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-HU

Enrollment

200

Registered

2018-07-17

Start date

2018-09-05

Completion date

Unknown

Last updated

2021-11-15

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: 21.1 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: S95008 Other descriptive name: BUMETANIDE Concentration unit: mg/ml m

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 - 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
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 ;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;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

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 ;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;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

Australia, Belgium, Brazil, Czech Republic, France, Germany, Hungary, Ireland, Italy, Netherlands, Poland, Portugal, Slovakia, Spain, United Kingdom, United States

Contacts

Public ContactClinical Studies Department

Institut de Recherches Internationales Servier

clinicaltrials@servier.com+33155 72 43 66

Outcome results

None listed

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