Fecal Microbiota Transplant for Autism

Overseen By Sabine Hazan, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: ProgenaBiome

Must be taking: Vancomycin

Must not be taking: Non-study antibiotics, Opiates

Disqualifiers: Pregnancy, Rett syndrome, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether a fecal microbiota transplant, which introduces beneficial bacteria from a donor's stool into a patient's gut, can help adults with autism improve social skills and language. Participants will first undergo a 6-week course of the antibiotic Vancomycin to prepare for the transplant. The trial seeks adults diagnosed with autism who also experience digestive issues and currently receive community or school support for autism. The researchers aim to determine if these treatments can significantly enhance daily interactions and communication. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative approach.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you must refrain from taking non-study antibiotics and certain medications like loperamide, diphenoxylate/atropine, or cholestyramine. If you are on opiate treatment, you must be on a stable dose with no planned increase during the study.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that fecal microbiota transplant (FMT) has been explored as a treatment for autism spectrum disorder (ASD). This process involves transferring healthy gut bacteria from a donor to a patient to help balance their gut bacteria. Studies have found that FMT is generally safe for patients.

In earlier research, some patients experienced mild side effects like stomach discomfort and bloating, but these were temporary. FMT has also been used safely for other conditions like C. difficile infections, which supports its safety. However, since this is an early-phase study, researchers are closely monitoring the treatment to ensure its safety for participants. As with any medical treatment, there could be risks, so discussing any concerns with the study team is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Most treatments for autism focus on behavioral therapies and medications that target symptoms like irritability or hyperactivity. However, Fecal Microbiota Transplant (FMT) is unique because it targets the gut microbiome, which researchers believe may play a role in autism. This treatment involves transplanting healthy bacteria into the gut via colonoscopy, potentially improving gut health and, subsequently, behavior and cognitive function. Researchers are excited about FMT because it offers a novel approach that might address underlying biological factors rather than just the symptoms of autism.

What evidence suggests that fecal microbiota transplant might be an effective treatment for autism?

Research has shown that fecal microbiota transplant (FMT) might help people with autism spectrum disorder (ASD) by balancing the bacteria in the stomach and intestines. Studies have found that FMT can improve both gut health and autism symptoms. In one study with 328 patients, FMT led to positive results, suggesting it could help with behaviors related to ASD. Another study highlighted the long-term benefits of microbiota transfer therapy, showing promise for treating ASD in children with gut issues. These findings suggest that FMT could be a useful treatment for some autism symptoms. Participants in this trial will receive FMT administered by colonoscopy to evaluate its effectiveness in treating ASD. ^{1 2 3 6 7}

Who Is on the Research Team?

Sabine Hazan, MD

Principal Investigator

ProgenaBiome

Are You a Good Fit for This Trial?

This trial is for individuals at least two years old with a confirmed diagnosis of Autism Spectrum Disorder (ASD) who are stable on medications and receiving community or school interventions. Participants need a reliable caregiver to report side effects. Males with partners of childbearing potential must use birth control. Exclusions include pregnancy, nursing, unwilling caregivers, certain medical conditions like uncontrolled seizures, substance abuse, major surgeries like gastric bypass or total colectomy planned or done.

Inclusion Criteria

I am getting help for autism at school or in the community.

A reliable parent or caregiver who can report the side effects and communicate effectively with the research team

A reliable parent or caregiver who can report the side effects and communicate effectively with the research team.

Exclusion Criteria

I cannot stop taking my diarrhea medication before the study.

Hepatic values greater than 20

Unable to stop opiate treatment unless on a stable dose including PRN dosing, and no dose increase planned for the duration of the study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a 6-week treatment with Vancomycin

6 weeks

Fecal Microbiota Transplant

Participants are given fecal microbiota transplant by colonoscopy

1 week

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Fecal Microbiota Transplant

Trial Overview The study tests the effect of fecal microbiota transplant from family members in treating social deficits and language delays in ASD patients after a preliminary treatment with Vancomycin for six weeks. It's an open-label trial where everyone knows what treatment is being given.

How Is the Trial Designed?

Group I: Autism Subjects Experimental Treatment 1 Intervention

These subjects will be administered fecal microbiota transplant by colonoscopy

Fecal Microbiota Transplant is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Fecal Microbiota Transplantation for:

• Recurrent and refractory Clostridioides difficile infection

Approved in European Union as Faecal Microbiota Transplantation for:

• Recurrent and refractory Clostridioides difficile infection

Approved in Canada as Fecal Microbiota Transplantation for:

• Recurrent and refractory Clostridioides difficile infection

Who Is Running the Clinical Trial?

ProgenaBiome

Lead Sponsor

Trials

46

Recruited

5,400+

Microbiome Research Foundation

Collaborator

Ventura Clinical Trials

Collaborator

Trials

1

Recruited

30+

Published Research Related to This Trial

Fecal microbiota transplant (FMT) from tumor-bearing mice significantly improved cognitive function and short-term memory in Alzheimer's disease (AD) mice, indicating a potential therapeutic role for gut microbiota in cognitive impairment.

The FMT not only reduced Aβ plaque burden in the brain but also altered the gut microbiome composition, reversing specific microbial imbalances associated with AD, suggesting a complex interaction between gut health and cognitive function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transfer of Tumor-Bearing Mice Intestinal Flora Can Ameliorate Cognition in Alzheimer's Disease Mice. Hang, Z., Cai, S., Lei, T., et al.[2022]

Fecal microbiota transplantation (FMT) is highly effective for treating Clostridium difficile infection (CDI), with a 90% resolution of diarrhea reported in 867 patients across 33 studies, and a 94% resolution after repeated FMT in a randomized controlled trial with 16 participants.

FMT shows promise in treating ulcerative colitis, with remission rates varying from 0% to 68% in 106 patients, while its efficacy in Crohn's disease, chronic constipation, pouchitis, and irritable bowel syndrome remains inconclusive due to limited data.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation as novel therapy in gastroenterology: A systematic review. Rossen, NG., MacDonald, JK., de Vries, EM., et al.[2022]

Fecal microbiota transplantation (FMT) has a reported adverse event (AE) rate of 39.3%, primarily involving mild and self-limiting issues like abdominal pain and diarrhea, based on a meta-analysis of 9 high-quality studies involving 756 FMTs.

Serious adverse events (SAE) were relatively rare at 5.3%, with the most common being Clostridium difficile infection, indicating that while FMT is generally safe, monitoring and standardized reporting are essential for understanding its long-term safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events of fecal microbiota transplantation: a meta-analysis of high-quality studies. Michailidis, L., Currier, AC., Le, M., et al.[2022]

Fecal microbiota transplantation (FMT) is a generally safe and well-tolerated procedure for treating recurrent Clostridioides difficile infections, even in high-risk patients, with most short-term risks being mild and related to delivery methods.

Recent concerns have emerged regarding serious adverse events linked to FMT products from stool banks that do not screen for multi-drug resistant organisms, highlighting the need for improved safety protocols.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplantation: Is It Safe? Park, SY., Seo, GS.[2021]

Fecal microbiota transplantation (FMT) is an effective method for modifying the gut microbiome and has shown promise in treating various conditions, including Clostridiodes difficile infection (CDI) and other gastrointestinal and neurological disorders.

While FMT is currently approved for CDI in Taiwan, there is a growing interest in its potential applications for other diseases, particularly in children, where data is still limited.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current and future applications of fecal microbiota transplantation for children. Chen, CC., Chiu, CH.[2022]

Fecal microbiota transplantation (FMT) has a total adverse event (AE) incidence rate of 28.5%, with abdominal discomfort being the most common AE reported in 19 studies, highlighting the need for careful monitoring during the procedure.

