Fecal Microbiota Transplantation in Children With ASD

NCT07381374 | Recruiting DMC

• 1 other identifier: 202500102
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Autism Spectrum Disorder

Keywords

Autism Spectrum Disorder; Fecal Microbiota Transplantation; Microbiome; children

Outcome Measures

Primary Outcomes (1)

• Change in Total Score on the Childhood Autism Rating Scale (CARS)

  The change from baseline to Week 24 in the total score of the Childhood Autism Rating Scale (CARS). The CARS is a 15-item behavioral rating scale used to diagnose and assess the severity of autism. Each item is scored from 1 to 4. The total score ranges from 15 to 60. A higher score indicates more severe autism symptoms.

  Pre-FMT, 3 months post-FMT, and 6 months post-FMT

Secondary Outcomes (10)

• Change in Total Score on the Social Responsiveness Scale, Second Edition (SRS-2)

  Pre-FMT, at Week 12, 24, and 48 post-FMT.

• Change in Total Score on the Aberrant Behavior Checklist (ABC)

  Pre-FMT, at Week 12, 24, and 48 post-FMT.

• Change in Total Score on the Gastrointestinal Symptom Rating Scale (GSRS)

  Pre-FMT, during FMT, and at 2, 6, 12, 24, and 48 post-FMT.

• Daily Stool Record (DSR)

  Pre-FMT, during FMT, and at 2, 6, 12, 24 and 48 weeks post-FMT.

• Change in Total Score on the Short Sensory Profile (SSP)

  Pre-FMT, at12, 24 and 48 post-FMT.

Study Arms (3)

Group 1 (FMT-NJT)

ACTIVE COMPARATOR

active FMT via nasojejunal tube + sham colonoscopy

Procedure: ctive FMT via Nasojejunal Tube (FMT-Upper GI)

Group 2 (FMT-C)

ACTIVE COMPARATOR

active FMT via colonoscopy with placement of a transendoscopic enteral tube (TET) secured at the cecum during the first session, followed by two subsequent infusions via the indwelling TET + sham nasojejunal intubation.

Procedure: Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI)

Group 3 (Control)

PLACEBO COMPARATOR

placebo via nasojejunal tube + placebo via colonoscopy (sham procedures for both routes)

Procedure: Placebo via Sham Procedures (Sham-Control)

Interventions

Placebo via Sham Procedures (Sham-Control) PROCEDURE

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.

Group 3 (Control)

ctive FMT via Nasojejunal Tube (FMT-Upper GI) PROCEDURE

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.

Group 1 (FMT-NJT)

Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI) PROCEDURE

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.

Group 2 (FMT-C)

Eligibility Criteria

• Age: 3 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences: collaborator
• Shenzhen Medical Academy of Research and Translation: collaborator
• Shenzhen Children's Hospital: lead

Study Sites (1)

Children's Hospital, Shenzhen

Shenzhen, Guangdong, 518026, China

RECRUITING

Related Publications (19)

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MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Central Study Contacts

Dongling Dai, PhD

CONTACT

08618938690736; daidong3529@email.szu.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: January 25, 2026
• First Posted: February 2, 2026
• Study Start: March 21, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): April 12, 2027
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Date for public release of raw data: within no later than 3 years after the publication of research results.
• Access Criteria: To download data, contact must be made with the researcher.

Locations

CN (1)