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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Microbiota Transfer Therapy (MTT) for children with autism who also experience gut problems like constipation, diarrhea, or belly pain. The treatment involves taking an antibiotic, undergoing a bowel cleanse, and then using a special mix of gut bacteria to see if it improves both autism symptoms and gut issues. Participants will join one of several groups, trying different doses or a placebo (inactive treatment). Children who have had autism and gut issues for over a year and haven't found relief from standard treatments might be suitable for this trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to early findings.

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.

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

Research shows that Microbiota Transfer Therapy (MTT) is generally safe and well-tolerated for people with autism spectrum disorder (ASD) and digestive issues. Studies have found that MTT can help balance gut bacteria, potentially improving both digestive and ASD symptoms. For example, one study found that MTT had long-term safety benefits for children with ASD who have digestive problems. Another small trial noted that the therapy positively changed gut bacteria without causing serious side effects.

In the trial under consideration, MTT involves using antibiotics like vancomycin and treatments such as Miralax, an antacid, and MTP-101P. Similar studies have used these treatments, reporting no major safety concerns. However, as this is a Phase 2 trial, the research process is still in its early stages. The researchers are still ensuring the therapy's safety and effectiveness for a broader population. ^{1 2 3 4 5}

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

Researchers are excited about Microbiota Transplant Therapy because it offers a new approach for treating autism and gastrointestinal disorders by targeting the gut microbiome. Unlike traditional treatments that might focus on alleviating symptoms with medications, this therapy aims to restore a healthy balance of gut bacteria, potentially addressing underlying causes rather than just symptoms. By using a combination of antibiotics, laxatives, and antacids to prepare the gut, followed by introducing beneficial microbes, this method could lead to more comprehensive and lasting improvements in both gut health and behavioral symptoms associated with autism.

What evidence suggests that Microbiota Transplant Therapy could be an effective treatment for autism and gastrointestinal disorders?

Research has shown that Microbiota Transfer Therapy (MTT) can improve symptoms in people with autism spectrum disorder (ASD) who experience stomach issues like constipation and diarrhea. Studies have found that MTT may enhance both gut health and behaviors linked to autism. For example, one study found that participants continued to have better stomach health and saw further improvements in autism-related behaviors after the treatment ended. Another study suggested that MTT could be a promising way to ease both stomach and behavioral symptoms in people with ASD. Overall, the therapy aims to restore a healthy balance of bacteria in the gut, which is believed to affect both digestion and behavior.

In this trial, researchers will assign participants to different groups to receive varying doses of MTT or a placebo. This approach allows them to evaluate the therapy's effectiveness in improving symptoms associated with ASD and gastrointestinal disorders. ^{3 4 6 7 8}

Are You a Good Fit for This Trial?

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.

I have had a gastrointestinal disorder for at least 1 year.

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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. 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 ...

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

Long-term benefit of Microbiota Transfer Therapy on autism ... Notably, most improvements in GI symptoms were maintained, and autism-related symptoms improved even more after the end of treatment. Important ...

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

Faecal microbiota transplantation as a novel approach for ... Early clinical investigations, though limited, suggest that FMT may improve gastrointestinal health, core ASD symptoms, and comorbid anxiety. Mechanistically, ...

5. 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.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03408886

NCT03408886 | Microbiota Transfer Therapy for Adults ... This is a clinical trial of Microbiota Transplant Therapy (MTT) for adults with autism spectrum disorders (ASD) who have gastrointestinal problems.

7. 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 ...

8. microbiomejournal.biomedcentral.com microbiomejournal.biomedcentral.com/articles/10.1186/s40168-016-0225-7

Microbiota Transfer Therapy alters gut ecosystem and ... A small open-label clinical trial evaluated the impact of Microbiota Transfer Therapy (MTT) on gut microbiota composition and GI and ASD symptoms of 18 ASD- ...