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

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.

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-004420-30-NL

Enrollment

200

Registered

2018-06-20

Start date

2019-02-04

Completion date

Unknown

Last updated

2019-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autism Spectrum Disorder (ASD) MedDRA version: 20.0 Level: PT Classification code 10063844 Term: Autism spectrum disorder System Organ Class: 10037175 - Psychiatric disorders

Interventions

Product Name: S95008 Product Code: S95008 Pharmaceutical Form: Oral solution INN or Proposed INN: Bumetanide Current Sponsor code: S9500

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
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 - To assess the safety of bumetanide - To confirm the acceptability and palatability of the oral liquid formulation - To describe the bumetanide effects on patients quality of life - 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	—

Measure

Time frame

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 - To assess the safety of bumetanide - To confirm the acceptability and palatability of the oral liquid formulation - To describe the bumetanide effects on patients quality of life - 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

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Secondary

Measure	Time frame
Secondary end point(s): - Each individual CARS2 domain, SRS-2 total raw score, CGI-I score, VABS II subscores - Adverse events, Paediatric Adverse Event Rating Scale (PAERS), clinical laboratory evaluation, vital signs and clinical examination, electrocardiogram, renal ultrasound, Columbia Suicide Severity Rating Scale Children's version (C-SSRS-C) - Acceptability and palatability questionnaire - Paediatric Quality of Life Inventory (PedsQL), WHOQOL-Bref questionnaire - PK parameters of bumetanide ; Timepoint(s) of evaluation of this end point: SRS-2 total raw score: W000/W004/W012/W026/W038/W052 CGI-I score: W000+Day17/W004/W008/W012/W016/W020/W026/W038/W052/WEND VABS II subscores: W000/W026/W052 Each individual CARS2 domain: W000/W004/W012/W026/W038/W052/WEND Adverse events/PAERS/Clinical laboratory evaluation/Electrolytes monitoring (sodium, potassium)/Clinical examination: all along the study Vital signs: ASSE/W000/W00+D17/W012/W026/W026+Day17/W038/W052 Electrocardiogram: ASSE/W004/W008/W012, W026/W030/W034/W038/W052 Renal ultrasound: ASSE/W026/W052 C-SSRS-C: W000/W012/W026/W038/W052 Acceptability and palatability questionnaire: W026 PedsQL / WHOQOL-Bref questionnaire: W000/W004/W012/W026/W030/W038/W052 + W008/W034 for WHOQOL-Bref questionnaire PK parameters of bumetanide: W012/W026	—

Measure

Time frame

Secondary end point(s): - Each individual CARS2 domain, SRS-2 total raw score, CGI-I score, VABS II subscores - Adverse events, Paediatric Adverse Event Rating Scale (PAERS), clinical laboratory evaluation, vital signs and clinical examination, electrocardiogram, renal ultrasound, Columbia Suicide Severity Rating Scale Children's version (C-SSRS-C) - Acceptability and palatability questionnaire - Paediatric Quality of Life Inventory (PedsQL), WHOQOL-Bref questionnaire - PK parameters of bumetanide ; Timepoint(s) of evaluation of this end point: SRS-2 total raw score: W000/W004/W012/W026/W038/W052 CGI-I score: W000+Day17/W004/W008/W012/W016/W020/W026/W038/W052/WEND VABS II subscores: W000/W026/W052 Each individual CARS2 domain: W000/W004/W012/W026/W038/W052/WEND Adverse events/PAERS/Clinical laboratory evaluation/Electrolytes monitoring (sodium, potassium)/Clinical examination: all along the study Vital signs: ASSE/W000/W00+D17/W012/W026/W026+Day17/W038/W052 Electrocardiogram: ASSE/W004/W008/W012, W026/W030/W034/W038/W052 Renal ultrasound: ASSE/W026/W052 C-SSRS-C: W000/W012/W026/W038/W052 Acceptability and palatability questionnaire: W026 PedsQL / WHOQOL-Bref questionnaire: W000/W004/W012/W026/W030/W038/W052 + W008/W034 for WHOQOL-Bref questionnaire PK parameters of bumetanide: W012/W026

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Countries

Belgium, France, Germany, Hungary, Ireland, Italy, Netherlands, Poland, Portugal, Spain, United Kingdom

Contacts

Public ContactClinical Studies Department

Institut de Recherches Internationales Servier

clinicaltrials@servier.com+33155 72 70 63

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026