Autism Spectrum DisorderNCT06677840
Exploring the Gut-Brain-Behavior Axis With Biomarkers, Probiotic Efficacy, and Artificial Intelligence Algorithms
A study of Autism Spectrum Disorder, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
With an increased prevalence of autism spectrum disorder (ASD) worldwide and in Taiwan, ASD demands urgent attention due to its lasting impact, unclear causes, and limited diagnostic and treatment options. Recent studies highlight disruptions in the gut-brain axis, particularly in ASD. Read more about this study
- Who is it for?
- Ages 4 to 15 years
- Conditions the study is for
- Autism Spectrum Disorder
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 110 participants
- Where
- 1 siteTaiwan
- Study dates
- 1 May 2024to 30 Apr 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 4 to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children and adolescents aged 4 to 15 who are clinically diagnosed with ASD according to DSM-5 criteria and confirmed by the ADI-R/ADOS.
- Caregivers cooperate with all the assessments and stool and blood collection.
Who cannot join
- A history of major psychiatric disorders (e.g., schizophrenia, bipolar disorder, major depression), neurological disorders, and substances use disorders.
- Difficulty following instructions.
- Consumption of antibiotics and yogurt or probiotic products two weeks prior to enrollment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Hospital
Taipei
Susan Shur-Fen Gau, MD, PhD, + 886-2-23123456 ext. 266802, gaushufe@ntu.edu.tw
About this study
Official title: Precision Health in Autism: Exploring the Gut-Brain-Behavior Axis With Biomarkers, Probiotic Efficacy, and Artificial Intelligence Algorithms
With an increased prevalence of autism spectrum disorder (ASD) worldwide and in Taiwan, ASD demands urgent attention due to its lasting impact, unclear causes, and limited diagnostic and treatment options. Recent studies highlight disruptions in the gut-brain axis, particularly in ASD. Building on PI Gau's prior research on altered gut microbiota in autism spectrum disorder (ASD), this proposed 4-year study aims to develop evidence-based probiotic intervention (GKB7) and treatment biomarkers for behavioral interventions (Naturalistic Developmental Behavioral Interventions, NDBI, and Program for the Education and Enrichment of Relational Skills, PEERS®) through comprehensive assessments. The ultimate goal is to establish probiotic treatments and diagnostic/treatment biomarkers for ASD utilizing a deep-learning multimodal auxiliary platform.
Specific Aims:
Read more
S.I To conduct a randomized, double-blind, placebo-controlled trial to assess the treatment effects of Clostridium butyricum GKB7 on ASD.
S.II To investigate the effect of NDBI intervention trials using metagenomics as the outcome measure.
S.III To evaluate the effect of PEERS® interventional trials with metagenomics as the outcome measure.
S.IV To develop an artificial intelligence (machine/deep learning) multimodal auxiliary prediction platform that integrates environmental, behavioral/clinical, neurocognitive/imaging, and metagenomic data for ASD diagnosis, subtyping, and impairment prediction.
S.V To identify microbiome and metabolomic biomarkers and endophenotypes of ASD, and to establish Gut-Brain-Behavioral Axes in ASD.
Read the detailed description
We will enroll 110 ASD patients (age 4-15 years) in probiotic clinical trials with Clostridium butyricum GKB7 (S.I), 120 ASD children (2-6 years old) in treatment biomarker of NDBIs study (S.II), and 24 autistic adolescents and 15 young adults in treatment biomarker of PEERS® study (S.III). Assessments include diagnostic evaluations and dimensional measures regarding ASD and other diagnoses (ADI-R, ADOS, K-SADS-E, CGI), digestive symptoms and eating (GSQ, FFQ), autistic symptoms (SRS, CAST, GSQ, SP, EQ, SCQ), psychopathology (CBCL, SNAP-IV), social functions/quality of life (SAICA), neuropsychological functions (CPT, CANTAB, BRIEF), images (fNIRS, T1, T2, rfMRI, DTI, MRS), metabolome (blood) and microbiome (stool). We will conduct multidimensional data analysis to identify the mutual regulation pathways of the gut-brain-behavior axis and key biochemical molecules using machine/deep learning algorithms to establish a multimodal auxiliary platform for ASD classification and prediction (S.IV). We will also use the case-control and longitudinal data to identify microbiota and metabolomics biomarkers and endophenotypes of ASD (S.V).
