Microbiota Transfer Therapy for Autism and Gastrointestinal Issues

Overseen By James B Adams, PhD

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Arizona State University

Must not be taking: Antibiotics, Probiotics, Psychotropics, others

Disqualifiers: Single-gene disorders, Major brain malformation, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 14 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment called Microbiota Transfer Therapy (MTT) for children with Autism Spectrum Disorder (ASD) who also experience gastrointestinal issues like constipation or diarrhea. The treatment involves taking the antibiotic vancomycin, followed by a bowel cleanse, and then receiving healthy gut bacteria (known as Fecal Microbiota Transplantation or FMT) over 12 weeks. Two groups participate: one receives the full treatment, while the other receives placebos (inactive substances) for comparison. Children aged 5-17 with a diagnosis of autism and ongoing gastrointestinal problems for at least three years may be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that there be no changes in medications, supplements, diet, therapies, or education in the last 3 months and no intention to change them during the trial. If you are taking psychotropic medications that affect gastrointestinal function, you must have stopped them at least 2 months before joining the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

A previous study found fecal microbiota transplantation (FMT) to be safe and effective for children with autism spectrum disorder (ASD) who also have gut problems. Researchers observed improvements in both digestive and behavioral symptoms after FMT, indicating it is well-tolerated.

Research has shown that vancomycin, an antibiotic used in this trial, improved ASD behaviors during treatment, although these benefits often decreased after stopping the medication. Other studies have used it without major side effects, making it a good short-term treatment option.

Overall, both FMT and vancomycin appear safe and well-tolerated based on previous research, with encouraging results for improving symptoms in children with ASD. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about Microbiota Transfer Therapy because it offers a novel way to address autism and gastrointestinal issues by restoring gut health. Unlike standard treatments that focus on managing symptoms, this therapy uses fecal microbiota to potentially alter the gut environment, which could improve both digestive and behavioral symptoms. Additionally, the use of vancomycin and Miralax helps prepare the gut to better accept and maintain the new microbiota, which might enhance the treatment's effectiveness. This approach may provide a more comprehensive improvement in symptoms, which is why researchers are hopeful about its potential.

What evidence suggests that this trial's treatments could be effective for Autism and gastrointestinal issues?

This trial will compare the effects of Fecal Microbiota Transplantation (FMT) and vancomycin on individuals with Autism Spectrum Disorder (ASD) and gastrointestinal issues. Research has shown that FMT can improve both digestive and behavioral symptoms in people with ASD by restoring gut bacteria balance. Participants in one arm of this trial will receive vancomycin followed by FMT. Some research indicates that vancomycin can temporarily improve digestive and ASD symptoms by reducing harmful gut bacteria, but more studies are needed to confirm its long-term benefits. Both treatments in this trial aim to enhance gut health, potentially leading to better overall outcomes for people with ASD. ^{1 2 3 5 6}

Who Is on the Research Team?

James B Adams, PhD

Principal Investigator

Arizona State University

Are You a Good Fit for This Trial?

This trial is for children aged 5-17 with Autism Spectrum Disorder and gastrointestinal issues like constipation or diarrhea. They must be able to swallow pills, have stable health aside from GI problems, and not plan any treatment changes during the study. Kids on psychotropic meds, recent antibiotics, probiotics, or those with severe GI conditions are excluded.

Inclusion Criteria

I have had a gastrointestinal disorder for at least 3 years.

I have been diagnosed with autism based on specific assessments.

You can swallow pills without chewing them.

Exclusion Criteria

I have not taken any antibiotics in the last 3 months, except for creams or ointments.

I have been diagnosed with a chronic inflammatory bowel condition.

You are extremely underweight or malnourished, as determined by your doctor.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a 2-week treatment with oral vancomycin or placebo, followed by 1 day of Miralax, then 2 days of high-dose FM or placebo, and 12 weeks of maintenance dose FM or placebo

14 weeks

Extension

Group A undergoes a 12-week observation period, while Group B receives treatment similar to Part 1 without vancomycin

12 weeks

Follow-up

Participants are monitored for long-term safety and efficacy at 6, 12, and 18 months after treatment ends

18 months

What Are the Treatments Tested in This Trial?

Interventions

• Fecal Microbiota
• Miralax
• placebo intestinal microbiota
• placebo vancomycin
• vancomycin

Trial Overview The trial tests Microbiota Transfer Therapy (MTT) in kids with ASD and GI disorders. It starts with a 10-day course of vancomycin (an antibiotic), followed by a bowel cleanse, then a 12-week Fecal Microbiota treatment. Some participants will receive placebos instead of actual treatments for comparison.

