Efficacy of Bumetanide in Children With Autism Spectrum Disorder Guided by Peripheral Blood Biomarkers and Machine Learning Models

NCT07005414 | Completed Phase 2 DMC

• 1 other identifier: XHEC-C-2025-089
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this study is to learn if bumetanide could alleviate the clinical symptoms in children with autism spectrum disorder (ASD), who has been predicted as high responders to bumetanide via a cytokine-based predictive model. The Eligible ASD participants identified as high responder to bumetanide will be randomly assigned to either the experimental group or the control group. Participants in the experimental group will receive bumetanide interventions, along with the behavioral interventions, for three months. Participants in the control group will only undergo behavioral interventions. The clinical symptoms and potential adverse effects will be closely monitored throughout the intervention period.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• Social Responsiveness Scale, Second Edition (SRS-2)

  Social Responsiveness Scale, Second Edition (SRS-2) is a scale used to evaluate the presence and severity of social impairment. The scores of the SRS-2 rate from 0 to 195, and higher scores indicate more severe symptoms. Changes of the SRS-2 scores from baseline to 3 month after treatment will be analyzed to assess the effect of the intervention on children with ASD.

  From enrollment to the end of treatment at 3 months

Secondary Outcomes (5)

• Clinical Global Impressions Scale (CGI)

  From enrollment to the end of treatment at 3 months

• Vineland Adaptive Behavior Scale

  From enrollment to the end of treatment at 3 months

• Childhood Autism Rating Scale (CARS)

  From enrollment to the end of treatment at 3 months

• Aberrant Behavior Checklist (ABC)

  From enrollment to the end of treatment at 3 months

• Chinese Communicative Development Inventory (CCDI)

  From enrollment to the end of treatment at 3 months

Other Outcomes (2)

• Biomarkers (single nucleotide polymorphisms)

  From enrollment to the end of treatment at 3 months

• Biomarkers (metabolites)

  From enrollment to the end of treatment at 3 months

Study Arms (2)

bumetanide plus treatment as usual (TAU)

EXPERIMENTAL

Participants in experimental group will receive bumetanide, along with treatments as usual (i.e. behavioral interventions)

Drug: Bumetanide; Behavioral: Treatment as usual (TAU)

Treatment as usual (TAU)

OTHER

Participants will undergo treatment as usual, such as behavioral interventions

Behavioral: Treatment as usual (TAU)

Interventions

Bumetanide DRUG

Participants will receive bumetanide (0.5mg bid) for a period of three months.

bumetanide plus treatment as usual (TAU)

Treatment as usual (TAU) BEHAVIORAL

Participants will undergo treatment as usual, such as behavioral interventions

Treatment as usual (TAU); bumetanide plus treatment as usual (TAU)

Eligibility Criteria

• Age: 3 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children aged between 3 and 6 years old
• Meeting the ASD diagnostic criteria of the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5)
• Meeting the ASD diagnostic criteria of the Diagnostic Observational Scale for Autism, Second Edition(ADOS-2)
• CARS total score ≥ 30
• Identified as high responder to bumetanide by machine learning algorithms
• Obtaining informed consent from the legal guardian

You may not qualify if:

• Liver and kidney dysfunction
• With a history of allergy to sulfa drugs
• History of neurological diseases such as epilepsy
• Abnormal ECG
• Diagnosed with genetic or chromosomal abnormalities
• Brain structural abnormalities detected by MRI which required surgical intervention
• Using the melatonin treatment for sleep disorders or withdrawal less than three weeks.
• Received any new intervention within 8 weeks prior to enrollment

Sponsors & Collaborators

• Fei Li: lead

Study Sites (1)

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200092, China

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Bumetanide; Therapeutics

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; meta-Aminobenzoates; Aminobenzoates; Benzoates; Acids, Carbocyclic; Carboxylic Acids; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Sulfones; Sulfur Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Physician, Doctoral Supervisor

Study Record Dates

• First Submitted: May 25, 2025
• First Posted: June 5, 2025
• Study Start: June 4, 2025
• Primary Completion: April 21, 2026
• Study Completion: April 22, 2026
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)