Clinical trial NCT03156153: A Study of Bumetanide for the Treatment of Autism Spectrum Disorders
Official title: A Study of Bumetanide for the Treatment of Children With Autism Spectrum Disorder：a Randomized Double-blind Placebo-controlled Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Lead sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
Collaborators: name: Shanghai Jiao Tong University School of Medicine
First posted: 2017-05-17
Enrollment: 120
Sex: ALL
Minimum age (years): 3
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Childhood Autism Rating Scale(CARS)
timeFrame: Day 0 and Day 90
Secondary outcomes:
measure: Clinical Global Impressions Scale (CGI)
timeFrame: Day 0 and Day 90
measure: Social Responsiveness Scale (SRS)
timeFrame: Day 0 and Day 90
measure: Autism Diagnostic Observation Schedule (ADOS)
timeFrame: Day 0 and Day 90
measure: Short Sensory Profile Report
timeFrame: Day 0 and Day 90
measure: Symbolic Play Test
timeFrame: Day 0 and Day 90
measure: Chinese Communicative Development Inventory
timeFrame: Day 0 and Day 90
Other outcomes:
measure: MRI Structure Imaging and Spectrum imaging, data analysis
timeFrame: Day 0 and Day 90
measure: Multichannel EEG Signals
timeFrame: Day 0 and Day 90
measure: Genome wide association study from blood sample
timeFrame: Day 90
measure: Analysis of metabolites from blood sample
timeFrame: Day 0 and Day 90
Locations:
city: Shanghai
country: China
facility: Xinhua Hospital
state: Shanghai
Study officials:
affiliation: Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine
name: Fei Li, doctor
role: PRINCIPAL_INVESTIGATOR
References: