Fecal Microbiota Transplant for Chronic Pouchitis

Overseen By Maia Kayal

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Maia Kayal

Must be taking: Antibiotics

Must not be taking: Biologics, Immunomodulators, Steroids, others

Disqualifiers: Pregnancy, Breastfeeding, Fever, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

16 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this research study is to assess the safety and efficacy of fecal microbiota transplant (FMT) in the treatment of chronic pouchitis.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as biologics, immunomodulators, steroids, or any investigational drugs, to participate.

What data supports the effectiveness of the treatment Fecal Microbiota Transplant (FMT) for chronic pouchitis?

Some studies suggest that Fecal Microbiota Transplant (FMT) may help improve symptoms in patients with chronic pouchitis, with a few patients experiencing clinical remission and increased microbial diversity. However, results are mixed, and more well-designed studies are needed to confirm its effectiveness. ^{1 2 3 4 5}

Is fecal microbiota transplant (FMT) generally safe for humans?

FMT is considered generally safe for treating certain conditions like Clostridium difficile infection, but there is limited data on long-term safety. Short-term side effects can occur, often related to the method of delivery, and more research is needed to fully understand the safety profile. ^{1 2 4 6 7}

How is the treatment Fecal Microbiota Transplant (FMT) unique for chronic pouchitis?

FMT is unique because it involves transferring stool from a healthy donor to a patient to restore a balanced gut microbiome, which is different from traditional treatments that may use medications to reduce inflammation. This approach aims to directly alter the gut bacteria composition, potentially offering a novel way to manage chronic pouchitis. ^{1 2 4 8 9}

Research Team

Maia Kayal

Principal Investigator

Icahn School of Medicine at Mount Sinai

Eligibility Criteria

This trial is for adults with chronic pouchitis who rely on antibiotics to stay in remission and have tried stopping antibiotics at least twice, leading to flare-ups. It's not for those with certain pouch strictures, Crohn's-like inflammation, allergies to specific antibiotics used here, pregnant or breastfeeding women, or patients currently on biologics, immunomodulators, steroids or other trials.

Inclusion Criteria

I am 18 or older with UC and had surgery to create a pouch in my lower belly.

I need ongoing antibiotics for pouchitis and have had flare-ups when trying to stop them.

I have active pouchitis and have tried at least 4 antibiotic treatments in the past year.

Exclusion Criteria

I currently have a fever over 100.4F or show signs of being sick.

I am currently taking immunomodulators, steroids, or experimental drugs.

I have inflammation similar to Crohn's disease in my pouch.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive fecal microbiota transplant (FMT) for the treatment of chronic pouchitis

8 weeks

Regular visits for monitoring and assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Fecal Microbiota Transplant (FMT)

Trial Overview The study is testing the safety and effectiveness of fecal microbiota transplant (FMT) as a treatment option for people suffering from chronic pouchitis. Participants will receive FMT to see if it can help maintain their condition without the need for continuous antibiotic use.

Participant Groups

Group I: Fecal Microbiota Transplant (FMT) Experimental Treatment 1 Intervention

Single arm of 16-18 subjects, all of whom will receive the interventional FMT.

Fecal Microbiota Transplant (FMT) is already approved in United States for the following indications:

Approved in United States as Fecal Microbiota Transplant for:

• Recurrent Clostridioides difficile infection (CDI)

Who Is Running the Clinical Trial?

Maia Kayal

Lead Sponsor

Trials

1

Recruited

20+

Findings from Research

Fecal microbiota transplant (FMT) appears to be safe for patients with chronic pouchitis, as indicated by the four studies reviewed, but its efficacy is limited, with only a few patients achieving sustained clinical remission.

Out of the studies, only one showed a significant clinical response, with 75% of patients in that study reaching remission, while the overall results suggest that more research is needed to better understand the effectiveness of FMT for both acute and chronic pouchitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Systematic Review of Fecal Microbiota Transplant for the Management of Pouchitis. Kayal, M., Lambin, T., Pinotti, R., et al.[2023]

In a study involving 8 patients with chronic pouchitis, faecal microbiota transplantation (FMT) led to variable changes in the composition of gut microbiota, indicating potential shifts towards a healthier microbiota.