Serious adverse events (SAEs) occurred in 9.2% of patients, with a notable incidence of death (3.5%) and infections (2.5%), indicating that while FMT can be beneficial, it carries significant risks that require further investigation through high-quality randomized controlled trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Systematic Review: Adverse Events of Fecal Microbiota Transplantation. Wang, S., Xu, M., Wang, W., et al.[2023]

Fecal microbiota transplantation (FMT) is primarily studied for conditions like Clostridium difficile infection and inflammatory bowel disease, with 84% of the 85 reports focusing on these areas, but most studies (87%) were non-randomized controlled trials.

Important methodological details about FMT, such as donor eligibility criteria and stool collection methods, are often poorly reported, which hinders the ability to replicate studies and fully understand the safety and efficacy of FMT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Methods and Reporting Studies Assessing Fecal Microbiota Transplantation: A Systematic Review. Bafeta, A., Yavchitz, A., Riveros, C., et al.[2022]

Fecal microbiota transplantation (FMT) is recommended for treating recurrent Clostridium difficile infections and has shown benefits for various gastrointestinal disorders, but safety data is still limited due to the rapid adoption of the technique without extensive long-term studies.

Capsulized FMT therapy offers a promising solution for long-term maintenance and reduces the need for invasive procedures, indicating a shift towards safer and more tolerable methods of delivering FMT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Current research progress and thinking of fecal microbiota transplantation for the treatment of gastrointestinal disorders]. Li, N., Tian, H.[2020]

Fecal microbiota transplantation (FMT) is primarily used to treat gastrointestinal diseases caused by harmful microorganisms, but it is also being explored for conditions like metabolic syndrome, diabetes, cancer, and Parkinson's disease.

The review discusses the effects of FMT on weight gain, immune response, and various diseases, while also addressing potential side effects that recipients may experience.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation in disease therapy. Antushevich, H.[2020]

Faecal microbiota transplantation (FMT) has proven effective for treating recurrent Clostridium difficile infections, with established protocols and donor stool banks in Denmark.

However, FMT is not recommended for routine use in treating inflammatory bowel disease or irritable bowel syndrome due to insufficient clinical trial data supporting its efficacy for these conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Faecal microbiota transplantation for the treatment of bowel disease]. Rode, AA., Chehri, M., Petersen, AM., et al.[2019]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10017995/

Effect of fecal microbiota transplantation in children with ... Fecal microbiota transplantation (FMT) may be helpful in the treatment of autism spectrum disorder (ASD) as rebalancing the gut microbiome has been shown to ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35105621/

FTACMT study protocol: a multicentre, double-blind ... FTACMT study protocol: a multicentre, double-blind, randomised, placebo-controlled trial of faecal microbiota transplantation for autism spectrum disorder.

3. sciencedirect.com sciencedirect.com/science/article/pii/S2590097825000199

Faecal microbiota transplantation as a novel approach for ... Fecal microbiome transplantation from children with autism ... outcomes of 328 patients with of autism spectrum disorder after fecal microbiota transplantation].

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06290258

Fecal Microbiota Transplantation for Patients With Autism ... This study aims to evaluate the efficacy of fecal microbiota transplantation on the gastrointestinal symptoms, autistic symptoms and emotional behavior ...

5. nature.com nature.com/articles/s41598-019-42183-0

Long-term benefit of Microbiota Transfer Therapy on autism ... Our observations demonstrate the long-term safety and efficacy of MTT as a potential therapy to treat children with ASD who have GI problems.

6. autism.org autism.org/editorial-fecal-microbiota-transplantation-and-autism/

Editorial - Fecal Microbiota Transplantation and Autism This editorial discusses the various aspects of FMT, describes potential benefits, and outlines critical issues that must be addressed.

7. bmjopen.bmj.com bmjopen.bmj.com/content/12/1/e051613

FTACMT study protocol: a multicentre, double-blind ... We are undertaking the first-ever multicentre, double-blind, randomised controlled trial of FMT for the treatment of children with both ASD and ...