Abstract
AbstractThere 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.
Publisher
Springer Science and Business Media LLC
Subject
Developmental and Educational Psychology
Reference62 articles.
1. American Psychiatric Association. (2013). Diagnostic and statistical manual of mental disorders (DSM-5®). Washington, DC: American Psychiatric Publishing.
2. American Psychiatric Association. (2016). DSM-5 Update. Supplement to Diagnostic and statistical manual of mental disorders (DSM-5). Washington, DC: American Psychiatric Publishing.
3. Autism Genome Project Consortium, Szatmari, P., Paterson, A. D., Zwaigenbaum, L., Roberts, W., Brian, J., et al. (2007). Mapping autism risk loci using genetic linkage and chromosomal rearrangements. Nature Genetics, 39(3), 319–328. https://doi.org/10.1038/ng1985.
4. Bai, D., Yip, B. H. K., Windham, G. C., Sourander, A., Francis, R., Yoffe, R., et al. (2019). Association of genetic and environmental factors with autism in a 5-country cohort. JAMA Psychiatry, 76(10), 1035–1043. https://doi.org/10.1001/jamapsychiatry.2019.1411.
5. Baron-Cohen, S., Lombardo, M. V., Auyeung, B., Ashwin, E., Chakrabarti, B., & Knickmeyer, R. (2011). Why are autism spectrum conditions more prevalent in males? PLoS Biology, 9 (6), e1001081. https://doi.org/10.1371/journal.pbio.1001081.
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