S.I.1 Pilot Study-Open-Label in 20 autistic children： This pilot clinical trial will employ an open-label study design to explore the effects of a 12-week treatment with the probiotic Clostridium butyricum GKB7 on clinical symptoms as primary outcome, and cognitive function, brain activity, and gut microbiota as secondary outcome measures in 20 autistic children, aged 4-15 years. Participants and their parents will undergo clinical and neuropsychological assessments at baseline, Month 1, Month 2, and Month 3 or the last observation day. MRI scans, and stool and blood samples will be collected at baseline and at Month 3 or the last observation day SI.2 Double-Blind Randomized Placebo-Controlled Trial in 90 autistic children： The objectives of this double-blind randomized placebo-controlled trial are to evaluate the tolerability, safety, and efficacy of the probiotic intervention with Clostridium butyricum GKB7 among 90 4-15-year-old children and adolescents with a clinical diagnosis of ASD according to the DSM-5. They will be randomized to two treatment arms [GKB7, n = 60; the placebo comparison group, n = 30] for three months followed by another 3-month open-label clinical trial as follows. At the end of 3rd month, all the 90 participants will receive continuous treatment with GKB7 for 3 months. In summary, among the 90 ASD participants, 60 will receive GKB7 for 6 months, 30 will have routine follow-up assessments for three months, followed by GKB7 for 3 months. The participants and their parents will receive clinical and neuropsychological assessments at baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3, transition to open-label study), month 4 (V4), and month 6 (V5). Besides, the participants will undergo MRI scanning, blood and stool sampling at baseline (before treatment, V0), month 3 (V3), and month 6 (V5).
- Conditions
- Autism Spectrum Disorder
- Being tested
- probiotic (GKB7/placebo)
- 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.
probiotic (GKB7/placebo)Other
The objectives of this double-blind randomized placebo-controlled trial are to evaluate the tolerability, safety, and efficacy of the probiotic intervention with Clostridium butyricum GKB7 among 90 4-15-year-old children and adolescents with a clinical diagnosis of ASD according to the DSM-5
| Study group | Role | What they receive |
|---|---|---|
| GKB7 | Experimental | Not described Receives: Other: probiotic (GKB7/placebo) |
| Placebo | Placebo comparator | Not described Receives: Other: probiotic (GKB7/placebo) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Global Impression
The CGI-S (Clinical Global Impression-Severity Scale) and CGI-I (-Improvement Scale) are single-item ratings of the clinician's assessment of the global severity of clinical symptoms. Severity and improvement are rated on a 7-point scale (from 1 = normal, not at all ill, to 7 = among the most extremely ill) and (from 1 = very much improved, to 7 = very much worse).
Measured: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)
Social Responsiveness Scale (SRS)
The SRS is a 65-item questionnaire based on parent-reports on the child's social interactions with others (rating 0-3), consists of 5 subscales: social awareness, social cognition, social communication, social motivation, and autistic mannerisms
Measured: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)
Secondary outcomes (3)
Self-administered questionnaires
The self-administered questionnaires include autistic symptoms (Autism Spectrum Quotient, AQ; Sensory Profile, SP), psychopathology (Child Behavior Checklist, CBCL; Swanson, Nolan, and Pelham, version IV scale, SNAP-IV), social functions/quality of life (Social Adjustment Inventory for Children and Adolescents, SAICA), and executive functions (Behavior Rating Inventories of Executive Function, BRIEF)
Measured: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Neuropsychological functions: Continuous Performance Test(CPT)
The 4 dimensions of CCPT: focused attention, hyperactivity/impulsivity, sustained attention, and vigilance
Measured: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Neuropsychological functions: Cambridge Neuropsychological Test Automated Batteries(CANTAB)
The 4 main cognitive components of CANTAB: Visual Memory, Attention, Working and Planning Memory (Executive Functions), and Decision Making
Measured: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2024
- Study start1 May 2024
- Primary completion30 Apr 2027 (estimated)
- Study completion30 Apr 2028 (estimated)
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.
