NCT07005414

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

Efficacy of Bumetanide in Children With Autism Spectrum Disorder Guided by Peripheral Blood Biomarkers and Machine Learning Models: A Randomized Controlled Trial

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 66 (estimated)
• Sponsor: Fei Li · Academic / Other
• Sex: All
• Age: 3 Years – 6 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Autism spectrum disorder (ASD) affects about 1% children around the world and can cause lifelong disability and elevate premature mortality. Currently, no medication that can cure ASD or its core symptoms. The recent success of repurposing drugs for novel treatments in psychiatry has been highlighted, with one of the examples given being the use of bumetanide to improve the core symptoms in ASD. However, the heterogeneity in the treatment effect of bumetanide among ASD patients has been observed. A further study (Li et al., Translational Psychiatry, 2022) demonstrated that integrating the cytokine levels and behavioral symptoms could help to identify optimal responders to bumetanide in ASD, showing promise for biomarker- and machine learning model-guided precision medicine with bumetanide. This open-label clinical trial aimed at investigating whether bumetanide could alleviate the clinical symptoms in children with ASD 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 a period of 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

Interventions

Type	Name	Description
DRUG	Bumetanide	Participants will receive bumetanide (0.5mg bid) for a period of three months.
BEHAVIORAL	Treatment as usual (TAU)	Participants will undergo treatment as usual, such as behavioral interventions

Timeline

• Start date: 2025-06-04
• Primary completion: 2026-05-30
• Completion: 2026-05-30
• First posted: 2025-06-05
• Last updated: 2025-06-12

Locations

1 site across 1 country: China