Protocol for the efficacy and safety of fecal microbiota transplantation in children with autism spectrum disorder: a prospective single-center, single-arm interventional study

Baixian Lin ¹, Zhongsheng Zhu ¹, Xiao Yang ^2,3

⁰International Medical Center, Endoscopy Center and Gastroenterology Department, Shenzhen Children's Hospital, Shantou University Medical College, Shenzhen, China.

Frontiers in Pediatrics +

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January 5, 2026

View abstract on PubMed

Summary

Fecal microbiota transplantation (FMT) shows promise for improving gastrointestinal and autism spectrum disorder (ASD) symptoms in children. This study investigates FMT

Area Of Science

Neuroscience
Gastroenterology
Microbiome research

Background

Autism spectrum disorder (ASD) affects 0.7% of children globally.
90% of children with ASD experience gastrointestinal (GI) symptoms.
The microbiota-gut-brain axis (MGBA) may link gut health to ASD. Fecal microbiota transplantation (FMT) is being explored as a therapeutic option.

Purpose Of The Study

To evaluate the efficacy and safety of FMT in children with moderate-to-severe ASD.
To assess the impact of FMT on GI symptoms and ASD severity.
To explore changes in gut microbiota composition following FMT.

Main Methods

Open-label, single-arm trial with 30 children (2-12 years) diagnosed with moderate-to-severe ASD (CARS score ≥36).
Participants received 3 nasojejunal FMTs over 5 days.
Primary outcomes: GI symptom improvement (GSRS) and ASD severity (CARS). Secondary outcomes: social responsiveness (SRS), aberrant behaviors (ABC), and gut microbiota analysis (mNGS).

Main Results

Data on main results are not yet available as this is a prospective study registration.

Conclusions

Further research is needed to determine the full potential of FMT for managing ASD and associated GI symptoms.
FMT may offer a novel therapeutic avenue by modulating the gut microbiome and its influence on the MGBA.

Keywords:

autism spectrum disorder clinical protocol fecal microbiota transplantation gastrointestinal symptoms microbiome microbiota dysbiosis microbiota-gut-brain axis