- More trials run by National Taiwan University HospitalThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study consists of two sequential stages. Stage 1 is an open-label pilot study enrolling 20 participants to explore the effects of a 12-week treatment with the probiotic Clostridium butyricum GKB7 on clinical symptoms as primary outcome, and cognitive function, brain activity, and gut microbiota as secondary outcome measures. Following review of the pilot results, Stage 2 is a randomized, double-blind, placebo-controlled, parallel-group trial enrolling 90 participants.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 110 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06677840
- Sponsor's protocol ID
- 202404049RIPA
- Status last verified
- Jun 2026
- First submitted
- 4 Nov 2024
- Last updated
- 9 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Precision Health in Autism: Exploring the Gut-Brain-Behavior Axis With Biomarkers, Probiotic Efficacy, and Artificial Intelligence Algorithms
- ClinicalTrials.gov ID
- NCT06677840
- Acronym / sponsor ID
- 202404049RIPA
- Conditions
- Autism Spectrum Disorder
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 110 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- 1 May 2024 (actual)
- Primary completion
- 30 Apr 2027 (estimated)
- Study completion
- 30 Apr 2028 (estimated)
- First posted
- 7 Nov 2024 (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.
With an increased prevalence of autism spectrum disorder (ASD) worldwide and in Taiwan, ASD demands urgent attention due to its lasting impact, unclear causes, and limited diagnostic and treatment options. Recent studies highlight disruptions in the gut-brain axis, particularly in ASD. Building on PI Gau's prior research on altered gut microbiota in autism spectrum disorder (ASD), this proposed 4-year study aims to develop evidence-based probiotic intervention (GKB7) and treatment biomarkers for behavioral interventions (Naturalistic Developmental Behavioral Interventions, NDBI, and Program for the Education and Enrichment of Relational Skills, PEERS®) through comprehensive assessments. The ultimate goal is to establish probiotic treatments and diagnostic/treatment biomarkers for ASD utilizing a deep-learning multimodal auxiliary platform.
Specific Aims:
S.I To conduct a randomized, double-blind, placebo-controlled trial to assess the treatment effects of Clostridium butyricum GKB7 on ASD.
S.II To investigate the effect of NDBI intervention trials using metagenomics as the outcome measure.
S.III To evaluate the effect of PEERS® interventional trials with metagenomics as the outcome measure.
S.IV To develop an artificial intelligence (machine/deep learning) multimodal auxiliary prediction platform that integrates environmental, behavioral/clinical, neurocognitive/imaging, and metagenomic data for ASD diagnosis, subtyping, and impairment prediction.
S.V To identify microbiome and metabolomic biomarkers and endophenotypes of ASD, and to establish Gut-Brain-Behavioral Axes in ASD.
Detailed description
We will enroll 110 ASD patients (age 4-15 years) in probiotic clinical trials with Clostridium butyricum GKB7 (S.I), 120 ASD children (2-6 years old) in treatment biomarker of NDBIs study (S.II), and 24 autistic adolescents and 15 young adults in treatment biomarker of PEERS® study (S.III). Assessments include diagnostic evaluations and dimensional measures regarding ASD and other diagnoses (ADI-R, ADOS, K-SADS-E, CGI), digestive symptoms and eating (GSQ, FFQ), autistic symptoms (SRS, CAST, GSQ, SP, EQ, SCQ), psychopathology (CBCL, SNAP-IV), social functions/quality of life (SAICA), neuropsychological functions (CPT, CANTAB, BRIEF), images (fNIRS, T1, T2, rfMRI, DTI, MRS), metabolome (blood) and microbiome (stool). We will conduct multidimensional data analysis to identify the mutual regulation pathways of the gut-brain-behavior axis and key biochemical molecules using machine/deep learning algorithms to establish a multimodal auxiliary platform for ASD classification and prediction (S.IV). We will also use the case-control and longitudinal data to identify microbiota and metabolomics biomarkers and endophenotypes of ASD (S.V).
