Autism Spectrum Disorder (ASD) Clinical Trial
Official title:
Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder. A 6-month Randomised, Double-blind, Placebo Controlled Multicentre Parallel Group Study to Evaluate Efficacy and Safety of Bumetanide 0.5mg Twice a Day Followed by an Open Label Active 6-month Treatment Period With Bumetanide (0.5mg Twice a Day) and a 6 Weeks Discontinuation Period After Treatment Stop.
Verified date | May 2023 |
Source | Servier |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study was to evaluate the efficacity and the safety of bumetanide/S95008 in the improvement of Autism Spectrum Disorder core symptoms.
Status | Terminated |
Enrollment | 211 |
Est. completion date | October 26, 2021 |
Est. primary completion date | October 26, 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 6 Years |
Eligibility | Inclusion Criteria: - Male and female patients from 2 to less than 7 - Primary diagnosis of ASD as per Diagnostic and Statistical Manual of Mental Disorders - Fifth Edition (DSM-5) criteria - Criteria met for ASD on Autism Diagnostic Observation Schedule-Generic (ADOS-2) and Autism Diagnosis Interview Revised (ADI-R) - CGI (Clinical Global Impression) - Severity rating Score = 4 - Childhood Autism Rating Scale second edition (CARS2-ST or HF) total raw score = 34 - Social responsiveness Scale second edition (SRS-2) total score = 66 T-Score - Absence of diagnosis of Fragile X or Rett Syndrome - Absence of any clinically significant abnormality likely to interfere with the conduct of the study according to the judgment of the investigator. Exclusion Criteria: - Patients not able to follow the study assessments defined by the protocol, with the exception of self-rating questionnaires which will be assessed by parent/legal representative/caregiver for those patients unable to complete them - Patients having a high suicidal risk according to the investigator judgement - Chronic renal dysfunction - Chronic cardiac dysfunction - Patient with unstable psychotherapy, behavioural, cognitive or cognitive-behavioural therapy - Severe electrolyte imbalance that is likely to interfere with the study conduct or evaluation |
Country | Name | City | State |
---|---|---|---|
Australia | Liverpool Hospital | Liverpool | |
Australia | The Royal Children's Hospital Melbourne | Parkville | |
Brazil | Trial Tech em Pesquisas com Medicamentos Ltda | Curitiba | |
Brazil | Hospital Universitário Walter Cantídio-Universidade Federal do Ceará | Fortaleza | |
Brazil | Clínica Neurológica e Neurocirúrgica de Joinville | Joinville | |
Brazil | Hospital São Vicente de Paulo | Passo Fundo | |
Brazil | Faculdade de Medicina da Universidade de São Paulo - Departamento de psiquiatra | São Paulo | |
Brazil | Universidade Federal de São Paulo, Escola Paulista de Medicina | São Paulo | |
Czechia | University Hospital Brno, Department of Child Neurology | Brno | |
Czechia | University hospital of Ostrava, Department of Psychiatry | Poruba | Ostrava |
Czechia | Institute of Neuropsychiatric Care, Department of Child Psychiatry | Praha | |
France | Centre Hospitalier Charles Perrens CRA Aquitaine | Bordeaux | Nouvelle-Aquitaine |
France | Hôpital des Enfants-Pellegrin | Bordeaux | Nouvelle Aquitaine |
France | Centre d'Investigation Clinique de Lyon | Bron | Auvergne Rhone Alpes |
France | Hôpital Le Vinatier CRA Rhône-Alpes, Bat 211 | Bron Cedex | Auvergne-Rhône-Alpes |
France | GSC CHU-LENVAL Centre ressource autisme | Nice | Alpes-Maritimes |
France | Hôpital Robert Debré Service de Psychiatrie de l'enfant et de l'adolescent | Paris | Ile De France |
France | CHU Rouen | Rouen | Normandie |
France | Centre Hospitalier du Rouvray Centre de Ressources pour l'Autisme | Sotteville-lès-Rouen | Normandie |
France | Hôpitaux Universitaires de Strasbourg Service de Psychiatrie de l'Enfant et de l'Adolescent | Strasbourg | Alsace-Champagne-Ardenne-Lorraine |
Hungary | Magyar Református Egyház Bethesda Gyermekkórháza | Budapest | |
Hungary | Servus Salvus Kft. | Budapest | |
Hungary | Vadaskert Korhaz es Szakambulancia | Budapest | |
Hungary | Bekes Megyei Kozponti Korhaz Gyermek es ifjusagpszichiatriai osztaly | Gyula | |
Hungary | Szegedi Tudományegyetem Szent-Gyorgy Albert Klinikai Kozpont Gyermekgyógyászati Klinika Gyermek es Ifjusagpszichiátriai o | Szeged | |
Italy | Neuro Riabilitazione/Psicopatologia dell'età evolutiva Istituto Scientifico Medea - Bosisio Parini | Bosisio Parini | Lombardia |
Italy | Clinica di Neuropsichiatria dell'Infanzia e dell'Adolescenza Ospedale Pediatrico-Microcitemico | Cagliari | Sardegna |
Italy | U.O.C. Psichiatria dello Sviluppo IRCCS Fondazione Stella Maris | Calambrone | Toscana |
Italy | Programma Interdipartimentale "Autismo 0-90" A.O.U. Policlinico "Gaetano Martino" | Messina | Sicilia |
Italy | U.O. di Neuropsichiatria Infantile Fondazione Istituto Neurologico Nazionale Casimiro Mondino Istituto di Ricovero e Cura a Carattere Scientifico | Pavia | Lombardia |
