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
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ClinicalTrials.gov Identifier: NCT03715166 |
Recruitment Status :
Active, not recruiting
First Posted : October 23, 2018
Last Update Posted : January 20, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Autism Spectrum Disorder (ASD) | Drug: Bumetanide Oral Solution Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 211 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | 6-month, randomized, double-blind, placebo-controlled, parallel groups followed by an open label active 6-month treatment period with bumetanide. |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | 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. |
Actual Study Start Date : | September 24, 2018 |
Estimated Primary Completion Date : | December 8, 2021 |
Estimated Study Completion Date : | December 8, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Bumetanide/S95008 |
Drug: Bumetanide Oral Solution
Oral Solution dosed at 0.5mg/mL Taken twice daily |
Placebo Comparator: Placebo |
Drug: Placebo
Oral Solution Taken twice daily |
- Childhood Autism Rating Scale, Second Edition (CARS2) total raw score [ Time Frame: Change from baseline to 6 month ]Efficacy criterion The CARS2 is a 15 item rated instrument. The rating values given for the 15 areas are summed to produce a Total score.
- Social Responsiveness Scale, Second Edition (SRS-2) total raw score [ Time Frame: Change from baseline to 6 month ]Efficacy criterion 65 items scale measuring symptoms associated with autism
- Clinical Global Impression - Global Improvement (CGI-I) score [ Time Frame: At 6 months ]Efficacy criterion Scale which assesses the severity of the illness and the global improvement of the patient under study treatment
- Vineland Adaptative Behaviour Sales, Second Edition (VABS II) subscores [ Time Frame: Change from baseline to 6 month ]Efficacy criterion Scale designated to measure adaptative behaviour
- Adverse events and Paediatric Adverse Event Rating Scale (PAERS) [ Time Frame: On average of 52 weeks ]Safety criterion Inventory report used to identify signs/symptoms experienced by the patient since the study treatment initiation.
- Abnormalities in 12-leads electrocardiogram (ECG) parameters [ Time Frame: selection visit/Week004/Week008/Week012/Week 026/Week030/Week034/Week038/Week052 ]Safety criterion
- Renal ultrasound [ Time Frame: selection visit/Week026/Week052 ]Safety criterion Assessment of the renal function
- the Columbia Suicide Severity Rating Scale Children's version (C-SSRS-C) [ Time Frame: Week000/Week012/Week026/Week038/Week052 ]Scale which assesses suicidal ideation and suicidal behaviour
- Tanner stage [ Time Frame: Week000/Week026/Week052 ]Safety criterion Assessment of Pubertal development
- Acceptability and palatability questionnaire [ Time Frame: Week026 ]Acceptability and palatability criterion Assessment of the ease of use to use the dosing device
- Paediatric Quality of Life Inventory (PedsQL) questionnaire [ Time Frame: Week000/Week004/Week012/Week026/Week030/Week038/Week052 ]Quality of Life criterion Assessment of parent/legal representative perception of patient health related quality of life

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 7 Years to 17 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male and female patients from 7 to less than 18 years
- Out patients
- Primary diagnosis of ASD as per Diagnostic and Statistical Manual of Mental Disorders - Fifth Edition (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
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
- Severe electrolyte imbalance that is likely to interfere with the study conduct or evaluation

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03715166

Study Data/Documents: Individual Participant Data Set

Responsible Party: | Institut de Recherches Internationales Servier |
ClinicalTrials.gov Identifier: | NCT03715166 |
Other Study ID Numbers: |
CL3-95008-001 2017-004419-38 ( EudraCT Number ) |
First Posted: | October 23, 2018 Key Record Dates |
Last Update Posted: | January 20, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies:
In addition, access can be requested for all interventional clinical studies in patients:
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Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Informed Consent Form (ICF) Clinical Study Report (CSR) |
Time Frame: | After Marketing Authorisation in EEA or US if the study is used for the approval. |
Access Criteria: | Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed. |
URL: | https://clinicaltrials.servier.com/ |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Autism Spectrum Disorder Child Development Disorders, Pervasive Neurodevelopmental Disorders Mental Disorders Bumetanide Diuretics |
Natriuretic Agents Physiological Effects of Drugs Sodium Potassium Chloride Symporter Inhibitors Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |