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.

Phase 3

Withdrawn

Conditions: autism spectrum disorder
10010118

Registration Number: NL-OMON45966

Lead Sponsor: Servier R&D Benelux

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Withdrawn

Sex: Not specified

Target Recruitment: 4

Inclusion Criteria

- Male and female patients from 2 to less than 7 years
- Out patients
- Primary diagnosis of ASD as per 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
-Absence of electrolyte imbalance that is likely to interfere with the study conduct or evaluation

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

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Childhood Autism Rating Scale, Second Edition (CARS2) total raw score. Main<br /><br>expression will be change from baseline to 6 month.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>To assess the effect of bumetanide on the other efficacy endpoints<br /><br>To assess the safety of bumetanide<br /><br>To confirm the acceptability and palatability of the oral liquid formulation<br /><br>To describe the bumetanide effects on patients quality of life<br /><br>To improve existing pharmacokinetic model of bumetanide in this population.</p><br>