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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

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Abstract

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.

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Acknowledgements

We thank the patients and their families for their participation in this study.

Funding

This study was supported by Servier. Medical writing support (in the form of writing assistance, collating author comments, assembling tables/figures, grammatical editing and referencing) was provided by Fiona Goodwin and Rebecca Cunningham of Aura, a division of Spirit Medical Communications Group Limited (Manchester UK) and was funded by Servier.

Author information

Authors and Affiliations

  1. ReCognition Health, London, UK

    Christina Georgoula & Maite Ferrin

  2. Child and Adolescent Psychiatry Unit, Niepubliczny Zakład Opieki Zdrowotnej Gdańskie Centrum Zdrowia, Gdańsk, Poland

    Bozena Pietraszczyk-Kedziora

  3. Child and Adolescent Mental Health Unit, Hospital Universitari Mútua de Terrassa, and Global Institute of Neurodevelopment Integrated Care (IGAIN), Barcelona, Spain

    Amaia Hervas

  4. Department of Neonatal Pediatrics, Intensive Care, and Neuropediatrics, Rouen University Hospital and INSERM U 1245 Team 4 Neovasc, School of Medicine, Normandy University, Rouen, France

    Stéphane Marret

  5. Neurodevelopmental and Autism Unit From Child Developmental Center and Centro de Investigação e Formação Clínica, Hospital Pediátrico, Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal

    Guiomar Oliveira

  6. Faculty of Medicine, University Clinic of Pediatrics, University of Coimbra, Coimbra, Portugal

    Guiomar Oliveira

  7. Department of Neonatal Pediatrics, CHU de Rouen and CHU le Rouvray, Sotteville les Rouen, France

    Antoine Rosier

  8. Neuro Inflammation Therapeutic Area, Institut de Recherches Internationales Servier, 50 Rue Carnot, 92284, Suresnes CEDEX, France

    Véronique Crutel, Emmanuelle Besse & Cristina Albarrán Severo

  9. Neurochlore, Marseille, France

    Denis Ravel

  10. Child & Adolescent Psychiatry Service, Policlínica Gipuzkoa and Gipuzkoa Autism Society (GAUTENA), Donostia/San Sebastián, Spain

    Joaquin Fuentes

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  1. Christina Georgoula
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  6. Guiomar Oliveira
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  10. Cristina Albarrán Severo
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Contributions

MF, CG, BPK, AH, SM, GO, AR, and JF acquisition of data—critical review and revision of the manuscript—approval of final version for submission. VC, EB, CAS, DR, study conception and design—data analysis and interpretation—critical review and revision of the manuscript—approval of final version for submission.

Corresponding author

Correspondence to Cristina Albarrán Severo.

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Conflict of interest

Maite Ferrin—research support from Servier; advisory board participation for Novartis. Christina Georgoula, Bozena Pietraszczyk-Kedziora, Amaia Hervas, Stéphane Marret, Guiomar Oliveira, Antoine Rosier—research support from Servier. Véronique Crutel, Emmanuelle Besse, Cristina Albarrán Severo—employee of Servier. Denis Ravel—employee of Neurochlore. Joaquin Fuentes—research support from Policlínica Gipuzkoa Foundation, Servier and AIMS-2-Trials project ID 777394. Partial support for attending professional meetings from Policlínica Gipuzkoa Foundation, ESCAP and AACAP.

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Georgoula, C., Ferrin, M., Pietraszczyk-Kedziora, B. et al. 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. Child Psychiatry Hum Dev 54, 1360–1372 (2023). https://doi.org/10.1007/s10578-022-01328-5

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