Despite these changes, FMT did not result in significant improvements in metabolic, immunological responses, or clinical outcomes, suggesting that the current method of FMT administration may need further optimization for effective treatment of pouchitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Variable alterations of the microbiota, without metabolic or immunological change, following faecal microbiota transplantation in patients with chronic pouchitis. Landy, J., Walker, AW., Li, JV., et al.[2023]

A systematic review of nine studies involving 69 patients with chronic pouchitis found that fecal microbiota transplantation (FMT) resulted in clinical response in 31.8% and clinical remission in 22.7% of patients, indicating some potential efficacy.

Despite these findings, the overall quality of the studies was low, with only one randomized controlled trial showing no beneficial effect of FMT, highlighting the need for more rigorous research to establish the effectiveness of this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplantation in the Treatment of Chronic Pouchitis: A Systematic Review. Cold, F., Kousgaard, SJ., Halkjaer, SI., et al.[2020]

Faecal microbiota transplantation (FMT) showed a clinical response rate of 42.6% and a remission rate of 29.8% in treating chronic pouchitis, based on a systematic review of 9 studies involving 103 patients.

The treatment was well tolerated with minor, self-limiting adverse events reported, indicating a good safety profile, but the evidence is limited and calls for more rigorous studies to better assess FMT's efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Faecal microbiota transplantation (FMT) in the treatment of chronic refractory pouchitis: A systematic review and meta-analysis. Zaman, S., Akingboye, A., Mohamedahmed, AYY., et al.[2023]

In a pilot study involving nine patients with chronic pouchitis, fecal microbiota transplantation (FMT) led to significant clinical improvement in symptoms for four patients, with three maintaining remission for up to six months.

FMT treatment resulted in a notable increase in gut microbial diversity and successful engraftment of donor microbiota in six patients, suggesting a potential mechanism for its therapeutic effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical results and microbiota changes after faecal microbiota transplantation for chronic pouchitis: a pilot study. Kousgaard, SJ., Michaelsen, TY., Nielsen, HL., et al.[2021]

A systematic review of 129 studies involving 4241 patients found that 19% of fecal microbiota transplantation (FMT) procedures reported adverse events, with the most common being mild issues like diarrhea (10%) and abdominal discomfort (7%).

Serious adverse events (SAEs) occurred in 1.4% of patients, primarily in those with mucosal barrier injury, highlighting the need for improved FMT methodologies to enhance safety, especially for high-risk patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Systematic review: the global incidence of faecal microbiota transplantation-related adverse events from 2000 to 2020. Marcella, C., Cui, B., Kelly, CR., et al.[2021]

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 effective for treating recurrent Clostridioides difficile infection (CDI), but 16.3% of patients experienced treatment failure, with significant predictors including advanced age, severe CDI, and use of non-CDI antibiotics around the time of FMT.

Improving bowel preparation and antibiotic management before and after FMT can enhance treatment success, and patients with certain risk factors should be informed about their likelihood of FMT failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Predictors of Fecal Microbiota Transplant Failure in Clostridioides difficile Infection : An Updated Meta-analysis. Beran, A., Sharma, S., Ghazaleh, S., et al.[2023]

Fecal microbiota transplantation (FMT) is a cost-effective treatment for recurrent Clostridium difficile infection (rCDI), with all economic evaluations showing it to be more cost-effective than other standard treatments at a threshold of ≤$50,000 per quality-adjusted life year (QALY).

The systematic review included 9 high-quality economic evaluations, primarily focusing on rCDI and inflammatory bowel disease (IBD), indicating that while FMT is effective for rCDI, further studies are needed to explore its economic impact for other conditions like IBD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A systematic review of economic evaluation in fecal microbiota transplantation. Stalder, T., Kapel, N., Diaz, S., et al.[2021]

References

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

A Systematic Review of Fecal Microbiota Transplant for the Management of Pouchitis. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Variable alterations of the microbiota, without metabolic or immunological change, following faecal microbiota transplantation in patients with chronic pouchitis. [2023]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplantation in the Treatment of Chronic Pouchitis: A Systematic Review. [2020]

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

Faecal microbiota transplantation (FMT) in the treatment of chronic refractory pouchitis: A systematic review and meta-analysis. [2023]

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

Clinical results and microbiota changes after faecal microbiota transplantation for chronic pouchitis: a pilot study. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Systematic review: the global incidence of faecal microbiota transplantation-related adverse events from 2000 to 2020. [2021]

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

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

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

Predictors of Fecal Microbiota Transplant Failure in Clostridioides difficile Infection : An Updated Meta-analysis. [2023]

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

A systematic review of economic evaluation in fecal microbiota transplantation. [2021]