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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03715166




Registration number
NCT03715166
Ethics application status
Date submitted
12/10/2018
Date registered
23/10/2018
Date last updated
25/07/2024

Titles & IDs
Public title
Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children and Adolescents Aged From 7 to Less Than 18 Years Old With Autism Spectrum Disorder
Scientific title
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.
Secondary ID [1] 0 0
2017-004419-38
Secondary ID [2] 0 0
CL3-95008-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autism Spectrum Disorder (ASD) 0 0
Condition category
Condition code
Mental Health 0 0 0 0
Autistic spectrum disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Bumetanide Oral Solution
Treatment: Drugs - Placebo

Experimental: Bumetanide/S95008 -

Placebo comparator: Placebo -


Treatment: Drugs: Bumetanide Oral Solution
Oral Solution dosed at 0.5mg/mL Taken twice daily

Treatment: Drugs: Placebo
Oral Solution Taken twice daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Childhood Autism Rating Scale, Second Edition (CARS2) total raw score
Timepoint [1] 0 0
Change from baseline to 6 month
Secondary outcome [1] 0 0
Social Responsiveness Scale, Second Edition (SRS-2) total raw score
Timepoint [1] 0 0
Change from baseline to 6 month
Secondary outcome [2] 0 0
Clinical Global Impression - Global Improvement (CGI-I) score
Timepoint [2] 0 0
At 6 months
Secondary outcome [3] 0 0
Vineland Adaptative Behaviour Sales, Second Edition (VABS II) subscores
Timepoint [3] 0 0
Change from baseline to 6 month
Secondary outcome [4] 0 0
Adverse events and Paediatric Adverse Event Rating Scale (PAERS)
Timepoint [4] 0 0
On average of 52 weeks
Secondary outcome [5] 0 0
Abnormalities in 12-leads electrocardiogram (ECG) parameters
Timepoint [5] 0 0
selection visit/Week004/Week008/Week012/Week 026/Week030/Week034/Week038/Week052
Secondary outcome [6] 0 0
Renal ultrasound
Timepoint [6] 0 0
selection visit/Week026/Week052
Secondary outcome [7] 0 0
the Columbia Suicide Severity Rating Scale Children's version (C-SSRS-C)
Timepoint [7] 0 0
Week000/Week012/Week026/Week038/Week052
Secondary outcome [8] 0 0
Tanner stage
Timepoint [8] 0 0
Week000/Week026/Week052
Secondary outcome [9] 0 0
Acceptability and palatability questionnaire
Timepoint [9] 0 0
Week026
Secondary outcome [10] 0 0
Paediatric Quality of Life Inventory (PedsQL) questionnaire
Timepoint [10] 0 0
Week000/Week004/Week012/Week026/Week030/Week038/Week052

Eligibility
Key inclusion criteria
* Male and female patients from 7 to less than 18 years
* Out patients
* 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 (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 total score (SRS-2 T-Score) = 66
* Absence of known monogenic syndrome (Fragile X, Rett syndrome ...)
* Absence of any clinically significant abnormality likely to interfere with the conduct of the study according to the judgment of the investigator
Minimum age
7 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/09/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/09/2021
Sample size
Target
Accrual to date
Final
211
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Brazil
State/province [1] 0 0
Curitiba
Country [2] 0 0
Brazil
State/province [2] 0 0
Fortaleza
Country [3] 0 0
Brazil
State/province [3] 0 0
Passo Fundo
Country [4] 0 0
Brazil
State/province [4] 0 0
São Paulo
Country [5] 0 0
France
State/province [5] 0 0
Alpes-Maritimes
Country [6] 0 0
France
State/province [6] 0 0
Alsace-Champagne-Ardenne-Lorraine
Country [7] 0 0
France
State/province [7] 0 0
Auvergne Rhone Alpes
Country [8] 0 0
France
State/province [8] 0 0
Auvergne-Rhône-Alpes
Country [9] 0 0
France
State/province [9] 0 0
Il De France
Country [10] 0 0
France
State/province [10] 0 0
Normandie
Country [11] 0 0
France
State/province [11] 0 0
Nouvelle Aquitaine
Country [12] 0 0
France
State/province [12] 0 0
Nouvelle-Aquitaine
Country [13] 0 0
Germany
State/province [13] 0 0
Baden-Württemberg
Country [14] 0 0
Germany
State/province [14] 0 0
Saxony (Sachsen)
Country [15] 0 0
Hungary
State/province [15] 0 0
Budapest
Country [16] 0 0
Hungary
State/province [16] 0 0
Gyula
Country [17] 0 0
Hungary
State/province [17] 0 0
Szeged
Country [18] 0 0
Italy
State/province [18] 0 0
Campania
Country [19] 0 0
Italy
State/province [19] 0 0
Lombardia
Country [20] 0 0
Italy
State/province [20] 0 0
Sardegna
Country [21] 0 0
Italy
State/province [21] 0 0
Sicilia
Country [22] 0 0
Italy
State/province [22] 0 0
Toscana
Country [23] 0 0
Netherlands
State/province [23] 0 0
Groningen
Country [24] 0 0
Poland
State/province [24] 0 0
Wojewodztwo Lodzkie
Country [25] 0 0
Poland
State/province [25] 0 0
Wojewodztwo Mazowieckie
Country [26] 0 0
Poland
State/province [26] 0 0
Wojewodztwo Pomorskie
Country [27] 0 0
Poland
State/province [27] 0 0
Gdansk
Country [28] 0 0
Poland
State/province [28] 0 0
Warszawa
Country [29] 0 0
Portugal
State/province [29] 0 0
Coimbra
Country [30] 0 0
Spain
State/province [30] 0 0
Barcelona
Country [31] 0 0
Spain
State/province [31] 0 0
Guipuzcoa
Country [32] 0 0
Spain
State/province [32] 0 0
Madrid
Country [33] 0 0
Spain
State/province [33] 0 0
Alicante
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Birmingham
Country [35] 0 0
United Kingdom
State/province [35] 0 0
London
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Other
Name
Institut de Recherches Internationales Servier
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
ADIR, a Servier Group company
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
After Marketing Authorisation in EEA or US if the study is used for the approval.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://clinicaltrials.servier.com/


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT03715166