Fecal Microbiota Transplantation in Children With ASD
ConditionAutism Spectrum Disorder
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3-16
SponsorShenzhen Children's Hospital
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
ctive FMT via Nasojejunal Tube (FMT-Upper GI)
Procedure/SurgeryIntervention: 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.
Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI)
Procedure/SurgeryParticipants 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.
Placebo via Sham Procedures (Sham-Control)
Procedure/SurgeryParticipants 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.
Treatment groups
1 Participants
are divided into 3 treatment groupsGroup A: Group 1 (FMT-NJT)Active comparator 1 intervention
Group B: Group 2 (FMT-C)Active comparator 1 intervention
Group C: Group 3 (Control)Placebo comparator 1 intervention
Trial outcomes
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.
Time frame
Pre-FMT, 3 months post-FMT, and 6 months post-FMTSecondary outcomes
1
Change in Total Score on the Social Responsiveness Scale, Second Edition (SRS-2)
Change from baseline to Weeks 12, 24, and 48. The SRS-2 assesses social impairments associated with autism spectrum disorders. Raw scores are converted to T-scores. Higher T-scores indicate greater severity of social impairment.
Time frame
Pre-FMT, at Week 12, 24, and 48 post-FMT.2
Change in Total Score on the Aberrant Behavior Checklist (ABC)
Change from baseline to Weeks 12, 24, and 48. The ABC assesses problem behaviors in individuals with developmental disabilities. Higher scores indicate more severe problem behaviors.
Time frame
Pre-FMT, at Week 12, 24, and 48 post-FMT.3
Change in Total Score on the Gastrointestinal Symptom Rating Scale (GSRS)
Change from baseline to Weeks 2, 6, 12, 24, and 48. The GSRS evaluates common gastrointestinal symptoms. Each item is scored from 1 (no discomfort) to 7 (very severe discomfort). Higher scores indicate worse GI symptoms.
Time frame
Pre-FMT, during FMT, and at 2, 6, 12, 24, and 48 post-FMT.4
Daily Stool Record (DSR)
Daily documentation of stool characteristics using the Bristol Stool Scale. The percentage of days with abnormal bowel movements (e.g., abnormal consistency, absence of stool, or use of GI medications) is calculated, with higher percentages indicating worse symptoms.
Time frame
Pre-FMT, during FMT, and at 2, 6, 12, 24 and 48 weeks post-FMT.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Shenzhen Children's Hospital
Lead sponsor
Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences
Collaborator
Shenzhen Medical Academy of Research and Translation
Collaborator
Source ClinicalTrials.gov