Exploring the Gut-Brain-Behavior Axis With Biomarkers, Probiotic Efficacy, and Artificial Intelligence Algorithms

NCT06677840 | Recruiting DMC

• 1 other identifier: 202404049RIPA
• Study Type: interventional
• Target: 110
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (2)

• 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).

  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

  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

  At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)

• Neuropsychological functions: Continuous Performance Test(CPT)

  At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)

• Neuropsychological functions: Cambridge Neuropsychological Test Automated Batteries(CANTAB)

  At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)

Study Arms (2)

GKB7

EXPERIMENTAL

Other: probiotic (GKB7/placebo)

Placebo

PLACEBO COMPARATOR

Other: probiotic (GKB7/placebo)

Interventions

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

GKB7; Placebo

Eligibility Criteria

• Age: 4 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Taiwan University Hospital: lead

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan

RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Probiotics

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Dietary Supplements; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Central Study Contacts

Susan Shur-Fen Gau, MD, PhD

CONTACT

+886-2-23123456; gaushufe@ntu.edu.tw

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This study consists of two sequential stages with different masking procedures. Stage 1 is an open-label pilot study in which all participants receive GKB7, and both investigators and participants are aware of the assigned intervention. Stage 2 begins with a 3-month randomized, double-blind, placebo-controlled period during which participants are randomized in a 2:1 to receive either GKB7 (n = 60) or placebo (n = 30). Participants, care providers, investigators, and outcome assessors remain blinded to treatment allocation throughout this period. At the end of Month 3, all 90 participants enter a 3-month open-label extension and receive continuous treatment with GKB7. During the open-label extension, treatment assignment is no longer masked.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: November 4, 2024
• First Posted: November 7, 2024
• Study Start: May 1, 2024
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): April 30, 2028
• Last Updated: July 9, 2026

Record last verified: 2026-06

Locations

TW (1)