Effect of faecal microbiota transplant on the expression of autism spectrum disorder core symptoms in children – open-label phase II clinical trial (FMT-PAS)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Masarykova Univerzita

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10], Psychiatry and Psychology [F] - Mental Disorders [F03]

Decision date (initial)

2026-03-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Agency for Health Research of the Czech Republic (Ministry of Health of the Czech Republic) · Masaryk University

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

The primary objective of the trial is to describe the effect of faecal microbiota transplant (FMT) on autism spectrum disorder core symptoms.

Secondary objectives 4

1. Description of the safety profile of FMT
2. Description of the effect of FMT on gastrointestinal symptoms using Bristol stool scale and ATN-GISSI-17 questionnaire
3. Description of the effect of FMT on the quality of life (PEDS-QL)
4. Description of the effect of FMT on other psychological, social and behavioural symptoms (CARS2, SEQ, SDQ)

Conditions and MedDRA coding

Autism spectrum disorder

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Age 3–12 years
2. Diagnosis of ASD confirmed by a clinical psychologist or psychiatrist
3. Previous ADOS-2 examination result in the ASD range
4. Only minimum verbal ability (i.e. corresponding to ADOS-2, Module 1)
5. Informed consent of the parent (legal representative) and willingness to follow all planned study visits and instructions, especially for bowel preparation

Exclusion criteria 10

1. Congenital GIT defect or primary GIT disease, incl. medically confirmed coeliac disease (gluten enteropathy)
2. Early reaction to food in the patient’s medical history
3. Expected inability to undergo bowel preparation (e.g. child’s inability to use liquid medication declared by parent)
4. Contraindication for common sedative medication, or serious complications of sedation in the patient’s medical history
5. Use of systemic immunosuppressants at present or in the last 3 months, or present medically confirmed immunocompromised status
6. Use of antiplatelet drugs, anticoagulants, or systemic antibiotics at present or in the last 3 months
7. Use of probiotics, prebiotics, or postbiotics at present or in the last 3 months
8. Severe co-morbid conditions (e.g. psychosis, epilepsy uncontrolled by medication, presence of severe visual or hearing impairment) that may interact with study procedures (at discretion of the investigator)
9. Newly initiated or recently changed pharmacotherapy (less than 30 days before enrolment)
10. Newly initiated or recently changed formal behavioural, cognitive or cognitive-behavioural therapy (less than 30 days before enrolment)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The treatment response (improvement/worsening) will be defined as ± 2 calibrated severity scores (CSS) in ADOS-2, Module 1 examination, which quantifies core symptoms intensity.

Secondary endpoints 4

1. Adverse events of FMT
2. Bristol stool scale score, ATN-GISSI-17 questionnaire score
3. PEDS-QL questionnaire score
4. Scores in CARS2, SEQ and SDQ questionnaires

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Masarykova Univerzita

Sponsor organisation

Masarykova Univerzita

Address

Zerotinovo Namesti 617/9, Brno-Mesto Brno-Mesto

City

Brno-Stred

Postcode

602 00

Country

Czechia

Scientific contact point

Organisation

Masarykova Univerzita

Contact name

Regina Demlová

Public contact point

Organisation

Masarykova Univerzita

Contact name

Regina Demlová

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications