Clinical trial NCT06677840: Exploring the Gut-Brain-Behavior Axis With Biomarkers, Probiotic Efficacy, and Artificial Intelligence Algorithms
Official title: Precision Health in Autism: Exploring the Gut-Brain-Behavior Axis With Biomarkers, Probiotic Efficacy, and Artificial Intelligence Algorithms
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Autism Spectrum Disorder
Lead sponsor: National Taiwan University Hospital
Sponsor class: OTHER
First posted: 2024-11-07
Enrollment: 110
Sex: ALL
Minimum age (years): 4
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This groundbreaking human study on the ASD microbiome includes probiotic clinical trials, investigation of treatment biomarkers, machine learning/deep learning platform development for ASD classification and prediction, and identification of diagnostic biomarkers. Upon completion, the investigators anticipate publishing at least 12 SCI papers and/or patents and establishing an auxiliary diagnosis platform for both clinical and academic purposes. The findings will offer new insights into the pathogenetic mechanisms, improving early detection, diagnosis, and treatment, ultimately advancing precision medicine for ASD.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Clinical Global Impression
timeFrame: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)
measure: Social Responsiveness Scale (SRS)
timeFrame: At baseline (before treatment, V0), month 1 (V1), month 2 (V2), month 3 (V3), month 4 (V4), and month 6 (V5)
Secondary outcomes:
measure: Self-administered questionnaires
timeFrame: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
measure: Neuropsychological functions: Continuous Performance Test(CPT)
timeFrame: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
measure: Neuropsychological functions: Cambridge Neuropsychological Test Automated Batteries(CANTAB)
timeFrame: At baseline (before treatment, V0), month 3 (V3), and month 6 (V5)
Other outcomes:
Locations:
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
Study officials:
References: