Refined Fecal Microbiota Transplantation (FMT) for Ulcerative Colitis (UC) (REFOCUS)

Ari M Grinspan

Status and phase: Terminated; Early Phase 1

Conditions: Fecal Microbiota Transplant; FMT; Ulcerative Colitis

Treatments: Biological: Fecal Microbiota Transplantation; Drug: Metronidazole; Drug: Placebo; Drug: Vancomycin

Study type: Interventional

Funder types: Other

Identifiers

NCT04968951

GCO 18-1464

Details and patient eligibility

About

The researchers intend to prospectively study the safety, clinical efficacy and microbial outcomes in patients with recently diagnosed UC with FMT capsule therapy derived from pre-defined donors. Donors will be specifically screened for Fusobacterium and Sutterella species as well as for global diversity. It is unknown if treatment with antibiotics before FMT improves the engraftment and/or efficacy of FMT in UC, therefore the researchers plan to randomize subjects to receive pre-treatment with antibiotics or not before FMT therapy. The research team enroll patients from The Susan and Leonard Feinstein IBD Center and our established early diagnosis clinic at Mount Sinai Hospital (MSH).

Full description

This is an open-label two arm pilot study to measure the safety, microbiological and clinical impacts of FMT in patients with ulcerative colitis. The researchers will prospectively enroll 16 UC patients (up to 20 subjects accounting for subjects dropping out) with moderate-severe disease from one tertiary care referral center. The overall objective of the study is to collect robust clinical data and create a tissue repository including blood, stool and biopsies to understand the safety, efficacy and microbial changes FMT has on UC patients. The central hypothesis is that pre-defined oral capsule administered FMT is safe and effective for the treatment of UC.

Objectives: To determine the tolerability, feasibility, and safety of using fecal microbiota transplantation orally as an induction agent for patients with ulcerative colitis. To determine whether fecal microbiota transplantation (FMT) delivered via oral capsules can induce clinical remission in patients with mild to moderate ulcerative colitis. Assess whether pretreatment with antibiotics improves engraftment and efficacy of FMT in UC. To characterize the impact of orally administered FMT on the microbiota of patients with ulcerative colitis, particularly those changes associated with response or lack of response.

Study Outcomes: Clinical remission at Week 8, defined as: Steroid-free clinical remission (Total Mayo less 2) and Endoscopic remission (Mayo endoscopic subscore 0 or 1)

Enrollment: 4 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

• Adults aged 18 to 75 with UC, with initial diagnosis of UC >3 months prior to time of study enrollment visit
• Partial Mayo score of 4-10 with endoscopic subscore ≥2 on flexible sigmoidoscopy
• Permissible UC medications include oral or rectal administered mesalamines
• Corticosteroids must be discontinued at least 4 weeks before enrollment
• Documented negative C. difficile and GI PCR for enteric pathogens (BioFire) testing before commencement of fecal microbiota transplantation
• Previous documentation of ulcerative colitis based on colonoscopy/flexible sigmoidoscopy with compatible histology

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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4 participants in 2 patient groups, including a placebo group

FMT with Antibiotics

Active Comparator group

Description:

Participants will receive antibiotics before receiving Fecal Microbiota Transplantation

Treatment:

Drug: Metronidazole

Biological: Fecal Microbiota Transplantation

Drug: Vancomycin

FMT with placebo

Placebo Comparator group

Description:

Participants will receive placebo before receiving Fecal Microbiota Transplantation

Treatment:

Drug: Placebo

Biological: Fecal Microbiota Transplantation