REFOCUS: Refined Fecal Microbiota Transplantation (FMT) Delivered by Oral Capsules for Induction of Remission in Mild to Moderate Ulcerative Colitis - a Phase I Study
Trial Snapshot
- Phase
- Early Phase 1
- Status
- Terminated
- Sponsor
- Enrollment
- 4
- Locations
- 1
- Primary Endpoint
- Proportion of participants with an adverse
Study Overview
Brief Summary
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).
Detailed 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)
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Masking Description
Antibiotics and placebo will be disguised
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
FMT with Antibiotics
Participants will receive antibiotics before receiving Fecal Microbiota Transplantation
Intervention: Metronidazole (Drug)
FMT with Antibiotics
Participants will receive antibiotics before receiving Fecal Microbiota Transplantation
Intervention: Vancomycin (Drug)
FMT with Antibiotics
Participants will receive antibiotics before receiving Fecal Microbiota Transplantation
Intervention: Fecal Microbiota Transplantation (Biological)
FMT with placebo
Participants will receive placebo before receiving Fecal Microbiota Transplantation
Intervention: Placebo (Drug)
FMT with placebo
Participants will receive placebo before receiving Fecal Microbiota Transplantation
Intervention: Fecal Microbiota Transplantation (Biological)
Outcomes
Primary Outcomes
Proportion of participants with an adverse
Time Frame: 8 Weeks
Safety as measured by proportion of participants with an adverse event through week 8
Proportion of participants with a severe adverse
Time Frame: 8 Weeks
Safety as measured by proportion of participants with a severe adverse event through week 8
Secondary Outcomes
- ESR level(baseline and 8 weeks)
- Number of patients requiring escalation of medical therapies(8 weeks)
- Albumin levels(baseline and 8 weeks)
- Proportion of patients that achieve Mayo score 0 or 1(8 weeks)
- Number of patients with endoscopic remission(8 weeks)
- Change in Nancy score(8 weeks)
- Fecal calprotectin level(baseline and 8 weeks)
- C-reactive protein levels(baseline and 8 weeks)
- Hemoglobin levels(baseline and 8 weeks)
Investigators
Ari M Grinspan
Associate Professor of Medicine
Icahn School of Medicine at Mount Sinai