Autism Spectrum DisorderNCT07381374
Fecal Microbiota Transplantation in Children With ASD
A study testing ctive FMT via Nasojejunal Tube (FMT-Upper GI) and Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI) in people with Autism Spectrum Disorder, run by Shenzhen Children's Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 to 16 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- ctive FMT via Nasojejunal Tube (FMT-Upper GI) and Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1 participants
- Where
- 1 siteChina
- Study dates
- 21 Mar 2026to 12 Apr 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Children's Hospital, Shenzhen
Shenzhen, Guangdong, 518026
Daming Bai, 086-0755-83008158, 394807416@qq.com
About this study
Official title: Study Protocol for a Randomized Controlled of Fecal Microbiota Transplantation Via Different Routes in Children With Moderate-to-Severe Autism Spectrum Disorder
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.
Read the detailed description
Background:
Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder often accompanied by gastrointestinal (GI) symptoms and gut microbiota dysbiosis. Fecal Microbiota Transplantation (FMT) has shown promise in modulating the gut-brain axis and improving both behavioral and GI symptoms in preliminary ASD studies. However, the optimal route of FMT administration remains unclear, and high-quality comparative evidence is lacking.
Objectives:
Primary: To compare the efficacy of FMT delivered via NJT versus colonoscopy versus placebo in improving social interaction and communication, as measured by the change in CARS total score from baseline to Week 24.
Secondary: To evaluate effects on social responsiveness (SRS), aberrant behaviors (ABC), sensory processing (SSP), sleep quality (CSHQ), GI symptoms (GSRS), gut microbiota engraftment dynamics, and safety/tolerability.
Methods:
Design: Single-center, randomized, double-dummy, triple-blind, placebo-controlled, three-arm parallel-group trial.
Participants: 75 children aged 3-16 years with DSM-5-confirmed moderate-to-severe ASD (CARS ≥36) and stable behavioral intervention.
Interventions:
Group 1 (FMT-NJT): Active FMT via NJT + sham colonoscopy. Group 2 (FMT-C): Active FMT via colonoscopy with placement of a transendoscopic enteral tube (TET) in the cecum for subsequent infusions + sham NJT.
Group 3 (Control): Placebo via both NJT and colonoscopy (sham procedures). Dosage: 5 mL/kg (max 100 mL) per infusion, administered every other day for three sessions.
Blinding: Triple-blind-participants/guardians, outcome assessors, and data analysts are blinded. An independent pharmacy unit prepares identical active and placebo preparations.
Assessments: Behavioral scales (CARS-2, SRS-2, ABC, SSP, CSHQ), GI symptoms (GSRS), stool metagenomics, and safety monitoring at baseline, Weeks 2, 6, 12, 24, and 48.
Sample Size: 25 per group (total N=75), calculated to detect a 2.5-point difference in CARS change with 80% power, accounting for 15% dropout.
Randomization: Centralized block randomization stratified by age and baseline CARS severity.
Statistical Analysis: ANCOVA for primary outcome with baseline adjustment; mixed models for repeated measures; descriptive and inferential methods for secondary and safety outcomes.
Outcomes:
Primary: Change in CARS-2 total score from baseline to Week 24. Secondary: Changes in SRS-2, ABC, GSRS, SSP, CSHQ scores; microbiota composition/function; incidence and severity of adverse events (CTCAE v5.0).
Significance: This trial will provide high-level evidence on whether the therapeutic effect of FMT in ASD depends on the gastrointestinal delivery site. The novel TET-based protocol for repeated cecal delivery allows for a rigorous comparison of microbial engraftment in the lower versus upper GI tract. The results will guide the optimization of microbiota-targeted therapies for ASD and other conditions linked to the gut-brain axis.
- Conditions
- Autism Spectrum Disorder
- Being tested
- ctive FMT via Nasojejunal Tube (FMT-Upper GI), Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI), Placebo via Sham Procedures (Sham-Control)
- Keywords
- Autism Spectrum Disorder, Fecal Microbiota Transplantation, Microbiome, children
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ctive FMT via Nasojejunal Tube (FMT-Upper GI)Procedure or surgery
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.
Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI)Procedure or surgery
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.
Placebo via Sham Procedures (Sham-Control)Procedure or surgery
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.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 (FMT-NJT) | Active comparator | active FMT via nasojejunal tube + sham colonoscopy Receives: 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. Receives: 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) Receives: Procedure: Placebo via Sham Procedures (Sham-Control) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
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.
Measured: Pre-FMT, at Week 12, 24, and 48 post-FMT.
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.
Measured: Pre-FMT, at Week 12, 24, and 48 post-FMT.
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.
Measured: Pre-FMT, during FMT, and at 2, 6, 12, 24, and 48 post-FMT.
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.
Measured: 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)
Change from baseline to Weeks 12, 24, and 48. The SSP measures sensory processing patterns in children. Lower scores indicate greater sensory processing difficulties.
Measured: Pre-FMT, at12, 24 and 48 post-FMT.
Change in Total Score on the Children's Sleep Habits Questionnaire (CSHQ)
Change from baseline to Weeks 12, 24, and 48. The CSHQ assesses sleep problems in children. Higher scores indicate more disturbed sleep.
Measured: Pre-FMT, at12, 24 and 48 post-FMT.
Change in Gut Microbiota Alpha Diversity (Shannon Index)
Change from baseline to Weeks 2, 6, 12, 24, and 48 as measured by shotgun metagenomic sequencing. Alpha diversity measures the richness and evenness of microbial species within a sample.
Measured: Pre-FMT, and at 2, 6, 12, 24 and 48 weeks post-FMT.
Change in Relative Abundance of Specific Gut Microbial Taxa (at the genus level)
Change from baseline to Weeks 2, 6, 12, 24, and 48 as measured by shotgun metagenomic sequencing (e.g., using MetaPhlAn for taxonomic profiling).
Measured: Pre-FMT, and at 2, 6, 12, 24 and 48 weeks post-FMT.
Incidence of Treatment-Emergent Adverse Events (TEAEs)
The number and percentage of participants experiencing any adverse event during the study period, graded for severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.0. The relationship to the study intervention (FMT/placebo procedures) will be assessed by the investigator.
Measured: From date of randomization until the end of study visit (Week 48).
Incidence of Serious Adverse Events (SAEs)
The number and percentage of participants experiencing any serious adverse event during the study period, as defined by CTCAE v4.0 and ICH guidelines.
Measured: From date of randomization until the end of study visit (Week 48).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2026
- Study start21 Mar 2026
- Primary completion31 Dec 2026 (estimated)
- Study completion12 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (19)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Children's Hospital
- Sponsor type
- Other government
- Collaborators
- Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences, Shenzhen Medical Academy of Research and Translation
- Responsible party
- Principal investigator, Dongling Dai, Shenzhen Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07381374
- Sponsor's protocol ID
- 202500102
- Status last verified
- Jun 2026
- First submitted
- 25 Jan 2026
- Last updated
- 9 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Ghirardini A, Nanni-Costa A, Venturi S, Ridolfi L, Petrini F, Taddei S, Venturoli A, Pugliese MR, Monti M, Martinelli G. Efficiency of organ procurement and transplantation programs. Transpl Int. 2000;13 Suppl 1:S267-71. doi: 10.1007/s001470050339.
PubMed 11112010Li Y, Xiao P, Ding H, Wang H, Xu Q, Wang R, Zheng L, Song X, Wang Y, Zhang T. Fecal Microbiota Transplantation in Children with Autism. Neuropsychiatr Dis Treat. 2024 Dec 9;20:2391-2400. doi: 10.2147/NDT.S488001. eCollection 2024.
PubMed 39677507Kang DW, Adams JB, Gregory AC, Borody T, Chittick L, Fasano A, Khoruts A, Geis E, Maldonado J, McDonough-Means S, Pollard EL, Roux S, Sadowsky MJ, Lipson KS, Sullivan MB, Caporaso JG, Krajmalnik-Brown R. Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study. Microbiome. 2017 Jan 23;5(1):10. doi: 10.1186/s40168-016-0225-7.
