Fecal Microbiota Transplant for Patients With Chronic Pouchitis

Safety and Efficacy of Healthy to Inflamed Pouch Fecal Microbiota Transplantation

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

Study Overview

• Status: Recruiting
• Conditions: Pouchitis; Chronic Pouchitis
• Intervention / Treatment: Drug: Fecal Microbiota Transplant (FMT)

Detailed Description

The purpose of this research study is to assess the safety and efficacy of fecal microbiota transplant (FMT) in the treatment of chronic pouchitis. FMT has been successfully used in the treatment of recurrent Clostridiodes difficile infection and has shown benefit in the treatment of ulcerative colitis in clinical trials. The success of FMT in these patients is because of the reconstitution of the recipient's unhealthy gut bacteria with the donor's healthy gut bacteria.

Surgery to remove the colon is required in a subset of patients with ulcerative colitis that does not respond to medical therapy. In these patients, an internal pouch is created from small intestine to function as a stool reservoir and avoid an ostomy after the colon is removed. Inflammation of the pouch, pouchitis, is common after surgery and can manifest as diarrhea, pelvic pain, urgency and blood in the stool. Chronic pouchitis occurs in up to 20% of patients and there is no approved treatment. A number of studies have evaluated FMT in patients with chronic pouchitis, but have proven unsuccessful. This is likely because these studies have used stool from patients with a colon and transplanted it into patients with a pouch. This is problematic because the gut bacteria of the colon and pouch are not similar, and putting healthy stool from a colon may not reconstitute a healthy pouch microbiome. The specific purpose of this project is to transplant stool from patients with a healthy pouch to patients with an inflamed pouch.

• Study Type: Interventional
• Enrollment (Estimated): 16
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Maia Kayal, MD, MS; Phone Number: 212-241-0150; Email: Maia.Kayal@mountsinai.org

Study Locations

• United States
  • New York
    • New York, New York, United States, 10029
      • Recruiting
      • Icahn School of Medicine at Mount Sinai
      • Contact: Maia Kayal, MD, MS; Phone Number: 212-241-0150; Email: Maia.Kayal@mountsinai.org
      • Principal Investigator: Maia Kayal

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Patients age 18 or greater with UC who have undergone TPC with IPAA and have one of the following chronic pouchitis phenotypes, each defined as:

• Chronic antibiotic dependent pouchitis:
• The need for continuous antibiotic therapy (>4 weeks) to maintain clinical remission and a history of at least 2 attempts in the last 24 months to stop antibiotic therapy resulting in pouchitis episodes, OR
• Active pouchitis with a modified Pouchitis Disease Activity Index (mPDAI) ≥5 and a history of ≥4 antibiotic therapies in the last 12 months
• Chronic antibiotic refractory pouchitis:
• Active pouchitis with a modified Pouchitis Disease Activity Index (mPDAI) ≥5 with no response to antibiotics
• Crohn's disease like pouch inflammation on biologic or small molecule therapy with persistent symptoms:
• Pre-pouch ileal inflammation, strictures, and/or fistulae, AND
• Active biologic or small molecule therapy, AND
• Persistent symptoms with mPDAI clinical sub-score ≥ 2

Exclusion Criteria:

Patients with UC who underwent TPC with IPAA and meet one of the following criteria will be excluded:

• Allergy to vancomycin, metronidazole, or ingredients present in the FMT
• Women who are breastfeeding
• Women who are pregnant
• Participants with fever > 100.4F/38C or other signs of active illness
• Active treatment with biologics (infliximab, adalimumab, golimumab, vedolizumab, ustekinumab)
• Active treatment with immunomodulators (azathioprine, 6-mercaptopurine, methotrexate), steroids or any investigational drugs
• Crohn's disease like pouch inflammation
• Active enteric infection
• Isolated cuffitis
• Clinically significant strictures of the pouch inlet or outlet
• Participation in a clinical trial in the preceding 30 days
• Any condition that the physician investigators deems unsafe, including other conditions or medications that the investigator determines will put the participant at greater risk from FMT

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Fecal Microbiota Transplant (FMT); Single arm of 16-18 subjects, all of whom will receive the interventional FMT.	Drug: Fecal Microbiota Transplant (FMT); The intervention consists of the following steps: Step 1: Vancomycin 125 mg orally every 6 hours and metronidazole 250 mg orally every 6 hours for 5 days.; Step 2: Bowel preparation with 10 ounces of magnesium citrate.; Step 3: Two FMT doses will be administered via enema one week apart using stool from donors with a durably healthy pouch.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with FMT related adverse events	The number of patients with FMT related adverse events through week 8 classified according to the Medical Dictionary for Regulatory Activities (MedDRA) and categorized according to the NIH Criteria for Adverse Events.	8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients in clinical remission	The number of patients in clinical remission assessed via patient interview and defined as mPDAI clinical subscore ≤4 points and no need for antibiotic therapy at week 8.	8 weeks
Number of patients with endoscopic response	The number of patients with endoscopic response assessed via pouchoscopy and defined as a decrease from baseline in mPDAI endoscopic subscore > 2 points at week 8.	8 weeks
Change in recipient fecal microbial diversity via metagenomics analysis	The change in recipient fecal microbial diversity after FMT relative to baseline assessed via stool collection and using metagenomics analysis.	Baseline and up to 8 weeks
Change in recipient fecal microbial diversity via strain strain isolation	The change in recipient fecal microbial diversity after FMT relative to baseline assessed via strain isolation - to isolate and sequence the gut microbial strains from each donor	Baseline and up to 8 weeks
Change in recipient microbe tracking	The change in recipient fecal microbial diversity after FMT relative to baseline assessed by recipient microbe tracking.	Baseline and up to 8 weeks
Change in B cells	The change in the mucosal immune profile before and after FMT as measured by B cells	Baseline and 8 weeks
Change in myeloid cells	The change in the mucosal immune profile before and after FMT as measured by myeloid cells	Baseline and 8 weeks
Change in T cells	The change in the mucosal immune profile before and after FMT as measured by T cells	Baseline and 8 weeks
Change in NK cell subsets	The change in the mucosal immune profile before and after FMT as measured by NK cell subsets	Baseline and 8 weeks

Collaborators and Investigators

• Sponsor: Maia Kayal
• Investigators: Principal Investigator: Maia Kayal, MD, MS, Icahn School of Medicine at Mount Sinai

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026

Study Registration Dates

• First Submitted: March 30, 2023
• First Submitted That Met QC Criteria: April 12, 2023
• First Posted (Actual): April 25, 2023

Study Record Updates

• Last Update Posted (Actual): July 15, 2025
• Last Update Submitted That Met QC Criteria: July 10, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Enteritis; Ileitis; Ileal Diseases; Pouchitis
• Other Study ID Numbers: STUDY-22-01753

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices).
• IPD Sharing Time Frame: Beginning 9 months and ending 36 months following article publication.
• IPD Sharing Access Criteria: Researchers who provide a methodologically sound proposal. For individual participant data meta-analysis. Proposals should be directed to Maia.Kayal@mountsinai.org. To gain access, data requestors will need to sign a data access agreement. Data are available for 5 years at a third party website (tbd).
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No