Efficacy and safety of bumetanide in children with autism aged 7-17 .

• Research type

  Research Study

• Full title

  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.

• IRAS ID

  243977

• Contact name

  Latha Hackett

• Contact email

  Latha.Hackett@mft.nhs.uk

• Sponsor organisation

  Servier Research & Development Ltd

• Eudract number

  2017-004419-38

• ISRCTN Number

  ISRCTN00000000

• Clinicaltrials.gov Identifier

  NCT00000000

• Clinicaltrials.gov Identifier

  NA, NA

• Duration of Study in the UK

  2 years, 11 months, 8 days

• Research summary

  Research Summary

  For children and adults with Autism Spectrum Disorder (ASD) it is thought there is a difference in how the brain cells (neurons) have developed and process information. One theory is that the level of ‘chloride’ in the neurons is too high and this affects how they are stimulated and build connections.

  A preceding trial in 88 children, using bumetanide, which lowers chloride levels in the kidney and brain, has shown interesting results. In this trial children with moderate to severe ASD were treated for 3 months with 3 different doses of bumetanide or placebo. A recognised tool called the Childhood Autism Rating Scale (CARS), which assesses different aspects of ASD, showed significant improvements in children who received bumetanide and who completed the trial. Another tool called the Social Responsiveness Scale (SRS) also showed significant improvements, the most common being in social communication and restricted interest. Side effects typical of bumetanide, such as a drop in levels of potassium were seen, especially in the high dose 2.0mg bumetanide group.

  The main aims of this new trial are to see if a twice daily dose of 0.5mg bumetanide can improve core symptoms of ASD and also to assess the safety of 0.5mg bumetanide.

  Children with moderate to severe ASD will be treated for 12 months. For the first 6 months half will receive bumetanide and half will receive placebo. In the second 6 months all children will receive bumetanide. The primary assessment will be the change in CARS score at month 6.

  Summary of Results

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• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  18/NE/0169

• Date of REC Opinion

  2 Oct 2018

• REC opinion

  Further Information Favourable Opinion