PubMed 28122648Chen K, Fu Y, Wang Y, Liao L, Xu H, Zhang A, Zhang J, Fan L, Ren J, Fang B. Therapeutic Effects of the In Vitro Cultured Human Gut Microbiota as Transplants on Altering Gut Microbiota and Improving Symptoms Associated with Autism Spectrum Disorder. Microb Ecol. 2020 Aug;80(2):475-486. doi: 10.1007/s00248-020-01494-w. Epub 2020 Feb 26.
PubMed 32100127Flora SJ, Tripathi N. Hepatic and renal metallothionein induction following single oral administration of gallium arsenide in rats. Biochem Mol Biol Int. 1998 Sep;45(6):1121-7. doi: 10.1080/15216549800203342.
PubMed 9762410Tan Q, Orsso CE, Deehan EC, Kung JY, Tun HM, Wine E, Madsen KL, Zwaigenbaum L, Haqq AM. Probiotics, prebiotics, synbiotics, and fecal microbiota transplantation in the treatment of behavioral symptoms of autism spectrum disorder: A systematic review. Autism Res. 2021 Sep;14(9):1820-1836. doi: 10.1002/aur.2560. Epub 2021 Jun 26.
PubMed 34173726Goncalves CL, Doifode T, Rezende VL, Costa MA, Rhoads JM, Soutullo CA. The many faces of microbiota-gut-brain axis in autism spectrum disorder. Life Sci. 2024 Jan 15;337:122357. doi: 10.1016/j.lfs.2023.122357. Epub 2023 Dec 19.
PubMed 38123016Ullah H, Arbab S, Tian Y, Chen Y, Liu CQ, Li Q, Li K. Crosstalk between gut microbiota and host immune system and its response to traumatic injury. Front Immunol. 2024 Jul 31;15:1413485. doi: 10.3389/fimmu.2024.1413485. eCollection 2024.
PubMed 39144142Njeh CF, Fuerst T, Diessel E, Genant HK, Hausler KD. Association between QUS and structural parameters? Calcif Tissue Int. 2000 Sep;67(3):273-4. doi: 10.1007/s002230001136. No abstract available.
PubMed 10954784Ullah H, Arbab S, Tian Y, Liu CQ, Chen Y, Qijie L, Khan MIU, Hassan IU, Li K. The gut microbiota-brain axis in neurological disorder. Front Neurosci. 2023 Aug 4;17:1225875. doi: 10.3389/fnins.2023.1225875. eCollection 2023.
PubMed 37600019Morton JT, Jin DM, Mills RH, Shao Y, Rahman G, McDonald D, Zhu Q, Balaban M, Jiang Y, Cantrell K, Gonzalez A, Carmel J, Frankiensztajn LM, Martin-Brevet S, Berding K, Needham BD, Zurita MF, David M, Averina OV, Kovtun AS, Noto A, Mussap M, Wang M, Frank DN, Li E, Zhou W, Fanos V, Danilenko VN, Wall DP, Cardenas P, Baldeon ME, Jacquemont S, Koren O, Elliott E, Xavier RJ, Mazmanian SK, Knight R, Gilbert JA, Donovan SM, Lawley TD, Carpenter B, Bonneau R, Taroncher-Oldenburg G. Multi-level analysis of the gut-brain axis shows autism spectrum disorder-associated molecular and microbial profiles. Nat Neurosci. 2023 Jul;26(7):1208-1217. doi: 10.1038/s41593-023-01361-0. Epub 2023 Jun 26.
PubMed 37365313You M, Chen N, Yang Y, Cheng L, He H, Cai Y, Liu Y, Liu H, Hong G. The gut microbiota-brain axis in neurological disorders. MedComm (2020). 2024 Jul 20;5(8):e656. doi: 10.1002/mco2.656. eCollection 2024 Aug.