Italy | U.O. di Neuropsichiatria Infantile Azienda Ospedaliera Universitaria Senese | Siena | Toscana |
Poland | "Niepubliczny Zaklad Opieki Zdrowotnej Gdanskie Centrum Zdrowia Sp. z o.o. | Gdansk | |
Poland | Centrum Badan Klinicznych PI-House sp. z o.o | Gdansk | |
Poland | NAVICULA Centrum Diagnozy i Terapii Autyzmu w Lodzi | Lodz | |
Poland | Fundacja SYNAPSIS ul. | Warszawa | |
Poland | Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie Oddzial Kliniczny Psychiatrii Wieku Rozwojowego | Warszawa | |
Portugal | Centro Hospitalar e Universitario de Coimbra Hospital Pediatrico | Coimbra | |
Slovakia | National Institute of Children Diseases, Department of Child Psychiatry | Bratislava | |
Slovakia | EPAMED, s.r.o. | Košice | |
Spain | Hospital General Universitario de Alicante | Alicante | |
Spain | Hospital Clinic de Barcelona | Barcelona | |
Spain | Hospital Niño Jesus | Madrid | |
Spain | Hospital Universitario Gregorio Marañon | Madrid | |
Spain | Hospital Puerta de Hierro | Majadahonda | Madrid |
Spain | Policlinica Guipuzcoa | San Sebastian | Guipuzcoa |
Spain | Hospita Mutua de Terrassa | Terrassa | Barcelona |
United Kingdom | Colchester Hospital | Colchester | |
United Kingdom | ReCognition Health | London | |
United Kingdom | The Winnicott Centre 195-197 Hathersage Road | Manchester | |
United States | Richmond Behavioral Associates | Staten Island | New York |
Lead Sponsor | Collaborator |
---|---|
Institut de Recherches Internationales Servier | ADIR, a Servier Group company |
United States, Australia, Brazil, Czechia, France, Hungary, Italy, Poland, Portugal, Slovakia, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Childhood Autism Rating Scale, Second Edition (CARS2) Total Raw Score | The CARS2 total raw score range from 15 to 60. This scale is a behaviour rating scale intended to diagnose autism. A total score of 15 indicates that an individual behaviour is within normal limits, whereas a value of 60 indicates that the individual's behaviour is severly abnormal.
In term of change from baseline, the greater the mean value decreases, the better it is. |
Change from baseline to Week 26 | |
Secondary | Social Responsiveness Scale, Second Edition (SRS-2) Total Raw Score | The SRS-2 total raw score serves as an index of severity of social deficits in the autism spectrum.
The total raw score ranges from 65 to 260. A value of 65 represents no symptoms disorders, a value of 260 represents a severe autism spectrum disorder. In terms of change from baseline, the greater the mean value decreases, the better it is. |
Change from baseline to Week 26 | |
Secondary | Clinical Global Impression - Global Improvement (CGI-I) Score | Scale which assesses the severity of the illness and the global improvement of the patient under study treatment.
It ranges from 1 (normal) through to 7 (amongst the most severely ill patients). |
At Week 26 | |
Secondary | Vineland Adaptative Behaviour Scale II (VABS II) | Scale designated to measure adaptative behaviour The scale for behaviour ranges from 1 to 67. The more the score decreases, the better it is. | Change from baseline to Week 26 | |
Secondary | Number of Patients With Abnormalities in 12-leads Electrocardiogram (ECG) Parameters | Number of patients with clinically significant ECG abnormalities
The 12-lead electrocardiogram (ECG) is a medical test that is recorded using leads, or nodes, attached to the body. Electrocardiograms (ECGs), capture the electrical activity of the heart and transfer it to graphed paper where abnormalities are reported and interpretated by the cardiologist. |
Week 26 | |
Secondary | Columbia-Suicide Severity Scale Children's Version (C-SSRS-C) | Number of patients with suicidal ideation or suicidal behavior. The scale is 0 to 5 with the highest suicidal behavior being a 5 and the absence of suicidal behavior or very minor behaviors are 0. However, statistical analysis was done by looking at the number of patients with suicidal behavior or suicidal ideation during their lifetime and during the last 6 months of treatment. | Week 26 | |
Secondary | Acceptability and Palatability Questionnaires - Only Descriptive Analyses | Acceptability and palatability criterion Based on your child's reactions (indirect rating), do you think that he/she found the administration method to be | Week 26 | |
Secondary | Paediatric Quality of Life Inventory (PedsQL) Questionnaire | It represents the assessment of parent/legal representative perception of patient health related quality of life The values of the questionnaire range from 0 to 100. Higher scores indicate better HRQOL (Health-Related Quality of Life) | Change from baseline to week 26 | |
Secondary | Number of Participants Experiencing at Least 1 Treatment Emergent Adverse Event (TEAE) | through week 52 |
Status | Clinical Trial | Phase | |
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