Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials)

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...

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Bibliographic Details
Published in:Journal of autism and developmental disorders 2021-08, Vol.51 (8), p.2959-2972
Main Authors: Crutel, Véronique, Lambert, Estelle, Penelaud, Pierre-François, Albarrán Severo, Cristina, Fuentes, Joaquin, Rosier, Antoine, Hervás, Amaia, Marret, Stéphane, Oliveira, Guiomar, Parellada, Mara, Kyaga, Simon, Gouttefangeas, Sylvie, Bertrand, Marianne, Ravel, Denis, Falissard, Bruno
Format: Article
Language:English
Subjects:
Adolescent
Adolescents
Autism
Autism Spectrum Disorder - drug therapy
Autistic adolescents
Autistic children
Behavior Problems
Behavioral Science and Psychology
Bumetanide
Bumetanide - administration & dosage
Bumetanide - therapeutic use
Child
Child and School Psychology
Child, Preschool
Childhood
Children
Diagnosis
Dosage and administration
Double-Blind Method
Double-blind studies
Drug Therapy
Efficacy
Humans
Interpersonal Competence
Intervention
Male
Neurosciences
Original Paper
Pediatrics
Pervasive Developmental Disorders
Pharmacology
Placebos
Psychology
Public Health
Reciprocity
Repetitive behaviour
Research Design
Social Behavior
Symptoms (Individual Disorders)
Teenagers
Testing
Treatment methods
Treatment Outcome
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Description
Summary: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.
ISSN:0162-3257
1573-3432
1573-3432
DOI:10.1007/s10803-020-04709-8