A Study of Bumetanide for the Treatment of Autism Spectrum Disorders

A Study of Bumetanide for the Treatment of Children With Autism Spectrum Disorder:a Randomized Double-blind Placebo-controlled Trial

The investigators are going to carry out a randomized double-blind placebo-controlled trial to study the efficiency and mechanism of bumetanide on the treatment of children with Autism Spectrum Disorder.

Study Overview

Status

Completed

Intervention / Treatment

Detailed Description

In consideration of the increasing number of autistic children and poor intervention effect in China, it is an urgent to find some effective medicine. Some studies have reported bumetanide, a classic diuretic, could improve autistic behaviors in both animal model and humans; while the efficiency of bumetanide on Chinese autistic chilren is unkonwn and the underlying mechanisms remain unfolding. The investigators aim at investigating whether bumetanide would improve the clinical symptoms in Chinese children with autism within a safe dosage and further study the physiological mechanism beneath.The investigators will regularly assess the participants' autism-related symptoms during medication, as well as the adverse effects of each patient. The investigators will carry out genome-wide association analysis (GWAS) from blood sample, related metabolites in nervous system and compare the concentration of the neurotransmitter in autistic brain before and after 3 months' treatmeat,and also will collect the EEG signal in autistic chilldren when the participants performing certain tasks before and after 3 months' treatment.

Study Type

Interventional

Enrollment (Actual)

120

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Shanghai
      • Shanghai, Shanghai, China, 200000
        • Xinhua Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 6 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

The patients, aged from 3 to 6 years old, were given the diagnosis of ASD according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V) , by a team of autism experts; Scores for Children Autism Rating Scale (CARS) were more than 30; Signed Informed Consents were provided by parents.

Exclusion Criteria:

Liver and kidney dysfunction; With a history of allergy to sulfa drugs; abnormal ECG; chromosomal abnormality; suffering from nervous system diseases (such as epilepsy, schizophrenia, and so on); using the melatonin treatment for sleep disorders or withdrawal less than three weeks.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Bumetanide group
Double-blind phase: in the first 3 months, patients will receive the experimental treatment - bumetanide, oral intake, 0.5mg/time, twice a day; Open-label phase: after 3 month double-blined treatment, all the patients will receive 3-month bumetanide treatment - oral intake, 0.5mg/time, twice a day.
bumetanide tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm
Placebo Comparator: Control group
Double-blind phase: in the first 3 months, patients will receive the placebo - oral intake, 0.5mg/time, twice a day; Open-label phase: after 3 month double-blined treatment, patients in this group will receive 3-month bumetanide treatment - oral intake, 0.5mg/time, twice a day.
placebo tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Childhood Autism Rating Scale(CARS)
Time Frame: Day 0 and Day 90
CARS is a behaviour-rating scale used to assess the presence and severity of the symptoms of autism spectrum disorder
Day 0 and Day 90

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical Global Impressions Scale (CGI)
Time Frame: Day 0 and Day 90
CGI is a scale used to assess the severity of the illness and the global improvement of the patient under intervention
Day 0 and Day 90
Social Responsiveness Scale (SRS)
Time Frame: Day 0 and Day 90
SRS is a questionnaire used to assess the presence and severity of social impairment.
Day 0 and Day 90
Autism Diagnostic Observation Schedule (ADOS)
Time Frame: Day 0 and Day 90
The ADOS is a structured play session conducted by clinicians, with the total score combines the Social and Communication domain items.
Day 0 and Day 90
Short Sensory Profile Report
Time Frame: Day 0 and Day 90
The Sensory Profile is a measure of children's responses to sensory events in daily life. The caregiver completes the Sensory Profile by assessing the frequency of the child's responses to certain sensory processing, modulation, and behavioral/emotional events as described in the 125 items.
Day 0 and Day 90
Symbolic Play Test
Time Frame: Day 0 and Day 90
Symbolic Play Test is a nonverbal measure of symbolic functioning in participants aged 12-36 months. The test does not require any expressive speech, and is therefore appropriate for use with all participants with ASD. Participants are sequentially presented with four sets of toys, and their spontaneous manipulation of the objects is observed and recorded on a standardized checklist.
Day 0 and Day 90
Chinese Communicative Development Inventory
Time Frame: Day 0 and Day 90
The Chinese Communicative Development Inventories (CCDI) is a questionnaire that is used to direct measures of the participants' language.Change between day 0 and day 90 of the result of the Chinese Communicative Development Inventory
Day 0 and Day 90

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
MRI Structure Imaging and Spectrum imaging, data analysis
Time Frame: Day 0 and Day 90
Change of structure of the brain and the GABA-neurotransmitters in specific brain regions of the brain.
Day 0 and Day 90
Multichannel EEG Signals
Time Frame: Day 0 and Day 90
Change in brain Multichannel EEG signals
Day 0 and Day 90
Genome wide association study from blood sample
Time Frame: Day 90
Selection and study of susceptible genes
Day 90
Analysis of metabolites from blood sample
Time Frame: Day 0 and Day 90
Metabonomics research
Day 0 and Day 90

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Fei Li, doctor, Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 24, 2017

Primary Completion (Actual)

July 8, 2019

Study Completion (Actual)

July 10, 2019

Study Registration Dates

First Submitted

May 15, 2017

First Submitted That Met QC Criteria

May 15, 2017

First Posted (Actual)

May 17, 2017

Study Record Updates

Last Update Posted (Actual)

March 1, 2021

Last Update Submitted That Met QC Criteria

February 25, 2021

Last Verified

July 1, 2019

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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