S.I.1 Pilot Study-Open-Label in 20 autistic children： This pilot clinical trial will employ an open-label study design to explore the effects of a 12-week treatment with the probiotic Clostridium butyricum GKB7 on clinical symptoms as primary outcome, and cognitive function, brain activity, and gut microbiota as secondary outcome measures in 20 autistic children, aged 4-15 years. Participants and their parents will undergo clinical and neuropsychological assessments at baseline, Month 1, Month 2, and Month 3 or the last observation day. MRI scans, and stool and blood samples will be collected at baseline and at Month 3 or the last observation day SI.2 Double-Blind Randomized Placebo-Controlled Trial in 90 autistic children： The objectives of this double-blind randomized placebo-controlled trial are to evaluate the tolerability, safety, and efficacy of the probiotic intervention with Clostridium butyricum GKB7 among 90 4-15-year-old children and adolescents with a clinical diagnosis of ASD according to the DSM-5. They will be randomized to two treatment arms [GKB7, n = 60; the placebo comparison group, n = 30] for three months followed by another 3-month open-label clinical trial as follows. At the end of 3rd month, all the 90 participants will receive continuous treatment with GKB7 for 3 months. In summary, among the 90 ASD participants, 60 will receive GKB7 for 6 months, 30 will have routine follow-up assessments for three months, followed by GKB7 for 3 months. The participants and their parents will receive clinical and neuropsychological assessments at baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3, transition to open-label study), month 4 (V4), and month 6 (V5). Besides, the participants will undergo MRI scanning, blood and stool sampling at baseline (before treatment, V0), month 3 (V3), and month 6 (V5).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This study consists of two sequential stages. Stage 1 is an open-label pilot study enrolling 20 participants to explore the effects of a 12-week treatment with the probiotic Clostridium butyricum GKB7 on clinical symptoms as primary outcome, and cognitive function, brain activity, and gut microbiota as secondary outcome measures. Following review of the pilot results, Stage 2 is a randomized, double-blind, placebo-controlled, parallel-group trial enrolling 90 participants.
| Arm | Type | Receives |
|---|---|---|
| GKB7 | Experimental | Other: probiotic (GKB7/placebo) |
| Placebo | Placebo comparator | Other: probiotic (GKB7/placebo) |
Who may qualify
- Ages
- 4 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Children and adolescents aged 4 to 15 who are clinically diagnosed with ASD according to DSM-5 criteria and confirmed by the ADI-R/ADOS.
- Caregivers cooperate with all the assessments and stool and blood collection.
Key exclusion criteria (3 listed)
- A history of major psychiatric disorders (e.g., schizophrenia, bipolar disorder, major depression), neurological disorders, and substances use disorders.
- Difficulty following instructions.
- Consumption of antibiotics and yogurt or probiotic products two weeks prior to enrollment.
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 |
|---|---|---|
| probiotic (GKB7/placebo)The objectives of this double-blind randomized placebo-controlled trial are to evaluate the tolerability, safety, and efficacy of the probiotic intervention with Clostridium butyricum GKB7 among 90 4-15-year-old children and adolescents with a clinical diagnosis of ASD according to the DSM-5 | Other | GKB7, Placebo |
Primary outcome
Clinical Global Impression
Time frame: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)
The CGI-S (Clinical Global Impression-Severity Scale) and CGI-I (-Improvement Scale) are single-item ratings of the clinician's assessment of the global severity of clinical symptoms. Severity and improvement are rated on a 7-point scale (from 1 = normal, not at all ill, to 7 = among the most extremely ill) and (from 1 = very much improved, to 7 = very much worse).
Social Responsiveness Scale (SRS)
Time frame: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)
The SRS is a 65-item questionnaire based on parent-reports on the child's social interactions with others (rating 0-3), consists of 5 subscales: social awareness, social cognition, social communication, social motivation, and autistic mannerisms
3 secondary outcomes
Self-administered questionnaires
Time frame: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Neuropsychological functions: Continuous Performance Test(CPT)
Time frame: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Neuropsychological functions: Cambridge Neuropsychological Test Automated Batteries(CANTAB)
Time frame: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study of Autism Spectrum Disorder, run by National Taiwan University Hospital at 1 site in Taiwan.
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 2 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 9 Jul 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 Taiwan. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Susan Shur-Fen Gau, MD, PhD (+886-2-23123456 ext. 266802, gaushufe@ntu.edu.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Global Impression (time frame: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)). The study lists 2 primary outcomes in all. 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 9 Jul 2026.