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

50 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests a treatment called Microbiota Transfer Therapy (MTT) for children with Autism Spectrum Disorder and gut problems. It uses antibiotics to clear bad bacteria, cleans the bowel, and then adds good bacteria from donor stool over a few months. The goal is to see if this helps improve their symptoms. Microbiota Transfer Therapy (MTT) has shown significant improvements in gastrointestinal and autism symptoms in previous studies, with benefits persisting for some time after treatment.

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.

What data supports the effectiveness of the treatment Fecal Microbiota Transplantation (FMT) for autism and gastrointestinal issues?

Fecal Microbiota Transplantation (FMT) has shown promise in treating gastrointestinal diseases like Clostridium difficile infection and is being explored for neuropsychiatric disorders, including autism, due to its potential to improve gut health and related symptoms. Initial results are promising, but more research is needed to establish its effectiveness and safety for autism. ^{1 2 3 4 5}

Is fecal microbiota transplantation (FMT) safe for humans?

FMT is generally considered safe, with most side effects being mild and temporary, such as fever, abdominal pain, and bloating. However, there is limited data on long-term safety, and more research is needed to fully understand its safety profile, especially in vulnerable populations like children and immunocompromised individuals. ^{2 6 7 8 9}

How does Microbiota Transfer Therapy differ from other treatments for autism and gastrointestinal issues?

Microbiota Transfer Therapy (MTT) is unique because it involves transferring healthy gut bacteria from a donor to a patient, aiming to rebalance the gut microbiome, which may improve both gastrointestinal and autism symptoms. Unlike traditional treatments, MTT targets the gut bacteria directly, potentially offering long-term benefits by increasing microbial diversity and altering the gut ecosystem. ^{4 10 11 12 13}

Research Team

James B Adams, PhD

Principal Investigator

Arizona State University

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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) 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]

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]

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]

References

1. Korea (South) pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplantation and Its Usage in Neuropsychiatric Disorders. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplant in Recurrent Clostridium Difficile Infections: A Systematic Review. [2022]

3. China pubmed.ncbi.nlm.nih.gov

Encyclopedia of fecal microbiota transplantation: a review of effectiveness in the treatment of 85 diseases. [2023]

4. England pubmed.ncbi.nlm.nih.gov

FTACMT study protocol: a multicentre, double-blind, randomised, placebo-controlled trial of faecal microbiota transplantation for autism spectrum disorder. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Human microbiome: From the bathroom to the bedside. [2020]

6. Germany pubmed.ncbi.nlm.nih.gov

[Fecal Microbiota Transfer (FMT) in Children and Adolescents - Review and statement by the GPGE microbiome working group]. [2022]

7. China pubmed.ncbi.nlm.nih.gov

[Current research progress and thinking of fecal microbiota transplantation for the treatment of gastrointestinal disorders]. [2020]

8. England pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation for refractory diarrhea in immunocompromised diseases: a pediatric case report. [2020]

9. England pubmed.ncbi.nlm.nih.gov

Faecal microbiota transplantation: establishment of a clinical application framework. [2022]

10. England pubmed.ncbi.nlm.nih.gov

Microbiota transplant therapy and autism: lessons for the clinic. [2020]

11. Switzerland pubmed.ncbi.nlm.nih.gov

Effect of fecal microbiota transplantation in children with autism spectrum disorder: A systematic review. [2023]

12. England pubmed.ncbi.nlm.nih.gov

Long-term benefit of Microbiota Transfer Therapy on autism symptoms and gut microbiota. [2021]

13. England pubmed.ncbi.nlm.nih.gov

Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study. [2023]