Not yet recruitingPHASE2INTERVENTIONAL

Open Label Phase IIa Trial Evaluating the Effect and Safety of Transplantation of Fecal Microbiota in Children With Autism Spectrum Disorders and Gastrointestinal Symptoms

This study is investigating a treatment called Faecal Microbiota Transplantation (FMT) for children aged 3 to 6 who have both autism and ongoing tummy issues. We know that about a third of children with autism also experience gut problems, and there's growing evidence that the bacteria in our gut can influence both our digestion and brain. In this study, doctors want to see if transplanting healthy gut bacteria can improve not only these gut symptoms but potentially also social communication skills in young children with autism. It's an important step in understanding the connection between the gut and brain, building on earlier research that showed promise in adults.

At a glance

Status

Not yet recruiting

Phase

PHASE2

Sponsor

Assistance Publique - Hôpitaux de Paris

Enrolment target

Start

01 Sep 2025

Estimated completion

01 Apr 2027

Autism Spectrum Disorder Fecal Microbiota Transplantation (FMT)

What is this study about?

This research study is focused on children aged between 3 and 6 years old who have been diagnosed with Autism Spectrum Disorder (ASD) and also experience ongoing digestive problems, often called gastrointestinal (GI) symptoms. We know that ASD can affect how children communicate and interact with others, and some children also struggle with tummy troubles like constipation or diarrhoea. Recent research suggests there might be a link between the bacteria living in our gut – often called the gut microbiota – and how our brain works.

The main idea of this study is to explore if a treatment called Faecal Microbiota Transplantation (FMT) can help these children. FMT involves carefully transferring healthy gut bacteria from a donor to the child. The aim is to see if this can improve their tummy troubles and potentially even have a positive effect on their social and communication skills. Previous studies, especially in adults with ASD, have shown some promising results, hinting that changing the gut bacteria could make a difference.

This is a 'Phase IIa' study, which means it's an early-stage trial. Doctors are looking closely at how effective the treatment might be and also checking very carefully for any potential side effects to make sure it's safe. It's a key step in understanding whether FMT could be a helpful treatment option for this group of children in the future.

Key takeaways

This study is for children aged 3-6 with autism and persistent gut problems.
It investigates if 'Faecal Microbiota Transplantation' (FMT) can help tummy troubles and social skills.
FMT involves taking antibiotics, a laxative, and then receiving healthy donor stool via enema.
Participation involves hospital visits for treatments and assessments, plus phone check-ins.
It's an early-stage study to check effectiveness and safety.
You can withdraw your child from the study at any time.

Who may be eligible?

To join this study, children need to be between 3 and 6 years old and have a diagnosis of Autism Spectrum Disorder. They must also have experienced gut problems, like diarrhoea, tummy pain, or constipation, fairly regularly for at least a year.

There are also some reasons why a child might not be able to join. For example, if they have a very severe learning disability or another serious health problem like uncontrolled epilepsy. If they've recently started a new autism therapy or taken certain medications (like antibiotics or some mental health medicines) in the last few months, they also wouldn't be able to participate. Conditions like severe gut problems needing urgent care, being underweight, or having inflammatory bowel disease would also prevent a child from joining.

Before taking part, doctors will confirm your child doesn't have any allergies to the medications used in the study, and that they haven't been in another research study recently. Also, children currently on very strict diets (like gluten-free or sugar-free) would not be eligible.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child aged between 3 and 6 years old?
Does your child have a diagnosis of Autism Spectrum Disorder?
Has your child had ongoing gut problems (like tummy ache, diarrhoea, or constipation) for a year or more?
Does your child not have a very severe learning disability or uncontrolled fits (epilepsy)?
Has your child not just started a new autism therapy or taken certain medications (like antibiotics) recently?
Are you able to provide consent for your child to join the study?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, you'll first have a meeting at Robert Debré hospital to learn all about it and ask any questions. If you decide to proceed and give your consent, a child psychiatrist will confirm this about 1 to 2 weeks later.

Your child will then have several visits to the hospital (at the start, then at weeks 4, 18, 24, and month 12). During these visits, they will receive the FMT treatment, and doctors will check on their health, how well the treatment is working, and if there are any side effects. They will also collect stool, urine, and blood samples. Between these visits, there will be two phone calls (at week 11 and month 9) to check in on their safety and how acceptable the treatment is, mostly by asking you, the parents, some questions.

The FMT treatment itself involves taking an antibiotic for four days, followed by a special laxative. Then, healthy donor stool will be given as an enema (a liquid put into the bottom) three times: on day 5, day 12, and day 19. The first antibiotic dose and all three enema treatments will happen at the hospital, with the other antibiotic doses and the laxative taken at home. The study will last for approximately 12 months.

Potential risks and benefits

Participating in this study might benefit your child by potentially improving their long-standing gut problems and possibly even their social communication skills, based on early research. However, it's important to remember that this is a research study, and we don't yet know for sure if the treatment will be effective or safe for all children. Potential risks could include side effects from the antibiotic or laxative used before the FMT, or discomfort from the enema procedure. There's also a chance of side effects from the FMT itself, although healthy donor stools are carefully screened. You have the right to withdraw your child from the study at any time, for any reason, without affecting their future medical care.

Locations (1)

Robert Debré Hospital
Verified postcode
Paris, France

Common questions

What is Faecal Microbiota Transplantation (FMT)?

FMT is a treatment where healthy gut bacteria from a screened donor's stool are transferred to another person's gut, usually to restore a healthy balance of bacteria.

What kind of gut problems are you looking at?

We're focusing on common tummy troubles like diarrhoea, constipation, tummy aches, indigestion, or reflux that have been ongoing for at least a year.

Will my child need to stay in the hospital?

Most of the treatment happens at home, but your child will need to visit the hospital for the first antibiotic dose and all three FMT enema treatments, as well as for follow-up appointments.

How long will the study last for my child?

Your child's participation in the study will last for about 12 months, including initial treatments and follow-up checks.

What if we change our minds about participating?

You are free to withdraw your child from the study at any time, for any reason, without it affecting their other medical care.

How to find out more

Pierre ELLUL, MD

pierre.ellul@aphp.fr 0033140034635 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.