A Study of Bumetanide for the Treatment of Autism Spectrum Disorders

Autism Spectrum Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03156153
• Other study ID #: XH-16-048
• Status: Completed
• Phase: Phase 2
• Start date: May 24, 2017
• Est. completion date: July 10, 2019

Study information

• Verified date: July 2019
• Source: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 120
• Est. completion date: July 10, 2019
• Est. primary completion date: July 8, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 6 Years

Eligibility

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.

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder
• Child Development Disorders, Pervasive

Intervention

Drug:
• Bumetanide
bumetanide tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm
• Placebo
placebo tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm

Locations

Country	Name	City	State
China	Xinhua Hospital	Shanghai	Shanghai

Sponsors (2)

Lead Sponsor	Collaborator
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine	Shanghai Jiao Tong University School of Medicine

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	MRI Structure Imaging and Spectrum imaging, data analysis	Change of structure of the brain and the GABA-neurotransmitters in specific brain regions of the brain.	Day 0 and Day 90
Other	Multichannel EEG Signals	Change in brain Multichannel EEG signals	Day 0 and Day 90
Other	Genome wide association study from blood sample	Selection and study of susceptible genes	Day 90
Other	Analysis of metabolites from blood sample	Metabonomics research	Day 0 and Day 90
Primary	Childhood Autism Rating Scale(CARS)	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	Clinical Global Impressions Scale (CGI)	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
Secondary	Social Responsiveness Scale (SRS)	SRS is a questionnaire used to assess the presence and severity of social impairment.	Day 0 and Day 90
Secondary	Autism Diagnostic Observation Schedule (ADOS)	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
Secondary	Short Sensory Profile Report	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
Secondary	Symbolic Play Test	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
Secondary	Chinese Communicative Development Inventory	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