PubMed 39036341O'Brien CP, Testa T, O'Brien TJ, Greenstein R. Conditioning in human opiate addicts. Pavlov J Biol Sci. 1976 Oct-Dec;11(4):195-202. doi: 10.1007/BF03000314.
PubMed 995439Kodak T, Bergmann S. Autism Spectrum Disorder: Characteristics, Associated Behaviors, and Early Intervention. Pediatr Clin North Am. 2020 Jun;67(3):525-535. doi: 10.1016/j.pcl.2020.02.007. Epub 2020 May 4.
PubMed 32443991Penel C, Greppin H. Binding of plant isoperoxidases to pectin in the presence of calcium. FEBS Lett. 1994 Apr 18;343(1):51-5. doi: 10.1016/0014-5793(94)80605-5.
PubMed 8163016
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study Protocol for a Randomized Controlled of Fecal Microbiota Transplantation Via Different Routes in Children With Moderate-to-Severe Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT07381374
- Acronym / sponsor ID
- 202500102
- Conditions
- Autism Spectrum Disorder
- Keywords
- Autism Spectrum Disorder, Fecal Microbiota Transplantation, Microbiome, children
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- Shenzhen Children's HospitalOther government
- Collaborators
- Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences, Shenzhen Medical Academy of Research and Translation
- Study start
- 21 Mar 2026 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 12 Apr 2027 (estimated)
- First posted
- 2 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Background:
Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder often accompanied by gastrointestinal (GI) symptoms and gut microbiota dysbiosis. Fecal Microbiota Transplantation (FMT) has shown promise in modulating the gut-brain axis and improving both behavioral and GI symptoms in preliminary ASD studies. However, the optimal route of FMT administration remains unclear, and high-quality comparative evidence is lacking.
Objectives:
Primary: To compare the efficacy of FMT delivered via NJT versus colonoscopy versus placebo in improving social interaction and communication, as measured by the change in CARS total score from baseline to Week 24.
Secondary: To evaluate effects on social responsiveness (SRS), aberrant behaviors (ABC), sensory processing (SSP), sleep quality (CSHQ), GI symptoms (GSRS), gut microbiota engraftment dynamics, and safety/tolerability.
Methods:
Design: Single-center, randomized, double-dummy, triple-blind, placebo-controlled, three-arm parallel-group trial.
Participants: 75 children aged 3-16 years with DSM-5-confirmed moderate-to-severe ASD (CARS ≥36) and stable behavioral intervention.
Interventions:
Group 1 (FMT-NJT): Active FMT via NJT + sham colonoscopy. Group 2 (FMT-C): Active FMT via colonoscopy with placement of a transendoscopic enteral tube (TET) in the cecum for subsequent infusions + sham NJT.
Group 3 (Control): Placebo via both NJT and colonoscopy (sham procedures). Dosage: 5 mL/kg (max 100 mL) per infusion, administered every other day for three sessions.
Blinding: Triple-blind-participants/guardians, outcome assessors, and data analysts are blinded. An independent pharmacy unit prepares identical active and placebo preparations.
Assessments: Behavioral scales (CARS-2, SRS-2, ABC, SSP, CSHQ), GI symptoms (GSRS), stool metagenomics, and safety monitoring at baseline, Weeks 2, 6, 12, 24, and 48.
Sample Size: 25 per group (total N=75), calculated to detect a 2.5-point difference in CARS change with 80% power, accounting for 15% dropout.
Randomization: Centralized block randomization stratified by age and baseline CARS severity.
Statistical Analysis: ANCOVA for primary outcome with baseline adjustment; mixed models for repeated measures; descriptive and inferential methods for secondary and safety outcomes.
Outcomes:
Primary: Change in CARS-2 total score from baseline to Week 24. Secondary: Changes in SRS-2, ABC, GSRS, SSP, CSHQ scores; microbiota composition/function; incidence and severity of adverse events (CTCAE v5.0).
