Austistic spectrum… · Phase II (2025-520539-17-00)

Status Authorised, recruitment pending · 1 EU/EEA countries · 2 sites · Protocol APHP241418

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)

Status Authorised, recruitment pending

Participants planned 5

Countries 1

Sites 2

Austistic spectrum disorder

Assess initial efficacy on gastrointestinal symptoms of fecal microbiota transplantation in children (36 to 72 months) with autism spectrum disorders at week 18

Key facts

Sponsor: Assistance Publique Hopitaux De Paris
Participant type: Pediatric, Patients
Age range: 0-17 years
Gender: Male and Female
Therapeutic area: Phenomena and Processes [G] - Digestive System and Oral Physiological Phenomena [G10], Psychiatry and Psychology [F] - Mental Disorders [F03]
Decision date (initial): 2025-05-07
Transition trial: No
Low-intervention: No
Rare-disease indication: No
Vulnerable population: Yes
Funding sources: Fondation Erié · Fondation Initiatives Autisme · Biocodex Microbiota Foundation

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

Assess initial efficacy on gastrointestinal symptoms of fecal microbiota transplantation in children (36 to 72 months) with autism spectrum disorders at week 18

Secondary objectives 5

Estimate changes in autistic core symptoms
Estimate FMT safety: side effects occurrence
Estimate FMT tolerability
Estimate FMT impact on children and parent’s quality of life
Measure impact on GM diversity and immune markers

Conditions and MedDRA coding

Austistic spectrum disorder

Version	Level	Code	Term	System organ class
20.1	PT	10017944	Gastrointestinal disorder	100000004856
21.1	PT	10063844	Autism spectrum disorder	100000004873

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

ASD diagnosis based on DSM-5 criteria
Gastrointestinal symptoms: at least 1 a score of 5 on 1 of the 5 GSRS domains (diarrhea, abdominal pain, constipation, indigestion, reflux) for over a period of 1 year or more
Age between 36 and 72 months
Consent from all legal guardians
Social insurance affiliation

Exclusion criteria 13

Severe intellectual deficit (IQ<30) or another uncontrolled comorbidity (e.g. epilepsy)
Recent change in ASD global care (cognitive behavioral therapy 6 weeks before inclusion)
Actual or past psychotropic medication
Contraindications to colon preparation (Picoprep®) and/or to Meopa®)
Severe or uncontrolled gastrointestinal symptoms (e.g. urgent treatment needed)
Undernutrition (age related BMI < 3rd percentile)
Inflammatory bowel disease
Exclusive diet (gluten- or sugar-free)
Rectal enema contraindication
Antibiotic or antifungal therapy 3 months before inclusion
Probiotics 2 months before inclusion or FMT 12 months before inclusion
Vancomycin or Picoprep® or Meopa® (including excipients) known allergy or intolerance
Experimental therapeutic trial participation 3 months before inclusion

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

Rate of children with a success, determined by the reduction of more than 50% of the composite score in the Gastrointestinal Symptoms Rating Scale (GSRS) evaluated by parents between inclusion and week 18. GSRS is validated in French and evaluates the intensity of 5 gastrointestinal symptoms (diarrhea, abdominal pain, constipation, indigestion, and reflux).

Secondary endpoints 4

Improvement on autistic core symptoms and global improvement [at W0, W4, W18, W24, M12] by multiples questionnaires and scales
FMT side effects (antibiotic therapy, GM transplantation from a healthy donor): Pediatric Adverse Event Rating Scale [evaluated by the pediatrician at day (D) 0, D5, W4, W18, W24, M12]
Adherence to instruction to be followed during the FMT process, drop out numbers, reasons of drop out, acceptability questionnaire - Stool consistency: Bristol Stool Form Scale [by parents from W0 to W4 and from W17 to W18] and global improvement: Caregiver Strain index [by a psychologist at W0, W4, W18, W24, M12]
Pediatric Quality of Life Inventory (PedsQL) [at W0, W18, M12]

Investigational products

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

Test 1

Enema transplant of fecal microbiota 25g/100mL

PRD11975721 · Product

Active substance: Allogeneic Faecal Microbiota, Pooled
Substance synonyms: MaaT 033, Allogeneic fecal microbiota, pooled, Pooled allogeneic faecal microbiota
Pharmaceutical form: ENEMA
Route of administration: RECTAL USE
Max daily dose: 25 g gram(s)
Max total dose: 75 g gram(s)
Max treatment duration: 14 Day(s)
Authorisation status: Not Authorised
MA holder: ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS
Paediatric formulation: No
Orphan designation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation: Assistance Publique Hopitaux De Paris
Address: Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux
City: Paris Cedex 10
Postcode: 75475
Country: France

Scientific contact point

Organisation: Assistance Publique Hopitaux De Paris
Contact name: Pierre Ellul

Public contact point

Organisation: Assistance Publique Hopitaux De Paris
Contact name: Thibaut Vanrietvelde

Locations

1 EU/EEA country · 2 investigational sites

By country

Country	MS status	Planned subjects	Sites
France	Authorised, recruitment pending	5	2
Rest of world	—	0	—

Investigational sites

France

2 sites · Authorised, recruitment pending

Hôpital Robert Debré

Pedopsychiatry, 48 Boulevard Sérurier, 75019, Paris