A Study of Bumetanide for the Treatment of Autism Spectrum Disorders

Shanghai Jiao Tong University

Status and phase: Completed; Phase 2

Conditions: Autism Spectrum Disorder

Treatments: Drug: Bumetanide; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT03156153

XH-16-048

Details and patient eligibility

About

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.

Full 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.

Enrollment: 120 patients

Sex: All

Ages: 3 to 6 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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120 participants in 2 patient groups, including a placebo group

Bumetanide group

Experimental group

Description:

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.

Treatment:

Drug: Bumetanide

Control group

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo