Efficacy and safety of bumetanide oral liquid formulation in children aged from 2 to less than 7 years old with Autism Spectrum Disorder.

Phase 1

• Conditions: Autism Spectrum Disorder (ASD); MedDRA version: 21.1Level: PTClassification code 10063844Term: Autism spectrum disorderSystem Organ Class: 10037175 - Psychiatric disorders; Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]

• Registration Number: EUCTR2017-004420-30-CZ
• Lead Sponsor: Institut de Recherches Internationales Servier

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-12-03
• Last Update Posted: 4 January 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 200

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 200
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To demonstrate the superiority of bumetanide (0.5mg BID) oral liquid formulation compared to placebo in the improvement of ASD core symptoms after 6 months of treatment in ASD children aged from 2 to less than 7 years old;Secondary Objective: - To assess the effect of bumetanide on the other efficacy endpoints<br>- To assess the safety of bumetanide<br>- To confirm the acceptability and palatability of the oral liquid formulation<br>- To describe the bumetanide effects on patients quality of life<br>- To improve existing pharmacokinetic model of bumetanide in this population<br>;Primary end point(s): CARS2 total raw score;Timepoint(s) of evaluation of this end point: Change from baseline to 6 month