Study Stopped
The 6-month efficacy analysis did not show any significant difference between bumetanide versus placebo in the treatment of ASD in the overall studied population. No unexpected safety concerns were identified.
Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.
2 other identifiers
interventional
211
12 countries
50
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Oct 2018
Typical duration for phase_3
50 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 4, 2018
CompletedFirst Submitted
Initial submission to the registry
October 12, 2018
CompletedFirst Posted
Study publicly available on registry
October 23, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 26, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
October 26, 2021
CompletedResults Posted
Study results publicly available
June 5, 2023
CompletedJune 5, 2023
May 1, 2023
3.1 years
October 12, 2018
October 26, 2022
May 9, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (8)
Social Responsiveness Scale, Second Edition (SRS-2) Total Raw Score
Change from baseline to Week 26
Clinical Global Impression - Global Improvement (CGI-I) Score
At Week 26
Vineland Adaptative Behaviour Scale II (VABS II)
Change from baseline to Week 26
Number of Patients With Abnormalities in 12-leads Electrocardiogram (ECG) Parameters
Week 26
Columbia-Suicide Severity Scale Children's Version (C-SSRS-C)
Week 26
- +3 more secondary outcomes
Study Arms (2)
BUMETANIDE (S95008) followed by Open-Label S95008
EXPERIMENTALParticipants will receive S95008 for 6 months, 26 weeks, and then they will begin an open-label 6 month treatment period with S95008.
PLACEBO followed by Open-Label S95008
PLACEBO COMPARATORParticipants will receive placebo for 6 months, 26 weeks, and then they will begin an open-label 6 month treatment period with S95008.
Interventions
Oral solution dosed at 0.5 mg/mL Taken twice daily.
Oral solution dosed at 0.5 mg/mL Taken twice daily.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (50)
Richmond Behavioral Associates
Staten Island, New York, 10312, United States
Liverpool Hospital
Liverpool, 2170, Australia
The Royal Children's Hospital Melbourne
Parkville, 3052, Australia
Trial Tech em Pesquisas com Medicamentos Ltda
Curitiba, 80240-280, Brazil
Hospital Universitário Walter Cantídio-Universidade Federal do Ceará
Fortaleza, 60430-370, Brazil
Clínica Neurológica e Neurocirúrgica de Joinville
Joinville, 89202-190, Brazil
Hospital São Vicente de Paulo
Passo Fundo, 99010-080, Brazil
Universidade Federal de São Paulo, Escola Paulista de Medicina
São Paulo, 04017-030, Brazil
Faculdade de Medicina da Universidade de São Paulo - Departamento de psiquiatra
São Paulo, 054030-010, Brazil
University hospital of Ostrava, Department of Psychiatry
Poruba, Ostrava, Czechia
University Hospital Brno, Department of Child Neurology
Brno, 613 00, Czechia
Institute of Neuropsychiatric Care, Department of Child Psychiatry
Prague, 186 00, Czechia
GSC CHU-LENVAL Centre ressource autisme
Nice, Alpes-Maritimes, 6200, France
Centre d'Investigation Clinique de Lyon
Bron, Auvergne-Rhône-Alpes, 69677, France
Hôpital Le Vinatier CRA Rhône-Alpes, Bat 211
Bron, Auvergne-Rhône-Alpes, 69678, France
Hôpitaux Universitaires de Strasbourg Service de Psychiatrie de l'Enfant et de l'Adolescent
Strasbourg, Grand Est, 67091, France
CHU Rouen
Rouen, Normandy, 76000, France
Centre Hospitalier du Rouvray Centre de Ressources pour l'Autisme
Sotteville-lès-Rouen, Normandy, 76301, France
Centre Hospitalier Charles Perrens CRA Aquitaine
Bordeaux, Nouvelle-Aquitaine, 33076, France
Hôpital des Enfants-Pellegrin
Bordeaux, Nouvelle-Aquitaine, 33076, France
Hôpital Robert Debré Service de Psychiatrie de l'enfant et de l'adolescent
Paris, Île-de-France Region, 75019, France
Vadaskert Korhaz es Szakambulancia
Budapest, 1021, Hungary
Servus Salvus Kft.
