Clinical trial NCT04968951: Refined Fecal Microbiota Transplantation (FMT) for Ulcerative Colitis (UC)
Official title: REFOCUS: Refined Fecal Microbiota Transplantation (FMT) Delivered by Oral Capsules for Induction of Remission in Mild to Moderate Ulcerative Colitis - a Phase I Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: EARLY_PHASE1
Conditions: Ulcerative Colitis
FMT
Fecal Microbiota Transplant
Keywords: FMT
Fecal Microbiota Transplantation
Fecal Microbiota Transplant
Fecal Matter Transplant
Ulcerative colitis
UC
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
First posted: 2021-07-20
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants are assigned to one of two groups. One group will be assigned to receive pre-treatment with antibiotics before Fecal Microbiota Transportation. The other group will be assigned to receive pre-treatment without antibiotics (with placebo) before Fecal Microbiota Transplantation.
Primary purpose: TREATMENT

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)

Eligibility 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

Primary outcomes:
measure: Proportion of participants with an adverse
timeFrame: 8 Weeks
measure: Proportion of participants with a severe adverse
timeFrame: 8 Weeks
Secondary outcomes:
measure: Number of patients requiring escalation of medical therapies
timeFrame: 8 weeks
measure: Proportion of patients that achieve Mayo score 0 or 1
timeFrame: 8 weeks
measure: Number of patients with endoscopic remission
timeFrame: 8 weeks
measure: Change in Nancy score
timeFrame: 8 weeks
measure: Fecal calprotectin level
timeFrame: baseline and 8 weeks
measure: C-reactive protein levels
timeFrame: baseline and 8 weeks
measure: Hemoglobin levels
timeFrame: baseline and 8 weeks
measure: Albumin levels
timeFrame: baseline and 8 weeks
measure: ESR level
timeFrame: baseline and 8 weeks
Other outcomes:
Locations:
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
Study officials:
affiliation: Icahn School of Medicine at Mount Sinai
name: Ari Grinspan, MD
role: PRINCIPAL_INVESTIGATOR
References: