Fecal Microbiota Transplantation for C. diff Infection

Overseen By Anne Foley

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Krunal Patel

Disqualifiers: Age <16, Acute colonic dilation

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

500 Participants Needed

Trial Summary

Do I need to stop my current medications for this trial?

The trial information does not specify if you need to stop taking your current medications. However, since it involves patients who have failed standard therapy with certain antibiotics, you might need to discuss your current medications with the trial team.

What data supports the effectiveness of the treatment Fecal Microbiota Transplantation for C. diff Infection?

Research shows that Fecal Microbiota Transplantation (FMT) is highly effective for treating recurrent C. difficile infections, with cure rates over 90% and fewer recurrences compared to standard antibiotic treatments. It is also promising for severe cases, reducing the need for surgery and lowering death rates. ^{1 2 3 4 5}

Is fecal microbiota transplantation (FMT) safe for humans?

FMT is generally considered safe and well-tolerated, even in high-risk patients, with most short-term risks being mild. However, long-term side effects are not well-established, and serious adverse events have been linked to inadequate donor screening for drug-resistant organisms. ^{6 7 8 9 10}

How is fecal microbiota transplantation different from other treatments for C. diff infection?

Fecal microbiota transplantation (FMT) is unique because it involves transplanting stool from a healthy donor to restore the natural balance of bacteria in the gut, which is different from traditional antibiotic treatments. FMT has shown high cure rates and lower recurrence rates for recurrent C. diff infections compared to antibiotics, and it can be particularly effective for severe cases where other treatments have failed. ^{1 3 5 10 11}

What is the purpose of this trial?

The purpose of this study is to see if stool transplant performed by colonoscopy is effective at treating recurrent Clostridium difficile (C. diff) infection of the colon. During the procedure a stool sample is taken from a healthy donor (usually family member or close friend) and transplanted directly into the colon of the patient with C. diff infection. The goal of this experimental procedure (called fecal microbiota transplantation) is to replenish the good bacteria in the colon that can help prevent C. diff infection from coming back after treatment.

Research Team

Krunal Patel

Principal Investigator

UMass Medical School

Eligibility Criteria

This trial is for people who've had at least two recurrences of C. diff infection, with symptoms coming back within 2-8 weeks after treatment. It's also for those whose severe C. diff led to hospitalization and didn't improve with standard antibiotics. Participants must be over 16 and not at risk of colon perforation.

Inclusion Criteria

I've been hospitalized for a severe C. diff infection that didn't improve with antibiotics.

I have had C. difficile infection recur at least twice after treatment.

I have tried and not responded to standard treatments like metronidazole or vancomycin.

Exclusion Criteria

I am younger than 16 years old.

I am at risk for a tear in my colon due to severe swelling.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive fecal microbiota transplantation via colonoscopy to treat recurrent Clostridium difficile infection

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after fecal microbiota transplantation

12 months

Periodic visits (in-person)

Treatment Details

Interventions

• Fecal Microbiota Transplantation

Trial Overview The study tests fecal microbiota transplantation (FMT) via colonoscopy to treat recurrent C. diff infections by transferring stool from a healthy donor into the patient's colon, aiming to restore beneficial bacteria.

Participant Groups

Group I: Fecal Microbiota Transplantation Experimental Treatment 1 Intervention

Subjects receive intervention of stool transplanted to the colon via colonoscopy.

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

Approved in United States as Fecal Microbiota Transplantation for:

• Graft-versus-host disease (GVHD)
• Clostridioides difficile infection (CDI)

Approved in European Union as Fecal Microbiota Transplantation for:

• Graft-versus-host disease (GVHD)
• Clostridioides difficile infection (CDI)
• Other gastrointestinal disorders

Approved in Canada as Fecal Microbiota Transplantation for:

• Graft-versus-host disease (GVHD)
• Clostridioides difficile infection (CDI)

Who Is Running the Clinical Trial?

Krunal Patel

Lead Sponsor

Trials

1

Recruited

500+

Findings from Research

Fecal microbiota transplantation (FMT) has a cure rate of over 90% for recurrent Clostridioides difficile infection (CDI) and is more effective than antibiotics in preventing recurrence.

Emerging evidence suggests that FMT is beneficial for severe and fulminant CDI, potentially reducing mortality and the need for colectomy, and should be considered early in treatment for critically-ill patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplantation: Redefining Surgical Management of Refractory Clostridium difficile Infection. Cheng, YW., Fischer, M.[2021]

Fecal microbiota transplantation (FMT) significantly reduces the recurrence of Clostridium difficile infection (CDI) in patients, according to a mathematical model based on data from hospitals in the southeastern United States.

