Terrassa, Mútua Terrassa, Inserm, Centre Hospitalier Universitaire de Rouen, Université de Rouen Normandie, Unidade Local de Saúde de Coimbra, University of Coimbra, Hospital General Universitario Gregorio Marañón, Centro de Investigación Biomédica en Red de Salud Mental, Servier (France), Neurochlore (France), Assistance Publique – Hôpitaux de Paris, Université Paris Descartes, Santé mentale et santé publique
Language: English
Subfield: Cognitive Neuroscience
Field: Neuroscience
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $2,750
Funder: Servier
OpenAlex ID: W4240469607