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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03715166
Registration number
NCT03715166
Ethics application status
Date submitted
12/10/2018
Date registered
23/10/2018
Date last updated
29/03/2022
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
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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.
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Secondary ID [1]
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2017-004419-38
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Secondary ID [2]
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CL3-95008-001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Autism Spectrum Disorder (ASD)
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Condition category
Condition code
Mental Health
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Autistic spectrum disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Childhood Autism Rating Scale, Second Edition (CARS2) total raw score
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Assessment method [1]
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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.
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Timepoint [1]
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Change from baseline to 6 month
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Secondary outcome [1]
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Social Responsiveness Scale, Second Edition (SRS-2) total raw score
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Assessment method [1]
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Efficacy criterion 65 items scale measuring symptoms associated with autism
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Timepoint [1]
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Change from baseline to 6 month
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Secondary outcome [2]
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Clinical Global Impression - Global Improvement (CGI-I) score
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Assessment method [2]
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Efficacy criterion Scale which assesses the severity of the illness and the global improvement of the patient under study treatment
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Timepoint [2]
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At 6 months
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Secondary outcome [3]
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Vineland Adaptative Behaviour Sales, Second Edition (VABS II) subscores
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Assessment method [3]
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Efficacy criterion Scale designated to measure adaptative behaviour
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Timepoint [3]
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Change from baseline to 6 month
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Secondary outcome [4]
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Adverse events and Paediatric Adverse Event Rating Scale (PAERS)
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Assessment method [4]
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Safety criterion Inventory report used to identify signs/symptoms experienced by the patient since the study treatment initiation.
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Timepoint [4]
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On average of 52 weeks
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Secondary outcome [5]
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Abnormalities in 12-leads electrocardiogram (ECG) parameters
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Assessment method [5]
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Safety criterion
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Timepoint [5]
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selection visit/Week004/Week008/Week012/Week 026/Week030/Week034/Week038/Week052
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Secondary outcome [6]
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Renal ultrasound
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Assessment method [6]
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Safety criterion Assessment of the renal function
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Timepoint [6]
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selection visit/Week026/Week052
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Secondary outcome [7]
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the Columbia Suicide Severity Rating Scale Children's version (C-SSRS-C)
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Assessment method [7]
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Scale which assesses suicidal ideation and suicidal behaviour
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Timepoint [7]
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Week000/Week012/Week026/Week038/Week052
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Secondary outcome [8]
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Tanner stage
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Assessment method [8]
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Safety criterion Assessment of Pubertal development
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Timepoint [8]
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Week000/Week026/Week052
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Secondary outcome [9]
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Acceptability and palatability questionnaire
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Assessment method [9]
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Acceptability and palatability criterion Assessment of the ease of use to use the dosing device
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Timepoint [9]
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Week026
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Secondary outcome [10]
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Paediatric Quality of Life Inventory (PedsQL) questionnaire
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Assessment method [10]
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Quality of Life criterion Assessment of parent/legal representative perception of patient health related quality of life
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Timepoint [10]
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Week000/Week004/Week012/Week026/Week030/Week038/Week052
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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
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Minimum age
7
Years
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/09/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
13/09/2021
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Sample size
Target
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Accrual to date
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Final
211
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Brazil
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State/province [1]
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Curitiba
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Brazil
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State/province [2]
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Fortaleza
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Brazil
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State/province [3]
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Passo Fundo
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Country [4]
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Brazil
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São Paulo
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Country [5]
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France
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State/province [5]
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Alpes-Maritimes
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France
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Alsace-Champagne-Ardenne-Lorraine
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France
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Auvergne Rhone Alpes
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France
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Auvergne-Rhône-Alpes
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Country [9]
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France
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State/province [9]
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Il De France
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Country [10]
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France
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State/province [10]
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Normandie
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France
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Nouvelle Aquitaine
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France
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Nouvelle-Aquitaine
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Germany
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Baden-Württemberg
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Germany
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State/province [14]
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Saxony (Sachsen)
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Hungary
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Budapest
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Hungary
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Gyula
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Hungary
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Szeged
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Italy
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State/province [18]
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Campania
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Italy
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Lombardia
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Italy
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Sardegna
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Country [21]
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Italy
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State/province [21]
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Sicilia
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Italy
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State/province [22]
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Toscana
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Country [23]
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Netherlands
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Groningen
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Poland
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Wojewodztwo Lodzkie
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Poland
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Wojewodztwo Mazowieckie
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Poland
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Wojewodztwo Pomorskie
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Poland
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State/province [27]
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Gdansk
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Poland
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State/province [28]
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Warszawa
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Portugal
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Coimbra
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Spain
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State/province [30]
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Barcelona
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Country [31]
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Spain
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State/province [31]
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Guipuzcoa
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Spain
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Madrid
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Spain
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State/province [33]
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Alicante
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Country [34]
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United Kingdom
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State/province [34]
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Birmingham
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Country [35]
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United Kingdom
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London
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Country [36]
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United Kingdom
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State/province [36]
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Manchester
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Funding & Sponsors
Primary sponsor type
Other
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Name
Institut de Recherches Internationales Servier
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Address
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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ADIR, a Servier Group company
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Address [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03715166
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Address
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Phone
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Fax
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Email
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Contact person for public queries
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03715166
Download to PDF