Significance: This trial will provide high-level evidence on whether the therapeutic effect of FMT in ASD depends on the gastrointestinal delivery site. The novel TET-based protocol for repeated cecal delivery allows for a rigorous comparison of microbial engraftment in the lower versus upper GI tract. The results will guide the optimization of microbiota-targeted therapies for ASD and other conditions linked to the gut-brain axis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 (FMT-NJT) | Active comparator | Procedure: ctive FMT via Nasojejunal Tube (FMT-Upper GI)active FMT via nasojejunal tube + sham colonoscopy |
| Group 2 (FMT-C) | Active comparator | Procedure: Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI)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. |
| Group 3 (Control) | Placebo comparator | Procedure: Placebo via Sham Procedures (Sham-Control)placebo via nasojejunal tube + placebo via colonoscopy (sham procedures for both routes) |
Who may qualify
- Ages
- 3 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ctive 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. | Procedure or surgery | Group 1 (FMT-NJT) |
| Active 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. | Procedure or surgery | Group 2 (FMT-C) |
| Placebo 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. | Procedure or surgery | Group 3 (Control) |
Primary outcome
Change in Total Score on the Childhood Autism Rating Scale (CARS)
Time frame: Pre-FMT, 3 months post-FMT, and 6 months post-FMT
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.
10 secondary outcomes
Change in Total Score on the Social Responsiveness Scale, Second Edition (SRS-2)
Time frame: Pre-FMT, at Week 12, 24, and 48 post-FMT.
Change in Total Score on the Aberrant Behavior Checklist (ABC)
Time frame: Pre-FMT, at Week 12, 24, and 48 post-FMT.
Change in Total Score on the Gastrointestinal Symptom Rating Scale (GSRS)
Time frame: Pre-FMT, during FMT, and at 2, 6, 12, 24, and 48 post-FMT.
Daily Stool Record (DSR)
Time frame: 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)
Time frame: Pre-FMT, at12, 24 and 48 post-FMT.
Change in Total Score on the Children's Sleep Habits Questionnaire (CSHQ)
Time frame: Pre-FMT, at12, 24 and 48 post-FMT.
Change in Gut Microbiota Alpha Diversity (Shannon Index)
Time frame: Pre-FMT, and at 2, 6, 12, 24 and 48 weeks post-FMT.
Change in Relative Abundance of Specific Gut Microbial Taxa (at the genus level)
Time frame: Pre-FMT, and at 2, 6, 12, 24 and 48 weeks post-FMT.
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: From date of randomization until the end of study visit (Week 48).
Incidence of Serious Adverse Events (SAEs)
Time frame: From date of randomization until the end of study visit (Week 48).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2026.
- Results publication: PubMed 11112010Ghirardini A, Nanni-Costa A, Venturi S, Ridolfi L, Petrini F, Taddei S, Venturoli A, Pugliese MR, Monti M, Martinelli G. Efficiency of organ procurement and transplantation programs. Transpl Int. 2000;13 Suppl 1:S267-71. doi: 10.1007/s001470050339.
- Results publication: PubMed 39677507Li Y, Xiao P, Ding H, Wang H, Xu Q, Wang R, Zheng L, Song X, Wang Y, Zhang T. Fecal Microbiota Transplantation in Children with Autism. Neuropsychiatr Dis Treat. 2024 Dec 9;20:2391-2400. doi: 10.2147/NDT.S488001. eCollection 2024.
- Results publication: PubMed 28122648Kang DW, Adams JB, Gregory AC, Borody T, Chittick L, Fasano A, Khoruts A, Geis E, Maldonado J, McDonough-Means S, Pollard EL, Roux S, Sadowsky MJ, Lipson KS, Sullivan MB, Caporaso JG, Krajmalnik-Brown R. Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study. Microbiome. 2017 Jan 23;5(1):10. doi: 10.1186/s40168-016-0225-7.
- Results publication: PubMed 32100127Chen K, Fu Y, Wang Y, Liao L, Xu H, Zhang A, Zhang J, Fan L, Ren J, Fang B. Therapeutic Effects of the In Vitro Cultured Human Gut Microbiota as Transplants on Altering Gut Microbiota and Improving Symptoms Associated with Autism Spectrum Disorder. Microb Ecol. 2020 Aug;80(2):475-486. doi: 10.1007/s00248-020-01494-w. Epub 2020 Feb 26.
