Microbiota Transfer Therapy for Autism and Gastrointestinal Disorders

Overseen By James B Adams

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Gut-Brain-Axis Therapeutics Inc.

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

Disqualifiers: Single-gene disorder, Major brain malformation, Severe GI problems, others

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

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

Trial Summary

What is the purpose of this trial?

The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Autism-spectrum disorder with Gastrointestinal disorders (constipation, diarrhea, and/or abdominal pain). MTT involves a combination of 14 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using Miralax, followed by 5 days of high dose MTP-101P with an antacid, followed by 12 weeks of a lower maintenance dose of MTP-101P with an antacid.

Will I have to stop taking my current medications?

The trial requires that you do not change your medications, supplements, diet, therapies, or education for 2 months before starting and during the trial. If you are taking psychotropic medications (drugs affecting mood or behavior) that interfere with gut function, you must stop them at least 2 months before starting the trial.

What data supports the effectiveness of the treatment Microbiota Transplant Therapy for autism and gastrointestinal disorders?

Research suggests that Microbiota Transplant Therapy (MTT) can significantly reduce gastrointestinal issues and autism symptoms by changing the gut microbiome. Studies have shown that MTT is relatively safe and effective, with improvements in symptoms maintained even two years after treatment. ^{1 2 3 4 5}

Is Microbiota Transfer Therapy safe for humans?

Microbiota Transfer Therapy (MTT) has been shown to be relatively safe in studies, with most adverse reactions being mild to moderate, such as abdominal discomfort, nausea, and diarrhea, which improved after stopping the treatment. No serious adverse reactions were reported in the studies reviewed. ^{1 5 6 7 8}

How is Microbiota Transplant Therapy different from other treatments for autism and gastrointestinal disorders?

Microbiota Transplant 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-related symptoms. Unlike other treatments, MTT combines antibiotics, a bowel cleanse, and a stomach-acid suppressant with the transplant, and has shown long-term improvements in symptoms and gut health. ^{1 2 3 5 9}

Eligibility Criteria

This trial is for children with Autism Spectrum Disorder who also have gastrointestinal issues like constipation, diarrhea, or abdominal pain. Participants must meet specific criteria that the study outlines.

Inclusion Criteria

I haven't changed my medications, supplements, diet, or therapies in the last 2 months.

I am in good health except for issues with my digestive system.

My child, aged 4-17, is diagnosed with autism.

Exclusion Criteria

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

I have had recent surgery or have certain stomach or intestine conditions.

Current participation in other clinical trials

Timeline

Screening

Participants are screened for eligibility to participate in the trial

0-120 days

Placebo-Controlled Treatment

Randomized, double-blind, placebo-controlled trial with 14 days of oral vancomycin, 1 day of bowel cleanse using Miralax, followed by 5 days of high dose MTP-101P with an antacid, and 12 weeks of a lower maintenance dose of MTP-101P with an antacid

15 weeks

Open-Label Observation and Cross-Over

Observation for Groups A and B; Groups C and D receive treatment similar to Groups A and B respectively

12 weeks

Follow-up

Participants are monitored for long-term efficacy and possible adverse effects

15 weeks

Treatment Details

Interventions

• Microbiota Transplant Therapy

Trial Overview The study tests Microbiota Transfer Therapy (MTT), which includes a 14-day vancomycin course to remove harmful bacteria, a day of bowel cleanse with Miralax, then high dose MTP-101P with an antacid for five days followed by a maintenance dose for 12 weeks.

Participant Groups

Group I: Group B: Treatment (Dose 2) Experimental Treatment 4 Interventions

Part 1: Blinded Treatment (15 weeks) Vancomycin, Miralax, Antacid, MTP-101P

Group II: Group A: Treatment (Dose 1) Experimental Treatment 4 Interventions

Part 1: Blinded Treatment (15 weeks) Vancomycin, Miralax, Antacid, MTP-101P

Group III: Group D: Placebo Placebo Group 3 Interventions

Part 1: Blinded Placebo (15 weeks) Placebo Vancomycin, Real Miralax, Real Antacid, Placebo MTP-101P

Group IV: Group C: Placebo Placebo Group 3 Interventions

Part 1: Blinded Placebo (15 weeks) Placebo Vancomycin, Real Miralax, Real Antacid, Placebo MTP-101P

Who Is Running the Clinical Trial?

Gut-Brain-Axis Therapeutics Inc.

Lead Sponsor

Trials

2

Recruited

80+

Findings from Research

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]

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) in children has an overall effective rate of 85.75%, with a particularly high efficacy of 91.22% for treating Clostridium difficile infections, indicating that FMT is a promising treatment option.

While the overall adverse event rate associated with FMT is 28.86%, most events are mild to moderate, such as bellyache and diarrhea, and are typically self-limiting, suggesting that FMT is generally safe for use in children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The global incidence of adverse events associated with fecal microbiota transplantation in children over the past 20 years: A systematic review and meta-analysis. Wang, JG., Liang, Q., Dou, HH., et al.[2022]

Washed microbiota transplantation (WMT) using colonic transendoscopic enteral tubing (TET) was successfully performed in 38 children with autism spectrum disorder (ASD), achieving a 100% success rate across 124 procedures.

The TET method was found to be safe, with no severe adverse events reported, and parents expressed high satisfaction with the procedure, indicating its feasibility for treating symptoms associated with ASD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Colonic Endoscopic Tubing Is Safe and Effective Approach for Washed Microbiota Transplantation in Autistic Children. Yuan, QF., Wu, HY., Chen, XY., et al.[2023]

References

1. England pubmed.ncbi.nlm.nih.gov

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

2. Switzerland pubmed.ncbi.nlm.nih.gov

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

3. 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]

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

Fecal microbiota transplantation in children: current concepts. [2020]

5. England pubmed.ncbi.nlm.nih.gov

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

6. China pubmed.ncbi.nlm.nih.gov

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

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

[Long-term outcomes of 328 patients with of autism spectrum disorder after fecal microbiota transplantation]. [2022]

8. Australia pubmed.ncbi.nlm.nih.gov

The global incidence of adverse events associated with fecal microbiota transplantation in children over the past 20 years: A systematic review and meta-analysis. [2022]

9. Egypt pubmed.ncbi.nlm.nih.gov

Colonic Endoscopic Tubing Is Safe and Effective Approach for Washed Microbiota Transplantation in Autistic Children. [2023]