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.
- Conditions
- Autism Spectrum Disorder (ASD)MedDRA version: 20.0Level: PTClassification code 10063844Term: Autism spectrum disorderSystem Organ Class: 10037175 - Psychiatric disordersTherapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]
- Registration Number
- EUCTR2017-004419-38-ES
- Lead Sponsor
- Institut de Recherches Internationales Servier
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 211
- Male and female patients from 7 to less than 18 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
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
- 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
- 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 and adolescents aged from 7 to less than 18 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;Primary end point(s): CARS2 total raw score;Timepoint(s) of evaluation of this end point: Change from baseline to 6 month
- Secondary Outcome Measures
Name Time Method