- Results publication: PubMed 9762410Flora SJ, Tripathi N. Hepatic and renal metallothionein induction following single oral administration of gallium arsenide in rats. Biochem Mol Biol Int. 1998 Sep;45(6):1121-7. doi: 10.1080/15216549800203342.
- Results publication: PubMed 34173726Tan Q, Orsso CE, Deehan EC, Kung JY, Tun HM, Wine E, Madsen KL, Zwaigenbaum L, Haqq AM. Probiotics, prebiotics, synbiotics, and fecal microbiota transplantation in the treatment of behavioral symptoms of autism spectrum disorder: A systematic review. Autism Res. 2021 Sep;14(9):1820-1836. doi: 10.1002/aur.2560. Epub 2021 Jun 26.
- Results publication: PubMed 38123016Goncalves CL, Doifode T, Rezende VL, Costa MA, Rhoads JM, Soutullo CA. The many faces of microbiota-gut-brain axis in autism spectrum disorder. Life Sci. 2024 Jan 15;337:122357. doi: 10.1016/j.lfs.2023.122357. Epub 2023 Dec 19.
- Results publication: PubMed 39144142Ullah H, Arbab S, Tian Y, Chen Y, Liu CQ, Li Q, Li K. Crosstalk between gut microbiota and host immune system and its response to traumatic injury. Front Immunol. 2024 Jul 31;15:1413485. doi: 10.3389/fimmu.2024.1413485. eCollection 2024.
- Results publication: PubMed 10954784Njeh CF, Fuerst T, Diessel E, Genant HK, Hausler KD. Association between QUS and structural parameters? Calcif Tissue Int. 2000 Sep;67(3):273-4. doi: 10.1007/s002230001136. No abstract available.
- Results publication: PubMed 37600019Ullah H, Arbab S, Tian Y, Liu CQ, Chen Y, Qijie L, Khan MIU, Hassan IU, Li K. The gut microbiota-brain axis in neurological disorder. Front Neurosci. 2023 Aug 4;17:1225875. doi: 10.3389/fnins.2023.1225875. eCollection 2023.
- Results publication: PubMed 37365313Morton JT, Jin DM, Mills RH, Shao Y, Rahman G, McDonald D, Zhu Q, Balaban M, Jiang Y, Cantrell K, Gonzalez A, Carmel J, Frankiensztajn LM, Martin-Brevet S, Berding K, Needham BD, Zurita MF, David M, Averina OV, Kovtun AS, Noto A, Mussap M, Wang M, Frank DN, Li E, Zhou W, Fanos V, Danilenko VN, Wall DP, Cardenas P, Baldeon ME, Jacquemont S, Koren O, Elliott E, Xavier RJ, Mazmanian SK, Knight R, Gilbert JA, Donovan SM, Lawley TD, Carpenter B, Bonneau R, Taroncher-Oldenburg G. Multi-level analysis of the gut-brain axis shows autism spectrum disorder-associated molecular and microbial profiles. Nat Neurosci. 2023 Jul;26(7):1208-1217. doi: 10.1038/s41593-023-01361-0. Epub 2023 Jun 26.
- Results publication: PubMed 39036341You M, Chen N, Yang Y, Cheng L, He H, Cai Y, Liu Y, Liu H, Hong G. The gut microbiota-brain axis in neurological disorders. MedComm (2020). 2024 Jul 20;5(8):e656. doi: 10.1002/mco2.656. eCollection 2024 Aug.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ctive FMT via Nasojejunal Tube (FMT-Upper GI) and Active FMT via Colonoscopy and Transendoscopic Tube (FMT-Lower GI) in people with Autism Spectrum Disorder, run by Shenzhen Children's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dongling Dai, PhD (08618938690736, daidong3529@email.szu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Total Score on the Childhood Autism Rating Scale (CARS) (time frame: Pre-FMT, 3 months post-FMT, and 6 months post-FMT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07381374, which also lists 19 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jun 2026.