RecruitingNot ApplicableNCT07381374

Fecal Microbiota Transplantation in Children With ASD

Study Protocol for a Randomized Controlled of Fecal Microbiota Transplantation Via Different Routes in Children With Moderate-to-Severe Autism Spectrum Disorder

Sponsor

Shenzhen Children's Hospital

Enrollment

1 participants

Start Date

Mar 21, 2026

Study Type

INTERVENTIONAL

Conditions

Autism Spectrum Disorder

Summary

This is a single-center, randomized, double-dummy, triple-blind, placebo-controlled, three-arm parallel-group superiority trial. The study aims to compare the efficacy and safety of Fecal Microbiota Transplantation (FMT) administered via two different invasive routes-nasojejunal tube (NJT) and colonoscopy-versus a placebo control in children aged 3-16 years with moderate-to-severe Autism Spectrum Disorder (ASD). A total of 75 participants will be randomized in a 1:1:1 ratio to receive either active FMT via NJT with sham colonoscopy, active FMT via colonoscopy with sham NJT, or placebo via both routes. All participants will continue their stable behavioral interventions throughout the study. The primary outcome is the change from baseline to Week 24 in the total score of the Childhood Autism Rating Scale (CARS). Secondary outcomes include changes in other behavioral and gastrointestinal symptom scores, gut microbiota profiling, and safety assessments over 48 weeks.

Eligibility

Min Age: 3 YearsMax Age: 16 Years

Inclusion Criteria5

Aged 3-16 years.
Diagnosed with ASD according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5), with a Childhood Autism Rating Scale (CARS) total score ≥36 (moderate-to-severe autism).
Legal guardians fully comprehend the trial's informed consent and voluntarily provide written consent.
Compliance with follow-up visits, examinations, and specimen collection.
No probiotic supplements consumed within the preceding 3 months.

Exclusion Criteria9

Use of probiotics or prebiotics within 3 months prior to enrollment.
Antibiotic usage within 1 month prior to enrollment.
Presence of fever (axillary temperature ≥37.5°C).
Dependency on tube feeding.
Severe gastrointestinal conditions requiring immediate intervention (e.g., life-threatening intestinal obstruction, perforation, hemorrhage, ulcerative colitis, Crohn's disease, celiac disease, or eosinophilic esophagitis).
Diagnosis of severe malnutrition, underweight status (BMI-for-age <3rd percentile), or severe immunodeficiency disorders.
History of severe allergic reactions (e.g., anaphylaxis).
Monogenic disorders (e.g., Fragile X syndrome, Rett syndrome).
Comorbid psychiatric diagnoses, including depression, developmental speech/language disorders, intellectual disability, attention-deficit/hyperactivity disorder (ADHD), selective mutism, reactive attachment disorder, or childhood schizophrenia.

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Interventions

PROCEDUREctive FMT via Nasojejunal Tube (FMT-Upper GI)

Intervention: Active FMT via Nasojejunal Tube (FMT-Upper GI) Intervention Type: Procedure + Biological Intervention Name: Upper Gastrointestinal-Targeted Fecal Microbiota Transplantation Description: Participants in this group receive active fecal microbiota suspension delivered to the jejunum (upper gastrointestinal tract). Under endoscopic guidance, a nasojejunal tube is placed with its tip positioned past the Ligament of Treitz. The active FMT preparation is then infused slowly through this tube. Additionally, participants undergo a sham colonoscopy (simulated procedure under anesthesia where the scope is inserted to the rectosigmoid junction with minimal water/air insufflation, but no FMT is administered). Dosage: 5 mL per kilogram of body weight, with a maximum total volume of 100 mL per infusion. Frequency: Administered once every other day, for a total of three sessions over 5 days.

PROCEDUREActive FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI)

Participants in this group receive active fecal microbiota suspension delivered to the cecum (lower gastrointestinal tract). The intervention involves two phases: First Session (Day 0): Under general anesthesia, a full colonoscopy is performed to reach the cecum. The active FMT preparation is infused directly into the cecum. Subsequently, a transendoscopic enteral tube (TET) is advanced through the colonoscope and its tip is secured in the cecum using endoscopic clips. Second \& Third Sessions (Days 2 \& 4): The active FMT preparation is infused through the indwelling TET at the bedside, without the need for repeat colonoscopy or general anesthesia. Additionally, participants undergo a sham nasojejunal intubation (a tube is placed into the stomach and secured, and a placebo is infused). Dosage: 5 mL per kilogram of body weight, with a maximum total volume of 100 mL per infusion. Frequency: Administered once every other day, for a total of three sessions over 5 days.

PROCEDUREPlacebo via Sham Procedures (Sham-Control)

Participants in this control group undergo both sham procedures with infusion of an inactivated placebo suspension, which is visually and physically identical to the active FMT preparation but contains no viable microbiota. Sham Nasojejunal Intubation: A tube is placed into the stomach (not the jejunum) and secured. The placebo suspension is infused. Sham Colonoscopy: Under anesthesia, a simulated colonoscopy is performed (scope inserted to rectosigmoid junction with minimal insufflation). No substance is infused during this sham procedure. This double-sham design ensures that both potential delivery routes are "simulated" for the control group. Dosage: Volume-matched to the active FMT arms (5 mL/kg, max 100 mL) for the nasogastric infusion. No infusion during sham colonoscopy. Frequency: The placebo infusion (during sham NJ intubation) occurs once every other day, for a total of three sessions over 5 days, coinciding with the two sham procedures.