The purpose of this study was to evaluate the efficacy and the safety of bumetanide/S95008 in the improvement of Autism Spectrum Disorder core symptoms.
The present study (CL3-95008-001) was performed in children and adolescents from 7 to less than 18 years old presenting with ASD. A 6-month double-blind treatment period was performed in which efficacy and safety of bumetanide 0.5mg BID were assessed versus placebo. This double-blind period was followed by a 6-month open label treatment period of bumetanide 0.5mg BID in which long term safety was evaluated.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
211
Oral Solution dosed at 0.5mg/mL Taken twice daily
Oral Solution Taken twice daily
Childhood Autism Rating Scale, Second Edition (CARS2) total raw score
Efficacy criterion The CARS2 is a 15 item rated instrument. The rating values given for the 15 areas are summed to produce a Total score.
Time frame: Change from baseline to 6 month
Social Responsiveness Scale, Second Edition (SRS-2) total raw score
Efficacy criterion 65 items scale measuring symptoms associated with autism
Time frame: Change from baseline to 6 month
Clinical Global Impression - Global Improvement (CGI-I) score
Efficacy criterion Scale which assesses the severity of the illness and the global improvement of the patient under study treatment
Time frame: At 6 months
Vineland Adaptative Behaviour Sales, Second Edition (VABS II) subscores
Efficacy criterion Scale designated to measure adaptative behaviour
Time frame: Change from baseline to 6 month
Adverse events and Paediatric Adverse Event Rating Scale (PAERS)
Safety criterion Inventory report used to identify signs/symptoms experienced by the patient since the study treatment initiation.
Time frame: On average of 52 weeks
Abnormalities in 12-leads electrocardiogram (ECG) parameters
Safety criterion
Time frame: selection visit/Week004/Week008/Week012/Week 026/Week030/Week034/Week038/Week052
Renal ultrasound
Safety criterion Assessment of the renal function
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Trial Tech em Pesquisas com Medicamentos Ltda
Curitiba, Brazil
Hospital Universitário Walter Cantídio - Universidade Federal do Ceará
Fortaleza, Brazil
Hospital São Vicente de Paulo
Passo Fundo, Brazil
Universidade Federal de São Paulo, Escola Paulista de Medicina
São Paulo, Brazil
Faculdade de Medicina da Universidade de São Paulo - Departamento de Psiquiatria
São Paulo, Brazil
GSC CHU-LENVAL Centre ressource autisme
Nice, Alpes-Maritimes, France
Centre d'Investigation Clinique de Lyon
Bron, Auvergne-Rhône-Alpes, France
Hôpital Le Vinatier CRA Rhône-Alpes, Bat 211
Bron, Auvergne-Rhône-Alpes, France
Hôpitaux Universitaires de Strasbourg Service de Psychiatrie de l'Enfant et de l'Adolescent
Strasbourg, Grand Est, France
Hôpital Robert Debré Service de Psychiatrie de l'enfant et de l'adolescent
Paris, Il de France, France
...and 35 more locations
Time frame: selection visit/Week026/Week052
the Columbia Suicide Severity Rating Scale Children's version (C-SSRS-C)
Scale which assesses suicidal ideation and suicidal behaviour
Time frame: Week000/Week012/Week026/Week038/Week052
Tanner stage
Safety criterion Assessment of Pubertal development
Time frame: Week000/Week026/Week052
Acceptability and palatability questionnaire
Acceptability and palatability criterion Assessment of the ease of use to use the dosing device
Time frame: Week026
Paediatric Quality of Life Inventory (PedsQL) questionnaire
Quality of Life criterion Assessment of parent/legal representative perception of patient health related quality of life
Time frame: Week000/Week004/Week012/Week026/Week030/Week038/Week052