How Is the Trial Designed?

Group I: Group A - Treatment Group Experimental Treatment 1 Intervention

This group will receive vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota for 2 days at high dose, then intestinal microbiota for 12 weeks at a maintenance dose

Group II: Group B - Control Group (Miralax only for 1 day) Placebo Group 1 Intervention

This group will receive placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota for 2 days at high dose, then placebo intestinal microbiota for 12 weeks at a maintenance dose

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

Approved in United States as Fecal Microbiota Transplantation for:

• Recurrent Clostridioides difficile (C. diff) infection

Approved in European Union as Fecal Microbiota Transplantation for:

• Recurrent Clostridioides difficile (C. diff) infection

Approved in Canada as Fecal Microbiota Transplantation for:

• Recurrent Clostridioides difficile (C. diff) infection

Who Is Running the Clinical Trial?

Arizona State University

Lead Sponsor

Trials

311

Recruited

109,000+

University of Minnesota

Collaborator

Trials

1,459

Recruited

1,623,000+

Finch Therapeutics

Collaborator

Trials

1

Recruited

50+

Arizona State University Foundation

Collaborator

Trials

1

Recruited

50+

Published Research Related to This Trial

Fecal microbiota transplantation (FMT) was successfully performed in two immunocompromised children with refractory diarrhea, leading to symptom remission without any infectious complications.

The procedure resulted in the reconstruction of a diverse microbiota, indicating that FMT is a safe and effective treatment option for managing diarrhea in immunocompromised patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation for refractory diarrhea in immunocompromised diseases: a pediatric case report. Zhong, S., Zeng, J., Deng, Z., et al.[2020]

Fecal Microbiota Transplantation (FMT) has shown high efficacy rates (68% to 93%) in treating recurrent Clostridium difficile infections, particularly in elderly patients with multiple health issues and in younger individuals with inflammatory bowel disease.

The procedure is generally safe, with only minor side effects reported, such as fever and abdominal discomfort, and no significant increase in the incidence of other diseases, indicating a favorable safety profile for FMT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplant in Recurrent Clostridium Difficile Infections: A Systematic Review. Gupta, K., Tappiti, M., Nazir, AM., et al.[2022]

Faecal microbiota transplantation (FMT) is being established as a safe second-line treatment for recurrent Clostridium difficile infections, with a comprehensive framework developed to ensure donor screening and clinical application comply with the European Tissue Act.

The framework includes rigorous donor recruitment and screening processes, laboratory protocols for processing faecal samples, and a structured follow-up system to monitor patient outcomes, ensuring safety and traceability in FMT procedures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Faecal microbiota transplantation: establishment of a clinical application framework. Jørgensen, SMD., Hansen, MM., Erikstrup, 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) has shown clinical benefits in treating a wide range of dysbiosis-related diseases, with 782 studies reviewed covering 85 specific conditions, indicating its potential effectiveness beyond just gut health.

Innovative methodologies like washed microbiota transplantation (WMT) and colonic transendoscopic enteral tubing (TET) are enhancing the delivery and efficacy of FMT, suggesting a promising future for this therapy in various medical fields.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Encyclopedia of fecal microbiota transplantation: a review of effectiveness in the treatment of 85 diseases. Wang, Y., Zhang, S., Borody, TJ., et al.[2023]

Microbiota Transplant Therapy (MTT) has shown to be relatively safe and effective in reducing gastrointestinal disorders and autism symptoms in children, as indicated by an open-label study and a two-year follow-up.

MTT resulted in significant changes to the gut microbiome structure and increased microbial diversity, suggesting a potential mechanism for its therapeutic effects in individuals with autism spectrum disorders and chronic gastrointestinal issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Microbiota transplant therapy and autism: lessons for the clinic. Adams, JB., Borody, TJ., Kang, DW., et al.[2020]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9762410/

Fecal Microbiota Transplantation in Autism Spectrum Disorder This review paper will therefore focus on FMT in the treatment of ASD, and FMT is effective in improving gastrointestinal and neurobehavioral symptoms in ...

2. frontiersin.org frontiersin.org/journals/cellular-and-infection-microbiology/articles/10.3389/fcimb.2021.759435/full

Fecal Microbiota Transplantation Relieves Gastrointestinal ... Our data suggest that FMT might be a promising therapeutic strategy to improve the GI and behavioral symptoms of patients with ASD.

3. 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 ...

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

Faecal microbiota transplantation as a novel approach for ... This review critically examines the existing evidence, explores the mechanistic pathways linking gut microbiota to anxiety in ASD, and discusses the future ...

5. 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 ...

6. 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.