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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
Sponsor:
Collaborator:
ADIR, a Servier Group company
Information provided by (Responsible Party):
Servier ( Institut de Recherches Internationales Servier )

Study Description
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Brief Summary:
The purpose of this study is to evaluate the efficacy and the safety of bumetanide/S95008 in the improvement of Autism Spectrum Disorder core symptoms.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder (ASD) Drug: Bumetanide Oral Solution Drug: Placebo Phase 3

Detailed Description:
The present study (CL3-95008-001) will be performed in children and adolescents from 7 to less than 18 years old presenting with ASD. A 6-month double-blind treatment period will be performed in which efficacy and safety of bumetanide 0.5mg BID will be assessed versus placebo. This double-blind period will be followed by a 6-month open label treatment period of bumetanide 0.5mg BID in which long term safety will be evaluated.
Study Design
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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

Resource links provided by the National Library of Medicine

Drug Information available for: Bumetanide

Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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

  2. 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

  3. 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

  4. 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.

  5. Abnormalities in 12-leads electrocardiogram (ECG) parameters [ Time Frame: selection visit/Week004/Week008/Week012/Week 026/Week030/Week034/Week038/Week052 ]
    Safety criterion

  6. Renal ultrasound [ Time Frame: selection visit/Week026/Week052 ]
    Safety criterion Assessment of the renal function

  7. 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

  8. Tanner stage [ Time Frame: Week000/Week026/Week052 ]
    Safety criterion Assessment of Pubertal development

  9. Acceptability and palatability questionnaire [ Time Frame: Week026 ]
    Acceptability and palatability criterion Assessment of the ease of use to use the dosing device

  10. 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


Eligibility Criteria
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Ages Eligible for Study:   7 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
Institut de Recherches Internationales Servier
ADIR, a Servier Group company
More Information
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Additional Information:

Study Data/Documents: Individual Participant Data Set  This link exits the ClinicalTrials.gov site
Study Protocol  This link exits the ClinicalTrials.gov site
Statistical Analysis Plan  This link exits the ClinicalTrials.gov site
Informed Consent Form  This link exits the ClinicalTrials.gov site
Clinical Study Report  This link exits the ClinicalTrials.gov site
study-level clinical trial data  This link exits the ClinicalTrials.gov site

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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:

  • used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
  • where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

  • sponsored by Servier
  • with a first patient enrolled as of 1 January 2004 onwards
  • for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.
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/

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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