While FMT helps prevent recurrent CDI, it does not lower the overall incidence of new cases, indicating that additional strategies are needed to prevent transmission of the infection.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A mathematical model to evaluate the routine use of fecal microbiota transplantation to prevent incident and recurrent Clostridium difficile infection. Lofgren, ET., Moehring, RW., Anderson, DJ., et al.[2022]

In a study of 35 patients with recurrent C. difficile infection, 85.7% experienced resolution of their symptoms within 6-8 weeks after undergoing fecal microbiota transplantation (FMT), indicating its effectiveness as a treatment option.

Among patients with a history of inflammatory bowel disease, 75% reported symptom resolution post-FMT, suggesting that FMT may also be beneficial for this high-risk group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation in recurrent Clostridium difficile infection: A retrospective single-center chart review. Kim, P., Gadani, A., Abdul-Baki, H., et al.[2022]

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]

Fecal microbiota transplantation (FMT) is an effective treatment for recurrent Clostridium difficile infections, helping to restore healthy gut bacteria and resolve symptoms.

The chapter presents a rapid and cost-effective method for preparing FMT using simple, disposable materials, making the procedure more accessible for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Practical Method for Preparation of Fecal Microbiota Transplantation. Perez, E., Lee, CH., Petrof, EO.[2020]

Fecal microbiota transplantation (FMT) is a generally safe and well-tolerated procedure for treating recurrent Clostridioides difficile infections, even in high-risk patients, with most short-term risks being mild and related to delivery methods.

Recent concerns have emerged regarding serious adverse events linked to FMT products from stool banks that do not screen for multi-drug resistant organisms, highlighting the need for improved safety protocols.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal Microbiota Transplantation: Is It Safe? Park, SY., Seo, GS.[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]

In 2018, OpenBiome, a large stool bank, processed 4,271 fecal microbiota transplants (FMT) from 7,536 donations, indicating a robust system for providing FMT material to treat recurrent Clostridioides difficile infections.

The median time from donation to shipment of FMT preparations was 8.9 months, highlighting the need for improved efficiency in the stool banking process to enhance patient access to this therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stool Banking for Fecal Microbiota Transplantation: Methods and Operations at a Large Stool Bank. Chen, J., Zaman, A., Ramakrishna, B., et al.[2021]

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]

Out of 114 evaluated donor candidates for fecal microbiota transplantation (FMT), only 29 (25%) qualified as stool donors after a rigorous four-step screening process, highlighting the challenges in donor recruitment.

The screening process resulted in a total of 127 usable stool aliquots, but 15 samples were excluded after further testing, indicating the importance of thorough testing to ensure safety and efficacy in FMT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Donor program for fecal microbiota transplantation: A 3-year experience of a large-volume Italian stool bank. Ianiro, G., Porcari, S., Bibbò, S., et al.[2021]

Fecal microbiota transplantation (FMT) is an effective treatment for recurrent Clostridioides difficile infection and has become more accessible due to the availability of stool banks.

Recent studies suggest that FMT may also be beneficial for other conditions like ulcerative colitis, irritable bowel syndrome, and hepatic encephalopathy, indicating its potential for broader therapeutic applications, although more research is needed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Application and Potential of Fecal Microbiota Transplantation. Ooijevaar, RE., Terveer, EM., Verspaget, HW., et al.[2022]

References

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

Fecal Microbiota Transplantation: Redefining Surgical Management of Refractory Clostridium difficile Infection. [2021]

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

A mathematical model to evaluate the routine use of fecal microbiota transplantation to prevent incident and recurrent Clostridium difficile infection. [2022]

3. Australia pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation in recurrent Clostridium difficile infection: A retrospective single-center chart review. [2022]

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

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

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

A Practical Method for Preparation of Fecal Microbiota Transplantation. [2020]

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

Fecal Microbiota Transplantation: Is It Safe? [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. Switzerland pubmed.ncbi.nlm.nih.gov

Stool Banking for Fecal Microbiota Transplantation: Methods and Operations at a Large Stool Bank. [2021]

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

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

10. Netherlands pubmed.ncbi.nlm.nih.gov

Donor program for fecal microbiota transplantation: A 3-year experience of a large-volume Italian stool bank. [2021]

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

Clinical Application and Potential of Fecal Microbiota Transplantation. [2022]