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The efficacy of bumetanide (oral liquid formulation 0.5 mg bid) as a treatment for the core symptoms of autism spectrum disorders in children and adolescents aged 7-17 years is being investigated in an international, randomised, double-blind, placebo-controlled phase III study. The primary endpoint is the change in Childhood Autism Rating Scale 2 (CARS2) total raw score after 6 months of treatment. At baseline, the 211 participants analysed are broadly representative of autistic subjects in this age range: mean (SD) age, 10.4 (3.0) years; 82.5% male; 47.7% with intelligence quotient ≥ 70. Mean CARS2 score was 40.1 (4.9) and mean Social Responsiveness Scale score was 116.7 (23.4). Final study results will provide data on efficacy and safety of bumetanide in autistic children and adolescents.

Titel:	A Phase III Study of Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents Aged Between 7 and 17 Years with Autism Spectrum Disorder (SIGN 1 Trial): Participant Baseline Characteristics.
Autor/in / Beteiligte Person:	Georgoula, C ; Ferrin, M ; Pietraszczyk-Kedziora, B ; Hervas, A ; Marret, S ; Oliveira, G ; Rosier, A ; Crutel, V ; Besse, E ; Severo, CA ; Ravel, D ; Fuentes, J
Link:	Full Text Finder (Volltext)
Zeitschrift:	Child psychiatry and human development, Jg. 54 (2023-10-01), Heft 5, S. 1360-1372
Veröffentlichung:	1999- : New York : Kluwer Academic/Plenum Publishers ; <i>Original Publication</i>: New York, Behavioral Publications, 2023
Medientyp:	academicJournal
ISSN:	1573-3327 (electronic)
DOI:	10.1007/s10578-022-01328-5
Schlagwort:	Humans Child Male Adolescent Female Bumetanide adverse effects Double-Blind Method Treatment Outcome Autism Spectrum Disorder drug therapy Autistic Disorder diagnosis
Sonstiges:	Nachgewiesen in: MEDLINE Sprachen: English Publication Type: Randomized Controlled Trial; Clinical Trial, Phase III; Journal Article; Research Support, Non-U.S. Gov't Language: English [Child Psychiatry Hum Dev] 2023 Oct; Vol. 54 (5), pp. 1360-1372. <i>Date of Electronic Publication: </i>2022 Mar 16. MeSH Terms: Autism Spectrum Disorder* / drug therapy ; Autistic Disorder* / diagnosis ; Humans ; Child ; Male ; Adolescent ; Female ; Bumetanide / adverse effects ; Double-Blind Method ; Treatment Outcome References: American Psychiatric Association (2013) Diagnostic and statistical manual of mental disorders (DSM-5 ® ) (American Psychiatric Pub). ; Chiarotti F, Venerosi A (2020) Epidemiology of autism spectrum disorders: a review of worldwide prevalence estimates since 2014. Brain Sci 10:274. (PMID: 728802210.3390/brainsci10050274) ; Elsabbagh M, Divan G, Koh YJ, Kim YS, Kauchali S, Marcín C et al (2012) Global prevalence of autism and other pervasive developmental disorders. 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(PMID: 10.1016/S0140-6736(09)61803-1) Contributed Indexing: Keywords: Autism spectrum disorder; Bumetanide; Paediatrics; Randomised controlled trial Substance Nomenclature: 0Y2S3XUQ5H (Bumetanide) Entry Date(s): Date Created: 20220316 Date Completed: 20230818 Latest Revision: 20230822 Update Code: 20240513

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A Phase III Study of Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents Aged Between 7 and 17 Years with Autism Spectrum Disorder (SIGN 1 Trial): Participant Baseline Characteristics.