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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03715153




Registration number
NCT03715153
Ethics application status
Date submitted
12/10/2018
Date registered
23/10/2018

Titles & IDs
Public title
Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.
Scientific 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.
Secondary ID [1] 0 0
2017-004420-30
Secondary ID [2] 0 0
CL3-95008-002
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
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BUMETANIDE (S95008) for week 0 - 26
Treatment: Drugs - PLACEBO for week 0 - 26
Treatment: Drugs - Open-Label BUMETANIDE (S95008) for weeks 26 - 52

Experimental: BUMETANIDE (S95008) followed by Open-Label S95008 - Participants will receive S95008 for 6 months, 26 weeks, and then they will begin an open-label 6 month treatment period with S95008.

Placebo comparator: PLACEBO followed by Open-Label S95008 - Participants will receive placebo for 6 months, 26 weeks, and then they will begin an open-label 6 month treatment period with S95008.


Treatment: Drugs: BUMETANIDE (S95008) for week 0 - 26
Oral solution dosed at 0.5 mg/mL Taken twice daily.

Treatment: Drugs: PLACEBO for week 0 - 26
Oral solution Taken twice daily.

Treatment: Drugs: Open-Label BUMETANIDE (S95008) for weeks 26 - 52
Oral solution dosed at 0.5 mg/mL 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 Week 26
Secondary outcome [1] 0 0
Social Responsiveness Scale, Second Edition (SRS-2) Total Raw Score
Timepoint [1] 0 0
Change from baseline to Week 26
Secondary outcome [2] 0 0
Clinical Global Impression - Global Improvement (CGI-I) Score
Timepoint [2] 0 0
At Week 26
Secondary outcome [3] 0 0
Vineland Adaptative Behaviour Scale II (VABS II)
Timepoint [3] 0 0
Change from baseline to Week 26
Secondary outcome [4] 0 0
Number of Patients With Abnormalities in 12-leads Electrocardiogram (ECG) Parameters
Timepoint [4] 0 0
Week 26
Secondary outcome [5] 0 0
Columbia-Suicide Severity Scale Children's Version (C-SSRS-C)
Timepoint [5] 0 0
Week 26
Secondary outcome [6] 0 0
Acceptability and Palatability Questionnaires - Only Descriptive Analyses
Timepoint [6] 0 0
Week 26
Secondary outcome [7] 0 0
Paediatric Quality of Life Inventory (PedsQL) Questionnaire
Timepoint [7] 0 0
Change from baseline to week 26
Secondary outcome [8] 0 0
Number of Participants Experiencing at Least 1 Treatment Emergent Adverse Event (TEAE)
Timepoint [8] 0 0
through week 52

Eligibility
Key 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.
Minimum age
2 Years
Maximum age
6 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
4/10/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/10/2021
Sample size
Target
Accrual to date
Final
211
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [2] 0 0
The Royal Children's Hospital Melbourne - Parkville
Recruitment postcode(s) [1] 0 0
2170 - Liverpool
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
New York
Country [2] 0 0
Brazil
State/province [2] 0 0
Curitiba
Country [3] 0 0
Brazil
State/province [3] 0 0
Fortaleza
Country [4] 0 0
Brazil
State/province [4] 0 0
Joinville
Country [5] 0 0
Brazil
State/province [5] 0 0
Passo Fundo
Country [6] 0 0
Brazil
State/province [6] 0 0
São Paulo
Country [7] 0 0
Czechia
State/province [7] 0 0
Ostrava
Country [8] 0 0
Czechia
State/province [8] 0 0
Brno
Country [9] 0 0
Czechia
State/province [9] 0 0
Praha
Country [10] 0 0
France
State/province [10] 0 0
Alpes-Maritimes
Country [11] 0 0
France
State/province [11] 0 0
Alsace-Champagne-Ardenne-Lorraine
Country [12] 0 0
France
State/province [12] 0 0
Auvergne Rhone Alpes
Country [13] 0 0
France
State/province [13] 0 0
Auvergne-Rhône-Alpes
Country [14] 0 0
France
State/province [14] 0 0
Ile De France
Country [15] 0 0
France
State/province [15] 0 0
Normandie
Country [16] 0 0
France
State/province [16] 0 0
Nouvelle Aquitaine
Country [17] 0 0
France
State/province [17] 0 0
Nouvelle-Aquitaine
Country [18] 0 0
Hungary
State/province [18] 0 0
Budapest
Country [19] 0 0
Hungary
State/province [19] 0 0
Gyula
Country [20] 0 0
Hungary
State/province [20] 0 0
Szeged
Country [21] 0 0
Italy
State/province [21] 0 0
Lombardia
Country [22] 0 0
Italy
State/province [22] 0 0
Sardegna
Country [23] 0 0
Italy
State/province [23] 0 0
Sicilia
Country [24] 0 0
Italy
State/province [24] 0 0
Toscana
Country [25] 0 0
Poland
State/province [25] 0 0
Gdansk
Country [26] 0 0
Poland
State/province [26] 0 0
Lodz
Country [27] 0 0
Poland
State/province [27] 0 0
Warszawa
Country [28] 0 0
Portugal
State/province [28] 0 0
Coimbra
Country [29] 0 0
Slovakia
State/province [29] 0 0
Bratislava
Country [30] 0 0
Slovakia
State/province [30] 0 0
Košice
Country [31] 0 0
Spain
State/province [31] 0 0
Barcelona
Country [32] 0 0
Spain
State/province [32] 0 0
Guipuzcoa
Country [33] 0 0
Spain
State/province [33] 0 0
Madrid
Country [34] 0 0
Spain
State/province [34] 0 0
Alicante
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Colchester
Country [36] 0 0
United Kingdom
State/province [36] 0 0
London
Country [37] 0 0
United Kingdom
State/province [37] 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?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03715153