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Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.

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ClinicalTrials.gov Identifier: NCT03715153
Recruitment Status : Recruiting
First Posted : October 23, 2018
Last Update Posted : January 18, 2019
Sponsor:
Collaborator:
ADIR, a Servier Group company
Information provided by (Responsible Party):
Servier ( Institut de Recherches Internationales Servier )

Brief Summary:
The purpose of this study is to evaluate the efficacity 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/S95008 Drug: PLACEBO Phase 3

Detailed Description:
The present study (CL3-95008-002) will be performed in children from 2 to less than 7 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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 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: 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.
Actual Study Start Date : October 4, 2018
Estimated Primary Completion Date : April 2021
Estimated Study Completion Date : April 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Bumetanide

Arm Intervention/treatment
Experimental: BUMETANIDE/S95008 Drug: BUMETANIDE/S95008
Oral solution dosed at 0.5 mg/mL Taken twice daily.

Placebo Comparator: PLACEBO Drug: PLACEBO
Oral solution Taken twice daily.




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 Scale II (VABS II) [ Time Frame: Change from baseline to 6 month ]
    Efficacy criterion Scale designated to measure adaptative behaviour

  4. Adverse Event and Pediatric 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, Week026, Week030, Week034, Week038, Week052 ]
    Safety criterion

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

  7. Columbia-Suicide severity scale Children's version (C-SSRS-C) [ Time Frame: Week000/Week012/Week026/Week038/Week052 ]
    Scale which assesses suicidal ideation and suicidal behaviour

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

  9. Paediatric Quality of Life Inventory (PedsQL) questionnaire [ Time Frame: Week000/Week004/Week012/v026/Week030/Week038/Week052 ]
    Quality of Life questionnaire Assessment of parent/legal representative perception of patient health related quality of life



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Ages Eligible for Study:   2 Years to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female patients from 2 to less than 7
  • 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-Generic (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 (SRS-2) total score ≥ 66 T-Score
  • Absence of diagnosis of Fragile X or 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

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): NCT03715153


Contacts
Contact: Institut de Recherches Internationales Servier Clinical Studies Department +33 1 55 72 43 66 clinicaltrials@servier.com

  Show 41 Study Locations
Sponsors and Collaborators
Institut de Recherches Internationales Servier
ADIR, a Servier Group company

Additional Information:
Study Data/Documents: Individual Participant Data Set  This link exits the ClinicalTrials.gov site

Responsible Party: Institut de Recherches Internationales Servier
ClinicalTrials.gov Identifier: NCT03715153     History of Changes
Other Study ID Numbers: CL3-95008-002
2017-004420-30 ( EudraCT Number )
First Posted: October 23, 2018    Key Record Dates
Last Update Posted: January 18, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Researchers can ask for a study protocol, patient-level and/or study-level clinical trial data including clinical study reports (CSRs).

They can ask for all interventional clinical studies:

  • submitted for new medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
  • Where Servier or an affiliate are the Marketing Authorization Holders (MAH). The date of the first Marketing Authorization of the new médicine (or the new indication) in one of the EEA Member States will be considered within this scope.
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: http://clinicaltrials.servier.com/

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
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