Autism Spectrum DisorderPhase 2NCT07005414
Efficacy of Bumetanide in Children With Autism Spectrum Disorder Guided by Peripheral Blood Biomarkers and Machine Learning Models
A Phase 2 study testing Bumetanide and Treatment as usual (TAU) in people with Autism Spectrum Disorder, run by Fei Li at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 6 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Bumetanide and Treatment as usual (TAU)
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 1 siteChina
- Study dates
- 4 Jun 2025to 22 Apr 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
China1 site
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200092
About this study
Official title: Efficacy of Bumetanide in Children With Autism Spectrum Disorder Guided by Peripheral Blood Biomarkers and Machine Learning Models: A Randomized Controlled Trial
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.
Read the 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
- Being tested
- Bumetanide, Treatment as usual (TAU)
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BumetanideDrug
Participants will receive bumetanide (0.5mg bid) for a period of three months.
Treatment as usual (TAU)Behavioural
Participants will undergo treatment as usual, such as behavioral interventions
| Study group | Role | What they receive |
|---|---|---|
| bumetanide plus treatment as usual (TAU) | Experimental | Participants in experimental group will receive bumetanide, along with treatments as usual (i.e. behavioral interventions) Receives: Drug: Bumetanide; Behavioral: Treatment as usual (TAU) |
| Treatment as usual (TAU) | Other | Participants will undergo treatment as usual, such as behavioral interventions Receives: Behavioral: Treatment as usual (TAU) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From enrollment to the end of treatment at 3 months
Secondary outcomes (5)
Clinical Global Impressions Scale (CGI)
Clinical Global Impressions Scale (CGI) is a scale used to assess the severity of the illness and the global improvement of the participants under intervention.The Clinical Global Impression - Severity scale (CGI-S) is a clinician rated 7-point measure of overall severity. Scores range from 1 (Normal, not at all ill) through 4 (Moderately ill) to 7 (Among the most extremely ill patients). The Clinical Global Impression - Improvement scale (CGI-I) is the companion measure that evaluates the change in the patient's symptoms relative to baseline. Scores range from 1 (Very much improved) through 4 (No Change) to 7 (Very much worse).
Measured: From enrollment to the end of treatment at 3 months
Vineland Adaptive Behavior Scale
The VABS is used to measure adaptive functioning across domains such as communication, daily living skills, socialization, and motor skills. Changes in VABS scores from baseline to 3 months post-treatment will be analyzed to assess the intervention's impact on adaptive behaviors in children with ASD.
Measured: From enrollment to the end of treatment at 3 months
Childhood Autism Rating Scale (CARS)
CARS is a behavior-rating scale used to assess the presence and severity of the symptoms associated autism spectrum disorder. The scores of the CARS rate from 15 to 60, and higher scores indicate more severe symptoms. The changes in the CARS scores from baseline to 3 months after the treatment will be analyzed to assess the impact of the intervention on participants with ASD.
Measured: From enrollment to the end of treatment at 3 months
Aberrant Behavior Checklist (ABC)
Aberrant Behavior Checklist (ABC) is a 58-item instrument used to measure behavior problems across five sub-scales: hyperactivity, irritability, social withdrawal, stereotypic behavior and inappropriate speech. Each item rates behavior severity on a 4 point scale from 0 (Not at all a problem) to 3 (The problem is severe in degree). A higher score indicates more frequent aberrant behaviors.Changes of the total scores and scores of subscales from baseline to 3 month after treatment will be analyzed.
Measured: From enrollment to the end of treatment at 3 months
Chinese Communicative Development Inventory (CCDI)
Chinese Communicative Development Inventory (CCDI) is a questionnaire used to measures the language abilities. Higher scores suggest better language level. The changes in the CCDI between baseline and 3 month after treatment will be analyzed.
Measured: From enrollment to the end of treatment at 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2025
- Study start4 Jun 2025
- Primary completion21 Apr 2026
- Study completion22 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fei Li
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Fei Li, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07005414
- Sponsor's protocol ID
- XHEC-C-2025-089
- Status last verified
- Apr 2026
- First submitted
- 25 May 2025
- Last updated
- 29 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The Study Protocol, Statistical Analysis Plan, Informed Consent Form, and other relevant documents will be available under reasonable and ethically approved request to the corresponding authors.
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Bumetanide in Children With Autism Spectrum Disorder Guided by Peripheral Blood Biomarkers and Machine Learning Models: A Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT07005414
- Acronym / sponsor ID
- XHEC-C-2025-089
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Fei LiUniversity, hospital or other
- Study start
- 4 Jun 2025 (actual)
- Primary completion
- 21 Apr 2026 (actual)
- Study completion
- 22 Apr 2026 (actual)
- First posted
- 5 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| bumetanide plus treatment as usual (TAU) | Experimental | Drug: Bumetanide, Behavioral: Treatment as usual (TAU)Participants in experimental group will receive bumetanide, along with treatments as usual (i.e. behavioral interventions) |
| Treatment as usual (TAU) | Other | Behavioral: Treatment as usual (TAU)Participants will undergo treatment as usual, such as behavioral interventions |
Who may qualify
- Ages
- 3 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BumetanideParticipants will receive bumetanide (0.5mg bid) for a period of three months. | Drug | bumetanide plus treatment as usual (TAU) |
| Treatment as usual (TAU)Participants will undergo treatment as usual, such as behavioral interventions | Behavioural | Treatment as usual (TAU), bumetanide plus treatment as usual (TAU) |
Primary outcome
Social Responsiveness Scale, Second Edition (SRS-2)
Time frame: From enrollment to the end of treatment at 3 months
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.
5 secondary outcomes
Clinical Global Impressions Scale (CGI)
Time frame: From enrollment to the end of treatment at 3 months
Vineland Adaptive Behavior Scale
Time frame: From enrollment to the end of treatment at 3 months
Childhood Autism Rating Scale (CARS)
Time frame: From enrollment to the end of treatment at 3 months
Aberrant Behavior Checklist (ABC)
Time frame: From enrollment to the end of treatment at 3 months
Chinese Communicative Development Inventory (CCDI)
Time frame: From enrollment to the end of treatment at 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2026.
- Individual participant dataSharing planned: No. The Study Protocol, Statistical Analysis Plan, Informed Consent Form, and other relevant documents will be available under reasonable and ethically approved request to the corresponding authors.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bumetanide and Treatment as usual (TAU) in people with Autism Spectrum Disorder, run by Fei Li at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Social Responsiveness Scale, Second Edition (SRS-2) (time frame: From enrollment to the end of treatment at 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Apr 2026.