Budapest, 1026, Hungary
Magyar Református Egyház Bethesda Gyermekkórháza
Budapest, 1143, Hungary
Bekes Megyei Kozponti Korhaz Gyermek es ifjusagpszichiatriai osztaly
Gyula, 5700, Hungary
Szegedi Tudományegyetem Szent-Gyorgy Albert Klinikai Kozpont Gyermekgyógyászati Klinika Gyermek es Ifjusagpszichiátriai o
Szeged, 6725, Hungary
Neuro Riabilitazione/Psicopatologia dell'età evolutiva Istituto Scientifico Medea - Bosisio Parini
Bosisio Parini, Lombardy, 23842, Italy
U.O. di Neuropsichiatria Infantile Fondazione Istituto Neurologico Nazionale Casimiro Mondino Istituto di Ricovero e Cura a Carattere Scientifico
Pavia, Lombardy, 27100, Italy
Clinica di Neuropsichiatria dell'Infanzia e dell'Adolescenza Ospedale Pediatrico-Microcitemico
Cagliari, Sardinia, 09131, Italy
Programma Interdipartimentale "Autismo 0-90" A.O.U. Policlinico "Gaetano Martino"
Messina, Sicily, 98125, Italy
U.O.C. Psichiatria dello Sviluppo IRCCS Fondazione Stella Maris
Calambrone, Tuscany, 56128, Italy
U.O. di Neuropsichiatria Infantile Azienda Ospedaliera Universitaria Senese
Siena, Tuscany, 53100, Italy
"Niepubliczny Zakład Opieki Zdrowotnej Gdańskie Centrum Zdrowia Sp. z o.o.
Gdansk, 80-542, Poland
Centrum Badań Klinicznych PI-House sp. z o.o
Gdansk, 80-546, Poland
NAVICULA Centrum Diagnozy i Terapii Autyzmu w Łodzi
Lodz, 91-126, Poland
Fundacja SYNAPSIS ul.
Warsaw, 02-085, Poland
Samodzielny Publiczny Dziecięcy Szpital Kliniczny w Warszawie Oddział Kliniczny Psychiatrii Wieku Rozwojowego
Warsaw, 02-091, Poland
Centro Hospitalar e Universitario de Coimbra Hospital Pediatrico
Coimbra, 300-062, Portugal
National Institute of Children Diseases, Department of Child Psychiatry
Bratislava, 833 40, Slovakia
EPAMED, s.r.o.
Košice, 040 01, Slovakia
Hospita Mutua de Terrassa
Terrassa, Barcelona, 08221, Spain
Policlinica Guipuzcoa
Donostia / San Sebastian, Guipuzcoa, 20014, Spain
Hospital Puerta de Hierro
Majadahonda, Madrid, 28222, Spain
Hospital General Universitario de Alicante
Alicante, 03007, Spain
Hospital Clinic de Barcelona
Barcelona, 08036, Spain
Hospital Niño Jesus
Madrid, 28009, Spain
Hospital Universitario Gregorio Marañon
Madrid, 28009, Spain
Colchester Hospital
Colchester, CO4 5JL, United Kingdom
ReCognition Health
London, W1G 9JF, United Kingdom
The Winnicott Centre 195-197 Hathersage Road
Manchester, M13 0JE, United Kingdom
Related Publications (2)
Hawken N, Falissard B, Choquet C, Francois C, Tardu J, Schmid R. Exit interviews from two randomised placebo-controlled phase 3 studies with caregivers of young children with autism spectrum disorder. Front Child Adolesc Psychiatry. 2024 Jun 5;3:1236340. doi: 10.3389/frcha.2024.1236340. eCollection 2024.
PMID: 39816573DERIVEDFuentes J, Parellada M, Georgoula C, Oliveira G, Marret S, Crutel V, Albarran C, Lambert E, Penelaud PF, Ravel D, Ben Ari Y. Bumetanide oral solution for the treatment of children and adolescents with autism spectrum disorder: Results from two randomized phase III studies. Autism Res. 2023 Oct;16(10):2021-2034. doi: 10.1002/aur.3005. Epub 2023 Oct 4.
PMID: 37794745DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
The sponsor decided to stop the S95008 development and prematurely discontinue the extension period. This decision was not related to unexpected safety concerns.
Results Point of Contact
- Title
- Dr. Pierre-François PENELAUD
- Organization
- Institut de Recherches Internationales Servier (I.R.I.S.)
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 12, 2018
First Posted
October 23, 2018
Study Start
October 4, 2018
Primary Completion
October 26, 2021
Study Completion
October 26, 2021
Last Updated
June 5, 2023
Results First Posted
June 5, 2023
Record last verified: 2023-05
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Time Frame
- After Marketing Authorisation in EEA or US if the study is used for the approval.
- Access Criteria
- Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: * sponsored by Servier * with a first patient enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.